Personalized prophylaxis in people with hemophilia A: challenges and achievements.
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.
a Cerrahpasa Medical Faculty, Department of Internal Medicine, Division of Hematology , Istanbul University , Istanbul , Turkey.
Muhlis Cem
MC
Ar
a Cerrahpasa Medical Faculty, Department of Internal Medicine, Division of Hematology , Istanbul University , Istanbul , Turkey.
Zafer
Z
Baslar
a Cerrahpasa Medical Faculty, Department of Internal Medicine, Division of Hematology , Istanbul University , Istanbul , Turkey.
Teoman
T
Soysal
D003362:Cost-Benefit Analysis; D005169:Factor VIII / Q000008:administration & dosage / Q000627:therapeutic use; D006395:Hemarthrosis / Q000209:etiology / Q000517:prevention & control; D006467:Hemophilia A / Q000150:complications*; D006470:Hemorrhage / Q000209:etiology* / Q000517:prevention & control*; D006801:Humans; D000071184:Pharmacogenomic Variants; D057285:Precision Medicine; D011292:Premedication
D005169:Factor VIII
Hemophilia; adherence; hemophilic arthropathy; personalized prophylaxis; pharmacokinetics