ROUND TABLE: Management of constitutional Bone Marrow Failure
ROUND TABLE: Management of constitutional Bone Marrow Failure
Speakers: Antonio M Risitano, Carlo Dufour, Régis Peffault de Latour, Camilla Frieri, Francesca Fioredda, Thierry Leblanc, Tim H. Brümmendorf
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
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.
Dr Camilla Frieri received her MD degree from the University of Rome "Sapienza", in Italy, in 2014. She specialized in Hematology at the University of Naples "Federico II" in 2019. In 2018 she began her PhD in Hematology, with a special focus on novel treatments in paroxysmal nocturnal hemoglobinuria under the supervision of the tutor Prof. Antonio Maria Risitano. During the three-year PhD course, she has carried out a period of international training at the Saint Louis Hospital under the direction of Prof Régis Peffault de Latour. Her basic and clinical research activities mainly focus on paroxysmal nocturnal hemoglobinuria, acquired and inherited bone marrow failure syndromes and bone marrow transplantation. Dr Frieri now carries out her medical activity at the Moscati Hospital in Avellino, Italy, and she is completing her PhD thesis at the University of Naples "Federico II".
Dr. Francesca Fioredda is a senior doctor of the Hemato-Oncology team of IRCCS Ospedale Giannina Gaslini, Genova Italy. She gained her medical degree and double specializations in Infectious Disease and Pediatrics in 1993 and 1999 respectively. Dr Fioredda spent the first twelve years of her career in the Infectious Disease ward of Giannina Gaslini Hospital managing patients and developing research projects mainly on pediatric HIV /HCV infections and supporting the activity of the European Registries. Since 2020 she joined the Hematology Unit, where she is operating now. Her commitments are both clinical and scientific. Dr Fioredda area of expertise is represented by congenital bone marrow failure and autoimmune cytopenias with a particular focus on Neutropenia.
The main achievements acquired over time are the leadership of the Italian Neutropenia Registry (founded in 2004) and the coordination of the Italian project on Auto-Immune Lymphoprolipherative Syndrome and of the newborn Italian Registry of Telomers Diseases.
These projects converge in the activities of the Bone Marrow Failure Working Group of AIEOP (Associazione Italiana Ematologia Oncologia Pediatrica) which she has been coordinating since 2018.
As for external collaboration, she is in the board of Granulocytes & constitutional marrow failure disorders study Group of EHA and she has contributed to a couple of projects within the Severe Aplastic Anemia Working Party of the EBMT (European Group of Blood and Bone Marrow Transplantation)
In the last two years, Dr Fioredda is actively involved in European Net-INNOCHRON which is a big project on a different aspect of Neutropenia.
Dr Thierry Leblanc trained In Paris (Faculté de médecine Saint-Antoine, Paris VI University) and is registered as a pediatrician and haematologist. His main interest is non-malignant haematology. His clinical practice includes constitutional and acquired aplastic anaemia, other inherited blood disorders (except sickle cell disease) and autoimmune blood cytopenias. He runs pediatric and adult outpatient clinics in Hospital Robert-Debre and Hospital Saint-Louis. He is part of the leading team of the French national reference centre for aplastic anaemia and inherited bone marrow failure syndromes. He is deeply involved in training and education in this field.
Since 2009, Prof. Tim Brümmendorf is head of the Department of Hematology and Oncology at University Hospital Aachen, and director of the Center for Integrated Oncology Aachen (CIOA), Germany, which is part of the Cancer Center Network CIOABcD, a member of the German
Comprehensive Cancer Centers (CCC) of Excellence funded by the Deutsche Krebshilfe (DKH).
He earned his medical degree from the University of Heidelberg, Germany. He did his residency in haematology, oncology, and immunology at University Hospital Tübingen, Germany, followed by a post-doctoral research fellowship at the Terry Fox Laboratory, Vancouver, BC, Canada.
In 2004, he received an appointment as attending and then deputy head, Department of Hematology and Oncology, University Hospital Eppendorf, Hamburg, Germany. In Hamburg, he was co-founder of the University Cancer Center Hamburg (UCCH).
Prof Brümmendorf's research focus is on translational and clinical studies in the field of chronic myelogenous leukaemia, myeloproliferative neoplasms, hereditary and acquired bone marrow failure and myelodysplastic syndromes.
He serves as a referee for several international scientific journals and funding organizations. Prof Brümmendorf is a member of the German Cancer Society (DKG), the German Society of Hematology and Oncology (DGHO), and the American Society of Hematology (ASH) and
current chair of the German Association of Academic Hematology and Oncology (VUHO).