ONLINE eevventent th th 14 18 SEPTEMBER 2020 (CEST) PRESIDENT Joan Bladé Hospital Clínic. Barcelona, Spain www.isa2020.org 2 International Symposium on Amyloidosis WELCOME LETTER On behalf of the international Society of Amyloidosis (ISA) and the PETHEMA Foundation it is my pleasure to invite you to the XVII ISA Symposium. It will be held from September 14th to 18th, 2020 in an on-line format. It was planned for March 1-5 in Tarragona as a regular meeting, but it was postponed and later transformed at the virtual form because of the COVID-19 pandemic. The Symposium will consist of a five days scientific event. All types of amyloidosis will be covered: TTR (hereditary and wild-type), light chain (AL), AA as well as less frequent forms. There will be key lectures, plenary sessions, industry sponsored symposia, oral presentations plus meet-the-expert sessions and poster exhibition. This Symposium is attended by all the outstanding researchers in all the fields of amyloidosis plus about 800 delegates particularly interested in these diseases. The ISA 2020 is a unique opportunity to learn on basic biology, early diagnosis and recognition of unusual forms, best prognostication and response assessment, new drug targets and the role of novel agents, including immunotherapy, as well as the updated results from early phase and large randomized clinical trials. Please join us for this exciting conference on amyloidosis. Welcome to ISA 2020!!! Joan Bladé President ISA Symposium 2020 Hematology Department, Hospital Clinic. Barcelona Spain International Symposium on Amyloidosis 3 COMMITTEES Principal Organizers of the XVII International Symposium on Amyloidosis : • Joan Bladé, President. Barcelona, Spain • Josep Mª Campistol, Vicepresident 1. Barcelona, Spain • Alfonso Santiago, Vicepresident 2. Madrid, Spain • Mª Teresa Cibeira, Barcelona, Spain • Carlos Fernández de Larrea Rodríguez, Barcelona, Spain • Luis Quintana, Barcelona, Spain Board of Directors of ISA: • Giovanni Palladini, President and Representative of the Italian Society. Pavia, Italy • Stefan Schönland, President Elect and Representative of the German Society. Heidelberg, Germany • Vaishali Sanchorawala, Secretary. Massachusetts, U.S.A. • Shaji Kumar, Treasurer. Rochester, U.S.A. • Yukio Ando, Past President and Representative of the Japanese Society. Nagasaki, Japan • Per Westermark, Chair of the Nomenclature Committee and Editor-in-Chief of AMYLOID. Uppsala, Sweden • Giampaolo Merlini, Representative of the Italian Society. Pavia, Italy • Ute Hegenbart, Member at Large. Heidelberg, Germany • Paolo Milani, Member at Large. Pavia, Italy • Ashutosh Wechalekar, Member at Large. London, U.K. 4 International Symposium on Amyloidosis COMMITTEES Board of Directors of PETHEMA: • Joaquín Díaz Mediavilla, HonoraryPresident. Madrid, Spain • José Mª Ribera Santasusana, President. Barcelona, Spain • Joan Bladé Creixenti, Vicepresident. Barcelona, Spain • Juan José Lahuerta Palacios, Secretary. Madrid, Spain • José Francisco Tomás Martínez, Treasurer. Madrid, Spain • Jesús F. San Miguel Izquierdo, Chair. Pamplona, Spain • Miguel Ángel Sanz Alonso, Chair. Valencia, Spain • Jorge Sierra Gil, Chair. Barcelona, Spain • Albert Oriol Rocafiguera, Chair. Barcelona, Spain • Pau Montesinos Fernández, Chair. Valencia, Spain • Enrique María Ocio San Miguel, Chair. Santander, Spain • Jordi Estapé Rodríguez, Honorary Chairs. Barcelona, Spain • Emilio Montserrat Costa, Honorary Chairs. Barcelona, Spain • Luis Hernández Nieto, Honorary Chairs. Santa Cruz de Tenerife, Spain • José García Laraña, Honorary Chairs. Madrid, Spain • International Scientific Committee: • Arnaud Jaccard. Limoges, France • Ashutosh Wechalekar. London, UK • Bouke PC Hazenberg. Groningen, The Netherlands • Claudio Rapezzi. Bologna, Italy • David Adams. Paris, France • Efstathios Kastritis. Athens, Greece • Eli Muchtar. Rochester, USA • Gunilla Westermark. Uppsala, Sweden • Heather Landau. New York, USA • Joel N. Buxbaum. La Jolla, USA • John Berk. Boston, USA International Symposium on Amyloidosis 5 COMMITTEES • Julian Gillmore. London, UK • Laura Obici. Pavia, Italy • Marina Ramírez-Alvarado. Rochester, USA • Martha Skinner. Boston, USA • Merrill D Benson. Indianapolis, USA • Mitsuharu Ueda. Kumamoto, Japan • Morie Gertz. Rochester, USA • Ole Suhr. Umea, Sweden • Philip N. Hawkins. London, UK • Raymon Comenzo. Boston, USA • Robert A. Kyle. Rochester, USA • Rodney H. Falk. Boston, USA • Teresa Coelho. Porto, Portugal • Vaishali Sanchorawala. Boston, USA • Yoshiki Sekijima. Matsumoto, Japan Local Organizing Committee: • Alberto Martínez Vea. Tarragona, Spain • Antoni Raventós Estellé. Tarragona, Spain • Carlos Fernández de Larrea Rodríguez. Barcelona, Spain • Clara Salas Anton. Madrid, Spain • Jordi Colmenero Arroyo. Barcelona, Spain • Esther Gonzalez García. Gijón, Spain • Esther Gonzalez López. Madrid, Spain • Felipe de Arriba de la Fuente. Murcia, Spain • Ferrán Martínez Valle. Barcelona, Spain • Francisco Muñoz Beamut. Huelva, Spain • Inés Losada López. Palma Mallorca, Spain • Isabel Krsnik Castelló. Madrid, Spain 6 International Symposium on Amyloidosis COMMITTEES • Jesús San Miguel Izquierdo. Pamplona, Spain • Joan Buades Reinés. Palma de Mallorca, Spain • Jordi Yagüe Ribes. Barcelona, Spain • José Tomás Ortiz Pérez. Barcelona, Spain • Joseba Barcena Llona. Bizkaia, Spain • Josep M. Campistol Plana. Barcelona, Spain • Josep Sarrà Escarré. Tarragona, Spain • Juan González Moreno. Palma Mallorca, Spain • Juan Ignacio Aróstegui Gorospe. Barcelona, Spain • Juan José Lahuerta Palacios. Madrid, Spain • Laura Rosiñol Dachs. Barcelona, Spain • Lourdes Escoda Teigell. Tarragona, Spain • Lucía Galán Dávila. Madrid, Spain • Luis Quintana Porras. Barcelona, Spain • Manel Solé Arqués. Barcelona, Spain • María Casanovas Espinosa. Málaga, Spain • Maria Eugenia Cisneros Barroso. Palma de Mallorca, Spain • María-Victoria Mateos Manteca. Salamanca, Spain • María Teresa Cibeira López. Barcelona, Spain • Pablo García-Pavía. Madrid, Spain • Rafael Ríos Tamayo. Granada, Spain • Ramón Lecumberri Villamediana. Pamplona, Spain • Sebastián Fernando Garzón López. Cádiz, Spain • Sonia González Péres. Santiago de Compostela, Spain • Sunil Lakhwani Lakhwani. Canarias, Spain • Tomàs Ripoll Vera. Palma Mallorca, Spain International Symposium on Amyloidosis 7 SPONSORS PLATINUM Akcea Therapeutics Alnylam Pharmaceuticals Janssen Oncology Pfizer GOLD Eidos Thanks to the unrestricted grant of: Celgene | A Bristol-Myers Squibb Company 8 International Symposium on Amyloidosis International Symposium on Amyloidosis 9 ORAL AND POSTER PRESENTATIONS ORAL PRESENTATIONS INDEX ORAL PRESENTATIONS OP01 – OP40 BASIC SCIENCE I: OP01 – OP06 OP01 - HIGH RESOLUTION CRYO-EM STRUCTURE OF A TRANSTHYRETIN-DERIVED AMYLOID FIBRIL FROM A PATIENT WITH HEREDITARY VAL30MET ATTR AMYLOIDOSIS 34 OP02 - DEFINING THE CARDIAC AMYLOID PROTEOME AND ITS ASSOCIATION WITH PATIENT CLINICAL CHRACTERISTICS AND OUTCOMES 36 OP03 - IMMUNOGENETIC PROFILE OF PURIFIED PATHOLOGICAL PLASMA CELLS OF PATIENTS WITH LIGHT CHAIN AMYLOIDOSIS 37 OP04 - FROM PROTEIN-PROTEIN INTERACTION TO PROTEIN CO-EXPRESSION NETWORKS: A SYSTEMS BIOLOGY-BASED PERSPECTIVE TO INVESTIGATE AMYLOIDOSIS DISEASES 39 OP05 - TARGETING DEUBIQUITYLATING ENZYMES USP14 AND UCHL5 IN SYSTEMIC IMMUNOGLOBULIN LIGHT CHAIN (AL) AMYLOIDOSIS 40 OP06 - MEMBRANE AND SOLUBLE B-CELL MATURATION ANTIGEN (BCMA) IN SYSTEMIC LIGHT-CHAIN AMYLOIDOSIS 41 ATTR AMYLOIDOSIS: OP07 – OP15 OP07 - SKIN BIOPSY IN HEREDITARY TRANSTHYRETIN AMYLOIDOSIS WITH POLYNEUROPATHY IN FRANCE 42 OP08 - LONG-TERM SAFETY AND EFFICACY OF PATISIRAN: GLOBAL OPEN-LABEL EXTENSION 24-MONTH DATA IN PATIENTS WITH HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS 43 OP09 - LONG-TERM IMPACT OF TAFAMIDIS IN PATIENTS WITH LATE-ONSET HEREDITARY TRANSTHYRETIN AMYLOIDOSIS WITH STAGE I POLYNEUROPATHY 44 OP10 - EXTERNAL VALIDATION OF THE NATIONAL AMYLOIDOSIS CENTRE SCORE IN AN INTERNATIONAL COHORT OF PATIENTS WITH TRANSTHYRETIN CARDIAC AMILOIDOSIS. 45 OP11 - EVALUATION OF PATISIRAN WITH CONCOMITANT OR PRIOR USE OF TRANSTHYRETIN STABILIZERS IN PATIENTS WITH HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS 46 OP12 - OPEN-LABEL STUDY OF PATISIRAN IN PATIENTS WITH HEREDITARY TRANSTHYRETIN- MEDIATED AMYLOIDOSIS WITH POLYNEUROPATHY POST-ORTHOTOPIC LIVER TRANSPLANT 47 OP13 - HIGH RESOLUTION NERVE ULTRASOUND AS A DIAGNOSTIC TOOL FOR DIFFERENTIAL DIAGNOSIS AND PROGRESSION RECOGNITION IN TTR-RELATED FAMILIAL AMYLOIDOSIS 48 OP14 - ORIGIN OF VAL30MET IN FAMILIAL AMYLOID POLYNEUROPATHY (TTR-FAP) IN PORTUGAL: A WALK THROUGH THE MUTATIONAL PATH 49 OP15 - 99MTC-DPD SCINTIGRAPHY PREDICTS AMYLOID FIBRIL TYPE IN HEREDITARY TRANSTHYRETIN AMYLOIDOSIS 50 BASIC SCIENCE II: OP16 – OP20 OP16 - HEPATIC EXPRESSION OF MUTANT TRANSTHYRETIN REMODELS PROTEOSTASIS MACHINERY IN HEREDITARY ATTR AMYLOIDOSIS 51 OP17 - DIAGNOSTIC POTENTIAL OF A NOVEL RT-qPCR-BASED ASSAY TO MEASURE CCND1 mRNA EXPRESSION LEVELS IN BONE MARROW PLASMA CELLS FROM AL AMYLOIDOSIS PATIENTS 52 OP18 - MACHINE LEARNING PREDICTS IMMUNOGLOBULIN LIGHT CHAIN TOXICITY THROUGH SOMATIC MUTATIONS 53 International Symposium on Amyloidosis 11 ORAL PRESENTATIONS INDEX OP19 - DROSOPHILA MELANOGASTER AS A MODEL ORGANISM FOR ATTR AMYLOIDOSIS 54 OP20 - ELEVEN DIFFERENT AMYLOID TYPES IDENTIFIED IN CUTANEOUS AMYLOIDOSIS BY PROTEOMICS-BASED TYPING 55 AL AMYLOIDOSIS I: OP21 – OP26 OP21 - NEW ORGAN RESPONSE CRITERIA FOR LIGHT CHAIN AMYLOIDOSIS: AN INTERNATIONAL VALIDATION STUDY 56 OP22 - THE QUEST FOR INDICATORS OF PROFOUND HEMATOLOGIC RESPONSE IN AL AMYLOIDOSIS: COMPLETE RESPONSE REMAINS