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CC BY-NC-ND 4.0 · Semin Thromb Hemost 2025; 51(01): 058-067
DOI: 10.1055/s-0044-1786358
Review Article

Nonsevere Hemophilia: The Need for a Renewed Focus and Improved Outcomes

Gerard Dolan
1   Centre for Haemostasis and Thrombosis, St Thomas' Comprehensive Care Centre, London, United Kingdom
,
Karin Fijnvandraat
2   Paediatric Haematology, Emma Children's Hospital, and UMC, University of Amsterdam, Amsterdam, The Netherlands
,
Peter J. Lenting
3   Université Paris-Saclay, INSERM, Hémostase Inflammation Thrombose HITh U1176, Le Kremlin-Bicêtre, France
,
Cristina Catarino
4   Immunochemotherapy Department, Congenital Coagulopathies Comprehensive Care Centre, Santa Maria University Hospital, Lisbon University, Lisbon, Portugal
,
Michelle Lavin
5   Irish Centre for Vascular Biology, School of Pharmacy and Biomolecular Sciences, Royal College of Surgeons in Ireland, Dublin, Ireland
6   National Coagulation Centre, St. James' Hospital, Dublin, Ireland
,
on behalf of the Factor Think Tank› Author Affiliations
Funding The Factor Think Tank is funded by Sobi. Medical writing and editorial support for this manuscript was funded by Sobi, who also funded support with manuscript submission.
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Abstract

People with nonsevere hemophilia (PWNSH) are phenotypically more diverse than those with severe hemophilia. Perceptions relating to a “nonsevere” phenotype have contributed to fewer research initiatives, fewer guidelines on optimal management, and a lack of standards for surveillance and clinical assessment for affected individuals. In many cases, episodes of abnormal bleeding could, if investigated, have led to earlier diagnosis. Furthermore, the major recent developments in therapy for hemophilia have largely focused on severe disease and, as a group, PWNSH have not been included in many key clinical trials. Benefiting people with severe disease, innovative replacement therapies have generally targeted factor levels that are above those present in a large proportion of PWNSH. Therapeutic advances can lead to improvement in phenotype for people with severe hemophilia over that currently experienced by many PWNSH. As a result, we are approaching a point where PWNSH may, in many countries, have a higher risk of bleeding and restriction in lifestyle than those with severe disease but with more limited therapeutic options. Given the multiple major advances in treatment for people with hemophilia, it is timely to review the aspects of nonsevere disease, to ensure equity in care and management for all individuals with this condition.

Keywords

disease management - factor VIII - factor IX - hemophilia A - hemophilia B

Authors' Contributions

All the authors developed the initial concept for this manuscript. G.D. and M.L. drafted a version that all the other authors critically revised for intellectual content. All the authors reviewed and approved the final document.


* All the members of the Factor Think Tank group are listed at factorthinktank.com.




Publication History

Article published online:
11 May 2024

© 2024. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/)

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