Patients with hemophilia benefit from maintaining prophylaxis as they transition from childhood to young adults, according to a systematic literature review published in the Orphanet Journal of Rare Diseases.

Prophylactic treatment with factor replacement therapy has been shown to reduce bleeds and joint damage for patients with severe hemophilia. However, in China, a small number of patients receive prophylaxis.

The authors sought to summarize factors that affect adherence to prophylactic treatment in young adults and the effects on patients’ health. The review includes 31 articles with a total of 2379 patients, most with severe hemophilia.


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Multiple studies found that use of prophylaxis declined in adulthood, with the transition beginning around age 16.

Studies on primary prophylaxis reported better joint health outcomes and better long-term health-related quality of life. Most studies included found that annualized bleeding rates (ABRs) were better for patients who received prophylaxis compared to on-demand treatment.

Based on the literature review and the guidelines of many hemophilia societies, it is preferred for patients to receive prophylaxis before joint bleeds and joint damage occur and that prophylaxis should continue into adulthood.

Patients who did not receive prophylactic treatment at a young age were more likely to have joint damage as adults. Adults who adhered to prophylaxis reported better health-related quality of life and physical functioning.

Cost, moving away from parents, and increased independence may affect adherence as patients transition out of childhood. Reduction in symptoms, lack of time, and self-treatment challenges were among the most frequently reported reasons for not continuing prophylaxis.

When compared to low-dose or intermediate-dose prophylaxis, the literature review found that the standard dose preserved joints, had better adherence, and improved quality of life. However, a low dose can still reduce bleeding events.

Overall, the authors recommended that adherence to prophylaxis into young adulthood is best for long-term outcomes.

Reference

Sun J, Zhou X, Hu N. Factor VIII replacement prophylaxis in patients with hemophilia A transitioning to adults: a systematic literature review. Orphanet J Rare Dis. 2021;16(1):287. doi:10.1186/s13023-021-01919-w