Curriculum Vitae

PERSONAL INFORMATION Francesco Rodeghiero

WORK EXPERIENCE

February 2004- Present Scientific Director Fondazione Progetto Ematologia (Italy)

August 2001-October 2015 Director of the Department of Cell Therapy and Hematology Azienda ULSS N.6 (Italy)

The Department includes a Unit for Bone Marrow Transplantation, a specialized Center for the diagnosis and treatment of Hemophilia and Thrombosis, and a Research Laboratory

1989- 2016 Professor at the Postgraduate School of Hematology on a contract-basis University of Verona (Italy)

February 1993-October 2015 Director of the Hematology Department Azienda ULSS N.6 (Italy)

December 1985-October 2015 Director of the Hemostasis and Trombosis Center Azienda ULSS N.6 (Italy)

September 2006-November 2016 Member; Chairman from June 2016 to November 2016 Ethics Committee on drugs research and investigational protocol studies of the Vicenza District (Italy)

EDUCATION AND TRAINING

July 1975- Degree in Medicine University School of Medicine (Italy)

December 1984- Postgraduate specialization in Laboratory Medicine University of Padova (Italy)

June 1981- Postgraduate specialization in Oncology University of Ferrara (Italy)

July 1978- Postgraduate specialization in Hematology University of Ferrara (Italy)

ADDITIONAL INFORMATION

15/12/2020 European Medicine Agency Page 1/46 Expertise He has been conducting clinical research in the fields of hematology and hemostasis since the early 1970s. His main interests include thrombocytopenia, hemophilia, von Willebrand disease, thrombophilia, acute promyelocytic leukemia, myeloma, policytemia vera, and the epidemiological aspects of haematological diseases. In the last decades he mainly devoted to clinical research in the field of ITP.

Publications 1.T. Barbui, F. Rodeghiero, E. Dini The aspirin tolerance test in von Willebrands disease. Tromb Haemost 38(2): 510-513, 1977 2.F. Rodeghiero, T. Barbui Two dimensional immunoelectrophoresis of antithrombin III during disseminated intravascular coagulation in acute leukemia. Thromb Res 12(1): 191-196, 1977 3.T. Barbui, F. Rodeghiero, E. Dini, G. Mariani, M. L. Papa, R. De Biasi, R. Cordero-Murillo, C. Montero-Umana Subunits A and S inheritance in four families with congenital factor XIII deficiency. Br J Haematol 38(2): 267-271, 1978 4.F. Rodeghiero, T. Barbui Fibrin cross-linking in congenital factor XIII deficiency. J Clin Pathol 33(5): 434-437, 1980 5.F. Rodeghiero,T. Barbui, R. Battista, T. Chisesi, G. Rigoni, E. Dini Molecular subunits and transamidase activity of factor XIII during disseminated intravascular coagulation in acute leukemia. Thromb Haemost 43(1): 6-7, 1980 6.T. Barbui, F. Rodeghiero Hereditary dysfunctional antithrombin III (AT-III Vicenza). Thromb Haemost 45(1): 97, 1981 7.F. Rodeghiero, M. Morbin, T. Barbui Subnit A of factor XIII regulates subunit S plasma concentration. Thromb Haemost 46(3): 621-622, 1981 8.F. Rodeghiero, S. Castronovo, E. Dini Disseminated intravascular coagulation after infusion of FEIBA (factor VIII inhibitor bypassing activity) in a patient with acquired haemophilia. Thromb Haemost 48(3): 339-340, 1982

9.T. Barbui, R. Bassan, T. Chisesi, R. Battista, S. Cortelazzo, F. Rodeghiero, G. Capnist, P. Viero, A. D'Emilio, E. Dini Retrospective analysis of treatment of acute myeloblastic leukemia in patients more than 55 years old. Acta Haematol 67: 170-174, 1982 10.T. Barbui, F. Rodeghiero, S. Meli, E. Dini Fatal pulmonary embolism and antithrombin III deficiency in adult lymphoblastic leukemia during L-Asparaginase therapy. Acta Haematol 69(3): 188-191, 1983

15/12/2020 European Medicine Agency Page 2/46 11.T. Barbui, G. Finazzi, F. Rodeghiero, E. Dini Immunoelectrophoretic evidence of a thrombin-induced abnormality in a new variant of hereditary dysfunctional antithrombin III (AT III Vicenza). Br J Haematol 54(4): 561-565, 1983 12.F. Rodeghiero, P. M. Mannucci, S. Viganò, T. Barbui, L. Gugliotta, M. Cortellaro, E. Dini Liver dysfunction rather than intravascular coagulation as the main cause of low protein C and antithrombin III in acute leukemia. Blood 63(4): 965-969, 1984 13.F. Rodeghiero, T. Barbui, A. Dal Belin-Peruffo, E. Dini Defective fibrin crosslinking in acute leukemia. Thromb Haemost 52(3): 343-346, 1984 14.T. Barbui, R. Bassan, T. Chisesi, R. Battista, S. Cortellazzo, F. Rodeghiero, G. Capnist, A. DEmilio, P. Viero, E. Dini Treatment of acute lymphoblastic leukemia in adults. Hematol Oncol 3(1): 49-53, 1985 15.F. Rodeghiero, G. Castaman, T. Chisesi, A. De Rossi, A. Dalla Gassa, A. Del Mistro, E. Dini, S. Castronovo, E. Pornaro, L. Chieco Bianchi One year follow-up study of T-cell subsets and incidence of seropositivity for HTLV-I and HTLV-III antibodies in patients treated "on demand" or sporadically with clotting concentrates. Thromb Haemost 54(3): 665-668, 1985. 16.G. Castaman, F. Rodeghiero, G. F. Ronconi, E. Dini Successful treatment of neonatal "autoimmune" thrombocytopenia with high-doses intravenous immunoglobulins. Haematologica 70(3): 276-7, 1985 17.G. Ballerini, S. Guerra, F. Rodeghiero, G. Castaman A contribution to the pathology of acquired plasma factor XIII deficiency. Semin Thromb Hemost 11 (4): 357-61, 1985. 18.F. Rodeghiero, G. Castaman, L. Tonellato, E. Dini Erythrocyte fragmentation in disseminated intravascular coagulation (DIC) in acute leukaemia. Br J Haematol 61(3): 593-594, 1985 19.F. Rodeghiero Treatment of Hemophilia (editorial) Ric Clin Lab 15(4): 289-303, 1985. 20.P. M. Mannucci, M. Colombo, F. Rodeghiero Non A-, non B- hepatitis after factor VIII concentrate treated by heating and cloroform. Lancet 2(2): 1013, 1985. 21.R. Zanetti, P. M. Mannucci, E. Tanzi, A. G. Moroni, De Paschale, M. Morfini, V. Carnelli, M. C. Tirindelli, R. De Biasi, N. Ciavarella, V. De Rosa, F. Rodeghiero, M. Colombo Hepatitis B vaccination of 113 hemophiliacs: lower antibodies responses in anti-LAV/HTLV-III-positive patients. Am J Hematol 23: 339-345, 1986. 22.F. Rodeghiero, G. Castaman, E. Dini

15/12/2020 European Medicine Agency Page 3/46 Epidemiological investigation of the prevalence of von Willebrand's disease. Blood 69(2): 454-459, 1987. 23.F. Rodeghiero, G. Castaman, A. Dal Belin Peruffo, E. Dini, A. Galletti, E. Barone, G. Gastaldi Fibrinogen Vicenza and Genova II: two new cases of congenital dysfibrinogenemia with isolated defect of fibrin monomer polymerization and inhibitory activity on normal coagulation. Thromb Haemost 57(3): 252-258, 1987. 24.F. Rodeghiero, G. Castaman, E. Di Bona, M. Ruggeri, E. Dini Successful pregnancy in a woman with congenital factor XIII deficiency treated with substitutive therapy. Report of a second case. Blut 5581): 45-48, 1987. 25.F. Rodeghiero, G. Castaman, G. Pesavento, F. Bonato, G. Muleo, C. Consarino Recurrent life-threatening epistaxis in a child with Bernard -Soulier syndrome controlled by bilateral ligation of external carotids and ethmoidal arteries. Acta Haematologica 77(3): 183-185, 1987. 26.F. Rodeghiero, G. Castaman Calibration of lyophilized standards for ristocetin cofactor activity of von Willebrand's factor (vWf) requires vWf -deficient plasma as diluent for dose-response curves. Thromb Haemost 58(4): 978-981, 1987 27.F. Rodeghiero, G. Castaman, E. Di Bona, M. Ruggeri, R. Lombardi, P. M. Mannucci Hyper-responsiveness to DDAVP for patients with with type I von Willebrand's disease and normal intra-platelet von Willebrand factor. Eur J Haematol 40(2): 163-167, 1988. 28.P. M. Mannucci, R. Lombardi, G. Castaman, J. Dent, A. Lattuada, F. Rodeghiero, T. S. Zimmerman Von Willebrand disease "Vicenza" with larger than normal ("super-normal") von Willebrand factor multimers in plasma. Blood 71 (1): 65-70, 1988. 29.F. Rodeghiero, G. Castaman, M. Vespignani, E. Dini, M. Bertazzoni Erythema multiforme after intravenous immunoglobulin. Blut 56(3): 145, 1988. 30.F. Rodeghiero, G. Castaman, R. Lombardi, P. M. Mannucci Von Willebrand factor abnormalities in two patients with uraemia. Lancet 2, 1016-1017, ottobre1988. 31.Gringeri, P. M. Mannucci and the Medical-Scientific Committee of the Fondazione dell'Emofilia (Milano). National Survey of human immunodeficiency virus infection in italian hemophiliacs: 1983-1987. Ric Clin Lab 18 (4): 275-280, 1988. 32.G. Castaman, F. Rodeghiero, E. Di Bona, M. Ruggeri Clinical effectiveness of desmopressin in a case of acquired von Willebrand's syndrome associated with benign monoclonal gammopathy. Blut 58(4): 211-213, 1989 33.Tripodi, P. M. Mannucci and the ad hoc Study Group A survey of inherited thrombotic syndromes in Italy. Res Clin Lab 19(1): 67-74, 1989

15/12/2020 European Medicine Agency Page 4/46 34.G. Castaman, F. Rodeghiero, A. D'Emilio, E. Dini Juvenile essential thrombocythemia: really a benign disease? Haematologica 74(4): 416, 1989 35.G. Castaman, M. Ruggeri, F. Rodeghiero Increased proteolysis of antithrombin III Vicenza during disseminated intravascular coagulation in acute leukemia. Thromb Res 55: 661-664, 1989 36.F. Rodeghiero, G. Castaman, G. Soffiati, R. Quaglio, S. Castronovo, S. Cortesi, E. Dini Clinical significance of fibrinopeptide A in acute lymphocytic and non-lymphocytic leukemia. Blut 59(2): 177-183, 1989 37.F. Rodeghiero, G. Castaman, A. Tosetto, E. Di Bona, M. Ruggeri Triton X present in platelet lysates critically affects von Willebrand factor ristocetin cofactor assay. Thromb Res 55: 521-526, 1989 38.F. Rodeghiero, G. Castaman, E. Di Bona, M. Ruggeri Consistency of responses to repeated DDAVP infusions in patients with von Willebrand's disease and hemophilia A. Blood 74 (6): 1997-2000, 1989 39.P. M. Mannucci, A. Lattuada, G. Castaman, R. Lombardi, M. L. Colibretti, N. Ciavarella, F. Rodeghiero Heterogeneous phenotypes of platelet and plasma von Willebrand factor in obligatory heterozygotes for severe von Willebrand's disease. Blood 74(7): 2433-2436, 1989 40.G. Castaman, M. Ruggeri, F. Rodeghiero A new Italian family with severe prekallikrein deficiency. DDAVP-induced fibrinolysis and coagulation changes in homozygous and heterozygous members. Ris Clin Lab 20 (4): 239-244, 1990 41.F. Rodeghiero, G. Castaman, E. Dini Fibrinopeptide A changes during remission induction treatment with l-asparaginase in acute lymphoblastic leukemia: evidence for activation of blood coagulation. Thromb Res 57(1): 31-38, 1990 42.P. D'Alessio, J. J. Zwaginga, H. C. DeBoer, A. Federici, F. Rodeghiero, G. Castaman, G. Mariani, P. M. Mannucci, P. G. De Groot, J. J. Sixma Platelet adhesion to collagen in subtypes of type I von Willebrand's disease is dependent on platelet von Willebrand factor. Thromb Haemost 64(2): 227-231, 1990 43.F. Rodeghiero, G. Castaman The von Willebrand factor. Ris Clin Lab 20 (2) : 143-153, 1990 44.S. Cortelazzo, P. Viero, G. Finazzi, A. D'Emilio, F. Rodeghiero, T. Barbui Incidence and risk factors for thrombotic complications in an historical cohort of 100 patients with essential thrombocythemia. J Clin Oncol 8 (3): 556-562, 1990 45.F. Rodeghiero, G. Castaman, A. Tosetto, A. Lattuada, P. M. Mannucci

15/12/2020 European Medicine Agency Page 5/46 Platelet von Willebrand factor assay:results using two methods for platelet lysis. Thromb Res 59(2): 259-267, 1990 46.F. Rodeghiero Clinical Use of DDAVP in Von Willebrand's disease. Hemophilia World 6 (2): 11-13, 1990 47.P. M. Mannucci, K. Schimpf, D. B. Brettler, N. Ciavarella, M. Colombo, F. Haschke, K. Lechner, J. Lusher, G. Weissbach and the International Study Group Low Risk for Hepatitis C in Hemophiliacs given a high-purity, Pasteurized Factor VIII Concentrate. Ann Intern Med, 113 (1): 27-32, 1990 48.F.Rodeghiero, G.Castaman, A.Tosetto Von Willebrand factor antigen is less sensitive than Ristocetin Cofactor for the diagnosis of type I von Willebrand Disease - Results based on an epidemiological investigation. Thromb Haemost 64(3): 349-352, 1990 49.G. Castaman, F. Rodeghiero, E. Dini Thrombotic complications during L-Asparaginase treatment for acute lymphocytic leukemia. Haematologica 75(6): 567-569, 1990 50.F. Rodeghiero, G. Avvisati, G. Castaman, T. Barbui, F. Mandelli Early Deaths and Anti-Hemorrhagic Treatments in Acute Promyelocytic Leukemia. A GIMEMA Retrospective Study in 268 Consecutive Patients. Blood 75(11): 2112-2117, 1990 51.F. Rodeghiero, G. Castaman, G. Avvisati, F. Mandelli,T. Barbui Treatment of DIC Associated with APL. Blood 76(11): 2418, 1990 52.F. Rodeghiero, A. Tosetto, E. Di Bona, G. Castaman Clinical Pharmacokinetics of a Placenta-Derived Factor XIII Concentrate in Type I and Type II Factor XIII Deficiency. American Journal of Hematology 36(1): 30-34, 1991 53.F. Rodeghiero, A. Tosetto Factor XIII Subunit B Deficiency (Correspondence). Br J Haematol 76 (2): 317, 1990 54.G. Finazzi, T. Barbui and the ad hoc Study Group A Retrospective Study on Oral Anticoagulant Prophylaxis in 103 Patients with Hereditary Thrombophilia and Thrombosis. Res Clin Lab 20(4): 245-252, 1990 55.R. Caso, D. A. Lane, E. A. Thompson, R. J. Olds, S. L. Thein, M. Panico, L.Blench, H.R.Morris, J.M. Freyssinet, M.Alach, F. Rodeghiero, G.Finazzi Antithrombin Vicenza, Ala 384 to pro (GCA to CCA) mutation,transforming the inhibitor into a substrate. Br J Haematol 77: 87-92, 1991 56.F. Rodeghiero, G. Castaman, M. Ruggeri, M. Cazzavillan, G. Ferracin, E. Dini Fibrinolytic studies in 13 unrelated families with factor XII deficiency. Haematologica 76 (1): 28-32, 1991

15/12/2020 European Medicine Agency Page 6/46 57.G. Castaman, F. Rodeghiero Case-Report:Acquired von Willebrand's syndrome: Clinical responses to vasopressin analogue and Haemate P. Biomedical Progress 4 (3): 46-47, 1991

58.N. Schinaia, A. Ghirardini, F. Chiarotti, A. Gringeri. P. M. Mannucci and the Italian Group Progression to AIDS among Italian HIV-sieropositive haemophiliacs. AIDS 5: 385-391, 1991 59.M. Colombo, P. M. Mannucci, D. B. Brettler, A. Girolami, Lian, F. Rodeghiero, I.Scharrer, P. S. Smith, G. C. White Hepatocellular Carcinoma in Hemophilia. Am J Hematol 37(4): 243-246, 1991 60.F. Rodeghiero, G. Castaman, P. M. Mannucci Clinical Indications for Desmopressin (DDAVP) in Congenital and Acquired von Willebrand Disease. Blood Reviews 5(3): 155-161, 1991 61.G. Palareti, M. Maccaferri, C. Manotti, A. Tripodi, V. Chantarangkul, F. Rodeghiero, M. Ruggeri, P. M. Mannucci Fibrinogen Assays: a Collaborative Study of Six Different Methods. Clinical Chemistry 37(5): 714-719, 1991 62.G. Castaman, F. Rodeghiero, M. Ruggeri, E. Di Bona, E. Dini Long-lasting remission after high-dose intravenous immunoglobulins in a case of relapsing thrombotic thrombocytopenic purpura. Haematologica 76(6): 511-512, 1991 63.A. Tosetto, F. Rodeghiero Parallel line bioassay in a coagulation laboratory. A program for personal computer use and an example application to Ristocetin Cofactor assay. Clin lab Haemat 13(4): 371-377, 1991 64.G. Castaman, F. Rodeghiero, A. Lattuada, P. M. Mannucci Subunit Composition of Plasma von Willebrand Factor (vWF) inTwo Uremic Patients with Acquired vWF Abnormalities. Thromb Haemost 67(1): 183, 1992 65.G. Castaman, F. Rodeghiero, A. Lattuada, P. M. Mannucci A new variant of von willebrand disease (type II I) with anormal degree of proteolytic cleavage of von Willebrand factor. Thromb Res 65(3): 343-351, 1992 66.P. A. d'Alessio, G. Castaman, F. Rodeghiero, H. C. de Boer, A. B. Federici, P. M. Mannucci, G. de Groot, J. J. Sixma, J. J. Zwaginga. In vivo experiments indicate that relatively high platelet deposition in von Willebrand's disease 'Vicenza is caused by normal platelet-vWF levels rather than by high vWF-multimers in plasma. Thromb Res 65: 221-228, 1992 67.F. Rodeghiero, G. Castaman, M. Ruggeri, A. Tosetto Thrombosis in Subjects with Homozygous and Heterozygous Factor XII Deficiency. Thromb Haemost 67(5): 590-91, 1992

15/12/2020 European Medicine Agency Page 7/46 68.P. M. Mannucci for Medical-Scientific Committee, Fondazione dell'Emofilia Outbreak of hepatitis A among Italian patients with Haemophilia. Lancet 339(8796): 819, 1992 69.F. Rodeghiero, G. Castaman, M. Ruggeri, A. Tosetto The bleeding time in normal subjects is mainly determined by platelet von Willebrand Factor and is indipendent from blood group. Thromb Res 65(4): 605-615, 1992 70.F. Rodeghiero, G. Castaman The Coagulopathy of Acute Leukemia. Leukemia & Lymphoma 7 suppl. 2: 42-48, 1992 71.Cooperative Group for the Study of Platelet Immunology Retrospective analysis of platelet serology investigations performed in 1990 by twenty laboraties belonging to the "Cooperative group for the study of platelet immunology". Haematologica 77(1): 35-39, 1992 72.G. Castaman, M. Ruggeri, R. Girardello, F. Rodeghiero An unusually prolonged case of heparin-induced thrombocytopenia and disseminated intravascular coagulation. Haematologica 77 (2): 174-176, 1992 73.F. Rodeghiero, M. Frezzato, C. Schiavotto, G. Castaman, E. Dini Fulminant sepsis in adults splenectomized for idiopathic thrombocytopenic purpura. Haematologica 77(3): 253-256, 1992 74.F. Rodeghiero, C. Schiavotto, G. Castaman, M. Vespignani, M. Ruggeri, E. Dini A follow-up study of 49 adult patients with idiopathic thrombocytopenic purpura treated with high-dose immunoglobulins and anti-D immunoglobulins. Haematologica 77(3): 248-252, 1992 75.P. M. Mannucci, P. M. Tenconi, G. Castaman, F. Rodeghiero Comparison of four virus-inactivated plasma concentrates for treatment of severe von Willebrand disease: a cross-over randomized trial. Blood 79 (12): 3130-3137, 1992 76.G. Castaman, M. Ruggeri, F. Rodeghiero A new Case of Passovoy Defect. Am J Hematol 41 (1): 66, 1992 77.B. Casali, F. Rodeghiero, A. Tosetto, B. Palmieri, R. Immovilli, C. Ghedini, P. Rivasi Fibrin glue from single-donation autologous plasmapheresis. Transfusion 32(7): 641-643, 1992 78.L. Gugliotta, M. G. Mazzucconi, G. Leone, M. Mattioli Belmonte, Defazio, L. Annino, S. Tura, F. Mandelli and the GIMEMA Group Incidence of thrombotic complications in adult patients with acute lymphoblastic leukaemia receiving L-asparaginase during induction therapy: A retrospective study Eur J Haematol 49(2): 63-66, 1992 79.G. Castaman, A. Tosetto, F. Rodeghiero Effectiveness of High-Dose Intravenous Immunoglobulin in a Case of Acquired von Willebrand Syndrome With Chronic Melena Not Responsive to Desmopressin and Factor VIII Concentrate. Am J Hematol 41(2): 132-136, 1992

15/12/2020 European Medicine Agency Page 8/46 80.G. Castaman, M. Ruggeri, F. Rodeghiero EDTA-Dependent Pseudothrombocytopenia (Letters and Correspondence). Am J Hematol 41(3): 226, 1992 81.G. Castaman, F. Rodeghiero Failure of DDAVP to shorten the prolonged bleeding time of two patients with congenital afibrinogenemia. Thromb Res 68(3): 309-315, 1992 82.F. Rodeghiero, G. Castaman, D. Meyer, P. M. Mannucci Replacement Therapy with Virus-Inactivated Plasma Concentrates in von Willebrand Disease. Vox sang 62(4): 193-199, 1992 83.G. Castaman, M. Ruggeri, F. Burei, F. Rodeghiero High Levels of histidine-rich glycoprotein and thrombotic diathesis. Report of two unrelated families. Thromb Res 69(3): 297-305, 1993 84.F. Rodeghiero, G. Castaman, L. Gugliotta, M. Mattioli-Belmonte, A. Falanga, B. Bottasso,T. Barbui, P. M. Mannucci Supranormal antithrombin III levels induced by concentrate administration are ineffective in quenching thrombin generation in acute promyelocytic leukemia. Tromb Res 69(4): 377-385, 1993 85.G. Castaman, F. Rodeghiero, A. Lattuada, P. M. Mannucci Desmopressin-Induced Thrombocytopenia in Type I Platelet Discordant von Willebrand Disease. Am J Hem 43(1): 5-9, 1993 86.G. Marchetti, P. Patracchini, D. Gemmati, G. Castaman, F. Rodeghiero, A. Wacey, D. N. Cooper, E. G. D. Tuddenham, F. Bernardi Symptomatic type II protein C deficiency caused by a missense mutation (Gly 381_1ASer) in the substrate-binding pocket. Br J Haem 84(2): 285-289, 1993 87.F. Rodeghiero, A. Tosetto Some remarks on the epidemiology of thrombotic disorders Tromb Haemost 69 (5): 527-529, 1993 88.G. Castaman, M. Ruggeri, F. Rodeghiero Histidine-Rich Glycoprotein changes during pregnancy in a woman with genetically transmitted high levels of the protein and thrombotic diathesis. Thromb Res 69(3): 329-332, 1993 89.Ghirardini, F. Chiarotti, N. Schinaia and the Gruppo Italiano Coagulopatie Congenite Testing practices and spread of HIV among sexual partners of HIV-positive haemophiliacs in Italy. AIDS 7 :573-577, 1993 90.M. Morfini, P. M. Mannucci, P. M. Tenconi, G. Longo, M. G. Mazzucconi, F. Rodeghiero, N. Ciavarella, V. De Rosa, A. Arter Pharmacokinetics of monoclonally-purified and recombinant factor VIII in patients with severe von Willebrand disease. Tromb Haemost 70 (2): 270-272, 1993 91.G. Castaman, F. Rodeghiero, A. Lattuada, G. La Greca, P. M. Mannucci

15/12/2020 European Medicine Agency Page 9/46 Multimeric Pattern of Plasma and Platelet von Willebrand Factor is Normal in Uremic Patients. Am J Hem 44(4): 266-269, 1993 92.A. Falanga, F. A. Ofosu, S. Cortelazzo, F. Delaini, R. Consonni, R. Caccia, S. Longatti, D. Maran, F. Rodeghiero, E. Pogliani, A. Marassi, A. D'Angelo, T. Barbui Preliminary study to identify cancer patients at high risk of venous thrombosis following major surgery. Br J Haem, 85(4): 745-750, 1993 93.M. Ruggeri, G. Castaman, G. De Nardi, F. Rodeghiero Acute renal failure after high-dose intravenous immune globulin in a patient with idiopathic thrombocytopenic purpura. Haematologica 78 (5): 338-339, 1993 94.Italian Registry of Antiphospholipid antibodies (IR-APA) Thrombosis and thrombocytopenia in antiphospholipid syndrome (idiopathic and secondary to sle): first report from the italian registry. Haematologica 78 (5): 313-318,1993 95.M. Randi, E. Sacchi, G. Castaman, F. Rodeghiero, P .M. Mannucci The genetic defect of type I von Willebrand disease Vicenza is linked to the von Willebrand factor gene. Thromb Haemostas 69(2): 173-176, 1993 96.G. Castaman, F. Rodeghiero Consistency of responses to separate desmopressin infusione in patients with storage pool disease and isolated prolonged bleeding time. Thromb Res 69(3): 407-412, 1993 97.M. Frezzato, M. Ruggeri, G. Castaman, F. Rodeghiero Polycythemia vera and essential thrombocythemia in young patients. Haematologica 78 suppl. 2 al n. 6: 11-17, 1993 98.M. Ruggeri, G. Castaman, F. Rodeghiero Is ticlopidine a safe alternative to aspirin for management of myeloproliferative disorders? Haematologica 78 suppl. 2 al n. 6: 18-21, 1993 99.C. Schiavotto, F. Rodeghiero Twenty years experience with treatment of idiopathic thrombocytopenic purpura in a single department: results in 490 cases. Haematologica 78 suppl. 2 al n. 6: 22-8, 1993 100.C. Schiavotto, G. Castaman, F. Rodeghiero Treatment of idiopathic thrombocytopenic purpura (ITP) in patients with refractoriness to or with contraindication for corticosteroids and/or splenectomy with immunosuppressive therapy and danazol. Haematologica 78 suppl. 2 al n. 6: 29-34, 1993 101.C. Schiavotto, M. Ruggeri, F. Rodeghiero Adverse reactions after high-dose intravenous immunoglobulin:incidence in 83 patients treated for idiopathic thrombocytopenic purpura (ITP) and review of literature. Haematologica 78 suppl. 2 al n. 6: 35-40, 1993

15/12/2020 European Medicine Agency Page 10/46 102.A.Tosetto, M. Ruggeri, C. Schiavotto, G. Pellizzari, F. Rodeghiero The clinical significance of the antiplatelet antobody test based on results for 265 thrombocytopenic patients. Haematologica 78 suppl. 2 al n. 6: 41-46, 1993 103.M. Ruggeri, M. Milan, G. La Greca, G. Castaman, F. Rodeghiero Adult patients with nephrotic syndrome: really at high risk for deep venous thromboembolism? Report of a series and review of the literature. Haematologica 78 suppl. 2 al n. 6: 47-51, 1993 104.A. Tosetto, G. Castaman, F. Rodeghiero Acquired plasma factor XIII deficiencies. Haematologica 78 suppl. 2 al n. 6: 5-10, 1993 105.G. Castaman, E. Galloni, A. V. Dri, F. Rodeghiero Fibrinogen survival and fibrinopeptide A in acute leucemia. Haematologica 78 suppl. 2 al n. 6: 52-56, 1993 106.G. Castaman, F. Rodeghiero Erwinia- and E. coli-derived L-asparaginase have similar effects on hemostasis. Pilot study in 10 patients with acute lymphoblastic leukemia. Haematologica 78 suppl. 2 al n. 6: 57-60, 1993 107.DEmilio, A. Montaldi, M. Stella, P. Celli, N. Guercini, L. Tremul, F. Rodeghiero Hematologic and cytogenetic analyses of 99 cases of de novo myelodysplastic syndromes. Haematologica 78 suppl. 2 al n. 6: 61-65, 1993 108.R. Raimondi, G. Pellizzari, F. Rodeghiero Single step immunophenotyping of acute leukemias not classifiable by syandard morphology and cytochemestry: a practical approach. Haematologica 78 suppl. 2 al n. 6: 66-72, 1993 109.M. Frezzato, G. Castaman, F. Rodeghiero Fulminant sepsis in adults splenectomized for Hodgkins disease. Haematologica 78 suppl 2 al n.6: 73-77, 1993 110.E. Fressinaud, A. Federici, G. Castaman, C. Rothschild, F. Rodeghiero, H. R. Baumgarten, P.M. Mannucci, D. Meyer The role of platelet von Willebrand factor in platelet adhesion and thrombus formation: a study of 34 patients with various subtypes of type I von Willebrand disease. Br J Haem 86(2): 327-332, 1994 111.V. De Stefano, G. Leone, S. Mastrangelo, D. A. Lane, A. Girolami, P. de Moerloose, G. Sas, U. Abigaard, Blajchman, F. Rodeghiero, B. Bizzi Microheterogeneity of antithrombin III: effect of single amino acid substitutions and relationship with functional abnormalities. Blood Coagulation and Fibrinolysis 5(1): 7-15, 1994 112.S. Bernardi, G. Castaman, R. Redaelli, M. Pinotti, B. Lunghi, F.Rodeghiero, G. Marchetti Topologically equivalent mutations causing dysfunctional coagulation factors VII (294Ala-Val) and X (334Ser-Pro). Human Molecular Genetics 3(7): 1175-1177, 1994 113.Ghirardini, N. Schinaia, F. Chiarotti, R. De Biasi, F. Rodeghiero, N. Binkin and the Italian group on congenital coagulation disorders

15/12/2020 European Medicine Agency Page 11/46 Epidemiology of hemophilia and of Hiv infection in Italy. J Clin Epidemiol 47 (11): 1297-1306, 1994 114.V. De Stefano, G. Leone, S. Mastrangelo, A. Tripodi, F. Rodeghiero, G. Castaman, T. Barbui, G. Finazzi, B. Bizzi, P. M. Mannucci Clinical manifestations and management of inherited thrombophilia: retrospective analysis and follow-up after diagnosis of 238 patients with congenital deficiency of antithrombin III, protein C, protein S. Thromb Haemost 72 (3): 352-358, 1994 115.F. Rodeghiero, G. Castaman The pathophysiology and treatment of hemorrhagic syndrome of acute promyelocytic leukemia. Leukemia 8 suppl. 2: S20-S26, 1994 116.G. Castaman, F. Rodeghiero Acquired transitory von Willebrand syndrome with ciprofloxacin. Lancet 343(8895): 492, 1994 117.G. Castaman, E. Di Bona, F. Rodeghiero Angiodysplasia and von Willebrands disease. Thromb Haemosts 71(4): 527-28, 1994 118.V. De Stefano, G. Leone, S. Mastrangelo, A. Tripodi, F. Rodeghiero, G. Castaman, T. Barbui, G. Finazzi, B. Bizzi, P. M. Mannucci Thrombosis during pregnancy and surgery in patients with congenital defieciency of antithrombin III, protein C, protein S. Thromb Haemosts 71(6): 799-800, 1994 119.A. Tosetto, E. Gatto, M. T. Masiero, F. Rodeghiero Parallel line bioassay or single-point calibration for the establishment of reference ranges. Thromb Res 75 (4): 479-483, 1994 120.G. Marchetti, G. Castaman, M. Pinotti, B. Lunghi, M. G. DiIasio, M. Ruggeri, F. Rodeghiero, F. Bernardi Molecular bases of CRM+ factor X deficiency: a frequent mutation (Ser334Pro) in the catalytic domain and a substitution (Glu102Lys) in the second EGF-like domain. Br J Haematol 90(4): 910-915, 1995 121.R. Centurioni, E. Bobbio-Pallavicini, C. Porta, F. Rodeghiero, L. Gugliotta, A. Billio, F. Tacconi, E. Ascari and the Italian Cooperative Group for TTP Treatment of thrombotic thrombocytopenic purpura with high-dose immunoglobulins. Results in 17 patients. Haematologica 80(1): 325-331, 1995 122.Poon, L. M. Aledort, K. Anderle, M. Kunschak, M. Morfini and the Factor IX Study Group Comparison of the recovery and half-life of a high-purity factor IX complex concentrate. Transfusion 35: 319-323, 1995 123.G. Castaman, J. C. J. Eikenboom, F. Rodeghiero, E. Briet, P. H. Reitsma A novel candidate mutation (Arg611-His) in type I "platelet discordant" von Willebrand's disease with desmopressin-induced thrombocytopenia. Br J Haematol 89(3): 656-658, 1995

15/12/2020 European Medicine Agency Page 12/46 124.G. Castaman, A. Lattuada, P. M. Mannucci, F. Rodeghiero Factor VIII: C increases after desmopressin in a subgroup of patients with autosomal recessive severe von Willebrand disease. Br J Haematol 89(1): 147-151, 1995 125.C. Schiavotto, M. Ruggeri, F. Rodeghiero Failure of repeated courses of high-dose intravenous immunoglobulin to induce stable remission in patients with chronic idiopathic thrombocytopenic purpura. Ann Hematol 70(2): 89-90, 1995 126.A. Tosetto, F. Rodeghiero, E. Gatto, C. Manotti, T. Poli An acquired hemorrhagic disorder of fibrin crosslinking due to IgG antibodies to FXIII, successfully treted with FXIII replacement and cyclophosphamide. Am J Hematol 48(1): 34-39, 1995 127.G. Castaman, F. Rodeghiero No influence of blood group on the responsiveness to desmopressin in type I "platelet normal" von Willebrand's disease. Thromb Haemost 73 (3): 551-552, 1995 128.G. Castaman, A. Lattuada, M. Ruggeri, A. Tosetto, P. M. Mannucci, F. Rodeghiero Platelet von Willebrand factor abnormalities in myeloproliferative syndromes. Am J Hematol 49(4): 289-293, 1995 129.G. Castaman, M. Ruggeri, F. Rodeghiero Congenital afibrinogenemia: successful prevention of recurrent hemoperitoneum during ovulation by oral contraceptive. Am J Haematol 49(4): 363-4, 1995 130.G. Castaman, F. Rodeghiero Current management of von Willebrand's disease. Drugs 50(4): 602-614: 1995 131.A. Tosetto, M. Frezzato, F. Rodeghiero Family history and inherited thrombophilia. Br J Haematol 89: 227-228, 1995 132.G. Castaman, A. Lattuada, P. M. Mannucci, F. Rodeghiero Characterization of two cases of acquired transitory von Willebrand syndrome with ciprofloxacin: evidence for heightened proteolysis of von Willebrand factor. Am J Hematol 49(1): 83-86, 1995 133.S. Cortelazzo, G. Finazzi, M. Ruggeri, O. Vestri, M. Galli, F. Rodeghiero, T. Barbui Hydroxyurea for patients with essential thrombocythemia and a high risk of thrombosis. N Engl J Med 332 (17): 1132-1136, 1995 134.Arcieri, M.Puopolo, F.Baudo, F.Chiarotti, V.De Rosa, N.Schinaia, P.G.Mori, A.Ghirardini and the Italian Group of Congenital Coagulopathies The impact of antiviral therapy with zidovudine: a retrospective study on Hiv-positive hemophiliacs in Italy. Haematologica 80 (1): 25-30, 1995 135.G. Castaman, F. Rodeghiero Platelet ristocetin cofactor (ricof) in patients with type I "platelet normal" von Willebrand disease

15/12/2020 European Medicine Agency Page 13/46 correlates with plasmaricof peak after desmopressin infusion. Thromb Res 77 (4): 383-384, 1995 136.Ghirardini, M. Puopolo, G. Rossetti, G. Mancuso, L. Perugini, G. Piseddu and F. Chiarotti for the Italian Group on Congenital Coagulopathies Survival after AIDS among Italian haemophiliacs with HIV infection. AIDS 9 (12): 1351-1356, 1995 137.G. Castaman, F. Rodeghiero, P. M. Mannucci On the discrepant post-DDAVP increase of FVIII:C and von Willebrand factor in some patients with severe von Willebrand's disease. Br J Haematol 91: 511-520, 1995 138.A. Tosetto, F. Rodeghiero Diagnosis of APC Resistance in patients on oral anticoagulant therapy. Thromb and Haem 73 (4): 732-733, 1995 139.G. Castaman, Li Yu-Feng, F.Rodeghiero A bleeding disorder characterised by isolated deficiency of platelet microvesicle generation. Lancet 347(9002): 700-701, 1996 140.T. Izumi, T. Hashiguchi, G. Castaman, A. Tosetto, F. Rodeghiero, A. Girolami, A. Ichinose Type I Factor XIII deficiency is caused by a genetic defect of its b subunit: insertion of triplet AAC in Exon III leads to premature termination in the second Sushi domain. Blood 87 (7): 2769-2774, 1996 141.G. Castaman, F. Rodeghiero Desmopressin and type II B von Willebrand disease. Hemophilia 2: 73-77, 1996 142.M. Frezzato, A. Tosetto, F. Rodeghiero Validated questionnaire for the identification of previous personal or familial venous thromboembolism. Am J Epidemiol 143 (12): 1257-1265, 1996 143.L. Trentin, A. Cerutti, R. Zambello, R. Sancetta, C. Tassinari, M. Facco, F. Adami, F. Rodeghiero, C. Agostini, G. Semenzato Interleukin-15 promotes the growth of leukemic cells of patients with B-cell chronic l lymphoproliferative disorders. Blood 87 (8): 3327-3335, 1996 144.F. Rodeghiero, G. Castaman Successful surgical treatment of cerebral tumor and clearing of subsequent inhibitor in severe hemophilia A Review in "International Monitor on Hemophilia": 1: 20-21, 1996 145.G. Castaman, F. Rodeghiero, J. Eikenboom Desmopressin-induced thrombocytopenia in von Willebrand Disease patients with the Arg611HIs mutation in the A1 domain of von Willebrand Factor. Blood 87 (7): 3061, 1996 146.M. Frezzato, F. Rodeghiero Pregnancy in women with essential thrombocythaemia. Br J Haematol 93(4): 977, 1996

15/12/2020 European Medicine Agency Page 14/46 147.G. Castaman, M. Ruggeri, F. Rodeghiero Clinical usefulness of desmopressin for prevention of surgical bleeding in patients with symptomatic heterozygous factor XI deficiency. Br J Haematol 94(1): 168-170, 1996 148.R. Bassan, E. Di Bona, T. Lerede, E. Pogliani, G. Rossi, A. D'Emilio, M. Buelli, A. Rambaldi, P. Viero, F. Rodeghiero, T. Barbui Age-adapted moderate-dose induction and flexible outpatient postremission therapy for elderly patients with acute lymphoblastic leukemia. Leukemia and Lymphoma 22: 295-301, 1996 149.F. Bernardi, G. Castaman, M. Pinotti, P. Ferraresi, M. G. Di Iasio, B. Lunghi, F. Rodeghiero, G. Marchetti Mutation pattern in clinically asymptomatic coagulation factor VII deficiency. Human Mutation 88(2): 108-115, 1996 150.F. Rodeghiero, A. Tosetto The VITA Project: population-based distribution of protein C, Antithrombin III, Heparin-cofactor II and Plasminogen-relationship with physiological variables and establishment of reference ranges. Thrombosis and Haemostasis 76(2): 226-233, 1996 151.G. Castaman, M. Ruggeri, A. Tosetto, E. Missiaglia, F. Rodeghiero Thrombosis in patients with heterozygous and homozygous factor XII deficiency is not explained by the associated presence of Factor V Leiden Thrombosis ad Haemostasis 76(2): 275, 1996 152.C. Schiavotto, M. Ruggeri, G. Castaman, F. Rodeghiero High-dose dexamethasone in adult refractory idiopathic thrombocytopenic purpura Br J Haematol 93(2): 491-492, 1996 153.F. Rodeghiero, G. Castaman, P. M. Mannucci Prospective multicenter study on subcutaneous concentrated desmopressin for home treatment of patients with von Willebrand Disease and mild or moderatate Hemophilia A. Thromb Haemost 76 (5): 692-696, 1996 154.F. Rodeghiero, G. Castaman In vitro ad in vivo properties of plasma-derived factor IX concentrates. Review in "International Monitor on Hemophilia"4(2): 14-16, 1996 155.Di Bona, A. Montaldi, N. Guercini, V. Rossi, A. Luciano, A. Biondi, F. Rodeghiero A (15;17) traslocation not associated with acute promyelocytic leukaemia. Br J Haematol 95(4): 706-709, 1996 156.G. Castaman, M. Ruggeri, A. Tosetto, F. Bernardi, F. Rodeghiero The Ser460Pro substitution of the protein S (PS) gene is rare in Italian patients with type IIa PS deficiency. Blood 88 (9): 3666-3667, 1996 157.T. Lerede, R. Bassan, A. Rossi, E. Di Bona, G. Rossi, E. M. Pogliani, T. Motta, V. Torri, M. Buelli, B. Comotti, P. Viero, A. Rambaldi, S. Cortellazzo, F. Rodeghiero, T. Barbui Therapeutic impact of adult-type acute lymphoblastic leukemia regimens in B-cell/L3 acute leukemia and advanced-staf large Burkitts lymphoma. Haematologica 81 (5): 442-449, 1996

15/12/2020 European Medicine Agency Page 15/46 158.R.Seitz, F. Duckert, S. Lopaciuk, L. Muszbek, F. Rodeghiero, U. Seligsohn ETRO Working Party on factor XIII questionnaire on congenital factor XIII deficiency in Europe:status and perspective Seminars Thromb Hemost 22(5): 415-18, 1996 .

159.G. Castaman, Li Yu-Feng, E. Battistin, F. Rodeghiero Characterization of a novel bleeding disorders with isolated prolonged bleeding time and deficiency of platelet microvesicle generation. Br J Haematol 96(3): 458-463, 1997 160.M. Ruggeri, G. Castaman, A. Tosetto, F. Rodeghiero Low prevalence of thrombophilic coagulation defects in patients with deep vein thombosis of the upper limbs. Blood Coagulation and Fibrinolysis 8: 191-194, 1997 161.Gruppo Italiano Studio Policitemia (GISP) Low-dose aspirin in polycythaemia vera: a pilot study. Br J Haematol 97: 453-456, 1997 162.GITMO (Gruppo Italiano Trapianti di Midollo Osseo) Mobilization and collection of PBCS in healthy donors: a retrospective analysis of the italian bone marrow transplantation group (GITMO). Haematologica 82 (1): 47-52, 1997 163.A. Tosetto, E. Gatto, F. Rodeghiero Influence of cholesterol, triglycerides and fibrinogen on AT PC, HC-II and plasminogen measurement. Thromb Haemost 77(4): 804, 1997 164.E. Di Bona, M. Schiavoni, G. Castaman, N. Ciavarella, F. Rodeghiero Acquired haemophilia: experience of two Italian centres with new cases. Haemophilia 3(3): 183-188, 1997 165.A. Tosetto, E. Missiaglia, M. Frezzato, F. Rodeghiero The VITA Project: C667 mutation in the methylene-tetrahydrofolate reductase gene and risk of venous thromboembolism. British Journal of Hematology 97(4): 804-806, 1997 166.A. Tosetto, E. Missiaglia, E. Gatto, F. Rodeghiero The VITA Project: phenotypic resistance to activated protein C and FV Leiden mutation in the general population. Thrombosis and Haemostasis 78: 859-863, 1997 167.F. Rodeghiero, A. Tosetto The epidemiology of inherited thrombophilia: the VITA project. Thrombosis and Haemostasis 78(1): 636-640, 1997 168.A. Tosetto, M. Ruggeri, G. Castaman, F. Rodeghiero Inherited abnormalities of blood coagulation in juvenile stroke. A case-control study. Blood Coagulation and Fibrinolysis 8(7): 397-402, 1997 169.Mandelli, D. Diverio, G. Avvisati, A. Luciano,T. Barbui, C. Bernasconi, G. Broccia, R. Cerri, M. Falda,G. Fioritoni, F. Leoni, V. Liso, M. C. Petti, F. Rodeghiero, G. Saglio, M. L. Vegna, G. Visani, U. Ehn, R. Willemze, P. Muus, P. G. Pellicci, A. Biondi, F. Lo Coco per GIMEMA e AIEOP

15/12/2020 European Medicine Agency Page 16/46 Molecular remission in PML/RAR-positive promyelocytic leukemia by combined all-trans retinoic acid and idarubicin (AIDA) therapy. Blood 90(3): 1014-1021, 1997 170.G. Castaman, E. Di Bona, C. Schiavotto, L. Trentin, A. D' Emilio, F. Rodeghiero Pilot Study on the safety and efficacy of desmopressin for the treatment or prevention of bleeding in patients with hematological malignancies. Haematologica 82(5): 584-587, 1997 171.G. Castaman, B. Lunghi, E. Missiaglia, F. Bernardi, F. Rodeghiero Phenotypic homozygous activated protein C resistance associated with compound heterozygosity for Arg506Gln (factor V Leiden) and His1299Arg substitutions in factor V. Br J Haematol 99(2): 257-261, 1997 172.M. Ruggeri, C. Schiavotto, G. Castaman, A. Tosetto, F. Rodeghiero Gestational thrombocytopenia: a prospective study. Haematologica 82(3): 341-342, 1997 173.R. Zambello, M. Facco, L. Trentin, R. Sancetta, C. Tassinari, A. Perin, A. Milani, G. Pizzolo, F. Rodeghiero, C. Agostini, R. Meazza, S. Ferrini, G. Semenzato Interlukin-15 triggersthe proliferation and cytotoxicity of granular lymphocytes inpatients with lymphoproliferative disease of granular lymphocytes. Blood 89 (1): 201-11, 1997 174.R. Vangelisti, O. Pagnacco, G. Ristagno, M. Ruggeri, A. Tosetto, G. Castaman, F. Rodeghiero Prevention of hemorrhage and dental treatment of patients with congenital or acquired coagulopathies. Minerva Stomatol 46 (11): 621-626, 1997 175.R. Bassan, T. Lerede, E. Di Bona, G. Rossi, E. Pogliani, A. Rambaldi. M. Buelli, P. Viero, F. Rodeghiero, T. Izzi, G. Corneo, T. Barbui Granulocyte colony-stimulating factor (G-CSF, filgrastim) after or during an intensive remission induction therapy for adult acute lymphoblastic leukaemia: effects, role of patient pretreatment characteristics, and costs. Leuk Lymphoma 26 (1-2): 153-161, 1997 176.Eikenboom, G. Castaman, H. L. Vos, R. M. Bertina, F. Rodeghiero Characterization of the genetic defects in recessive type 1 and type 3 von Willebrand disease patients of italian origin. Thromb Haemost 79(4): 709-717, 1998 177.G. Castaman, M. Ruggeri, A. Tosetto, F. Rodeghiero Low risk of venous thrombosis in two families with combined type 1 plasminogen deficiency and factor V R506Q mutation. Am J Hematol 57(4): 344-347, 1998 178.N. Takahashi, H. Tsukamoto, H. Umeyama, G. Castaman, F. Rodeghiero, A. Ichinose Molecular mechanisms of type II factor XIII deficiency: novel Gly562-Arg mutation and C-terminal truncation of the A subunit cause factor XIII deficiency as characterized in a mammalian expression system. Blood 91(8): 2830-2838, 1998 179.M. Ruggeri, G. Finazzi, A. Tosetto, S. Riva, F. Rodeghiero, T. Barbui

15/12/2020 European Medicine Agency Page 17/46 No treatment for low-risk thrombocythaemia: results from a prospective study. Br J Haematol 103(3): 772-777, 1998 180.A. Tosetto, F. Rodeghiero, I. Martinelli, V. De Stefano, E. Missiaglia, P. Chiusolo, P. M. Mannucci Additional genetic risk factors for venous thromboembolism in carriers of the factor V Leiden mutation. Br J Haematol 103(3): 871-876, 1998 181.R. Bassan, R. Raimondi, T. Lerede, A. DEmilio, M. Buerilli, G. Borleri, A. Personeni, P. Bellavita, F. Rodeghiero, T. Barbui Outcome assessment of age group-specific (+/- 50 years) post-remission consolidation with high-dose cytarabine or bone marrow autograft for adult acute myelogenous leukemia Haematologica 83 (7): 627-35, 1998 182.Italian Federation of Anticoagulation Clinics (FCSA) A guide to oral anticoagulant therapy International Journal on Haemostasis and Thrombosis Research. Haemostasis 28 suppl.1: 1-46, 1998 183.Gisslinger, F. Rodeghiero, M. Ruggeri, N. Heis-Vahidi Fard, C. Mannhalter, M. Papagiannopoulos, C. Rintelen, W. Lalouschek, P. Knöbl, K. Lechner, I. Pabinger. Homocysteine levels in polycythaemia vera and essential thrombocythaemia. Br J Hamatol 105(2): 551-555, 1999 184.F. Rodeghiero, A. Tosetto Activated protein C resistance and factor V Leiden mutation are independent risk factors for venous thromboembolism. Ann Intern Med 130(8): 643-650, 1999 185.G. Castaman, A. Tosetto, M. Ruggeri, F. Rodeghiero Pseudohomozygosity for activated protein C resistance is a risk factor for venous thrombosis. Br J Haematol 106(1): 232-236, 1999 186.G. Castaman, J. C. J. Eikenboom, R. Bertina, F. Rodeghiero Inconsistency of association between type 1 von Willebrand Disease phenotype

and genotype in families identified in an epidemiological investigation. Thromb Haemost 82(3): 1065-1070, 1999 187.L. Trentin, L. Imberti, R. Zambello, A. Sottini, R. Raimondi, M. Facco, S. Cazzavillan, E. Bonoldi, S. Signorini, A. Bacigalupo, G. Semenzato, F. Rodeghiero, D. Primi Detection of identical T-cell clonotype expansions in both the donor and recipient after allogeneic bone marrow transplantation. Br J Haematol 106(1): 119-127, 1999 188.E. Di Bona, F. Rodeghiero, B. Bruno, A. Gabbas, P. Foa, A. Locasciulli, C. Rosanelli, L. Camba, P. Saracco, A. Lippi, A. P. Iori, F. Posta, V. de Rossi, B. Comotto, P.Iacopino, C. Dufour, A. Bacigalupo for the Gruppo Italiano Trapianto di Midollo Osseo (GITMO) Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anaemia patients unresponsive to a first course of intensive immunosuppressive therapy. Br J Haematol 107(2): 330-334, 1999 189.A. Tosetto, E. Missiaglia, M. Frezzato, F. Rodeghiero The VITA project:Prothrombin G20210A mutation and venous thromboembolism in the general population.

15/12/2020 European Medicine Agency Page 18/46 Tromb Haemost 82(5): 1395-1398, 1999 190.M. Ruggeri, A. Tosetto, F. Rodeghiero, G. Finazzi, T. Barbui The management of Low-Risk and Intermediate -Risk patients with primary thrombocythaemia. Br J Haematol 106 (3): 834, 1999 191.F. Rodeghiero, A. Tosetto Risk factors for thromboembolism. Annals of Internal Medicine 131 (10): 790-791, 1999 192.E. Sadler, P. M. Mannucci, E. Berntorp, N. Bochkov, V. Boulyjenkov, D. Ginsburg, D. Mayer, I. Peake, F. Rodeghiero, A. Srivastava Impact , diagnosis and treatment of von Willebrand disease. Thromb Haemost 84(2): 160-174, 2000 193.G. Castaman, J. C. J. Eikenboom, A. Lattuada, P. M. Mannucci, F. Rodeghiero Heightened proteolysis of the von Willebrand factor subunit in patients with von Willebrand disease hemizygous or homozygous for the C2362F mutation. Br J Haematol 108: 188-190, 2000 194.E. Di Bona, G. Avvisati, G. Castaman, M. L. Vegna, V. De Sanctis, F. Rodeghiero, F.Mandelli for the Gruppo Italiano per le Malattie Ematologiche dellAdulto (GIMEMA) Early haemorrhagic morbidity and mortality during remission induction with or without all-trans retinoic acid in acute promyelocytic leukaemia. Br J Haematol 108(4): 689-695, 2000 195.G. Castaman, J. E. J. Eikenboom, E. Missiaglia, F. Rodeghiero Autosomal dominant type 1 von Willebrand disease due to G3639T mutation (C1130F) in exon 26 of von Willebrand factor gene: description of five Italian families and evidence for a founder effect. Br J Haematol 108(4): 876-879, 2000 196.G. Castaman, A. Tosetto, A. Cappellari, M. Ruggeri, F. Rodeghiero The A20210 allele in the prothrombin gene enhances the risk of venous thrombosis in carriers of inherited protein S deficinecy. Blood Coagulation and Fibrinolysis 11(4): 321-326, 2000 197.R. Schneppenheim, A. B. Federici, U. Budde, G. Castaman, E. Dewke, S. Krey, P. M. Mannucci, G. Riesen, F. Rodeghiero, B. Zieger, R. Zimmermann Von Willebrand disease type 2M Vicenza in Italian and German patients:Identification of the first candidate mutation (G3864A;R1205H) in 8 families. Tromb Haemost 83(1): 136-40, 2000 198.G. Finazzi, M. Ruggeri, F. Rodeghiero, T. Barbui Second malignancies in patients with essential thrombocythaemia trated with busulphan and hydroxyurea: long-term follow-up of a randomized clinical trial. British Journal of Haematology 110(3): 577-583, 2000 199.G. Castaman, J. C. J. Eikenboom, A. Lattuada, F. Rodeghiero Grossly abnormal proteolysis of von Willebrand Factor (VWF) in a patient heterozygous for a gene deletion and mutation in the Demerization area of VWF. Thromb Haemost 84(4): 729-730, 2000 200.Z. S. Rohatiner, R. Bassan, R. Raimondi, J. A. L. Amess, S. Arnott, A. Personen, F. Rodeghiero, T. Barbui, M. J. Bradburn, M. Carter & T. A. Lister

15/12/2020 European Medicine Agency Page 19/46 High-dose treatment with autologous bone marrow support as consolidation of first remission in younger patients with acute myelogenous leukaemia. Annals of Oncology 11(8): 1007-1015, 2000 201.G. Castaman, M. Ruggeri, A. Tosetto, F. Rodeghiero Heterogeneity of activated protein C resistance phenotype in subjects with compound heterozygosity for HR2 haplotype and FV Leiden mutation (R506Q) in factor V gene Thromb Haemost 84(2): 357-358, 2000 202.G. Castaman, J. C. J. Eikenboom, A. Contri, F. Rodeghiero Pregnancy in women with type 1 von Willebrand disease caused by heterozygosity for von Willebrand factor mutation C1130F. Thromb Haemost 84: 351-352, 2000 203.G. Castaman, E. Missiaglia, A. B. Federici, R. Schneppenheim, F. Rodeghiero An additional unique candidate mutation (G2470A; M740I) in the original families with von Willebrand disease type 2 M Vicenza and the G3864A (R1205H) mutation. Thromb Haemost 84(2): 350-351, 2000 204.B. Federici, J. H. Rand, P. Bucciarelli, U. Buddle, P. J. van Genderen, H. Mohri, D. Meyer, F.Rodeghiero, J.E. Sadler

Acquired von Willebrand syndrome: data from an international registry. Thromb Haemost 84(2): 345-9, 2000 205.M. A. Sanz, F. Lo Coco, G. Martin, G. Avvisati, C. Rayon, T. Barbui, J. Diaz-Mediavilla, G. Fioritoni, J. D. Gonzales, V. Liso, J. Esteve, F. Ferrara, P. Bolufer, C. Bernasconi, M. Gonzales, F. Rodeghiero, D. Colomer, M. C. Petti, J. M. Ribera and F. Mandelli for the Spanish PETHEMA and the Italian GIMEMA Groups Definition of relapse risk and role of nonanthrcycline drugs for consolidation in patients with acute promyelocitic leukemia: a joint study of the PETHEMA and GIMEMA groups. Blood 96 (4): 1247-1253, 2000 206.A. Tosetto, G. Castaman, A. Cappellari, F. Rodeghiero The VITA Project: Heritability of resistance to activated protein C. Thromb Haemost 84(4): 811-814, 2000 207.I. Giaretta, D. Madeo, R. Bonaguro, A. Cappellari, F. Rodeghiero, G. Palù A comparative evaluation of gene transfer into blood cells using the same retroviral backbone for independent expression of the EGFP and ^LNGFR marker genes Haematologica 85(7): 680-689, 2000 208.M. Ruggeri, G. Castaman, A. Tosetto, F. Rodeghiero Two families with combined homozygous/heterozygous factor V Leiden mutation and hetorozygous G20210A factor II variant. Blood Coagul Fibrinolysis 11 (8): 783-784, 2000 209.M. Ruggeri. A. Tosetto, G. Castaman, F. Rodeghiero Congenital absence of the inferior vena cava: a rare risk factor for idiopathic deep-vein thrombosis. The Lancet 357 (9254): 441, 2001

15/12/2020 European Medicine Agency Page 20/46 210.F. Rodeghiero Cellular mechanism for DDAVP-dependent release of von Willebrand factor. The International Monitor on Hemophilia Reviews of current key literature on hemophilia 9(1): 35, 2001 211.M. Ruggeri, A. Tosetto, G. Castaman, F. Rodeghiero Pulmonary embolism after pregnancy in a patient with polycythemia vera American Journal of Hematology 67(3): 216-217, 2001 212.F. Rodeghiero, G. Castaman Congenital von Willebrand disease type 1: definition, pheotypes, clinical and laboratory assessment. Best Practice & Research Clinical Haematology 14(2): 321-335, 2001 213.G. Castaman, A. Tosetto, M. Simioni, M. Ruggeri, D. Madeo, F. Rodeghiero Phenotypic APC resistance in carriers of the A20210 prothrombin mutation is associated with an increased risk of venous thrombosis Thromb Haemost 86(3): 804-808, 2001 214.C. Goodeve, J. C. J. Eikenboom, D. Ginsburg, L. Hilbert, C. Mazurier, I. R. Peake, J. E. Sadler, F. Rodeghiero A standard nomenclature for von Willebrand factor gene mutations and polimorphirsms. Thromb.Haemost 85(5): 929-931, 2001 215.C. Mazurier, F. Rodeghiero Recommended abbreviations for von Willebrand factor and its activities. Thromb Haemost 86(2): 712, 2001 216.M. Lazzarino, A. Corso, L. Barbarano, E. P. Alessandrino, R. Cairoli, G. Pinotti, G. Ucci, L. Uziel, F. Rodeghiero, S. Fava, D. Ferrari, M. Fumanò, G. Frigerio, L. Isa, A. Luraschi, S. Montanara, S. Morandi, D. Perego, A. Santagostino, M. Savarè, A. Vismara, E. Morra DCEP (Dexamethasone, cyclophosphamide, etoposide, and cisplatin) is an effective regimen for peripheral blood stem cell collection in multiple myeloma. Bone Marrow Transplant 28(9): 835-839, 2001 217.F. Rodeghiero The Coagulopathy of Acute Promyelocytic Leucemia Haemostasis 31 suppl 1 : 49-51, 2001 218.F. Rodeghiero Heparins and tumors Haematologica 87 suppl 2 al n. 5: 37-44, 2002 219.M. Ruggeri, H. Gisslinger, A. Tosetto, C. Rintelen, C. Mannhalter, I. Pabinger, N. Heis, G. Castaman, E. Missiaglia, K. Lechner, F. Rodeghiero Factor V Leiden Mutation Carriership and Venous Thromboembolism in Polythemia Vera and Essential Trombocythemia American Journal of Hematology 71(4): 1-6, 2002 220.E. Di Bona, R. Sartori, R. Zambello, N. Guercini, D. Madeo, F. Rodeghiero Prognostic significance of CD56 antigen expression in acute myeloid leukemia. Haematologica 87 (3): 250-56, 2002 221.F. Rodeghiero

15/12/2020 European Medicine Agency Page 21/46 Von Willebrand disease: still an intriguing disorder in the era of molecular medicine. Haemophilia 8(3): 292-300, 2002 222.G. Castaman, E. Novella, E. Castiglia, J. C. J. Eikenboom, F. Rodeghiero A novel family with recessive von Willebrand disease due to compound heterozygosity for a splice site mutation and a missense mutation in the von Willebrand factor gene. Thromb Research 105 (2): 135-38, 2002 223.G. Castaman, E. Novella, L. Contino, M. Demicheli, F. Rodeghiero First Italian families with homozygous R854Q type 2 N von Willebrand disease. Thromb Haemost. Vol.88 (3): 534-5, 2002 224.G. Castaman, F. Rodeghiero, P. M. Mannucci The elusive pathogenesis of von Willebrand disease Vicenza. Blood 99 (11): 4243-4, 2002 225.E. M. Faioni, F. Franchi, G. Castaman, E. Biguzzi, F. Rodeghiero Mutations in the thrombomodulin gene are rare in patients with severe thrombophilia. Br J Haematol 118 (2): 595-9, 2002 226.E. Novella, I. Giaretta, F. Elice, D. Madeo, A. Piccin, G. Castaman, F. Rodeghiero Fluorescent polymerase chain reaction and capillary electrophoresis for IgH rearrangement and minimal residual disease evaluation in multiple myeloma. Haematologica 87 (11): 1157-64, 2002 227.G. Castaman, A. B. Federici, F. Rodeghiero, P. M. Mannucci von Willebrands disease in the year 2003: toward the complete identification of gene defects for correct diagnosis and treatment. Haematologica 88 (01): 94-108, 2003 228.F. Rodeghiero, C. Borghero, M. Ruggeri Idiopathic thrombocytopenic purpura: an old disease revisited in the era of evidence-based medicine. Haematologica 88 (10): 1081-87 , Oct. 2003 229.G. Finazzi, M. Ruggeri, F. Rodeghiero, T. Barbui Efficacy and safety of long-term use of hydroxyurea in young patients with essential thrombocythemia and a high risk of thrombosis. Blood 101 (9): 3749, 2003 230.F. Rodeghiero, C. Borghero, M. Ruggeri Management of ITP. 8th Congress of EHA, Lyon June 2003 The Hematology Journal 4 (suppl.3): 76-83, 2003 231.D. Madeo, A. Cappellari, G. Castaman, R. Raimondi, F. Rodeghiero Multiplex amplification and fluorimetric detection of short tandem repeats for mixed chimerism after bone marrow transplant. Leukemia & Lymphoma 44 (8): 1395-1404, 2003 232.A. Tosetto, M. Frezzato and F. Rodeghiero Prevalence and risk factors of non-fatal venous thromboembolism in the active population of the VITA Project. J Thromb Haemost 1 (8): 1724-29, 2003 233.F. Rodeghiero, F. Elice Ifosfamide in hematological malignancies of adults. Oncology 65 (suppl.2): 85-93, 2003

15/12/2020 European Medicine Agency Page 22/46 234.M. Ruggeri, A. Tosetto, M. Frezzato, F. Rodeghiero The rate of progression to polycythemia vera or essential thrombocythemia in patients with erythrocytosis or thrombocytosis. Ann Intern Med 139 (6): 470-75, 2003 235.F. Rodeghiero, F. Elice Thalidomide and thrombosis. Phatophysiol Haemost Thromb 33 (suppl.1): 15-18, 2003 236.F. Mandelli, R. Latagliata, G. Avvisati, P. Fazi, F. Rodeghiero, F. Leoni, M. Gobbi, F. Nobile, E. Gallo et al. Treatment of elderly patients (>60 years) with newly diagnosed acute promyelocytic leukemia. Results of the Italian multicenter group GIMEMA with ATRA and idarubicin (AIDA) protocols. Leukemia 17(6): 1085-1090, 2003 237.V. Scurti, A. Di Matteo, E. Sasso, M. Nigro, T. Barbui, G. Tognoni, M. Romero, per il Gruppo di Lavoro (Vicenza: A. Dacomo, A. Bagaccin, F. Rodeghiero ) Luso del Rituximab nella pratica clinica. Giornale italiano di Farmacia clinica 17(4): 70-8, 2003 238.E. M. Faioni, G. Castaman, D. Asti, F. Lussana, F. Rodeghiero Association of factor V deficiency with factor V HR2. Haematologica 89 (2): 195-200, 2004 239.T. Barbui, G. Barosi, A. Grossi, L. Gugliotta, L. N. Liberato, M. Marchetti, M. G. Mazzucconi, F. Rodeghiero, S. Tura Practice guidelines for the therapy of essential thrombocythemia. A statement from the Italian Society of Hematology, the Italian Society of Experimental Hematology and the Italian Group for Bone Marrow Transplantation. Haematologica 89 (2): 215-232, 2004 240.E. Novella, D. Madeo, E. Albiero, S. Roberti, G. Castaman, F. Elice, F. Rodeghiero Effect of DCEP mobilizing regimen in in vivo purging of PBSC harvests in multiple myeloma. Leukemia & Lymphoma 45 (7): 1497-99, 2004 241.D. Gemmati, A. Ongaro, G. L. Scapoli, M. Della Porta, S. Tognazzo, M. L. Serino, E. Di Bona, F. Rodeghiero, G. Gilli, R. Reverberi, A. Caruso, M. Pasello, A. Pellati, M. De Mattei Common gene polymorphisms in the metabolic folate and methylation pathway and the risk of acute lymphoblastic leukemia and non-Hodgkins lymphoma in adults. Cancer Epidemiol Biomarkers Prev. 13(5): 787-794, 2004 242.A. Tosetto, M. Simioni, D. Madeo, F. Rodeghiero Intraindividual consistency of the activated protein C resistance phenotype. Br J Haematol. 126(3): 405-409, 2004 243.M. Galli, F. Elice, C. Crippa, B. Comotti, F. Rodeghiero, T. Barbui Recombinant human erythropoietin and the risk of thrombosis in patients receiving thalidomide for multiple myeloma. Haematologica 89 (9): 1141-42, 2004 244.R. Raimondi, M. Soli, T. Lamparelli, A. Bacigalupo, W. Arcese, M. Belloni, F. Rodeghiero, on behalf of the Gruppo Italiano Midollo Osseo (GITMO) ABO-incompatible bone marrow transplantation: a GITMO survey of current practice in Italy and comparison with the literature.

15/12/2020 European Medicine Agency Page 23/46 Bone Marrow Transpl. 34(4): 321-29, 2004 245.F. Rodeghiero The incidence of renal disease in patients with hemophilia. Review of Renal disease among males with haemophilia, by R. Kulkarni, Jm Soucie, B. Evatt; Hemophilia Surveillance System Project Investigators. Haemophilia 9(6): 703-710, 2003 The International Monitor on Hemophilia Vol.12 no.2: 16-17, October 2004 246.F. Rodeghiero, G. Castaman Treatment of von Willebrand disease. Semin Hematol 42(1): 29-35, 2005 247.G. Castaman, R. Ghiotto, A. Tosetto, F. Rodeghiero B 14 Arg ? Cys variant dysfibrinogen and its association with thrombosis. J Thromb Haemost 3(2): 409-10, 2005 248.G. Castaman, K. Bertoncello, M. Bernardi, F. Rodeghiero Pregnancy and delivery in patients with homozygous or heterozygous R854Q type 2N von Willebrand disease. J Thromb Haemost 3 (2): 391-92, 2005 249.G. Castaman, R. Ghiotto, S. Duga, F. Rodeghiero A novel fibrinogen chain mutation ( 239 Gln? His ) is the cause of dysfibrinogenemia Vicenza. J Thromb Haemost 3(3): 600-01, 2005 250.A. Tosetto, P. Prati, C. Baracchini, R. Manara, F. Rodeghiero Age-adjusted reference limits for carotid intima-media thickness as better indicator of vascular risk: population-based estimates from the VITA project. J Thromb Haemost 3(6): 1224-30, 2005 251.E. Di Bona, E. Pogliani, G. Rossi, T.Lerede, A. DEmilio, M. Vespignani, F. Rodeghiero, T. Barbui, R. Bassan Transplant-finalized salvage of adult acute lymphoblastic leukemia: results of a mitoxantrone-and methotrexate-based regimen in 36 patients. Leukemia & Lymphoma 46(6): 879-884, 2005 252.J.J. Goedert for the Second Multicenter Hemophilia Cohort Study Prevalence of conditions associated with human immunodeficiency and hepatitis virus infections among persons with haemophilia, 2001-2003 Haemophilia 11(5): 516-528, 2005 253.A. Cortelezzi, M. Moia, E.M. Pogliani, G. Agnelli, E. Bonizzoni, G. Gussoni, T. Barbui e P. M. Mannucci on behalf of the CATHEM Study Group Incidence of thrombotic complications in patients with haematological malignancies with central venous catheters: a prospective multicentre study. Br J Haematol 129(6): 811-817, 2005 254.The Fibrinogen Studies Collaboration Plasma fibrinogen level and the risk of major cardiovascular diseases and nonvascular mortality: an individual participant meta-analysis. Jama 294(14): 1799-1809, 2005 255.A. Srivastava, F. Rodeghiero Epidemiology of von Willebrand disease in developing countries. Semin Thromb and Haemost 31(5): 569-576, 2005 256.F. Rodeghiero, G. Castaman, A. Tosetto, J. Battle, F. Baudo, A. Cappelletti, P. Casana, N. De Bosch, J.C.J. Eikenboom, A.B.Federici, S. Lethagen, S. Linari and A. Srivastava The discriminant power of bleeding history for the diagnosis of type 1 von Willebrand disease: an international, multicenter study. J Thromb Haemost 3(12): 2619-2626, 2005 257.J. E. Sadler and F. Rodeghiero on behalf of the ISTH SSC subcommittee on von Willebrand factor

15/12/2020 European Medicine Agency Page 24/46 Provisional criteria for the diagnosis of VWD type 1. J Thromb Haemost 3(4): 775-777, 2005 258.G. Castaman, A. B. Federici, M. Bernardi, B. Moroni, K. Bertoncello, F. Rodeghiero Factor VIII and von Willebrand factor changes after desmopressin and during pregnancy in type 2M von Willebrand disease Vicenza: a prospective study comparing patients with single (R1205H) and double (R1205H-M7401) defect. J Thromb Haemost 4 (2): 357-360, 2006 259.A. Tosetto, F. Rodeghiero, G. Castaman, A. Goodeve, A.B. Federici, J. Battle, D. Meyer, E. Fressinaud, C. Mazurier, J. Goudemand, J. Eikenboom et al. A quantitative analysis of bleeding symptoms in type 1 von Willebrand disease: results from a multicenter European study (MCMDM-1 VWD). J Thromb Haemost 4 (4 ): 766-773, 2006 260.J. Eikenboom, V. Van Marion, H. Putter, A. Goodeve, F. Rodeghiero, G. Castaman, A.B. Federici, J. Battle, D. Meyer, C. Mazurier, J. Goudemand, et al. Linkage analysis in families diagnosed with type 1 von Willebrand disease in the European study, molecular and clinical markers for the diagnosis and management or type 1 VWD. J Thromb Haemost 4(4 ): 774-782, 2006 261.R. Cairoli, A. Beghini, G. Grillo, G. Nadali, F. Elice, C.B. Ripamonti, P. Colapietro, M. Nichelatti, L. Pezzetti, M. Lunghi, A. Cuneo, A. Viola, F. Ferrara, M. Lazzarino, F. Rodeghiero, G. Pizzolo, L. Larizza, E. Morra Prognostic impact of c-KIT mutations in core binding factor leukemias: an Italian retrospective study. Blood 107 (9): 3463-3468, 2006 262.F. Elice, R. Raimondi, A. Tosetto, A. DEmilio, E. Di Bona, A. Piccin, F. Rodeghiero Prolonged overall survival with second on-demand autologous transplant in multiple

myeloma. Am J Hematol 81(6): 426-431, 2006 263.D. Lillicrap, S.C. Nair, A. Srivastava, F. Rodeghiero, I. Pabinger, A.B. Federici Laboratory issues in bleeding disorders. Haemophilia 12 (suppl.3): 68-75, 2006 264.A. Palumbo, C. Rus, J.B. Zeldis, F. Rodeghiero, M. Boccadoro, for the Italian Multiple Myeloma Network, GIMEMA Enoxaparin or aspirin for the prevention of recurrent thromboembolism in newly diagnosed myeloma patients treated with melphalan and prednisone plus thalidomide or lenalidomide J Thromb Haemost 4 (8): 1842-45, 2006 265.M. Ruggeri, S. Fortuna, F. Rodeghiero Heterogeneity of terminology and clinical definitions in adult idiopathic thrombocytopenic purpura: a critical appraisal from literature analysis. Pediatric Blood & Cancer, Suppl. to Vol. 47 (5): 653-56, 2006 266.J.E. Sadler, U. Budde, J.C.J Eikenboom, E.J. Favaloro, F.G.H. Hill, L. Holmberg, J. Ingerslev, C. A. Lee, D. Lillicrap, P.M. Mannucci, C. Mazurier, D. Meyer, W.L. Nichols, M. Nishino I.R. Peake, F. Rodeghiero , R. Schneppenheim, et al. Update on the pathophysiology and classification of von Willebrand disease: a report of the Subcommittee on von Willebrand Factor. J Thromb Haemost 4 (10):2103-2114, 2006 267.G. Castaman, F. Rodeghiero, A. Tosetto, A. Cappelletti, F. Baudo, J.C.J. Eikenboom, A.B. Federici, S. Lethagen, S. Linari, J. Lusher, M. Nishino, P. Petrini, A. Srivastava, J.S.Ungerstedt Hemorrhagic symptoms and bleeding risk in obligatory carriers of type 3 von Willebrand disease: an international, multicenter study. J Thromb Haemost 4 (10): 2164-69, 2006 268.C. Visco, L. Arcaini, E. Brusamolino, S. Burcheri, A. Ambrosetti, M. Merli, E. Bonoldi, M. Chilosi, A. Viglio, M. Lazzarino, G. Pizzolo, F. Rodeghiero

15/12/2020 European Medicine Agency Page 25/46 Distinctive natural history in hepatitis C virus positive diffuse large B-cell lymphoma: analysis of 156 patients from northern Italy. Ann Oncology 17 (9): 1434-1440, 2006 269.A. Goodeve, J. Eikenboom, G. Castaman, F. Rodeghiero, AB Federici, J. Battle, D. Meyer, C. Mazurier, J. Goudemand, R. Schneppenheim, U. Budde, et al. Phenotype and genotype of a cohort of families historically diagnosed with type 1 von Willebrand disease in the European study, Molecular and Clinical Markers for the Diagnosis and Management of Type 1 von Willebrand Disease (MCMDM-1VWD) Blood 109 (1): 112-21, 2007 270.MG Mazzucconi, P. Fazi, S. Bernasconi, G. De Rossi, G. Leone, L. Gugliotta, N. Vianelli, G. Avvisati, F. Rodeghiero, A. Amendola, C. Baronci, C. Carbone, S. Quattrin, G. fioritoni, G. DAlfonso and F. Mandelli for the GIMEMA Thrombocytopenia Working Party Therapy with high-dose dexametasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience. Blood 109 (4): 1401-07, 2007 271.A. Tosetto, F. Rodeghiero, G. Castaman, M. Bernardi, K. Bertoncello, A. Goodeve, A.B. Federici, J. Battle, D. Meyer, C. Mazurier, J. Goudemand, J. Eikenboom, et al. Impact of plasma von Willebrand factor levels in the diagnosis of type 1 von Willebrand disease: results from a multicenter European study (MCMDM-1VWD). J Thromb Haemost 5 (4): 715-721, 2007 272.A. Tosetto, G. Castaman, F. Rodeghiero Assessing bleeding in von Willebrand disease with bleeding score. Blood Reviews 21(2): 89-97, 2007 273.G. Castaman, K. Bertoncello, M. Bernardi, J.C.J. Eikenboom, U.Budde, F. Rodeghiero Autosomal recessive von Willebrand disease associated with compound heterozygosity for a novel nonsense mutation (2908 Del C) and the missense mutation C2362F: definite evidence for the non-penetrance of the C2362F mutation. Am J Hematol 82(5): 376-80, 2007 274.G. Castaman, S.H. Giacomelli, R. Ghiotto, C. Boseggia, K. Pojani, A. Bulo, D. Madeo, F. Rodeghiero Spectrum of mutations in Albanian patients with haemophilia A: identification of ten novel mutations in the factor VIII gene. Haemophilia 13(3): 311-316, 2007 275.A. Piccin, R. Raimondi, S. Laspina, M. Marchi, F. Rodeghiero, U. Rovigatti Erythroleukaemia, diabetes insipidus and hypophyseal damage: two cases reports. Leukemia Research 31(8) : 1143-47, 2007 276.C. Visco, I. Giaretta, M. Ruggeri, D. Madeo, A. Tosetto, F. Rodeghiero Un-mutated IgVH in chronic lymphocytic leukemia is associated with a higher risk of immune thrombocytopenia. Leukemia 21(5): 1092-3, 2007 277.E. Albiero, D. Madeo, N. Bolli, I. Giaretta, E. Di Bona, M.F. Martelli, I. Nicoletti, F. Rodeghiero, B. Falini Identification and functional characterization of a cytoplasmic nucleophosmin leukaemic mutant generated by a novel exon-11 NPM1 mutation. Leukemia 21(5): 1099-103, 2007 278.F. Rodeghiero, A. Tosetto, G. Castaman How to estimate bleeding risk in mild bleeding disorders. J Thromb Haemost 5 (Suppl.1): 157-166, 2007 279.B. Falini, E. Albiero, N. Bolli, MF De Marco, D. Madeo, M. Martelli, I. Nicoletti, F. Rodeghiero Aberrant cytoplasmic expression of C-terminal-truncated NPM leukaemic mutant is dictated by

15/12/2020 European Medicine Agency Page 26/46 tryptophans loss and a new NES motif. Leukemia 21 (9): 2052-54, 2007 280.B. Falini, E. Albiero, N. Bolli, MF De Marco, D. Madeo, M. Martelli, I. Nicoletti, F. Rodeghiero Reply to Pitiot et al. (Reply to Aberrant cytoplasmic .....) Leukemia 21 (9): 2055-56, 2007 281.The Fibrinogen Studies Collaboration Association of plasma fibrinogen levels with established cardiovascular disease risk factors, inflammatory markers, and other characteristics: individual participant meta-analysis of 154,211 adults in 31 prospective studies: the fibrinogen studies collaboration. Am J Epidemiol 166(8): 867-79, 2007 282.The Emerging Risk Factors Collaboration The Emergin Risk Factors Collaboration: anlysis of individual data on lipid, inflammatory and other markers in over 1.1 million participants in 104 prospective studies of cardiovascular diseases. European J Epidemiology 22(12): 839-69, 2007 283.G. Castaman, S.H.Giacomelli, V. Ivaskevicius, V. Schroeder, H.P. Kohler, A. Dragani, C. Biasioli, J. Oldenburg, D. Madeo, F. Rodeghiero Molecular characterization of five Italian families with inherited severe factor XIII deficiency. Haemophilia 14(1): 96-102, 2008 284.M. Ruggeri, S. Fortuna, F. Rodeghiero Heterogeneity of terminology and clinical definitions in adult idiopathic thrombocytopenic purpura: a critical appraisal from a systematic review of the literature. Haematologica 93(1): 98-103, 2008 285.F. Rodeghiero Management of menorrhagia in women with inherited bleeding disorders: general principles and use of desmopressin. Haemophila, 14 (Suppl. 1): 21-30, 2008 286.M. Ruggeri, F. Rodeghiero, A. Tosetto, G. Castaman, F. Scognamiglio, G. Finazzi, F. Delaini, C. Micò, A.M. Vannucchi, E. Antonioli, V. De Stefano, T. Za, et al. Postsurgery outcomes in patients with polycythemia vera and essential thrombocythemia: a retrospective survey. Blood 111(2): 666-671, 2008 287.G. Castaman, S.H.Giacomelli, R. Ghiotto, K. Pojani, F. Rodeghiero First factor IX mutations in Albanian hemophilia B patients. Am J Haematol 83(1): 88-9, 2008 288.F. Rodeghiero, M. Ruggeri Is splenectomy still the gold standard for the treatment of chronic ITP? Am J Haematol 83(2): 91, 2008 289.C. Visco, M. Ruggeri, M. Laura Evangelista, R. Stasi, R. Zanotti, I. Giaretta, A. Ambrosetti, D. Madeo, G. Pizzolo, F. Rodeghiero Impact of immune thrombocytopenia on the clinical course of chronic lymphocytic leukemia. Blood, 111(3): 1110-6, 2008 290.F. Rodeghiero First line therapies for immune thrombocytopenic purpura: re-evaluating the need to treat. European J Haematol, 80 (Suppl.69): 19-26, 2008 291.V. De Stefano, T. Za, E. Rossi, A.M. Vannucchi, M. Ruggeri, E. Elli, C. Micò, A. Tiegni, R. R. Cacciola, C. Santoro, G. Gerli, N. Vianelli, P. Guglielmelli, L. Pieri, F. Scognamiglio, F. Rodeghiero, E.M. Pogliani, G. Finazzi, L. Gugliotta, R. Marchioli, G. Leone, T. Barbui Recurrent thrombosis in patients with polycythemia vera and essential thrombocythemia: incidence, risk factors, and effect of treatmens. Haematologica, 93(3): 372-380, 2008

15/12/2020 European Medicine Agency Page 27/46 292.F. Scognamiglio, C. Corso, D. Madeo, G. Castaman, C. Visco, C. Borghero, M. Ruggeri, F. Rodeghiero Flow cytometry in the diagnosis of drug-induced thrombocytopenia: two illustrative cases Am J Haematol, 83(4): 326-329, 2008 293.G. Castaman, S. Lethagen, AB Federici, A. Tosetto, A. Goodeve, U. Budde, J. Battle, D. Meyer, C. Mazurier, E. Fressinaud, J. Goudemand, J. Eikenboom, J. Ingerslev, R. Schneppenheim, Z. Vorlova, D. Habart, L. Holmberg, J. Pasi, F. Hill, I. Peake, F. Rodeghiero Response to desmopressin is influenced by the genotype and phenotype in type 1 von Willebrand (VWD): results from the European Study MCMDM-1VWD. Blood, 111(7): 3531-9, 2008 294.A. Tosetto, G. Castaman, F. Rodeghiero Evidencebased diagnosis of type 1 von Willebrand disease: a Bayes theorem approach. Blood, 111(8): 3998-4003, 2008 295.P. Sonneveld, R. Hajek, A. Nagler, A. Spencer, J. Bladé, T. Robak, SH Zhuang, Jl. Harousseau, RZ Orlowski; DOXIL-MMY-3001 Study Investigators Combined pegylated liposomal doxorubicin and bortezomib is highly effective in patients with recurrent or refractory multiple myeloma who received prior thalidomide/lenalidomide therapy. Cancer, 112(7): 1529-37, 2008 296.G. Castaman, M. Ruggeri, F. Rodeghiero Acquired transitory factor XI inhibitor after gynaecological surgery. Haemophilia, 14(3): 643-644, 2008 297.G. Castaman, S.H. Giacomelli, S. Duga, F. Rodeghiero Congenital hypofibrinogenemia associated with novel heterozygous fibrinogen B

and chain mutations. Haemophilia, 14(3): 630-633, 2008 298.A. Tosetto, G. Castaman, F. Rodeghiero Bleeding scores in inherited bleeding disorders: clinical or research tools? Haemophilia, 14(3): 415-422, 2008 299.S.L. Haberichter, G. Castaman, U. Budde, I. Peake, A. Goodeve, F. Rodeghiero, A.B. Federici, J. Batlle, D. Meyer, C. Mazurier, J. Goudemand, J. Eikenboom, et al. Identification of type 1 von Willebrand disease patients with reduced von Willebrand factor survival by assay of the VWF propeptide in the European study: Molecular and Clinical Markers for the Diagnosis and Management of Type 1 VWD (MCMDM-1VWD). Blood, 111(10): 4979-4985, 2008 300.U. Budde, R. Schneppenheim, J. Eikenboom, A. Goodeve, K. Will, E. Drewke, G. Castaman, F. Rodeghiero, A.B. Federici, J. Batlle Detailed von Willebrand factor multimer analysis in patients with von Willebrand disease in the European study, molecular and clinical markers for the diagnosis and management of type 1 von Willebrand disease (MCMDM-1VWD). J Thromb and Haemost, 6(5): 762-771, 2008 301.G. Castaman, SH Giacomelli, A. Dragani, O. Iuliani, S. Duga, F. Rodeghiero Severe factor XI deficiency in the Abruzzo region of Italy is associated to different FXI gene mutations. Haematologica, 93(6): 957-8, 2008 302.F. Rodeghiero, R.A. Kadir, A. Tosetto, P.D. James Relevance of quantitative assessment of bleeding in haemorrhagic disorders. Haemophilia, 14 (Suppl.3): 68-75, 2008 303.F. Rodeghiero, A. Ruiz-Saez, P.H. Bolton-Maggs, C.P.Hayward, S.C. Nair, A. Srivastava Laboratory issues in bleeding disorders. Haemophilia, 14 (Suppl.3): 93-103, 2008 304.E. Albiero, M. Bernardi, D. Madeo, M. Ruggeri, F. Rodeghiero

15/12/2020 European Medicine Agency Page 28/46 A new TMHA-DHPLC assay for the rapid mutation screening of JAK2 exon 14 in myeloproloferative disorders. Am J Hematol, 83 (7): 603-4, 2008 305.E. Albiero, D. Madeo, M. Ruggeri, M. Bernardi, A. Giorgetti, F. Rodeghiero Loss of the JAK2 intramolecular auto-inhibition mechanism is predicted by structural modelling of a novel exon 12 insertion mutation in a case of idiopathic erythrocytosis. British J Haematol, 142 (6): 986-990, 2008 306.A. Carobbio, G. Finazzi, E. Antonioli, P. Guglielmelli, AM Vannucchi, F. Delaini, V. Guerini, M. Ruggeri, F. Rodeghiero, A. Rambaldi , T. Barbui Thrombocytosis and leukocytosis interaction in vascular complications of essential

thrombocythemia. Blood, 112(8): 3135-7, 2008 307.F. Elice, J. Jacoub, F.R. Rickles, A. Falanga, F. Rodeghiero Hemostatic complications of angiogenesis inhibitors in cancer patients. Am J Hematol, 83(11): 862-870, 2008 308.G. Castaman, H.S. Giacomelli, D. Habart, R. Asselta, S. Duga, F. Rodeghiero Factor XI gene mutations in factor XI deficient patients of the Czech Republic. Am J Hematol, 83(12): 916-919, 2008 309.C. Visco, E. Marchioni, F. Pomponi, P. Ferrante, S. Delbue, G. Pellizzer, F. Rodeghiero Progressive multifocal leuconcephalopathy and autoimmune haemolytic anemia in chronic lymphocytic leukaemia: more than a fortuitous combination? Ann Hematol, 88 (2):189-191, 2009 310.F. Rodeghiero, M. Ruggeri Chronic Immune Thrombocytopenic Purpura New Agents Hamostaseologie, 29(1): 76-9, 2009 311.The Fibrinogen Studies Collaboration Measures to assess the prognostic ability of the stratified Cox proportional hazards model. Statistics in Medicine, 28 (3): 389-411, 2009 312.G. Castaman, M. Lunardi, L. Rigo, V. Mastroeni, E. Bonoldi, F. Rodeghiero Severe spontaneous arterial thrombotic manifestations in patients with inherited hypo- and afibrinogenemia. Haemophilia, 15(2): 533-537, 2009 313.F. Elice, F. Rodeghiero, A. Falanga, F.R. Rickles Thrombosis associated with angiogenesis inhibitors. Best Pract Res Clinl Haem, 22(1): 115-128, 2009 314.F. Rodeghiero, R. Stasi, T. Gernsheimer, M. Michel, D. Provan, D.M. Arnold, J.B. Bussel, D.B. Cines, B.H. Chong, N. Cooper, B. Godeau, M. Ruggeri, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood, 113(11): 2386-2393, 2009 315.M. Bernardi, M. Ruggeri, E. Albiero, D. Madeo, F. Rodeghiero Isolated erythrocytosis in V617F negative patients with JAk2 exon 12 mutations: Report of a new mutation. American J Hematol, 84(4): 258-60, 2009 316.A.M. Risitano, R. Notaro, L. Marando, B. Serio, D. Ranaldi, E. Seneca, P. Ricci, F. Alfinito, A. Camera, G. Gianfaldoni, A. Amendola, C. Boschetti, E. Di Bona, G. Fratellanza, F. Barbano, F. Rodeghiero, A. Zanella, A.P. Iori, C. Selleri, L. Luzzatto, B. Rotoli Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab.

15/12/2020 European Medicine Agency Page 29/46 Blood, 113(17): 4094-4100, 2009 317.M.E. Daly, B.B. Dawood, W.A. Lester, I.R. Peake, F. Rodeghiero, A.C. Goodeve, M. Makris, J.T. Wilde, A.D. Mumford, S.P. Watson, S.J. Mundell Identification and characterization of a novel P2Y12 variant in a patient diagnosed with type 1 von Willebrand disease in the European MCMDM-1VWD study. Blood, 113(17): 4110-13, 2009 318.The Fibrinogen Studies Collaboration Systematically missing confounders in individual partecipant data meta-analysis of observational cohort studies. Statistics in Medicine, 28(8): 1218-1237, 2009 319.G. Castaman, S. Giacomelli, F. Rodeghiero Autosomal recessive von Willebrand disease type 1 or 2 due to homozygous or compound heterozygous mutations in the von Willebrand factor gene. A single centre experience on molecular heterogeneity and laboratory features in 12 families. Acta Haematol, 121(2-3): 106-10, 2009 320.M.Platé, S. Duga, G. Castaman, F. Rodeghiero Recurrence of the deep-intronic FGG IVS6-320A>T mutation causing quantitative fibrinogen deficiency in the Italian population of Veneto. Blood Coagul Fibrinolysis, 20(5): 381-4, 2009 321.The Emerging Risk Factors Collaboration Lipoprotein(a) concentration and the risk of coronary Heart disease, stroke, and nonvascular mortality. Jama, 302(4): 412-423, 2009 322.J. Eikenboom, L. Hilbert, A.S. Ribba, A. Hommais, D. Habart, S. Messenger, W. Lester, C. Mazurier, D. Meyer, U. Budde, G. Castaman, F. Rodeghiero et al. Expression of 14 von Willebrand factor mutations identified in patients with type 1 von Willebrand disease from the MCMDM-1VWD study. J Thromb Haemost, 7(8): 1304-1312, 2009 323.G. Castaman, A. Tosetto, F. Rodeghiero Reduced von Willebrand factor survival in von Willebrand disease: pathophysiologic and clinical relevance. J Thromb Haemost, 7 (Suppl.1): 71-74, 2009 324.F. Rodeghiero, G. Castaman, A. Tosetto How I treat von Willebrand disease Blood, 114(6): 1158-1165, 2009 325.F. Rodeghiero The revival of clinical wisdom : The case of oral anticoagulation management Am J Haematol, 84(9): 546-7, 2009 326.T. Robak, A. Salama, L. Kovaleva, Y. Vyhovska, S.V. Davies, M.G. Mazzucconi, O. Zenker, P. Kiessling, for the International Privigen® in ITP Study Group Efficacy and safety of Privigen®, a novel liquid intravenous immunoglobulin formulation, in adolescent and adult patients with chronic immune thrombocytopenic purpura. Hematology, 14(4): 227-236, 2009 327.M. Michel, V. Chanet, A. Dechartres, A. Morin, J. Piette, L. Cirasino, G. Emilia, F. Zaja, M. Ruggeri, E. Andres, P. Bierling, B. Godeau, F. Rodeghiero The spectrum of Evans syndrome in adults: new insight into the disease based on the analysis of 68 cases. Blood, 114(15): 3167-3172, 2009 328.G. Castaman, M.E. Mancuso, S.H. Giacomelli, A. Tosetto, E. Santagostino, P.M. Mannucci, F. Rodeghiero Molecular and phenotypic determinants of the response to desmopressin in adult patients with mild hemophilia A. J Thromb Haemost, 7(11): 1824-31, 2009 - Premio Bayer

15/12/2020 European Medicine Agency Page 30/46 329.B. Cosmi, A. Alatri, M. Cattaneo, P. Gresele, M. Marietta, F. Rodeghiero, A. Tripodi, L. Ansaloni, M. Fusari, S. Taddei Assessment of the risk of bleeding in patients undergoing surgery or invasive procedures: Guidelines of the Italians Society for Haemostasis and Thrombosis (SISET). Thromb Res, 124(5): e6-e12, 2009 330.Emerging Risk Factors Collaboration Major lipids, apolipoproteins, and risk of vascular disease. Jama, 302(18): 1993-2000, 2009 331.C. Visco, F. Rodeghiero Immune thrombocytopenia in lymphoproliferative disorders. Hematol Oncol Clin North Am, 23(6): 1261-74, 2009 332.L. Pagano, M. Caira, A. Nosari, G. Rossi, F. Locatelli, P. Viale, F. Aversa for the Hema E-Chart Group, Italy Hema e-Chart: Italian Registry for Prospective analysis of Epidemiology, Management and Outcome of Febrile Events in Patients with Hematological Malignancies. J Chemotherapy, 22(1): 20-24, 2010 333.D. Provan, R. Stasi, A.C. Newland, V.S. Blanchette, P. Bolton-Maggs, J.B. Bussel, B.H. Chong, D.B. Cines, T.B. Gernsheimer, B. Godeau, J. Grainger, I. Greer, B.J. Hunt, P.A. Imbach, G. Lyons, R. McMillan, F. Rodeghiero, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood, 115 (2): 168-186, 2010 334.V. De Stefano, T. Za, E. Rossi, A.M. Vannucchi, M. Ruggeri, E. Elli, C. Micò, A. Tieghi, R. R. Cacciola, C. Santoro, N. Vianelli, P. Guglielmelli, L. Pieri, F. Scognamiglio, E. Cacciola, F. Rodeghiero, E.M. Pogliani, G. Finazzi, L. Gugliotta, G. Leone, T. Barbui Increased risk of recurrent thrombosis in patients with essential thrombocythemia carrying the omozygous JAK2 V617F mutation. Ann Hematol, 89 (2): 141-6, 2010 335.V. De Stefano, T. Za, E. Rossi, A.M. Vannucchi, M. Ruggeri, E. Elli, C. Micò, A. Tieghi, R. R. Cacciola, C. Santoro, G. Gerli, P. Guglielmelli, L. Pieri, F. Scognamiglio, F. Rodeghiero, E.M. Pogliani, G. Finazzi, L. Gugliotta, G. Leone, T. Barbui Leukocythosis is a risk factor for recurrent arterial thrombosis in young patients with polycythemia vera and essential thrombocythemia. Am J Hematol, 85(2): 97-100, 2010 336.F. Elice, F. Rodeghiero Bleeding complications of antiangiogenic therapy: pathogenetic mechanisms and clinical impact. Thromb Research, 125 (Suppl.2): 55-57, 2010 337.The Emerging Risk Factors Collaboration C-reactive protein concentration and risk of coronary heart disease, stroke, and mortality: an individual participant meta-analysis. Lancet, 375 (9709): 132-40, 2010 338.F. Rodeghiero, J. Besalduch, M. Michel, D. Provan, K. Grotzinger, G. Thompson Treatment practices in adults with chronic immune thrombocytopenia a European perspective. Eur J Haematol, 84(2): 160-8, 2010 339.F. Rodeghiero, M. Ruggeri Standardization of definitions and terminology in immune thrombocytopenia (ITP) J Platelets, 1(1): 4-8, 2010 340.F. Rodeghiero Treatment of primary immune thrombocytopenia: highlights from the 51st ASH Annual Meeting and Exposition J Platelets, 1(1): 33-6, 2010

15/12/2020 European Medicine Agency Page 31/46 341.G. Castaman, A. Tosetto, F. Rodeghiero Pregnancy and delivery in women with von Willebrands disease and different von Willebrand factor mutations. Haematologica, 95(6): 963-969, 2010 342.C. Visco, E. Novella, E.Peotta, R. Paolini, I. Giaretta, F. Rodeghiero Autoimmune hemolytic anemia in patients with chronic lymphocytic leukemia associated with IgVH status. Haematologica, 95(7): 1230-2, 2010 343.The Emerging Risk Factors Collaboration Diabetes mellitus, fasting blood glucose concentration, and risk of vascular disease:

a collaborative meta-analysis of 102 prospective studies. Lancet, 375(9733): 2215-22, 2010 344.A. Carobbio, G. Finazzi, E. Antonioli, A.M. Vannucchi, G. Barosi, M. Ruggeri, F. Rodeghiero, F. Delaini, A. Rambaldi, T. Barbui Hydroxyurea in essential thrombocythemia: rate and clinical relevance of responses by European LeukemiaNet criteria. Blood, 116(7): 1051-55, 2010 345.G.Castaman, A. Tosetto, A. Cappelletti, A. Goodeve, A.B. Federici, J. Battle, D. Meyer, J. Goudemand, J.C.J. Eikenboom, R. Schneppenheim, U. Budde, J. Ingerslev, S. Lethagen, F. Hill, I.R. Peake, F. Rodeghiero Validation of a rapid test (VWF-LIA) for the quantitative determination of von Willebrand factor antigen in type 1 von Willebrand disease diagnosis within the European multicenter study MCMDM-1VWD. Thrombosis Research, 126(3): 227-231, 2010 346.G. Castaman, SH Giacomelli, ME Mancuso, S. Sanna, E. Santagostino, F. Rodeghiero F8 mRNA studies in haemophilia A patients with different splice site mutations. Haemophilia, 16(5): 786-90, 2010 347.M. Ruggeri, S. Finotto, S. Fortuna, F. Rodeghiero Treatment outcome in a cohort of young patients with polycythemia vera. Intern Emerg Med, 5(5): 411-13, 2010 348.F. Rodeghiero, A. Tosetto, T. Abshire, D.M. Arnold, B. Coller, P. James, C. Neunert and D. Lillicrap on behalf of the ISTH/SSC joint VWF and Perinatal/Pediatric Hemostasis Subcommittees Working Group ISTH/SSC bleeding assessment tool: a standardized questionnaire and a proposal for a new bleeding score for inherited bleeding disorders. J Thromb Haemost, 8(9): 2063-65, 2010 349.N. Hickson, D. Hampshire, P. Winship, J. Goudemand, R. Schneppenheim, U. Budde, G. Castaman, F. Rodeghiero, A.B. Federici, P. James, et al. von Willebrand factor variant p.Arg924GIn marks an allele associated with reduced von Willebrand factor and factor VIII levels. J Thromb Haemost, 8(9): 1986-1993, 2010 350.G. Castaman, S.H. Giacomelli, P. Jacobi, T. Obser, U. Budde, F. Rodeghiero, S.L. Haberichter and R. Schneppenheim Homozygous type 2N R854W von Willebrand factor is poorly secreted and causes a severe von Willebrand disease phenotype. J Thromb Haemost, 8(9): 2011-16, 2010 351.G. Castaman, A. Tosetto, J.C. Eikenboom and F. Rodeghiero Blood group significantly influences von Willebrand factor increase and half-life after desmopressin in von Willebrand disease Vicenza. J Thromb Haemost, 8(9): 2078-2080, 2010 352.G. Castaman, A. Tosetto, A. Goodeve, A.B. Federici, S. Lethagen, U. Budde,

15/12/2020 European Medicine Agency Page 32/46 J. Batlle, D. Meyer, J. Mazurier, J. Goudemand, J. Eikenboom, R. Schneppenheim, J. Ingerslev, D. Habart, F. Hill, I.Peake, F. Rodeghiero The impact of bleeding history, von Willebrand factor and PFA-100® on the diagnosis of type 1 von Willebrand disease: results from the European study MCMDM-1VWD. Br J Haematol, 151(3): 245-251, 2010 353.M. Frezzato, I. Giaretta, D. Madeo, F. Rodeghiero Identical IGHV-D-J rearrangement may precede the clinical onset of chronic lymphocytic leukemia by several years. Am J Hematol, 85(11): 868-71, 2010 354.D.J. Kuter, D. Phil, M. Rummel,R. Boccia, B.G. Macik, I. Pabinger, D. Selleslag, F. Rodeghiero, B.H. Chong, X. Wang, D.P. Berger Romiplostim or standard care of patients with immune thrombocytopenia. N England J Med, 363(20): 1889-1899, 2010 355.S. Thompson, S. Kaptoge, I. White, A. Wood, P. Perry, J. Danesh and The Emerging Risk Factors Collaboration Statistical methods for the timeto-event analysis of individual participant data from multiple epidemiological studies. Int J Epidemiology, 39 (5): 1345-1359, 2010 356.NC. Gorin, M. Labopin, J. Reiffers, N. Milpied, D. Blaise, F. Witz, T. de Witte, G. Meloni, M. Attal, T. Bernal and V. Rocha for The Acute Leukemia Working Party of The European Cooperative Group for Blood and Marrow Transplantation. Higher incidence of relapse in patients with acute myelocytic leukemia infused with higher doses of CD34+ cells from leukapheresis products autografted during the first remission. Blood, 116(17): 3157-3162, 2010 357.G. Castaman, M. Platè, S.H. Giacomelli, F. Rodeghiero, S. Duga Alterations of mRNA processing and stability as a pathogenic mechanism in von Willebrand factor quantitative deficiencies. J Thromb Haemost, 8(12): 2736-2742, 2010 358.M. Cavo, P.Tacchetti, F. Patriarca, M.T. Petrucci, L. Pantani, M. Galli, F. Di Raimondo, C. Crippa, E. Zamagni, A. Palumbo, M. Offidani, P. Corradini, F. Narni, A. Spadano, N. Pescosta, G. Lambertenghi Deliliers, A. Ledda, C. Cellini, T. Caravita, P. Tosi, M. Baccarani, for the GIMEMA Italian Myeloma Network Bortezomib with thalidomide plus dexamethasone compared with thalidomide plus dexamethasone as induction therapy before, and consolidation therapy after, double autologous stem-cell transplantation in newly diagnosed multiple myeloma: a randomised phase 3 study Lancet, 376(9758): 2075-2085, 2010 359.G. Castaman, F. Rodeghiero Advances in the diagnosis and management of type 1 von Willebrand disease. Expert Rev Hematol, 4(1): 95-106, 2011 360.A. Tosetto, F. Rodeghiero, G. Castaman, A. Goodeve, AB Federici, J. Batlle, D. Meyer, J. Goudemand, J. Eikenboom, et al. A comparison between two semi-quantitative bleeding scales for the diagnosis and assessment of bleeding severity in type 1 von Willebrand disease. Haemophilia, 17(1): 165-6, 2011 361.A. Tosetto, P. Prati, C. Baracchini, R. Manara, F. Rodeghiero Association of plasma fibrinogen, C-reactive protein and G-455>A polymorphism with early atherosclerosis in the VITA Project cohort. Thromb Haemost, 105(2): 329-35, 2011 362.N. Hickson, D. Hampshire, G. Castaman, J. Eikenboom, F. Rodeghiero, I. Peake and A. Goodeve on behalf of the MCMDM-1VWD and ZPMCB-VWD Study Groups Effect of the VWF promoter (GT)n repeat and single-nucleotide polymorphism c.-2527G>A on circulating von Willebrand factor levels under normal conditions.

15/12/2020 European Medicine Agency Page 33/46 J Thromb Haemost, 9(3): 603-5, 2011 363.The Emerging Risk Factors Collaboration Diabetes mellitus, fasting glucose, and risk of cause-specific death. N England J Med, 364(9): 829-41, 2011 364.E. Albiero, M. Ruggeri, S. Fortuna, M. Bernardi, S. Finotto, D. Madeo, F. Rodeghiero Analysis of the oxygen sensing pathway genes in familial chronic myeloproliferative neoplasms and identification of a novel EGLN1 germ-line mutation. Br J Haematol, 153(3): 405-8, 2011 365.G. Castaman, A. Tosetto, A.B. Federici, F. Rodeghiero Bleeding tendency and efficacy of anti-haemorrhagic treatments in patients with type 1 von Willebrand disease and increased von Willebrand factor clearance. Thromb Haemost, 105(4): 647-654, 2011 366.S. Castegnaro, K. Chieregato, M. Maddalena, E. Albiero, C. Visco, D. Madeo, M. Pegoraro, F. Rodeghiero Effect of platelets lysate on the functional and molecular characteristics of mesenchymal stem cells isolated from adipose tissue. Current Stem Cell Research & Therapy, 6: 105-14, 2011 367.A. Tosetto, G. Castaman, I. Plug, F. Rodeghiero, J. Eikenboom Prospective evaluation of the clinical utility of quantitative bleeding severity assessment in patients referred for hemostatic evaluation. J Thromb Haemost, 9(6): 1143-1148, 2011 368.A. Carobbio, J.Thiele, F. Passamonti, E. Rumi, M. Ruggeri, F. Rodeghiero, M.L. Randi, I. Bertozzi, A.M. Vannucchi, E. Antonioli, H. Gisslinger, et al. Risk factors for arterial and venous thrombosis in WHO-defined essential thrombocythemia: an international study of 891 patients. Blood, 117(22): 5857-5859, 2011 369.G. Castaman, SH Giacomelli, ME Mancuso, G. DAndrea, R. Santacroce, S. Sanna, E. Santagostino, PM Mannucci, A. Goodeve, F. Rodeghiero Deep intronic variations may cause mild hemophilia. J Thromb Haemost, 9(8): 1541-8, 2011 370.T. Barbui, J.Thiele, F. Passamonti, E. Rumi, E. Boveri, M. Ruggeri, F. Rodeghiero, ES DAmore, ML Randi, I. Bertozzi, F. Marino, AM Vannucchi, V. Carrai, et al. Survival and disease progression in essential thrombocythemia are significantly influenced by accurate morphologic diagnosis: an international study of 1,104 pts. J Clin Oncol, 29(23): 3179-84, 2011 371.AR Hubbard, M. Hamill, M. Beeharry, SA Bevan, AB Health, SSC sub-committee on von Willebrand Factor of ISTH Value assignement of the WHO 2nd International Standard von Willebrand factor, concentrate (09/182) J Thromb Haemost, 9(8): 1638-40, 2011 372.A. Giampaolo, F. Abbonizio, M. Puopolo, R. Arcieri, PM Mannucci, HJ Hassan, A. Accorsi, PC Ettorre, M. Schiavoni, G. Palareti, G. Rodorigo, L. Valdré, G. Amaddii, A. Billio, L. Notarangelo, P. Iannacaro, P. Muleo, C. Biasioli, V. Rossi, A. Alatri, S. Testa, D. Vincenzi, G. Scapoli, M. Morfini, G. Castaman, F. Rodeghiero, et al. Consumption of clotting factors in severe haemophilia patients undergoing prophylaxis and on-demand treatment in Italy. Transfus Med, 21(4): 280-4, 2011 373.K. Chieregato, S. Castegnaro, D. Madeo, G. Astori, M. Pegoraro, F. Rodeghiero Epidermal growth factor, basic fibroblast growth factor and platelet-derived growth factor-bb can substitute for fetal bovine serum and compete with human platelet-rich plasma in the ex vivo expansion of mesenchymal stromal cells derived from adipose tissue. Cytotherapy, 13(8): 933-43, 2011

15/12/2020 European Medicine Agency Page 34/46 374.AR. Hubbard, M. Hamill, M. Beeharry, SA. Bevan, and AB. Heath on behalf of the SSC Sub-Committees on Factor VIII/factor IX and von Willebrand factor of ISTH Value assignment of the WHO 6th International Standard for blood coagulation factor VIII and von Willebrand factor in plasma (07/316) J Thromb Haemost, 9(10): 2100-2102, 2011 375.T. Kuhne, W. Berchtold, L. A. Michaels, R. Wu, H. Donato, B. Espina, H. Tamary, F. Rodeghiero, M. Chitlur, J. Rischewski, and Paul Imbach on behalf of the Intercontinental Cooperative ITP Study Group Newly diagnosed immune thrombocytopenia in children and adults: a comparative prospective observational registry of the Intercontinental Cooperative Immune Thrombocytopenia Study Group. Haematologica, 96(12): 1831-37, 2011 376.G. Castaman, S.H. Giacomelli, V. Schroeder, S. Sanna, L. Valdrè, M. Morfini, L. Banov, H.P. Kohler, F. Rodeghiero

Further evidence of heterogeneity of gene defects in Italian families with factor XIII deficiency. Haemophilia, 18(1): e6-8, 2012 377.E. Albiero, M. Ruggeri, S. Fortuna, S. Finotto, M. Bernardi, D.Madeo, F. Rodeghiero Isolated erythrocytosis: study of 67 patients and identification of three novel germ-line mutations in the prolyl hydroxylase domain protein 2 (PHD2) gene. Haematologica, 97(1): 123-7, 2012 378.C. Visco, S. Castegnaro, K. Chieregato, M. Bernardi, E. Albiero, C. Zanon, D. Madeo, F. Rodeghiero The cytotoxic effects of bendamustine in combination with cytarabine in mantle cell lymphoma cell lines. Blood Cells Mol Dis, 48(1): 68-75, 2012 379.F. Elice, F. Rodeghiero Hematologic malignancies and thrombosis Thrombosis Research, 129(3): 360-66, 2012 380.D. Tormene, E. Grandone, V. De Stefano, A. Tosetto, G. Palareti, M. Margaglione, G. Castaman, E. Rossi, A. Ciminello, L. Valdrè, C. Legnani, G. L.Tiscia, V. Bafunno, S. Carraro, F. Rodeghiero, P. Simioni Obstetric complications and pregnancy-related venous thrombo-embolism: The effect of low-molecular-weight heparin on their prevention in carriers of factor V Leiden or prothrombin G20210A mutation. Thromb Haemost, 107(3): 477-484, 2012 381.F. Elice, F. Rodeghiero Side effects of anti-angiogenic drugs Thrombosis Research, 129 (suppl.1): S50-S53, 2012 382.G. Castaman, A. B. Federici, A. Tosetto, S . La Marca, F . Stufano, P .M. Mannucci, F. Rodeghiero Different bleeding risk in type 2A and 2M von Willebrand disease: a 2-year prospective study in 107 patients. J Thromb Haemost, 10(4): 632-38, 2012 383.C. Visco, F. Maura, G.Tuana, L. Agnelli, M. Lionetti, S. Fabris, E. Novella, I. Giaretta, G. Reda, W. Barcellini, L. Baldini, A. Neri, F. Rodeghiero, A.Cortelezzi Immune Thrombocytopenia in Patients with Chronic Lymphocytic Leukemia is associated with Stereotyped B-cell receptors. Clin Cancer Res, 18(7): 1870-8, 2012 384.G. Finazzi, A Carobbio1, J. Thiele, F. Passamonti, E. Rumi, M. Ruggeri, F. Rodeghiero, ML. Randi, I. Bertozzi, AM. Vannucchi, E. Antonioli, H. Gisslinger, V. Buxhofer-Ausch, N. Gangat, A. Rambaldi, A. Tefferi, T Barbui Incidence and risk factors for bleeding in 1104 patients with essential

15/12/2020 European Medicine Agency Page 35/46 thrombocythemia or prefibrotic myelofibrosis diagnosed according to the 2008 WHO criteria. Leukemia, 26(4): 716-19, 2012 385.E. Antonioli, P. Guglielmelli, L. Pieri, MC. Finazzi, E. Rumi, V. Martinelli, N. Vianelli, ML.Randi, I. Bertozzi, V. De Stefano, T. Za, E. Rossi, M. Ruggeri, E. Elli, R. Cacciola, E. Cacciola, .E. Pogliani, F. Rodeghiero, M. Baccarani, F. Passamonti, G. Finazzi, A. Rambaldi, A. Bosi, M. Cazzola, T. Barbui, AM. Vannucchi, and On behalf of the AGIMM Investigators Hydroxyurea-related toxicity in 3,411 patients with Ph-negative MPN Am J Hematol, 87(5): 552-54, 2012 386.G. Castaman, S.H. Giacomelli, P.M. Jacobi, T. Obser, U. Budde, F . Rodeghiero, R. Schneppenheim and S.L. Haberichter Reduced von Willebrand factor secretion is associated with loss of WeibelPalade body formation J Throm Haemost, 10(5): 951-958, 2012 387.F. Rodeghiero, G. Castaman Short-and long-term risks of splenectomy for benign haematological disorders: should we revisit the indications? Br J Haematol, 158(1): 16-29, 2012 388.V. Buxhofer-Ausch, H. Gisslinger, J. Thiele, B. Gisslinger, H.M. Kvasnicka, L. Müllauer, S. Frantal, A.Carobbio, F. Passamonti, E. Rumi, M. Ruggeri, F. Rodeghiero, M. L. Randi, I. Bertozzi, A.M. Vannucchi, E. Antonioli, G. Finazzi, N. Gangat, A.Tefferi, T. Barbui Leukocytosis as an important risk factor for arterial thrombosis in WHO-defined early/prefibrotic myelofibrosis: An international study of 264 patients Am J Hematol, 87(7): 669-672, 2012 389.T. Barbui, J. Thiele, A. Carobbio, F. Passamonti, E. Rumi, M.L. Randi, I. Bertozzi, A M. Vannucchi, H. Gisslinger, B. Gisslinger, G. Finazzi, M. Ruggeri, F. Rodeghiero, A. Rambaldi, N. Gangat, and A. Tefferi Disease characteristics and clinical outcome in young adults with essential thrombocythemia versus early/prefibrotic primary myelofibrosis Blood, 120(3): 569-571, 2012 390.Cavo M, Pantani L, Petrucci M T, Patriarca F, Zamagni E, Donnarumma D, Crippa C, Boccadoro M, Perrone G, Falcone A,Nozzoli C, Zambello R, Masini L, Furlan A, Brioli A, Derudas D, Ballanti S, Dessanti M L, et al., for the GIMEMA (Gruppo Italiano Malattie Ematologiche dellAdulto) Italian Myeloma Network Bortezomib-thalidomide-dexamethasone is superior to thalidomide-dexamethasone as consolidation therapy after autologous hematopoietic stem cell transplantation in patients with newly diagnosed multiple myeloma Blood, 120(1): 9-19, 2012 391.Passamonti F, Thiele J, Girodon F, Rumi E, Carobbio A, Gisslinger H, Kvasnicka H M, Ruggeri M, Randi M L, Gangat N, Vannucchi A M, Gianatti A, Gisslinger B, Mullauer L, Rodeghiero F, DAmore E, Bertozzi I, Hanson C A, Boveri E, Marino F, Maffioli M, Caramazza D, Antonioli E, Carrai V, Buxhofer-Ausch V, Pascutto C, Cazzola M, Barbui T, Tefferi A A prognostic model to predict survival in 867World Health Organizationdefined essential thrombocythemia at diagnosis: a study by the InternationalWorking Group on Myelofibrosis Research and Treatment Blood, 120 (6): 1197-1201, 2012 392.Raimondi R, Tosetto A, Rosi O, Cavazzina R, Rodeghiero F, Bacigalupo A, Fanin R, Rambaldi A, Bosi A Validation of the Hematopoietic Cell Transplantation-Specific Comorbidity Index: a prospective, multicenter GITMO study. Blood, 120(6): 1327-1333, 2012 393.Reda G, Maura F, Gritti G, Gregorini A, Binda F, Guidotti F, Piciocchi A, Visco C, Rodeghiero F, Cortelezzi A

15/12/2020 European Medicine Agency Page 36/46 Low-dose alemtuzumab-associated immune thrombocytopenia in chronic lymphocytic leukemia Am J Hematol, 87(9): 936-7, 2012 394.Chieregato K, Albiero E, Castegnaro S, Bernardi M, D'Amore E, Zanon C, Madeo D, Rodeghiero F, Astori G A study on mutual interaction between cytokine induced killer cells and umbilical cord-derived mesenchymal cells: Implication for their in-vivo use Blood Cells Mol Dis, 49(3-4): 159-165, 2012 395.Castegnaro S, Visco C, Chieregato K , Bernardi M, Albiero E , Zanon C, Madeo D, Rodeghiero F Cytosine arabinoside potentiates the apoptotic effect of bendamustine on several B- and T-cell leukemia/lymphoma cells and cell lines Leukemia & Lymphoma, 53(11): 2262-2268, 2012 396.Rodeghiero F TPO-receptor agonists in ITP: A clinicians navigation between Scylla and Charybdis Am J Haematol, 87(10): 943, 2012 397.The Emerging Risk factors Collaboration Adult height and the risk of cause-specific death and vascular morbidity in 1 million people: individual partecipant meta-analysis Int J Epidemiol, 41(5): 1419-1433, 2012 398.The Emerging Risk factors Collaboration C-reactive protein , fibrinogen, and cardiovascular disease prediction N Engl J Med, 367(14): 1310-1320, 2012 399.Barbui T, Finazzi G, Carobbio A, Thiele J, Passamonti F, Rumi E, Ruggeri M, Rodeghiero F, Randi ML, Bertozzi I, Gisslinger H, Buxhofer-Ausch V, De Stefano V, Betti S, Rambaldi A, Vannucchi AM, Tefferi A Development and validation of an International Prognostic Score of thrombosis in World Health Organizationessential thrombocythemia (IPSET-thrombosis) Blood, 120(26): 5128-36, 2012 400.Carobbio A, Finazzi G, Thiele J, Kvasnicka HM, Passamonti F, Rumi E, Ruggeri M, Rodeghiero F, Randi L, Bertozzi M, Vannucchi AM, Antonioli E, Gisslinger H, Buxhofer-Ausch V, Gangat N, Rambaldi A, Tefferi A, Barbui T Blood tests may predict early primary myelofibrosis in patients presenting with essential thrombocythemia Am J Hematol, 87(2): 203-4, 2012 401.Maura F, Visco C, Falisi E, Reda G, Fabris S, Agnelli L, Tuana G, Lionetti M, Guercini N, Novella E, Nichele I, Montaldi A, Autore F, Gregorini A, Barcellini W, Callea V, Mauro F, Laurenti L, Foa` R, Neri A, Rodeghiero F, Cortelezzi A B-cell receptor configuration and adverse cytogenetics are associated with autoimmune hemolytic anemia in chronic lymphocytic leukemia Am J Hematol, 88(1): 32-6, 2013 402.Marchioli R, Finazzi G, Specchia G, Cacciola R, Cavazzina R, Cilloni D, De Stefano V, Elli E, Iurlo A, Latagliata R, Lunghi F, Lunghi M, Marfisi RM, Musto P, Masciulli A, Musolino C, Cascavilla N, Quarta G, Randi ML, Rapezzi D, Ruggeri M, Rumi E, Scortechini AR, Santini S, Scarano M, Siragusa S, Spadea A, Tieghi A, Angelucci E, Visani G, Vannucchi AM, Barbui T, for the CYTO-PV Collaborative Group Cardiovascular Events and Intensity of Treatment in Polycythemia Vera N Engl J Med, 368(1): 22-23, 2013 403.Eikenboom J, Federici AB, Dirven RJ, Castaman G, Rodeghiero F, Budde U, Schneppenheim R, Batlle J, Canciani MT, Goudemand J, Peake I, Goodeve A, and the MCMDM-1VWD Study Group VWF propeptide and ratios between VWF, VWF propeptide, and FVIII in the characterization of type

15/12/2020 European Medicine Agency Page 37/46 1 von Willebrand disease Blood, 121(12): 2336-9, 2013 404.Visco C, Moretta F, Falisi E, Facco M, Maura F, Novella E, Nichele I, Finotto S, Giaretta I, Ave E, Perbellini O, Guercini N, Scupoli MT, Trentin L, Trimarco V, Neri A, Semenzato G, Rodeghiero F, Pizzolo G, Ambrosetti A Double productive immunoglobulin sequence rearrangements in patients with chronic lymphocytic leukemia Am J Hematol, 88(4): 277-82, 2013 405.Visco C, Finotto S, Pomponi F, Sartori R, Laveder F, Trentin L, Paolini R, Di Bona E, Ruggeri M, Rodeghiero F The combination of rituximab, bendamustine, and cytarabine for heavily pretreated relapsed/refractory cytogenetically high-risk patients with chronic lymphocytic leukemia Am J Hematol, 88(4): 289-93, 2013 406.F. Rodeghiero Von Willebrand disease: pathogenesis and management Thromb Res, 131(Suppl 1): S47-S50, 2013 407.Rodeghiero F, Michel M, Gernsheimer T, Ruggeri M, Blanchette V, Bussel JB, Cines DB, Cooper N, Godeau B, Greinacher A, Imbach P, Khellaf M, Klaassen RG, K¨uhne T, Liebman H, Mazzucconi MG, Newland A, Pabinger I, Tosetto A, Stasi R Standardization of bleeding assessment in immune thrombocytopenia: report from the International Working Group Blood, 121(4): 2596-606, 2013 408.Visco C, Finotto S, Zambello R, Paolini R, Menin A, Zanotti R, Zaja F, Semenzato G, Pizzolo G, DAmore ESG, Rodeghiero F Combination of Rituximab, Bendamustine, and Cytarabine for Patients With Mantle-Cell Non-Hodgkin Lymphoma Ineligible for Intensive Regimens or Autologous Transplantation J Clin Oncol, 31(11): 1442-9, 2013

409.Castaman G, Goodeve A, and J Eikenboom, on behalf of the European Group on von Willebrand disease (EUVWD) Principles of care for the diagnosis and treatment of von Willebrand disease Haematologica, 98(5): 667-74, 2013 410.Vianelli N, Palandri F, Polverelli N, Stasi R, Joelsson J, Johansson E, Ruggeri M, Zaja F, Cantoni S, Catucci AE, Candoni A, Morra E, Björkholm M, Baccarani M, Rodeghiero F Splenectomy as a curative treatment for immune thrombocytopenia: a retrospective analysis of 233 patients with a minimum follow up of 10 years Haematologica, 98(6): 875-880, 2013 411.Tosetto A, Castaman G, Rodeghiero F Bleeders, bleeding rates, and bleeding score J Thromb Haemost, 11 (suppl.1): 142-50, 2013 412.Bernardi M, Albiero E, Alghisi A, Chieregato K, Lievore C, Madeo D, Rodeghiero F, Astori G Production of human platelet lysate by use of ultrasound for ex vivo expansion of human bone marrow-derived mesenchymal stromal cells Cytotherapy, 15 (8): 920-9, 2013 413.Castaman G, Giacomelli SH, Tagliaferri A, Rodeghiero F A novel heterozygous missense mutation (His127Arg) in a family with inherited cross-reacting material positive factor XI deficiency Blood Coagul Fibrinolysis, 24(6): 670-2, 2013 414.Cairoli R, Beghini A, Turrini M, Bertani G, Nadali G, Rodeghiero F, Castagnola C, Lazzaroni F, Nichelatti M, Ferrara F, Pizzolo G, Pogliani E, Rossi G, Martinelli G, Morra E Old and new prognostic factors in acute leukemia with deranged core-binding factor beta Am J Hematol,88(7): 594-600, 2013 415.Tefferi A, Rumi E, Finazzi G, Gisslinger H, Vannucchi AM, Rodeghiero F, Randi ML, Vaidya R,

15/12/2020 European Medicine Agency Page 38/46 Cazzola M, Rambaldi A, Gisslinger B, Pieri L, Ruggeri M, Bertozzi I, Sulai NH, Casetti I, Carobbio A, Jeryczynski G, Larson DR, Mullauer L, Pardanani A, Thiele J, Passamonti F, Barbui T

Survival and prognosis among 1545 patients with contemporary polycythemia vera: an international study Leukemia, 27: 1874-1881, 2013 416.Rodeghiero F, Stasi R, Giagounidis A, Viallard JF, Godeau B, Pabinger I, Cines D, Liebman H, Wang X, Woodard P Long-term safety and tolerability of romiplostim in patients with primary immune thrombocytopenia (ITP): a pooled analysis of 13 clinical trials Eur J Haematol, 91: 423-36, 2013 417. Zaja F, Mian M,Volpetti S,Visco C, Sissa C, Nichele I, Castelli M, Ambrosetti A, Puglisi S, Fanin R, Cortelazzo S, Pizzolo G, Trentin L, Rodeghiero F, Paolini R, Vivaldi P, Sancetta R, Isola M, Semenzato G Bendamustine in chronic lymphocytic leukemia: Outcome according to different clinical and biological prognostic factors in the everyday clinical practice Am J Hematol, 88(11): 955-60, 2013 418.Bernardi M, Adami V, Albiero E, Madeo D, Rodeghiero F, Astori G Absence of micronucleus formation in CHO-K1 cells cultivated in platelet lysate enriched medium Exp Toxicol Pathol, 66 (2-3): 111-16, 2014 419.Rodeghiero F. Morfini M, Nozzoli C, Manotti C, Violi F, Castaldo G, Pane F, Biasioli B, Velati C Identificazione e ruolo dellesperto in emostasi e trombosi nel Sistema Sanitario Nazionale The expert in hemostasis and thrombosis in the Italian health system: role and requirements for a specific clinical and laboratory expertise Italian Journal of Medicine, 7(Suppl.8): 71-84, 2013 La Rivista Italiana della Medicina di Laboratorio, 9(3): 134-42, 2013 Biochimica Clinica, 38(1): 39-46, 2014 420.Falisi E, Novella E, Visco C, Guercini N, Maura F, Giaretta I, Pomponi F, Nichele I, Finotto S, Montaldi A, Neri A, Rodeghiero F B-cell receptor configuration and mutational analysis of patients with chronic lymphocytic leukaemia and trisomy 12 reveal recurrent molecular abnormalities Hematol Oncol, 32(1): 22-30, 2014 421.Rodeghiero F, Ruggeri M ITP and international guidelines: what do we know, what do we need? La Presse Medicale, 43 (4 part 2): e61-e67, 2014 422.Fedeli U, Schievano E, Rodeghiero F, Saugo M Mortality from myelodysplastic sydromes: a multiple causes of death approach Am J Hematol, 89 (4): 450-1, 2014

423.Imberti D, Dentali F, Ageno W, Crowther M, Garcia D, Huisman M, Rodeghiero F, DAngelo A, Palareti G Evidence and clinical judgement: vena cava filters Thromb Haemost ,111(4): 618-24, 2014 424.Perbellini O, Falisi E, Giaretta I, Boscaro E, Novella E, Facco M, Fortuna S, Finotto S, Amati E, Maniscalco F, Montaldi A, Alghisi A, Aprili F, Bonaldi L, Paolini R, Scupoli MT, Trentin L, Ambrosetti A, Semenzato G, Pizzolo G, Rodeghiero F, Visco C Clinical significance of LAIR1 (CD305) as assessed by flow cytometry in a prospective series of patients with chronic lymphocytic leukemia Haematol, 99(5): 881-7, 2014 425.Rodeghiero F, Castaman G

15/12/2020 European Medicine Agency Page 39/46 Inibitor devolopment: the last enemy to be defeated in hemophilia A and B Am J Hematol, 89(6): 569-70, 2014 426. Visco C, Cortelezzi A, Moretta F, Falisi E, Maura F, Finotto S, Barcellini W, Ambrosetti A, Neri A, Ruggeri M, Rodeghiero F Autoimmune cytopenias in chronic lymphocytic leukemia at disease presentation in the modern treatment era: is stage C always stage C? Leuk Lymphoma, 55(6): 1261-5, 2014 427.Zanon C, Stocchero M, Albiero E, Castegnaro S, Chieregato K, Madeo D, Rodeghiero F, Astori G Multivariate statistical data analysis as a tool to analyze ex vivo expansion dynamics of cytokine-induced killer cells Cytometry B Clin Cytom, 86(B): 257-62, 2014 428.Federici AB, Bucciarelli P, Castaman G, Mazzucconi MG, Morfini M, Rocino A, Schiavoni M, Peyvandi F, Rodeghiero F, Mannucci PM The bleeding score predicts clinical outcomes and replacement therapy in adults with von Willebrand disease: a prospective cohort study of 796 cases. Blood, 123(26): 4037-44, 2014 429.Abbonizio F, Giampaolo A, Coppola A; the Italian Association of Haemophilia Centres (AICE), Arcieri R, Hassan HJ. Therapeutic management and costs of severe haemophilia A patients with inhibitors in Italy. Haemophilia, 20(4): e243-50, 2014 430.Rodeghiero F Von Willebrand disease Hematology, 19(6): 370-1, 2014 431.Rodeghiero F Diagnosing type 1 von Willebrand disease: good for patients health or for doctors prestige? J Thromb Haemost, 12(8): 1234-7, 2014 432.Ruggeri M, Tosetto A, Palandri F, Polverelli N, Mazzucconi MG, Santoro C, Gaidano G, Lunghi M, Zaja F, De Stefano V, Sartori R, Fazi P, Rodeghiero F; the Gruppo Italiano Malattie Ematologiche dellAdulto (GIMEMA) Anemia and Thrombocytopenias Working Party. GIMEMA Study ITP0311 Thrombotic risk in patients with primary immune thrombocytopenia is only mildly increased and explained by personal and treatment-related risk factors J Thromb Haemost, 12(8): 1266-73, 2014 433.Visco C, Barcellini W, Maura F, Neri A, Cortelezzi A, Rodeghiero F Autoimmune cytopenias in chronic lymphocytic leukemia. Am J Hematol, 89(11): 1055-62, 2014 434.Rohatiner AZ, Smith ML, Spinelli O, Rambaldi A, Bassan R, Di Bona E, Rodeghiero F, Raimondi R, Bjorkholm M, Johnson S, Newland AC, Cavenagh Jd, Macdougall F, Waters R, Myeloblative therapy with autologous haemopoietic stemcell support as consolidation of first remission in acute myeloid leukemia very lonf follow-up Br J Haematol, 167(5): 724-6, 2014 435.Barcellini W, Fattizzo B, Zaninoni A, Radice T, Nichele I, Di Bona E, Lunghi M, Tassinari C, Alfinito F, Ferrari A, Leporace AP, Niscola P, Carpenedo M, Boschetti C, Revelli N, Villa AM, Consonni D, Scaramucci L, De Fabritiis P, Tagariello G, Gaidano G, Rodeghiero F, Cortelezzi A, Zanella A Clinical heterogeneity and predictors of outcome in primary autoimmune hemolytic anemia: a GIMEMA study of 308 patients Blood, 124(19): 2930-36, 2014 436.Barbui T, Carobbio A, Rumi E, Finazzi G, Gisslinger H, Rodeghiero F, Randi ML, Rambaldi A, Gisslinger B, Pieri L, Bertozzi I, Casetti I, Pardanani A, Passamonti F, Vannucchi Am, Tefferi A In contemporary patients with polycythemia vera, rates of thrombosis and risk factors delineate a new clinical epidemiology Blood, 124(19): 3021-23, 2014

15/12/2020 European Medicine Agency Page 40/46 437.Rodeghiero F Diagnosing type 1 von Willebrand disease: good for patients health or for doctors: reply. J Thromb Haemost, 12(12): 2134-6, 2014 438.F. Rodeghiero, M. Ruggeri Treatment of Immune Thrombocytopenia in Adults: The Role of Thrombopoietin-Receptor Agonists Semin Hematol, 52(1): 16-24, 2015

439.Barbui T, Vannucchi AM, Carobbio A, Thiele J, Rumi E, Gisslinger H, Rodeghiero F, Randi ML, Rambaldi A, Pieri L, Pardanani A, Passamonti F, Finazzi G, Tefferi A Patterns of presentation and thrombosis outcome in patients with polycythemia vera strictly defined by WHO-criteria and stratified by calendar period of diagnosis Am J Hematol, 90(5): 434-7, 2015

440.Bron D, Ades L, Fulop T, Goede V and Stauder R on behalf of the Elderly Task Force in Hematology EHA SWG Aging and blood disorders: new perspectives, new challenges Haematologica, 100(4): 415-17, 2015 441.Nichele I, Ruggeri M, Rodeghiero F Effectiveness of lenalidomide in a patient with refractory anemia with ring sideroblasts and thrombocytosis with jak2 (V617F) mutation Am J Hematol, 90(8): e148-9, 2015 442.Rodeghiero F Thrombocytopenias: really a rare encounter by the hematologist ? DCTH, 3(1): 28-35, 2015 443. Visco C, Falisi E, Young KH, Pascarella M, Perbellini O, CarliG, Novella E, Rossi D, Giaretta I, Cavallini C, Scupoli MT, De Rossi A, DAmore E, Rassu M, Gaidano G, Pizzolo G, Ambrosetti A, Rodeghiero F Epstein-Barr virus DNA load in chronic lymphocytic leukemia is an independent predictor of clinical course and survival Oncotarget, 6(21): 18653-63, 2015 444.Chieregato K, Alghisi A, Borghero C, Elice F, Raimondi R, Zanetti E, Rodeghiero F, Astori G Evaluation of lymphocytes inactivation by extracorporeal photopheresis using tetrazolium salt based-assay Transfusion and Apheresis Science, 53(2): 242-45, 2015 445.Castagnetti F, Gugliotta G, Breccia M, Stagno F, Iurlo A, Albano F, Abruzzese E, Martino B, Levato L, Intermesoli T, Pregno P, Rossi G, Gherlinzoni F, Leoni P, Cavazzini F, Venturi C, Soverini S, Testoni N, Alimena G, Cavo M, Martinelli G, Pane F, Saglio G, Rosti G, Baccarani M on behalf of the GIMEMA CML Working Party Long-term outcome of chronic myeloid leukemia patients treated frontline with imatinib Leukemia, 29(9): 1823-31, 2015 446.Barbui T, Vannucchi AM, Buxhofer-Ausch V, De Stefano V, Betti S, Rambaldi A, Rumi E, Ruggeri M, Rodeghiero F, Randi ML, Bertozzi I, Gisslinger H, Finazzi G, Carobbio A, Thiele J, Passamonti F, Falcone C, Tefferi A Practice-relevant revision of IPSET-thrombosis based on 1019 patients with WHO-defined essential thrombocythemia Blood Cancer J, 27(5): e369, 2015 447.Ruggeri M, Rodeghiero F Thrombotic risk in patients with immune haemolytic anaemia Br J Haematol, 172(1): 144-5, 2016 448.Rodeghiero F Is ITP a thrombophilic disorder ?

15/12/2020 European Medicine Agency Page 41/46 Am J Hematol, 91(1): 39-45, 2016 449.Engert A, Balduini C, Brand A, Coiffier B, Cordonnier C, Döhner H, Duyvené de Wit T,Eichinger S, Fibbe W, Green T, De Haas F, Iolascon A, Jaffredo T, Rodeghiero F, Salles G, Schuringa J, and the other authors of the EHA Roadmap for European Hematology Research The European Hematology Association Roadmap for European Hematology Research: a consensus document Haematologica, 101(2): 115-208, 2016 450.Carli G, Visco C, Falisi E, Perbellini O, Novella E, Giaretta I, Ferrarini I, Sandini A, Alghisi A, Ambrosetti A, Rodeghiero F Evans syndrome secondary to chronic lymphocytic leukaemia: presentation, treatment, and outcome Ann Hematol , 95(6): 863870, 2016 451.Castegnaro S, Dragone P, Chieregato K, Alghisi A, Rodeghiero F, Astori G Enumeration of residual white blood cells in leukoreduced blood products: Comparing flow cytometry with a portable microscopic cell counter Transf Apher Sci, 54(2): 266-70, 2016 452.Janssens A, Rodeghiero F, Anderson D, Chong BH, Boda Z, Pabinger I, Cervinek L, Terrel DR, Wang X, Franklin J Changes in bone marrow morphology in adults receiving romiplostim for the treatment of thrombocytopenia associated with primary immune thrombocytopenia Ann Hematol, 95(7): 1077-87, 2016 453.Astori G, Amati E, Bambi F, Bernardi M, Chieregato K, Schafer R, Sella S, Rodeghiero F Platelet lysate as a substitute for animal serum for the ex-vivo expansion of mesenchymal stem/cells: present and future Stem Cell Research & Therapy, 7(1): 93, 2016 454.Efficace F, Mandelli F, Fazi P, Santoro C, Gaidano G, Cottone F, Borchiellini A, Carpenedo M, Simula MP, Di Giacomo V, Bergamaschi M, Vincelli ID, Rodeghiero F, Ruggeri M, Scaramucci L, Rambaldi A, Cascavilla N, Forghieri F, Petrungaro A, Ditonno P, Caocci G, Cirrincione S, Mazzucconi MG Health-related quality of life and burden of fatigue in patients with primary immune thrombocytopenia by phase of disease Am J Hematol, 91(10): 995-1001, 2016 455.Albiero E, Amati A, Baumeister E, Einsele H, Grigoleit U, Rodeghiero F Heterogeneity of specific CD4+ and CD8+ T cells stimulated by CMV pp65 and IE1 antigens J Immunother, 39(9): 329-342, 2016 456. Amati E, Sella S, Perbellini O, Alghisi A, Bernardi M, Chieregato K, Lievore C, Peserico D, Rigno M, Zilio A, Ruggeri M, Rodeghiero F, Astori G. Generation of mesenchymal stromal cells from cord blood: evaluation of in vitro quality parameters prior to clinical use. Stem Cell Res Ther. 2017 Jan 24;8(1):14. doi: 10.1186/s13287-016-0465-2. 457. Rodeghiero F, Carli G Beyond immune thrombocytopenia: the evolving role of thrombopoietin receptor agonists. Ann Hematol. 2017 Sep;96(9):1421-1434. doi: 10.1007/s00277-017-2953-6. Epub 2017 458. Rodeghiero F. A hit to current "hit" wisdom: A century later, it's time for a change. Am J Hematol. 2017 Aug;92(8):727-729. doi: 10.1002/ajh.24789. Epub 2017 Jun 5. 459. Cines DB, Wasser J, Rodeghiero F, Chong BH, Steurer M, Provan D, Lyons R, Garcia-Chavez J, Carpenter N, Wang X, Eisen M. Safety and efficacy of romiplostim in splenectomized and nonsplenectomized patients with primary immune thrombocytopenia. Haematologica. 2017 Aug;102(8):1342-1351. doi: 10.3324/haematol.2016.161968. Epub 2017 Apr 14. 460. Cantoni S, Carpenedo M, Mazzucconi MG, De Stefano V, Carrai V, Ruggeri M, Specchia G,

15/12/2020 European Medicine Agency Page 42/46 Vianelli N, Pane F, Consoli U, Artoni A, Zaja F, D'adda M, Visentin A, Ferrara F, Barcellini W, Caramazza D, Baldacci E, Rossi E, Ricco A, Ciminello A, Rodeghiero F, Nichelatti M, Cairoli R. Alternate use of thrombopoietin receptor agonists in adult primary immune thrombocytopenia patients: A retrospective collaborative survey from Italian hematology centers. Am J Hematol, 93(1): 58-64, 2018 461.Amati E, Perbellini O, Rotta G, Bernardi M, Chieregato K, Sella S, Rodeghiero F, Ruggeri M, Astori G High-throughput immunophenotypic characterization of bone marrow- and cord blood-derived mesenchymal stromal cells reveals common and differentially expressed markers: identification of angiotensin-converting enzyme (CD143) as a marker differentially expressed between adult and perinatal tissue sources Stem Cell Res Ther, 9(1): 10, 2018 463.Rodeghiero F A critical appraisal of the evidence for the role of splenectomy in adults and children with ITP Br J Haematol, 181(2): 183-95, 2018 464.Berntorp E, Ågren A, Aledort L, Blombäck M, Cnossen MH, Croteau SE, von Depka M, Federici AB, Goodeve A, Goudemand J, Mannucci PM, Mourik M, Önundarson PT, Rodeghiero F, Szántó T, Windyga J Fifth Åland Island conference on von Willebrand disease Haemophilia, 24(Suppl.4): 5-19, 2018 465.De Stefano V, Rocca B, Tosetto A, Soldati D, Petrucci G, Beggiato E, Bertozzi I, Betti S, Carli G, Carpenedo M, Cattaneo D, Cavalca V, Dragani A, Elli E, Finazzi G, Iurlo A, Lanzarone G, Lissandrini L, Palandri F, Paoli C, Rambaldi A, Ranalli P, Randi MG, Ricco A, Rossi E, Ruggeri M, Specchia G, Timillero A, Turno L, Vianelli N, Vannucchi AM, Rodeghiero F, Patrono C The aspirin regimens in essential thrombocythemia (ARES) phase II randomized trial design: implementation of the serum thromboxane B2 assay as an evaluation tool of different aspirin dosing regimens in the clinical setting Blood Cancer J, 8(6): 49, 2018 466.Schifferli A, Holbro A, Chitlur M, Coslovsky M, Imbach P, Donato H, Elalfy M, Graciela E, Grainger J, Holzhauer S, Riccheri C, Rodeghiero F, Ruggeri M, Tamary H, Uglova T, Wu R, Kühne T; Intercontinental Cooperative ITP Study Group (ICIS). A comparative prospective observational study of children and adults with immune thrombocytopenia: 2-year follow-up Am J Hematol, 93(6): 751-59, 2018 467.Rodeghiero F, Pecci A, Balduini CL Thrombopoietin receptor agonists in hereditary thrombocytopenias J Thromb Haemost, 16(9): 1700-10, 2018 468.Rodeghiero F, Woszczykb D, Slamac B, Melikyan A, Viallarde J, Ouajaf R, Ousmane A C, Salamag A Efficacy and safety of IQYMUNE®, a ten percent intravenous immunoglobulin in adult patients with chronic, primary immune thrombocytopenia J Hematol, 7(3): 87-95, 2018 469.Kuter DJ, Newland A, Chong BH, Rodeghiero F, Romero MT, Pabinger I, Chen Y, Wang K, Mehta B, Eisen M Romiplostim in adult patients with newly diagnosed or persistent immune thrombocytopenia (ITP) for up to 1 year and in those with chronic ITP for more than 1 year: a subgroup analysis of integrated data from completed romiplostim studies Br J Haematol, 185(3): 503-13, 2019

470.Ghanima W, Cooper N, Rodeghiero F, Godeau B, Bussel JB Trombopoietin receptor agonists: ten years later Haematologica, 104(6): 1112-1123, 2019

15/12/2020 European Medicine Agency Page 43/46 471.Efficace F, Breccia M, Avvisati G, Cottone F, Intermesoli T, Borlenghi E, Carluccio P, Rodeghiero F, Fabbiano F, Luppi M, Romani C, Sborgia M, DArdia S, Nobile F, Cantore N, Crugnola M, Nadali G, Vignetti M, Amadori S, Lo Coco F Health-related quality of life, symptom burden, and comorbidity in long-term survivors of acute promyelocytic leukemia Leukemia, 33(7): 1598-1607, 2019 472.Barbui T, De Sefano V, Falanga A, Finazzi G, Martinelli I, Rodeghiero F, Vannucchi AM, Barosi G Addressing and proposing solutions for unmet clinical needs in the management of myeloproliferative neoplasm-associated thrombosis: A consensus-based position paper Blood Cancer Journal, 9, 2019 473.Rodeghiero F, Pabinger I, Ragni M, Abdul-Kadir R, Berntorp E, Blanchette V, Bodò I, Casini A, Gresele P, Lassila R, Leebeek F, Lillicrap D, Mezzano D, Noris P, Srivastava A, Tosetto A, Windyga J, Zieger B, Makris M, Key N Fundamentals for a systematic approach to mild and moderate inherited bleeding disorders: an EHA consensus report HemaSphere, 3:5, e286, 2019 474.Provan D, Arnold DM, Bussel JB, Chong BH, Cooper N, Gernsheimer T, Ghanima W, Godeau B, González-López TJ, Grainger J, Hou M, Kruse C, McDonald V, Michel M, Newland AC, Pavord S, Rodeghiero F, Scully M, Tomiyama Y, Wong RS, Zaja F, Kuter DJ Updated International consensus report on the investigation and management of primary immune thrombocytopenia Blood Adv, 3(22): 3780-3817, 2019 475.Visco C, Rodeghiero F, Romano A, Valeri F, Merli M, Quaresemini G, Volpetti S, Santi RM, Carli G, Lucchini L, Passamonti F, Rambaldi A, Motta G, Borchiellini A, dAmore E, Ruggeri M Eltrombopag for immune thrombocytopenia secondary to chronic lymphoproliferative disorders: a phase 2 multicenter study Blood, 134(20): 1708-11, 2019 476.Palandri F, Santoro C, Carpenedo M, Cantoni S, Barcellini W, Carli G, Carrai V, Rossi E, Rivolti E, Lucchesi A, Rotondo F, Baldacci E, Auteri G, Sutto E, Di Pietro C, Catani L, Bartoletti D, De Stefano V, Ruggeri M, Mazzucconi MG, Cavo M, Rodeghiero F, Vianelli N Management of elderly patients with immune thrombocytopenia: Real-word evidence from 451patients older than 60 years Thromb Res, 185: 88-95, 2019 477.Rodeghiero F, Marranconi E Management of immune thrombocytopenia in women: current standards and special considerations Expert Rev Hematol, 2020 (EPUB) 478.Kuter DJ, Arnold DM, Rodeghiero F, Janssens A, Selleslag D, Bird R, Newland A, Mayer J, Wang K, Olie R Safety and efficacy of self-administered romiplostim in patients with immune thrombocytopenia: results of an integrated data base of five clinical trials Am J Hematol, 95: 643-651, 2020 479.Rocca B, Tosetto A, Betti S, Soldati D, Petrucci G, Rossi E, Timillero A, Cavalca V, Porro B, Iurlo A, Cattaneo D, Bucelli C, Dragani A, Di Ianni M, Ranalli P, Palandri F, Vianelli N, Beggiato E, Lanzarone G, Ruggeri M, Carli G, Elli EM, Carpenedo M, Bertozzi I, Paoli C, Randi ML, Ricco A, Specchia G, Vannucchi AM, Rodeghiero F, Patrono C, De Stefano V. A randomized, double-blind trial of three aspirin regimen to optimized antiplatelet therapy in essential thrombocythemia Blood, 136(2): 171-82, 2020 480.Breccia M, Vignetti M, Annibali O, Cottone F, Luppi M, Borlenghi E, Rodeghiero F, Efficace F

15/12/2020 European Medicine Agency Page 44/46 Younger age at diagnosis of acute promyelocytic leukaemia is associated with better long-term cognitive functioning Br J Haematol (Epub) 481.Sommer K, Vignetti M, Cottone F, Breccia M, Annibali O, Luppi M, Intermesoli T, Borlenghi E, Carluccio P, Rodeghiero F, Fabbiano F, Romani C, Sborgia M, Martino B, Crugnola M, Efficace F Acute promyelocytic leukaemia long-term survivors: higher fatigue and greater overall sympton burden BMJ Support Palliat Cure (Epub)

Projects

Memberships He has been member of the Board of the European Hematology Association (EHA) and of the EHA Corporate Sponsor Committee from June 2013 to June 2017. He established the Working Group on Thrombocytopenias within the European Hematology Association, of which he still is member of the Executive Committee. He served as a member of the executive committee of the Italian Society of Hematology (years 1994-1997). He has been Chairman-elect of the Italian Society for the Study of Thrombosis and Haemostasis (SISET) from 2008 to 2010; Chairman from 2010 to 2012 and is currently Past-Chairman. He has been a member of the Subcommittee on vWF of the Scientific and Standardization Committee of the ISTH for the years 1985-1995; co-chairman for the years 1995-1997. Chairman of the same Subcommittee from 1997 to 2000. He has been a member of the the Scientific and Standardization Committee (SSC) of the International Society on Thrombosis and Hemostasis (ISTH) from 1999 to 2002; Chairman-elect of the Scientific and Standardization Committee of ISTH from 2000 to 2002, Chairman from 2002 to 2004 and Special Assistant to the Chairman from 2004 to 2006. As director of the research laboratory, he has participated in several international collaborative studies for calibration of Factor VIII/vWF activities in cooperation with the National Institute for Biological Standards and Control in United Kingdom and with the WHO. He is member of several national and international scientific societies including International Society on Thrombosis and Haemostasis, European Hematology Association, American Society of Hematology and serves as reviewer for several major hematological journals. He is Associate Editor of The American Journal of Hematology since January 2007. He is Associate Editor of The Journal of Clinical Investigations since October 2009. He is member of the Editorial Board of Haematologica/The Haematology Journal since January 2008. He is Section Editor for Hemostasis in the journal Drugs and therapies in haematology since March 2012. He is Associate Editor of Haematologica since January 2020. He is Associate Editor of eJHaem since January 2020. Hes Chairman of the Thrombocytopenias and Anemias Working Party of the Italian Group for Malignant Hematological Disorders in the Adult (GIMEMA). Member of the Scientific Committee of the GIMEMA Onlus Foundation from 2010 to 2015. Member of the Scientific Committee of the Italian Association against Leukemia-Lymphoma and Myeloma (AIL) since March 2013.

Other Relevant Information Associate Editor of The Journal of Clinical Investigations since October 2009. Section Editor for Hemostasis of the journal Drugs and therapies in haematology since March 2012. Member of the Advisory Board of The International Monitor on Hemophilia" He is member of the Editorial Board of Haematologica/The Haematology Journal since January 2008. Member of the Advisory Board of Haematologica, the official journal of the Italian Society of Hematology, from 1990 to 1998.

15/12/2020 European Medicine Agency Page 45/46 Associate Editor of The American Journal of Hematology from January 2007 to December 2019. He has been member of the Advisory Board of Haematologica, the official journal of the Italian Society of Hematology, from 1990 to 1998. He is Associate Editor of Haematologica since January 2020. He is Associate Editor of the eJHaem since January 20202. He has been conducting clinical research in the field of hematology and hemostasis since early seventies, has published as first or co-author 434 papers (indexed in Medline November 21, 2016). My main interests include hemophilia, von Willebrand disease, thrombocytopenia, thrombophilia, rare bleeding disorders, acute promyelocytic leukemia, myeloma, myeloproliferative disorders and the epidemiological aspects of haematological diseases.

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