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New Hopes and Opportunities for Hemophilia and other Bleeding Disorders

New Hopes and Opportunities for Hemophilia and other Bleeding Disorders

ISTH2020 Virtual Congress: Independent symposium webcast

Publishing date: 15th July 2020

Chaired by Steven Pipe
With Stacy Croteau, David Lillicrap and Johnny Mahlangu

This independent symposium, led by a faculty comprising international key opinion leaders, aimed to discuss the emergence of novel treatment options in hemophilia triggered by a better understanding of this condition. It also provided the latest information on these emerging strategies and their therapeutic targets, focusing on anti-TFPI inhibitors and the clinical implications of the latest clinical data.

Watch the webcast recording from the session here. Please note that only the live activity at the ISTH2020 conference was accredited by EACCME®.

Learning objectives

After participating in the symposium you will be able to:

  • Explain the drivers behind the new and emerging treatment options
  • Compare new and emerging treatment options and their therapeutic targets
  • Interpret the clinical implications of the latest anti-TFPI trial data


  • Welcome and introduction (5 min)
  • How has our current understanding of hemophilia driven new treatment options? (10 min)
  • What are these new treatment options? (15 min)
  • What do we know about anti-TFPI inhibitors? (30 min)
  • Faculty discussion and audience questions (15 min)


Steven Pipe
University of Michigan, Ann Arbor, MI, USA

Dr Steven Pipe is a Professor and the Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases, as well as Professor of Pathology at the University of Michigan, Ann Arbor, Michigan, USA. Dr Pipe is also the medical director of both, the Pediatric Hemophilia and Coagulation Disorders Program and the Special Coagulation Laboratory at the University of Michigan. His clinical interests include bleeding and thrombotic disorders and congenital vascular anomalies. He also directs a basic research lab investigating coagulation factor VIII and the molecular mechanism of hemophilia A.

Consultant fees: Apcintext, Bayer, Biomarin, Catalyst Biosciences, CSL, Behring, HEMA Biologics, Freeline, Novo Nordisk, Pfizer, Roche/Genentech, Sangamo, Sanofi, Roche, Spark, uniQure

Stacy Croteau
Boston Children’s Hospital, Boston, MA, USA

Dr Croteau is a pediatric hematologist and clinical investigator at Boston Children’s Hospital, and an Assistant Professor of Pediatrics at Harvard Medical School, where she specializes in bleeding and clotting disorders. She received her medical degree from Brown Medical School and completed her pediatric residency training at Boston Children’s Hospital and pediatric hematology/oncology fellowship at Boston Children’s Hospital/Dana-Farber Cancer Institute.

Dr Croteau is the medical director for the Boston Hemophilia Center and serves as a regional medical director for the national network of US hemophilia treatment centers (HTCs). Currently she serves as the principle investigator for several industry-sponsored and investigator-initiated clinical trials on hemophilia and other bleeding and clotting disorders.

Consultant fees: Bayer, Bioverative, CSL-Behring, Genentech, Novo Nordisk, Octapharma, Shire
Research support: Genentech, Novo Nordisk, Pfizer, Spark

David Lillicrap
Queen’s University, Kingston, Ontario, Canada

Dr David Lillicrap is a Professor in the Department of Pathology and Molecular Medicine at Queen’s University, Kingston, Canada. He is the recipient of a Senior Canada Research Chair in Molecular Hemostasis. Between 2008-2014 he served as an Associate Editor of Blood and is currently Co-Editor-in-Chief of the Journal of Thrombosis and Hemostasis.

Dr Lillicrap is a past member of the World Federation of Hemophilia’s (WFH) Medical Advisory Board and a past Chair of the WFH’s Research Committee. He is also a past Chairman of the International Society on Thrombosis and Hemostasis’ (ISTH) Scientific and Standardization Committee and is a current member of the Council of ISTH. His research interests are centered on molecular aspects of the hemostatic system, with a particular emphasis on the potential of molecular genetics and molecular biology to address questions relating to pathological hemostasis. The main foci of interest of his research group are the investigation of the immune response to FVIII, the development and evaluation of novel therapies for hemophilia A, and characterization of the biology and pathobiology of von Willebrand factor.

Research support: Bayer, Biomarin, Biorevativ/Sanofi, CSL-Behring and Octapharma

Johnny Mahlangu
University of the Witwatersrand and NHLS, Johannesburg, South Africa

Professor Mahlangu is a Personal Professor in Hematology and Head of School of Pathology in the Faculty of Health Sciences of the University of the Witwatersrand and the National Health Laboratory Service in Johannesburg, South Africa. He is also Director of the International Hemophilia Training Centre and Consultant Clinical Hematologist at the Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg. His main area of research is novel therapies in bleeding disorders in which he has served as Principal Investigator for over 70 international multicenter studies. He is currently doing several gene therapy studies in hemophilia.

Professor Mahlangu is current President of the College of Pathologists in South Africa, board member of the South African Medical Research Council, Wits Health Consortium and Poliomyelitis Research Foundation. His past leadership roles include Chair of the Medical and Scientific Advisory Council of the National Hemophilia Foundation of South Africa, President of SA Hematology Society for two non-consecutive terms and an elected Executive member of the World Federation of Hemophilia.

Research support: Biomarin, CSL, Freeline Therapeutics, Novo Nordisk, Novartis, Pfizer, Sanofi, Roche
Consultant fees: CSL Berhing, Catalyst Biosciences, Freeline Therapeutics, Novo Nordisk, Roche, Sanofi, Spark and Takeda


This program is targeted at specialists (primarily hematologists) treating patient with hemophilia in Europe, United States, Canada and Japan.


The live activity that took place at ISTH2020 “New hopes and opportunities for hemophilia and other bleeding disorders, 14/07/2020-14/07/2020” has been accredited by the European Accreditation Council for Continuing Medical Education (EACCME®) with 1 European CME credit (ECMEC®). Each medical specialist should claim only those hours of credit that he/she actually spent in the educational activity.

Through an agreement between the Union Européenne des Médecins Spécialistes and the American Medical Association, physicians may convert EACCME® credits to an equivalent number of AMA PRA Category 1 Credits™. Information on the process to convert EACCME® credit to AMA credit can be found at

Live educational activities, occurring outside of Canada, recognised by the UEMS-EACCME® for ECMEC®s are deemed to be Accredited Group Learning Activities (Section 1) as defined by the Maintenance of Certification Program of the Royal College of Physicians and Surgeons of Canada.

Date of expiration: 14th September 2020

Educational grant

This program is made possible thanks to an independent educational grant from Novo Nordisk A/S and Pfizer.

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James Hutton

Senior Project Director

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