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ROUND TABLE: Diagnosis and Management of Acquired Aplastic Anemia

ROUND TABLE: Diagnosis and Management of Acquired Aplastic Anemia

05/07/2022 13:00

Speakers: Régis Peffault de Latour, Andrea Bacigalupo, Austin Kulasekararaj, Beatrice Drexler, Britta Höchsmann

Target: Health professionals
Subnetworks: Bone marrow failure
Disease Groups: Paroxysmal nocturnal hemoglobinuria, Rare acquired aplastic anemia (Other than PNH)

Prof. Peffault De La Tour obtained his PhD in Medicine from the University of Paris - Lariboisière in 2003 and his "Doctorat d'Etat" in Immunology from the Pasteur Institute in 2006. He trained in Hematology at Paris Hospitals AP-HP and did his postdoctoral fellowship at the National Institutes of Health, USA, from 2008 to 2010. He obtained his position as Full Professor of Clinical Hematology in 2014.

He is currently working in the Hematology and Bone Marrow Transplant Department of AP-HP Hôpital Saint-Louis, Paris, France. He is in charge of the reference center for Aplastic Anemia and Nocturnal Paroxysmal Hemoglobinuria (PNH) and the French network on Hematology-immunology rare diseases (MaRIH). He is also coordinator of the Aplastic Anemia Working Group of the European Society for Blood and Marrow Transplantation (EMBT). He is coordinator of the Bone Marrow Failures Subnetwork for the ERN-EuroBloodNet.

His research interests include bone marrow transplantation, bone marrow failure and paroxysmal nocturnal hemoglobinuria. He is the author of over 200 articles cited in PubMed and is a regular reviewer for journals such as The New England Journal of Medicine, Blood, Haematologica, British Journal of Haematology, Biology of Blood and Marrow Transplantation and Bone Marrow Transplantation.

Andrea Bacigalupo has been the Head of Hematology at the San Martino Hospital Genova Italy (1990-2014), then at Gemelli -Catholic University Rome (2015-2019) and is currently a Consultant at Gemelli Hospital in Rome. He has been involved in stem cell transplants since 1976, and has played an active role in the European Group for Blood and Marrow Transplants (EBMT) since 1977. He was Secretary of EBMT 1982-1986, chairperson of the SAA WP 1984-1990 and President of EBMT 1998-2002. He has conducted several prospective clinical trials in patients with aplastic anemia and in patients undergoing allogeneic transplants: in particular, he has been the principal investigator of several prospective randomized studies in the field of GvHD. His main interests are bone marrow failure, GvHD and related complications. He is a member of several national and international hematology societies. He is Associate Editor of the journal Bone Marrow Transplantation and Biology of Blood and Marrow Transplantation and has published over 600 articles in peer-reviewed journals. His current H index is 100.

Professor Austin G Kulasekararaj is a Consultant Haematologist and Honorary Senior Clinical Lecturer at King's College Hospital in London.

His Clinical Interests are Aplastic anaemia and bone marrow failure syndromes, PNH, MDS and myeloid malignancies. He leads the King's National Paroxysmal Nocturnal Haemoglobinuria service.

Passionate about the delivery of clinical trials, he is Coordinating Investigator for a number of early/late phase clinical trials. His current drive is to bring novel therapies for PNH, AA, TP53 mutated myeloid neoplasms and the improvement of thrombocytopenia.

His Translational Research work is about molecular/immunological pathogenesis of MDS and aplastic anaemia, particular focus on overlap disorders. He is Bloodwise foundation and British Society of Haematology (BSH) senior clinical research fellow.

He is also scientific advisor for patient advocacy group (MDS UK) and member of the MDS NCRN working group, member of the European Society for Blood and Marrow Transplantation Severe Aplastic Anaemia Working Party and member of the ASH Scientific Committee on Bone Marrow Failure.

He has authored/co-authored over 130 articles in peer-reviewed journals, and he has contributed to book chapters in major textbooks.

Prof. Beatrice Drexler is an attending physician at the Hematology Department of the University Hospital Basel, Switzerland, responsible for the posttransplant and bone marrow failure outpatients. Her research focuses on clinical aspects of bone marrow failure syndromes and paroxysmal nocturnal hemoglobinuria. She is an active member of the Severe Aplastic Anemia Working Party (SAAWP) of the EBMT.

Dr. Höchsmann studied medicine and awarded her medical degree at the University of Ulm, Germany. She spent parts of her medical training at the Tom Baker Cancer Centre, Health Sciences Centre, Faculty of Medicine, University of Calgary, Canada.

Her appointment as medical specialist for Internal Medicine was in 2004, as medical specialist for Haematology /Internal Oncology in 2005, as medical specialist for Palliative Care in 2010 and her appointment as medical specialist for Transfusion medicine in 2012.

Since 2004 she is working at the Institute of Clinical Transfusion Medicine and Immunogenetics, Department of Transfusion Medicine of the University UIm & German Red Cross Blood Transfusion Service Baden-Württemberg/Hessen with the focus on rare cytopenias and anaemias as well as clinical haemotherapy.

She is responsible for the specialist outpatient clinic regarding rare cytopenias and anaemias, for flow cytometric GPI-AP diagnostics including the German round robin tests regarding diagnosis of paroxysmal nocturnal haemoglobinuria (PNH) and research projects regarding rare cytopenias, complement and haemotherapy. Since 1998 she is investigator for several randomized and not-randomized multicentre studies regarding these topics. Especially she is the coordinating investigator of the prospective randomized EMAA-trial evaluating efficacy and safety of eltrombopag in combination with ciclosporin in patients with acquired moderate aplastic anaemia.

She is actively involved in the WP-SAA of the society of European bone marrow transplantation (EBMT), the working party "non-malignant haematology" of the German society of haematology and oncology (DGHO) and the Global PNH patient registry. She is a member of the writing comitee of the German guidelines for aplastic anemia of the DGHO (German Society of Haematology and Oncology) as well as for the guidelines for PNH of the DGHO and the German & Austrian consensus recommendations for the diagnosis of PNH.

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