Females with hemophilia A (FHAs) and hemophilia A carriers (HACs) may be undermanaged and undertreated in the United States, according to a study published in Haemophilia.
Hemophilia A, although rare, leads to an enormous economic burden on the US healthcare system, as well as on patients regarding cost of treatment. Uncontrolled bleeding episodes may be life threatening, requiring emergency hospitalization.
Hemophilia, being an X-linked disorder, predominantly affects men. There is a misconception that females can only be asymptomatic carriers. Some females have been found to have factor VIII levels less than 40% of normal, which is the threshold by which hemophilia A is defined. Hence, regardless of sex, patients who meet this threshold should be managed in the same way as patients with hemophilia A. In addition, studies found that HACs can also suffer significant bleeding episodes that require close monitoring and care.
To better understand the demographics and clinical characteristics of males with hemophilia A (MHAs), FHAs, and HACs, the research team obtained data from administrative claims submitted to the IBM MarketScan Research Databases. Data were extracted from the time period between January 2016 and September 2019. Patients between birth and 89 years of age who fit any of the 3 categories mentioned were eligible for the study.
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A total of 1731 patients were included in this retrospective observational cohort study. The research team found that both FHAs and HACs as a group experienced a higher number of bleeds, greater use of desmopressin to control bleeding, and had more concomitant soft tissue and musculoskeletal pain. In addition, they found that there was no diagnostic/management framework for females with a bleeding disorder, complicating diagnostic efforts.
“Improving disease awareness among patients and healthcare providers, reaching an early diagnosis and using a multi-disciplinary management approach, including genetic consultation around pregnancy outcomes, is the way forward for FHAs and HACs,” the authors concluded.
Batt K, Xing S, Kuharic M, et al. Real-world analysis of patients with haemophilia A and haemophilia A carriers in the United States: demographics, clinical characteristics and costs. Haemophilia. Published online May 6, 2023. doi:10.1111/hae.14794