SSC Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders

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Factor VIII, Factor IX and Rare Coagulation Disorders

The Factor VIII, Factor IX and Rare Coagulation Disorders Subcommittee has the mission of providing a forum for consideration of practical issues related to haemophilia and other rare bleeding disorders, provide state of the art knowledge regarding the diagnosis and management of these disorders, and conduct projects that aim to standardize and harmonize available laboratory evaluation and clinical care measures for the assessment of therapeutic products and clinical outcomes.

If you are interested in the subcommittee and want to follow its activity, please click "join group" above to sign up as a member (follower). 

Mandate

  • To address issues of practical importance to the research community in the field.
  • To evaluate existing data, determine issues for which data is missing or deficient, identify areas of controversy or pressing clinical need and discuss methodologic approaches to answer questions raised.
  • To create international collaborations for the purpose of planning and executing projects related to the defined needs in the field.
  • To suggest or organize collaborative studies as a result of these activities.
  • To generate, publish and distribute reports, recommendations and other documents concerning the above.
  • To develop laboratory standards, methods, and nomenclature if appropriate.

Role

The subcommittee appoints experts into project groups with specific mandates:

  • To evaluate and standardize definition of clinical and laboratory criteria to assess the clinical severity and effectiveness of therapy
  • Standardization of the currently recommended methodologies for standard coagulation assays, global hemostasis tests and genetic analysis to assess their application in different conditions
  • To evaluate current and upcoming treatment products to prepare recommendations on their best clinical use and to foster the development of more effective products.
  • To provide recommendations for optimal and practical pre- and post- licensure clinical trial design for the new clotting factor concentrates.

Such groups work over 1-2 years, review the available information in the literature and in practice and prepare recommendations to be published as SSC communication. See specific form of each project.

If you are a member of the Society and would like to know how to participate in the work of this group, please join the group to receive updates on activity or submit an Expression of Interest Form to the Chairman or any of the Co-Chairmen. We would be pleased to learn of your interest.

Leadership

  • Chair - Susan Shapiro
  • Co-chair - Giancarlo Castaman
  • Co-chair - Shannon Meeks
  • Co-chair - Karin Fijnvandraat
  • Co-chair - Cedric Hermans
  • Co-chair - Ming Lim
  • Co-chair - Yesim Dargaud
  • Co-chair - Roberta Gualtierotti
  • Co-chair - Jesus Ardila
  • Co-chair - Miguel Escobar 

Projects

Official Communication

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Announcements

  • Revision of the Definition of Prophylaxis in Hemophilia: A Communication from the ISTH SSC

    Prophylactic hemostatic therapy to prevent bleeding-related joint and other organ dysfunction has fundamentally transformed the management of severe hemophilia. Before the widespread adoption of such prophylaxis, individuals with severe hemophilia experienced recurrent spontaneous bleeding beginning in early childhood, leading to progressive hemophilic arthropathy, chronic pain, disability, and reduced life expectancy. Based on extensive clinical trials and real-world evidence, regular prophylaxis with CFCs has been established as the only effective treatment strategy for hemophilia that can modify the bleeding phenotype in a clinically meaningful way.

    To standardize terminology across clinical practice and research, the ISTH Scientific and Standardization Committee (SSC) published definitions of prophylaxis in hemophilia in 2014. Prophylaxis was defined as the regular administration of hemostatic therapy intended to prevent bleeding. It was categorized into primary, secondary, and tertiary strategies based on the timing of prophylaxis initiation relative to bleeding events and their complications. They have been widely adopted in clinical studies, registries, and treatment guidelines.

    Over the past decade, the therapeutic landscape of hemophilia has evolved substantially with the emergence of non-factor therapies (NFTs) and extended half-life products. These therapies differ in mechanisms of action, pharmacokinetics, and administration, but share the ability to enhance hemostasis and prevent bleeding. An ultra-long half-life FVIII concentrate has further expanded available options. These advances enable much earlier initiation of prophylaxis, reduce treatment burden, and minimize the need for central venous access. While concerns regarding inhibitor development persist with CFC exposure, NFTs do not carry the same direct risk profile. Treatment goals have consequently shifted toward the prevention of all bleeding and further optimization of outcomes.

    The objective of this SSC communication is to update the definitions of prophylaxis in hemophilia while maintaining conceptual continuity with the widely adopted 2014 framework.

    Please send any feedback/comments to be sent to Johnny Mahlangu at the following email address: johnny.mahlangu@nhls.ac.za

  • Open for Public Comment: Diagnosing and evaluating women and girls with hemophilia

    Per the Guidelines and Guidance (G&G) Committee process for guidance manuscripts, draft manuscripts go through a public comment period and G&G Committee review and approval before submitting the manuscript to the Journal of Thrombosis and Haemostasis (JTH).

     

    The G&G Committee would like to announce that the guidance manuscript titled, " The Diagnosis and Evaluation of Women and Girls with Hemophilia and Hemophilia Carriers: Guidance from the SSC of the ISTH," is now open for public comment. Note: This manuscript is confidential and in draft form.

     

    Please click here to submit your comments by Thursday, February 19, 2026.

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Community Admins

University of Utah
United States
Oxford University Hospitals NHS Trust
United Kingdom