Personalized prophylaxis in people with hemophilia A: challenges and achievements

Expert Rev Hematol. 2016 Dec;9(12):1203-1208. doi: 10.1080/17474086.2016.1252670. Epub 2016 Nov 4.

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.

Publication types

  • Review

MeSH terms

  • Cost-Benefit Analysis
  • Factor VIII / administration & dosage
  • Factor VIII / therapeutic use
  • Hemarthrosis / etiology
  • Hemarthrosis / prevention & control
  • Hemophilia A / complications*
  • Hemorrhage / etiology*
  • Hemorrhage / prevention & control*
  • Humans
  • Pharmacogenomic Variants
  • Precision Medicine*
  • Premedication*

Substances

  • Factor VIII