Scientific Publications by FDA Staff

Per Med 2015;12(4):403-15

Personalized approaches to the treatment of hemophilia A and B.

Simhadri VL, Banerjee AS, Simon J, Kimchi-Sarfaty C, Sauna ZE

The recognition that individuals respond differently to the same medication is not new and dates almost to the founding of western medicine. In the last century it came to be recognized that genetic factors influence the heterogeneity of individual responses to medications with respect to both toxicity and efficacy. Nonetheless, given the complexity of biological systems, the selection of appropriate biomarkers and the study design remain initial hurdles. Here we present potential applications of pharmacogenetics in managing bleeding disorders. We discuss how predicting and circumventing immunogenicity, an important impediment to treating haemophilia patients, particularly lends itself to a pharmacogenetic approach. In addition we discuss new trends towards personalizing the management of haemophilia in clinical settings.