Personalized prophylaxis in people with hemophilia A: challenges and achievements

Abstract

Prophylactic factor replacement is the standard of care for all people with severe hemophilia to prevent bleeding and associated complications. Current weight-based fixed dose prophylaxis regimens are effective; however, they lack flexibility and usually fail to meet the individual needs and expectations of the patients. Recent developments in hemophilia treatment provide new opportunities for a more personalized prophylaxis. Areas covered: Rationale and methods of individualizing prophylaxis in hemophilia A on the basis of current evidence are discussed in this review. For this relevant literature in English and German was searched using PubMed database. Expert commentary: Major determinants of personalized prophylaxis include age, bleeding pattern, personal pharmacokinetics, joint health, co-morbidities, venous access and adherence. An ideal prophylaxis programme should take into account all of the aforementioned items and also be able to meet the needs. Extended half-life factor concentrates, new hemostatic molecules and tools using population pharmacokinetics to estimate personal factor requirements will serve individualizing prophylaxis in a more precise manner.

Keywords: Hemophilia; adherence; hemophilic arthropathy; personalized prophylaxis; pharmacokinetics.