Researchers discovered shortcomings associated with factor VIII (FVIII) prophylaxis treatment for patients with severe hemophilia A, as published in the Journal of Clinical Medicine.
Prophylactic exogenous FVIII is recommended for individuals with severe hemophilia A. However, reports of bleeding events suggest that it is not a cure.
“Although recent advances in hemophilia treatment and management have dramatically increased quality of life (QoL), individuals with severe hemophilia A still experience bleeding events, indicating an unfulfilled need for greater hemostatic control in this population,” Kenet et al wrote.
The authors of this study believed that better data are needed on prophylactic FVIII replacement therapy, specifically standardized data on bleeding, FVIII use, and QoL parameters. This will help physicians understand the true burden of both severe hemophilia A and its treatment, they said.
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They conducted a prospective, multinational, multicenter, noninterventional, and longitudinal study, and recruited male patients with severe hemophilia A above 18 years old who were on continuous prophylactic exogenous FVIII for more than 6 months.
Selected participants were required to provide 6 months of retrospective data. In addition, they were followed up prospectively for up to 12 months. The researchers successfully enrolled 294 participants, with 225 (76.5%) completing follow-up. The parameters studied were annualized bleeding rates, FVIII utilization, and infusion rates.
“Overall, our data combined with these previous results suggest that breakthrough bleeding events requiring additional treatment occur for many individuals with severe hemophilia A receiving FVIII prophylaxis,” Kenet et al summarized. In addition, QoL results demonstrated that substantial burden and impairments remained present among patients with severe hemophilia A, despite good adherence to treatment regimes prescribed.
“Despite high prophylaxis adherence, the continued occurrence of spontaneous and joint bleeding events requiring treatment and impaired physical functioning was evident in our study,” the researchers concluded.
“These results illustrate real-world shortcomings associated with regular FVIII prophylaxis for this cohort of individuals with severe hemophilia A, for whom additional hemostatic options are needed.”
Kenet G, Chen YC, Lowe G, et al. Real-world rates of bleeding, factor VIII use, and quality of life in individuals with severe hemophilia A receiving prophylaxis in a prospective, noninterventional study. J Clin Med. Published online December 18, 2021. doi:10.3390/jcm10245959