66th ASH Annual Meeting: 331. Thrombotic Microangiopathies/Thrombocytopenias: Clinical and Epidemiological: Poster III

Symposia: Thrombotic Microangiopathies/Thrombocytopenias: Clinical and Epidemiological Oral and Poster Abstracts
Poster

Research, Bleeding and Clotting, Combination therapy, Adult, Clinical Practice (Health Services and Quality), Genetic Disorders, Clinical Research, Pediatric, Diseases, Thrombocytopenias, Therapy sequence, Treatment Considerations, Thrombotic disorders, Real-world evidence, Registries, Young adult , Study Population, Human

Halls G-H (San Diego Convention Center)

3999

Epidemiology of Hereditary Thrombotic Thrombocytopenic Purpura (TTP) in Spain: Preliminary Results of a Study of the Spanish TTP Registry (REPTT)

Inés Gómez Seguí¹^*, Maria Cristina Pascual Izquierdo, MD, PhD²^*, Maria Eva Mingot³^*, Amanda Lopez Picado^4,5^*, Guillermo Sanz, MD, PhD¹, Javier de la Rubia¹^*, Ramon Salinas Argente, MD⁶^* and Julio Del Rio Garma, MD⁷^*

¹Hematology Department, Hospital Universitari i Politècnic La Fe, Valencia, Spain
²Servicio de Hematología, Hospital Universitario Gregorio Marañón, Instituto de Investigación Gregorio Marañón, Madrid, Madrid, Spain
³Servicio de Hematología, Hospital Universitario Virgen del Rocío, Malaga, Spain
⁴Facultad de Medicina, Universidad de Deusto, Bilbao, Spain
⁵Sociedad Española de Hematologia, Madrid, Spain
⁶Universidad Internacional de Cataluña, Barcelona, Spain
⁷Hospital Clinico Santiago de Compostela, Coruña, ESP

4000

External Validation of the French Score in Thrombotic Microangiopathies: Real-World Experience in a Large Retrospective Cohort of 1483 Patients

Alicia Cruz Gómez, MD¹^*, María Martínez Rodríguez²^*, Andrés Felipe Melo Arias³^*, Clara Fernández Arias⁴^*, Olatz Olabarri Etxebarria⁵^*, Laura Sánchez Calvo⁶^*, Maria José de la Osa Velacoracho⁶^*, Kenneth Herrera Robles⁷^*, Ana Tobalina García⁸^*, Stefania Carvajal Altamiranda⁹^*, Jorge Martínez Nieto¹⁰^*, Ana Villegas, MD³, Alejandro Sánchez Saito¹¹^*, Miguel Gómez Álvarez³^*, Sara Ferrer¹⁰^*, Rafael López del Amo³^*, Paloma Ropero Gradilla³^*, Celina Benavente Cuesta³^*, Fernando Ataulfo González Fernández³^* and Gonzalo Del Campo Balguerías, MD³^*

¹Hospital Universitario de Getafe, Madrid, Spain
²Hospital Universitario de Móstoles, Madrid, Spain
³Hospital Clínico San Carlos, Madrid, Spain
⁴Clínica Universitaria de Navarra, Pamplona, Spain
⁵Hospital Galdakao, Bilbao, Spain
⁶Hospital Universitario de la Princesa, Madrid, Spain
⁷Hospital Clínico Universitarios de Valladolid, Valladolid, AL, ESP
⁸Hospital Universitario Marqués de Valdecilla, Santander, Spain
⁹Hospital Universitario de Segovia, Segovia, Spain
¹⁰Instituto de Investigación Sanitaria San Carlos (IdISSC), Madrid, Spain
¹¹Hospital Universitario Infanta Leonor, Madrid, Spain

4001

Caplacizumab in Immune-Mediated Acquired Thrombotic Thrombocytopenic Purpura: A Systematic Literature Review and Meta-Analyses of Clinical Trials and Observational Studies

Paul Coppo, MD¹^*, Marie Scully, MD², Agathe Nevière³^*, Pauline Le Nouveau³^*, Prerna Patel⁴^*, Alaeddine Sidhom⁵^*, Srushhti Trivedi⁶^*, Rachel Chu⁷^*, Da Eun Ahn⁴^*, Gabriela Marcheva⁸^*, Divyesh Thakker⁹^*, Gaye Siliman¹⁰^* and Alix Arnaud⁸^*

¹Department of Hematology, Reference Center for Thrombotic Microangiopathies, Saint-Antoine University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
²University College London, London, ENG, United Kingdom
³HEMA, Amaris Consulting, Nantes, France
⁴HEMA, Amaris Consulting, London, United Kingdom
⁵HEMA, Amaris Consulting, Barcelona, Spain
⁶HEMA, Amaris Consulting, Toronto, ON, Canada
⁷HEMA, Amaris Consulting, Montreal, QC, Canada
⁸HEVA, Sanofi, Boston, MA
⁹Sanofi Global Hub, Sanofi, Hyderabad, Telangana, India
¹⁰HEVA, Sanofi, Mississauga, ON, Canada

4002

Mutation Analysis of the ADAMTS13 Gene in Patients with Congenital Thrombotic Thrombocytopenic Purpura from the rADAMTS13 Phase 3 Study

Dorothee Diogo, PhD¹^*, Pinghai Zhang¹^*, Wolf-Achim Hassenpflug, MD²^*, Björn Mellgård¹, Parth Patwari, MD, PhD¹^*, Wei Qu³^* and Linda T. Wang¹^*

¹Takeda Development Center Americas, Inc., Cambridge, MA
²Department of Pediatric Hematology and Oncology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany
³Takeda Pharmaceuticals Company Limited, Fujisawa, Japan

4003

Daratumumab for Refractory and Frequently Relapsing Immune Thrombotic Thrombocytopenic Purpura – a Case Series with Long-Term Follow-up

Roman R. Schimmer, MD¹^*, Jana Van Den Berg, MD²^*, Marissa Schraner, PhD³, Alice Trinchero, MD¹^*, Andreas Holbro, MD², Johanna A. Kremer Hovinga, MD⁴ and Jan-Dirk Studt, MD¹^*

¹Department for Medical Oncology and Hematology, University Hospital Zurich and University of Zurich, Zurich, Switzerland
²Division of Hematology, University Hospital Basel, Basel, Switzerland
³Department of Hematology and Central Hematology Laboratory, Inselspital Bern, University Hospital Bern and University of Bern, Bern, Switzerland
⁴Department of Hematology and Central Hematology Laboratory, Inselspital Bern, Bern University Hospital, University of Bern, Bern, Switzerland

4004

Caplacizumab Resistance in Immune Thrombotic Thrombocytopenic Purpura (TTP) Mediated By VWF A1 Domain Missense Mutation

Mari Thomas^1,2^*, Imogen Buckle³^*, Adela Constantinescu-Bercu, PhD⁴^*, Rens De Groot, PhD⁵^*, Arshia Latif³^*, Sabina McCann⁶^* and Marie Scully, MD^7,8

¹Department of Haematology, University College London Hospital, London, ENG, United Kingdom
²Cardiometabolic Program, National Institute for Health Research UCLH/UCL Biomedical Research Centre, London, United Kingdom
³UCL, London, United Kingdom
⁴UCL, London, GBR
⁵Institute Cardiovascular Science, UCL, London, GBR
⁶Special Coagulation, Health Services Laboratories, London, London, GBR
⁷University College London, London, ENG, United Kingdom
⁸Department of Haematology, University College London Hospitals, Cardiometabolic Program, National Institute for Health Research UCLH/UCL Biomedical Research Centre, London, United Kingdom

4005

The Quest for Effective and Well-Tolerated Treatment for Congenital TTP: Half a Century of Experience in the United Kingdom

Praveen Gounder¹^*, Louisa Keogh²^*, William Lester³^*, Amanda Clark⁴^*, Joost Vanveen⁵^*, Tina Dutt⁶^*, Michael Desborough, BSc, DPhil, FRCP, FRCPath⁷^*, Joannes Hermans⁸^*, Alice Taylor⁹^* and Marie Scully, MD¹⁰

¹Haematology Department, University College London Hospital, London, United Kingdom
²Hematology Department, University College London Hospital, London, United Kingdom
³University Hospital NHS Foundation Trust Birmingham, Birmingham, GBR
⁴Haematology Department, University Hospital Bristol, Bristol, United Kingdom
⁵Haemophilia & Thrombosis Centre, Royal Hallamshire Hospital, Sheffield, United Kingdom
⁶Liverpool University Hospitals NHS Foundation Trust, Liverpool, United Kingdom
⁷NHS Blood and Transplant, John Radcliffe Hospital, Oxford, United Kingdom
⁸Haematology Department, Nottingham University Hospital, Nottingham, United Kingdom
⁹Great Ormond Street Hospital for Children Foundation Trust, London, United Kingdom
¹⁰University College London, London, ENG, United Kingdom

4006

Pharmacodynamic Activity of Recombinant ADAMTS13 Versus Plasma-Based Therapies in Congenital Thrombotic Thrombocytopenic Purpura: Interim Results of a Phase 3 Randomized, Controlled, Open-Label Study

Munjal Patel^*, Björn Mellgård, Luying Pan^*, Linda T. Wang^*, Shan Xiao^* and Parth Patwari, MD, PhD^*

Takeda Development Center Americas, Inc., Cambridge, MA

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^*signifies non-member of ASH