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Research-Roadmap-Full.Pdf Published Ahead of Print on January 27, 2016, as doi:10.3324/haematol.2015.136739. Copyright 2016 Ferrata Storti Foundation. The European Hematology Association Roadmap for European Hematology Research: a consensus document by Andreas Engert, Carlo Balduini, Anneke Brand, Bertrand Coiffier, Catherine Cordonnier, Hartmut Döhner, Thom Duyvené de Wit, Sabine Eichinger, Willem Fibbe, Tony Green, Fleur de Haas, Achille Iolascon, Thierry Jaffredo, Francesco Rodeghiero, Gilles Salles, Jan Jacob Schuringa, and the other authors of the EHA Roadmap for European Hematology Research Haematologica 2016 [Epub ahead of print] Citation: Engert A, Balduini C, Brand A, Coiffier B, Cordonnier C, Döhner H, de Wit TD, Eichinger S, Fibbe W, Green T, de Haas F, Iolascon A, Jaffredo T, Rodeghiero F, Salles G, Schuringa JJ, 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. 2016; 101:xxx doi:10.3324/haematol.2015.136739 Publisher's Disclaimer. E-publishing ahead of print is increasingly important for the rapid dissemination of science. Haematologica is, therefore, E-publishing PDF files of an early version of manuscripts that have completed a regular peer review and have been accepted for publication. E-publishing of this PDF file has been approved by the authors. After having E-published Ahead of Print, manuscripts will then undergo technical and English editing, typesetting, proof correction and be presented for the authors' final approval; the final version of the manuscript will then appear in print on a regular issue of the journal. All legal disclaimers that apply to the journal also pertain to this production process. Roadmap for European hematology research OPINION ARTICLE The European Hematology Association Ferrata Storti Roadmap for European Hematology Research: EUROPEAN HEMATOLOGY Foundation a consensus document ASSOCIATION Andreas Engert,1 Carlo Balduini,2 Anneke Brand,3 Bertrand Coiffier,4 Catherine Cordonnier,5 Hartmut Döhner,6 Thom Duyvené de Wit,7 Sabine Eichinger,8 Willem Fibbe,3 Tony Green,9 Fleur de Haas,7 Achille Iolascon,10 Thierry Jaffredo,11 Francesco Rodeghiero,12 Gilles Salles,13 Jan Jacob Schuringa,14 and the other authors of the EHA Roadmap for European Hematology Research 1Universität zu Köln, Cologne, Germany; 2IRCCS Policlinico San Matteo Foundation, Pavia, Italy; 3Leids Universitair Medisch Centrum, Leiden, the Netherlands; 4Université Claude Bernard, Lyon, France; 5Hôpitaux Universitaires Henri Mondor, Créteil, France; 6Universitätsklinikum Ulm, Germany; 7European Hematology Association, The Hague, the Netherlands; 8Medizinische Universität Wien, Vienna, Austria; 9Cambridge Institute for Medical Research, United Kingdom; 10Università Federico II di Napoli, Italy; 11Université Pierre et Marie Curie, Paris, France; 12Ospedale San Bortolo, Vicenza, Italy; 13Hospices Haematologica 2016 Civils de Lyon/Université de Lyon, Pierre-Bénite, France; and 14Universitair Medisch Volume 101(2):115-208 Centrum Groningen, the Netherlands ABSTRACT he European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements Tin diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable bet- Correspondence: ter funded, more focused European hematology research. Initiated by [email protected] the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness Received: 15/12/2015. of the burden of blood disorders on European society, which purely in Accepted: 27/01/2016. economic terms is estimated at €23 billion per year, a level of cost that is not matched in current European hematology research funding. Pre-published: 27/01/2016. In recent decades, hematology research has improved our fundamental understanding of the biology of blood disorders, and has improved diag- doi:10.3324/haematol.2015.136739 nostics and treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap. Check the online version for the most updated information on this article, online supplements, The EHA Roadmap identifies nine ‘sections’ in hematology: normal and information on authorship & disclosures: hematopoiesis, malignant lymphoid and myeloid diseases, anemias and www.haematologica.org/content/101/2/115 related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders. ©2016 Ferrata Storti Foundation The EHA Roadmap identifies priorities and needs across the field of Material published in Haematologica is cov- hematology, including those to develop targeted therapies based on ered by copyright. All rights reserved to Ferrata genomic profiling and chemical biology, to eradicate minimal residual Storti Foundation. Copies of articles are malignant disease, and to develop cellular immunotherapies, combina- allowed for personal or internal use. A permis- sion in writing by the publisher is required for tion treatments, gene therapies, hematopoietic stem cell treatments, and any other use. treatments that are better tolerated by elderly patients. haematologica | 2016; 101(2) 115 Engert et al. Introduction Key milestones included the characterization of hemo- globin (Hb),7 induced pluripotent stem cells (iPSCs),8 and Blood can be described as one of the human body’s somatic driver mutations.9 The discovery of the largest organs. It is essentially a liquid tissue containing Philadelphia chromosome and the subsequent identifica- many different types of specialized cells needed for the tion of the BCR-ABL1 tyrosine kinase and its role in chron- normal functioning of the human body. When one or ic myeloid leukemia (CML)10 led to the successful develop- more of these cell types do not perform well, a wide vari- ment of potentially curative targeted treatment in this ety of blood disorders can result, ranging from blood can- form of blood cancer.11 This was an unprecedented rate of cers and coagulation and platelet disorders to very com- success and it occurred in a malignancy that previously mon diseases such as anemia. could only be treated by allogeneic transplant in a very Hematology is the medical discipline concerned with select number of patients. Acute promyelocytic leukemia diagnosing and treating all of these diseases. became one of the first malignancies that could be cured In the European Union (EU) alone, an estimated 80 mil- without conventional chemotherapy.12 lion people are currently affected with blood disorders. Another key development in hematology was that of a Various types of anemia affect more than 50 million wide range of monoclonal antibodies following the original children and adults in the World Health Organization’s invention by Köhler and Milstein in the UK.13 Humanized European region.1 Blood cancers, some of which mainly or fully human monoclonal antibodies are now used in affect young people, contribute strongly to premature can- hematology for both diagnostic and therapeutic purposes. cer-related mortality and lost productivity in Europe.2 The clinical breakthrough was a humanized monoclonal Among cancers, blood cancers [leukemia, Hodgkin and antibody targeting the CD20 antigen on B-cell lymphoma.14 non-Hodgkin lymphomas (HLs and NHLs), and multiple Today, monoclonal antibodies or antibody-based conju- myeloma] together rank third after lung cancer and col- gates are used successfully in most malignant lymphomas orectal cancer in terms of age-adjusted mortality in the and leukemias. They can, however, also be effective in non- European Economic Area.3 malignant blood disorders such as paroxysmal nocturnal Inherited blood diseases, such as thalassemia, sickle cell hemoglobinuria (PNH), a rare acquired clonal stem cell disease, and glucose-6-phosphate dehydrogenase deficien- defect leading to increased fragility of hematopoietic cells cy, also affect millions of people and cause substantial and hemolytic anemia (HA), thrombosis, and bone marrow morbidity and mortality. Rarer forms of congenital blood failure (BMF). Prognosis of patients with severe PNH used disorders represent an immense burden on those affected. to be less than five years, but changed radically with the Many infectious diseases affect various types of blood or advent of an anti-complement monoclonal antibody that blood-forming cells, causing widespread diseases such as counteracts membrane fragility.15 Today, PNH patients malaria and HIV/AIDS. treated with this antibody have a normal life expectancy. In recent decades, enormous progress has been made in Severe hemophilia represents another story of unprece- terms of diagnosis and treatment of these diseases. dented success. Patients used to be confined to wheel- Unfortunately, many blood disorders remain incurable. chairs or face the specter of death because of untreatable Approximately 115,000 patients die each year.4 hemorrhage or blood-born infections such as HIV/AIDS. Blood disorders have immense economic consequences Today, new recombinant substitutive therapy is complete- as well. The combined societal cost of
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