For individuals with hemophilia A, lifelong prophylactic treatment with FVIII replacement therapies or non-factor therapies (NFTs) is the standard of care.¹ Despite advancements in treatment, bleeding episodes persist, significantly impacting physical health and quality of life (QoL).¹ This study evaluated the unmet needs of patients with moderate to severe hemophilia A on prophylaxis by analyzing patient- and physician-reported outcomes and bleeding events.¹ Findings were presented at the ASH Annual Meeting & Exposition 2024 by Dr. Lynn Malec from the Comprehensive Center for Bleeding Disorders, Versiti Blood Center of Wisconsin, Wisconsin, United States (US).¹

This real-world analysis utilized data from the Adelphi Hemophilia Disease Specific Programme™, a cross-sectional survey conducted in the US from July 2023 to March 2024.¹ Physicians provided relevant data on patient demographics, treatment history, FVIII activity levels, annual bleeding rate (ABR), joint issues, and therapy use.¹ Patients also voluntarily reported the impact of hemophilia A on their QoL.¹ Patients included had no inhibitors at the time of the study, known baseline FVIII levels, and were on prophylaxis using NFTs or extended (EHL) or standard half-life (SHL) therapies.¹ ABR data were analyzed for patients treated for >365 days.¹

Data were collected from 47 physicians covering 244 patients (mean age ± SD: 27.4±14.7 years).¹ FVIII activity levels were reported as: 2.0%-5.0% (n=92), 1.0%-1.99% (n=90), and <1.0% (n=62).¹ A total of 113 patients received treatment with SHL therapy (n=39 for >12 months), 40 received EHL therapy (n=23 for >12 months) and 67 received NFT (n=29 for >12 months).¹ The overall mean ABR for patients treated for >12 months (n=91) was 0.80 (95% CI: 0.57-1.03).¹ Subgroup analyses revealed that mean ABRs was highest for EHL therapy at 1.09 (95% CI: 0.46-1.74), followed by SHL therapy at 0.77 (95% CI: 0.45-1.15), and 0.52 (95% CI: 0.22-0.78) for NFTs.¹ Overall, 39 patients (42.9%) experienced one or more bleeds in the prior year, with higher rates reported in patients on EHL therapy (52.2%) compared to SHL (41.0%) and NFTs (37.9%).¹ Joint problems were reported in 25.0% of patients in the prior 12 months.¹ Physicians indicated that 44.3% (n=108) of patients used therapies such as physical therapy (59.3%), yoga/pilates (31.5%), or massage (21.3%) to manage joint issues, while 23.0% (n=56) required aids like braces (46.4%) or splints (30.4%).¹ Among patients not working full-time (n=32), 28.1% cited hemophilia A as the primary reason for unemployment.¹

Additionally, patient-reported data (n=26) revealed high levels of psychological and physical burden.¹ Nervousness or anxiety affected 55.0% (n=11) of patients, while 40.0% (n=8) expressed fear or worry about hemophilia A.¹ Concerns about breakthrough bleeds were reported by 80.0% (n=20), and 73.7% (n=14) noted difficulty participating in sports or hobbies.¹ Most patients (n=21; 84.0%) avoided physical activities, with 12.0% (n=3) doing so frequently.¹

In conclusion, despite regular prophylactic and on-demand treatments, over one-third of patients with moderate or severe hemophilia A experienced at least one bleeding episode annually.¹ Joint problems and reduced QoL remain significant challenges, with many patients avoiding physical activity due to hemophilia A.¹ These findings underscore an unmet need for more effective therapies to reduce bleeds and improve QoL in this population.¹