Review paper on prophylaxis in children with haemophilia

Karinfijnvandraat is Professor of Paediatric Haematology at the University of Amsterdam and Director of the Haemophilia Treatment Centre Amsterdam. She trained in paediatrics and specialised in paediatric haematology at the University of Amsterdam. Her clinical focus is on haemophilia, allied bleeding disorders and sickle cell disease. After a fellowship in clinical epidemiology, her haemophilia research focused on non-severe haemophilia. In 2007 she initiated the international INSIGHT Consortium to enhance collaboration on non-severe haemophilia research among 34 haemophilia treatment centres in 10 European countries and Australia. Furthermore, she contributed to many multicentre studies in haemophilia, von Willebrand disease and sickle cell disease.

Christoph Male is consultant and associate professor at the Department of Paediatrics, Medical University of Vienna (MUW), Austria, where he heads the Paediatric Haemostasis & Thrombosis unit. He trained in General Medicine and Paediatrics, with sub-speciality degrees in Paediatric Cardiology and Paediatric Intensive Care. He was clinical and research fellow with the Paediatric Thrombophilia Programme at the Hospital for Sick Children, Toronto, and McMaster University, Hamilton, Ontario, Canada, where he also earned a M.Sc. degree in Health Research Methodology (1998-2000). Dr Male’s scientific interest is in paediatric haemostasis & thrombosis, anticoagulation, clinical pharmacology, drug evaluation and clinical trials methodology in children. He has more 20 years of experience in clinical research and is actively involved in several academic or industry-sponsored clinical trials in children. Dr Male currently acts as co-chair of the Paediatric Scientific and Standardization Subcommittee of the International Society of Thrombosis & Haemostasis (ISTH), chair of the Scientific Board of the Austrian Haemophilia Society, and past board member of the German-Austrian-Swiss Society of Thrombosis and Haemostasis Research (GTH). He served as Austrian delegate in the Paediatric Committee (PDCO) at the European Medicines Agency from 2007-2016, is a member of the Scientific Board of the Austrian Medicines Agency and paediatric member of the ethics committee of the MUW

Christoph Königs has been based at the Paediatric Haemophilia Centre, University Hospital Frankfurt, Germany since 2002. He received his degree in science from Monash University, Melbourne, Australia in 1998 and his medical degree from Goethe University Frankfurt, Germany in 2002. For both his MD and PhD thesis, Christoph isolated ligands to clinically relevant structures of protein receptors and RNA. The main clinical and scientific interest of his research team is the understanding and modification of the immune response to coagulations factors. His main clinical focus is developing and improving therapies for children and adolescents with coagulation disorders with a focus on patient education and shared decision making. Christoph specialises in paediatrics and adolescent medicine and also in haemostasis. He is a member of several national and international scientific societies and committees. He co-chairs the Standing Committee Haemophilia of the Society for Thrombosis and Haemostasis (GTH), the National Registry on PUPs with Newly Diagnosed Haemophilias in Germany “GEPHARD” and the Scientific Advisory Council of PedNet. Christoph works closely with German patient organizations i.e. as a deputy chair of the medical advisory board.

Director of the Israel National Haemophilia Centre and Thrombosis Institute, Sheba Medical Centre and Director of Amalia Biron Research Institute of Thrombosis and Haemostasis, Tel Aviv University (located at the Sheba Medical Centre). Gili Kenet is a paediatric haematologist, Professor and former Chair of the Haematology Department at the Sackler Medical School, Tel Aviv University. Professor Kenet received her Medical Degree from the Hebrew University in Jerusalem. Her research projects have been focused on neonatal haemostasis, childhood stroke and paediatric thrombophilia, as well as the research of new therapy modes applied for people with haemophilia and severe bleeding disorders. She was awarded national grants for paediatric haematology and cancer research, study of rFVIIa mode of action, epidemiology of inhibitor evolution in haemophilia and for personalised tailoring of bypass agent therapy, and applying global haemostasis assays. Prof. Kenet pioneered in gene therapy clinical trials in Israel and novel non replacement drugs in haemophilia. Professor Kenet is the author of over 200 peer reviewed scientific publications, review articles and book chapters. An active member of the World Federation of Haemophilia, Israeli Society of Paediatric Haematology and Oncology, and the American Society of Haematology, an active reviewer for high impact thrombosis journals and an editorial board member of the Haemophilia journal. Former Chairperson of the Israeli Society of Thrombosis and Haemostasis and Chairperson of the Paediatric and Peri-natal Haemostasis Scientific Subcommittee of ISTH. Currently co-chairs the FVIII/FIX and rare bleeding disorders of the SSC of ISTH.

Jan Blatný, MD, PhD, is Consultant Haematologist at the Department of Paediatric Haematology and Biochemistry, Centre for Thrombosis and Haemostasis and the Director of the Haemophilia Comprehensive Care Centre of University Hospital in Brno, Czech Republic. He is also an Associate Professor of Paediatrics at Masaryk University in Brno, Czech Republic. His research interests encompass paediatric haematology, thrombosis and haemostasis, and life-threatening bleeding. He is a member of the Working Party on population PK of the ISTH SSC Subcommittee on Factor VIII and IX and Rare Bleeding Disorders. In addition to ISTH, Dr Blatný is a member of numerous national and international haematology organizations. He serves on the steering committee and is a paediatric coordinator of the Czech National Haemophilia Programme, Vice-president and a member of Executive Committee of EAHAD, member of MAG (Medical advisory group) of EHC, a reviewer for several haematology journals, and is on the editorial board for an official journal of Czech Haematology society. Dr Blatný’s publication credits are 9 book chapters and over 100 articles published in peer-reviewed journals

Maria Elisa Mancuso MD, PhD is a Senior Consultant in Haematology with a special interest in haemostasis. She obtained a post-degree in Clinical and Experimental Haematology and a PhD in Clinical Methodology. She worked as a Clinical Assistant at the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center in Milan (Italy) from 2008 to 2020. Since May 2020 she has been Senior Consultant at the Center for Thrombosis and Haemorrhagic Diseases at IRCCS Humanitas Research Center in Rozzano, Milan (Italy). Dr Mancuso is involved in clinical research and has published several original articles in peer-reviewed journals such as The Lancet, Blood, Journal of Thrombosis and Haemostasis, Haematologica, Thrombosis and Haemostasis, British Journal of Haematology and Haemophilia. She is a member of the Editorial Board of the Journal of Thrombosis and Haemostasis and is a reviewer for several peer-reviewed journals in the field of Thrombosis and Haemostasis and is a member of several national and international scientific societies (AICE, SISET, ISTH, WFH, ASH, EAHAD). She has acted as Co-Chair of the Scientific and Standardization Subcommittee of ISTH (SSC-ISTH) on FVIII, FIX and rare bleeding disorders. Dr Mancuso has been involved as co-investigator in several clinical trials, and takes care of both children and adults with haemophilia with a specific scientific interest in inhibitors, chronic hepatitis C, optimisation of prophylaxis and novel therapies.

Kaan Kavakli, MD, is chair of the Department of Paediatrics and a professor of paediatrics and paediatric haematology at the Medical School of Ege University in Izmir, Turkey, where he previously was chair of the Department of Paediatric Haematology. Additionally, he is a paediatric haematologist in the Department of Paediatrics at Ege University Hospital and the director of the Ege Haemophilia Centre and the Ege Haemophilia Association. Dr Kavakli received his medical degree and training in paediatrics at Ege University Medical School. His special interests are haemostasis, thrombosis, and the care of patients with haemophilia. His recent work has included identification of certain mutations in Turkish haemophilia patients with inhibitors, radioisotope synovectomy for haemophilic arthropathy, the role of circumcision in boys with haemophilia, and treatment options for patients with inhibitors. Dr Kavakli has been a principal investigator for international clinical trials in haemophilia. Dr Kavakli is president of the Turkish Hemostasis and Thrombosis Association and the Hemophilia Federation of Turkey, which comprises 11 regional haemophilia associations. He is a member of many international societies and serves on the editorial boards and as a reviewer for several haematology journals. Dr Kavakli has published more than 100 papers in such journals as Blood, The New England Journal of Medicine, and Haemophilia, etc.