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ROUND TABLE: Diagnosis and Management of Paroxysmal Nocturnal Hemoglobinuria

ROUND TABLE: Diagnosis and Management of Paroxysmal Nocturnal Hemoglobinuria

15/11/2022 13:00

Speakers: Antonio M Risitano, Carlo Dufour, Régis Peffault de Latour, Lucio Luzzato, Flore Sicre de Fontbrune, Morag Griffin

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

Prof. Antonio M Risitano is Director of Hematology and Hematopoietic Stem Cell Transplantation at the AORN San Giuseppe Moscati in Avellino, and Professor of Hematology at the Federico II University of Naples. After his education at the Federico II University of Naples, Prof. Risitano spent 3 years at the Hematology Branch of the National Institutes of Health in Bethesda, where he dedicated to experimental work on the pathophysiology of bone marrow failure syndromes, under the guidance of Dr. Neal Young. In 2004 he moved back to Naples where he served as Research Associate and then Assistant Professor in the Hematology unit, focusing paroxysmal nocturnal hemoglobinuria, aplastic anemia as well as of stem cell transplantation. From 2010 to 2020 he was Director of the bone marrow transplantation unit and Associate Professor of Hematology at the Federico II University, before being appointed as Head of Hematology in Avellino in July 2020. Prof. Risitano pioneered the development of novel treatments for aplastic anemia and paroxysmal nocturnal hemoglobinuria through seminal preclinical and clinical works.

Prof. Carlo Dufour works in G.Gaslini Research Children's Hospital, Genova, Italy, the largest, multi-specialist pediatric hospital of the country, where he chairs the Hemo-Onco- HSCT Pole and the Hematology Unit where have seat the national Registry of Neutropenia, the national Registry of the Alps and related disorders and the national Data Base of Fanconi Anemia. He founded the Marrow Failure Syndromes Study Group within the Italian Pediatric Hemato-Oncology Association (AIEOP). He is the author or co-author of over 200 international publications and of various hematology textbooks and textbook chapters. He acts as a reviewer for top ranking international journals including New England Journal of Medicine, Journal of Clinical Oncology, Leukemia, Clinical Immunology and Blood. He serves as an expert evaluator for different institutions including the French Registry for Rare Diseases, the Fanconi Anemia Research Fund INC (US), the Leukemia and Lymphoma Research Foundation (UK), the American Society of Hematology Education Program. He acted as the chairman of the Working Party of the Severe Aplastic Anemia (WPSAA) and as a Board member of the European Society for Blood and Marrow Transplantation (EBMT). He currently serves as the chairman of the Scientific Working Group on Granulocyte and Constitutional Marrow Failure Disorders of the European Hematology Association (EHA). Within EHA he is a member of the SWG Unit, of the Education Committee and of the Guidelines Committee. In 2015 he received by the Fanconi Anemia Research Fund. INC (FARF) the discovery award for participating in the identification of gene FANCT. Since December 2019 is a member of the Scientific Committee of the American Society of Hematology.

In December 2019 he was awarded for scientific contribution in Fanconi Anemia by the Spanish "Fundacion en la Anemia de Fanconi". In August 2020 has been awarded by the EBMT with the Van Bekkum award, a recognition reserved for best scientific communication of the year, as Co-PI of the prospective randomized trial RACE (Comparison of classical IST ± Eltombopag) in Severe Aplastic Anemia.

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.

Prof. Lucio Luzzatto qualified as MD in 1959 from the University of Genoa Medical School; he then trained in Haematology and has Libera Docenza (PhD) in Biochemistry. He has held academic positions in Nigeria, Italy, Great Britain, USA, Tanzania.

The leitmotiv of Lucio Luzzatto's research and teaching has been the understanding of human disease at the molecular level; particularly in the area of blood diseases, for the ultimate purpose to improve their management. Dr Luzzatto and colleagues cloned the human G6PD gene in the early eighties, thus laying foundations for the molecular pathophysiology and population genetics of G6PD and G6PD deficiency, as well as mechanisms of malaria selection. He discovered that paroxysmal nocturnal haemoglobinuria (PNH) is a clonal disorder, introduced the concept of conditional clonal selection and provided supporting evidence: thus rationalising the relationship of clonal expansion with bone marrow failure, and the treatment of PNH.

Dr Lucio Luzzatto holds several honorary degrees and has received awards and prizes from Learned Societies; he is a member of EMBO, of HUGO, honorary member of the American Society of Hematology, and member of the American Academy of Arts and Sciences. He has 430 articles listed in PubMed, has contributed chapters to major international textbooks, and has written a book for the general public, Capire il Cancro.

Prof. Flore Sicre de Fontbrune has a current position in the department of Adult Hematology Bone Marrow transplant at the AP-HP Hôpital Saint-Louis (Paris, France).

After finishing her M.Sc focused on Cellular Biology (AP-HP Hôpital Saint-Louis, Paris, France) and her M.D. in Clinical Hematology (Université Paris Descartes, Paris, France) had the opportunity to join the team of the Adult Hematology Unit at the AP-HP Hôpital Necker-Enfants Malades, Paris, France.

Dr Morag Griffin is the joint service lead of the National PNH service in Leeds. She has a specialist interest in PNH, aplastic anaemia and histiocytic disorders. Dr Griffin is the chief investigator and principal investigator of PNH clinical trials. Dr Griffin is a Member of the International PNH interest group, Severe aplastic anaemia working party. She has coursed a MSc in medical leadership and completed the EHA clinical research trials in the haematology programme.

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