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Author Notes:

Karin P. M. van Galen, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Heidelberglaan 100, 3584 CX, P.O. Box 85500, 3508 GA Utrecht, the Netherlands. Email: k.p.m.vangalen@umcutrecht.nl

K.P.M. van Galen contributed to the concept and design, analysis, and/or interpretation of data and writing of drafts and final version the paper. R. d’Oiron, P. James, R. Kadir, P. A. Kouides, R. Kulkarni, F. Peyvandi, and R. Winikoff contributed to concept and design, analysis and/or interpretation of data, critical writing and revising the intellectual content. J. N. Mahlangu, M. Othman, and D. Rotellini contributed to revising the intellectual content and approval of the manuscript to be published. R.F. Sidonio contributed to concept and design, analysis and/or interpretation of data, critical writing, and final approval of the version to be published.

The project was supported intellectually by the International Society on Thrombosis and Haemostasis Scientific and Standardization Committees on FVIII/IX & Rare Coagulation Disorders and Women’s Health Issues in Thrombosis & Hemostasis. Specifically, we would like to thank Dr. Malinowski, Dr. Grandona, and Dr. Casais for their input and all other SSC co‐chairs for their support. There has been no commercial funding. Next to ISTH we’d like to acknowledge the Coalition for Hemophilia B; National Hemophilia Foundation; Hemophilia Federation of America; European Association for Haemophilia and Allied Disorders Women and Bleeding Disorders Working group, specifically Michelle Lavin; the European Haemophilia Consortium; Association of Hemophilia Clinic Directors of Canada (AHCDC) and Canadian Hemophilia Society; women committee of the French Society for Hemophilia Patients (Association Française des Hémophiles); and Dutch Society for Hemophilia Patients for their contribution and support.

Dr. van Galen has received research support from CSL Behring, Bayer, and Octapharma and speakers’ fees from Takeda, CSL Behring, and Bayer. Dr. d’Oiron has served as a consultant for Baxalta/Shire, Bayer, CSL Behring, LFB, NovoNordisk, Octapharma, Pfizer, Roche, and Sobi, Spark and was invited speaker for Baxalta/Shire, Bayer, CSL Behring, LFB, NovoNordisk, Octapharma, Pfizer, Roche, and Sobi, Spark. Dr. Sidonio has participated in advisory boards for Novo Nordisk, Sanofi, Genentech, Octapharma, Takeda, Bayer, Sigilon, Catalyst, Biomarin, and Pfizer. Dr. James has received research funding from Bayer. Dr. Mahlangu received research grants from BioMarin, Catalyst Biosciences, CSL, Freeline Therapeutics, Novo Nordisk, Novartis, Pfizer, Sanofi, Roche, Spark, Takeda, and was an advisory board/consultant for CSL Behring, Catalyst Biosciences, Freeline Therapeutics, Novo Nordisk, Roche, Sanofi, Spark, Takeda. Dr. Kulkarni has served on the advisory boards of Sanofi‐Genzyme, NovoNordisk, Genentech, CSL Behring, Octapharma, Pfizer, Shire/Takeda, Catalyst Bioscience, and Bayer. Prof Peyvandi has received speaker fees for participating in educational symposia and advisory boards for Roche, Sanofi, Sobi, and Takeda. D. Rotellini has served on advisory boards for Bayer, BioMarin, and Pfizer with National Hemophilia Foundation receiving all honorarium. Prof Abdul‐Kadir received lecture fees/educational grants from Pfizer, NovoNordisk, Takeda, and ViforParma. Dr. Sidonio has IIS funded by Takeda, Genentech, and Octapharma and is the PI for the Wil‐29/Wil‐31 studies (Octapharma) and BAX‐855 study and has served as a consultant for Bayer, Sigilon, Roche/Genentech, Octapharma, Grifols, Biomain, Sanofi, and Novo Nordisk. None of the other authors has any conflict of interest to declare.

Subjects:

Keywords:

  • Science & Technology
  • Life Sciences & Biomedicine
  • Hematology
  • Peripheral Vascular Disease
  • Cardiovascular System & Cardiology
  • bleeding
  • hemophilia
  • phenotype
  • women's health
  • A CARRIERS
  • FACTOR-VIII
  • BAT
  • PHENOTYPE
  • RANGE

A new hemophilia carrier nomenclature to define hemophilia in women and girls: Communication from the SSC of the ISTH

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Journal Title:

JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Volume:

Volume 19, Number 8

Publisher:

, Pages 1883-1887

Type of Work:

Article | Final Publisher PDF

Abstract:

Hemophilia A and B predominantly attracts clinical attention in males due to X-linked inheritance, introducing a bias toward female carriers to be asymptomatic. This common misconception is contradicted by an increasing body of evidence with consistent reporting on an increased bleeding tendency in hemophilia carriers (HCs), including those with normal factor VIII/IX (FVIII/IX) levels. The term HC can hamper diagnosis, clinical care, and research. Therefore, a new nomenclature has been defined based on an open iterative process involving hemophilia experts, patients, and the International Society on Thrombosis and Haemostasis (ISTH) community. The resulting nomenclature accounts for personal bleeding history and baseline plasma FVIII/IX level. It distinguishes five clinically relevant HC categories: women/girls with mild, moderate, or severe hemophilia (FVIII/IX >0.05 and <0.40 IU/ml, 0.01–0.05 IU/ml, and <0.01 IU/ml, respectively), symptomatic and asymptomatic HC (FVIII/IX ≥0.40 IU/ml with and without a bleeding phenotype, respectively). This new nomenclature is aimed at improving diagnosis and management and applying uniform terminologies for clinical research.

Copyright information:

© 2021 The Authors. Journal of Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis

This is an Open Access work distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International License (https://creativecommons.org/licenses/by-nc/4.0/rdf).
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