The Under-Evaluated Impacts of the Therapeutic Revolution of Hemophilia on Women and Girls

Authors: Hermans, C; Krumb, E; Rotellini, D; Pierce, GF.

Affiliations: Division of Haematology, Haemostasis and Thrombosis Unit, Saint-Luc University Hospital, Université catholique de Louvain (UCLouvain), Brussels, Belgium. World Federation of Hemophilia, Montreal, Canada; National Bleeding Disorders Foundation (NBDF), previously National Hemophilia Foundation (NHF).

Publication: Journal of thrombosis and haemostasis : JTH. 2023

ABSTRACT: The advent of new treatment options over the last decades has markedly improved the lives of male persons with hemophilia (PwH). However, this therapeutic revolution has not benefited women and girls with Hemophilia (WGH) and symptomatic carriers of the disease to the same extent as their male counterparts. This inequity is primarily due to the exclusion of WGH from clinical trials and a failure to fully recognize their specific treatment needs. Additionally, the indirect impact of innovative therapies, when used for male PwH, on the lives of mothers, other relatives, and partners of these individuals has been largely overlooked until now. In addition to improving access of WGH and carriers to new hemostatic treatments and comprehensive hemophilia care, it is imperative to strive for alleviating the mental burden imposed on them by this chronic disease. The recently proposed concept of a « hemophilia-free mind », primarily focused on male PwH, should therefore also be applied to WGH, symptomatic carriers, and the predominantly female support network of PwH.