Impact of an Individualized Prophylaxis Approach on Young Adults with Severe Hemophilia

Abstract

It is now well established that the treatment of choice for children with severe hemophilia is prophylaxis started early in life. Although, there is no consensus among the hemophilia management community to either stop or maintain prophylactic treatment in adulthood, experts, and centers advise individualized prophylaxis according to clinical bleeding pattern, condition of joints, pharmacokinetic profile, physical activity, type of employment, and patients' personal preferences. The aim of this article is to describe the impact of an individualized prophylaxis approach on young adults with severe hemophilia, in the setting of a Portuguese Haemophilia Comprehensive Care Centre. We proposed a tailored prophylaxis approach on a young adult cohort with 10 patients with severe hemophilia (7× type A and 3× type B) on standard prophylactic regimens in childhood, based on clinical outcome. Patients were evaluated and prophylaxis was adjusted (dose and/or frequency) to daily life activity and bleeding pattern. After 12 months of follow-up, one patient returned to the previous regimen due to breakthrough bleeds and the remaining nine patients maintained their new prophylaxis approach, without increasing bleeding episodes. With an individualized approach, in this cohort of nine patients, we observed no negative impact on clinical outcome, with a proposed improvement in quality of life and a reduction of costs.

Keywords

individualized prophylaxis - hemophilia - young adults - bleeding

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