Individually Tailored Prophylaxis in Patients with Severe Hemophilia

Dargaud Y, Negrier C.  Individually Tailored Prophylaxis in Patients with Severe Hemophilia.  Journal of Coagulation Disorders, July 2010; 2(2):  11-17

REVIEW ARTICLE

Yesim Dargaud^1,2 and Claude Negrier^1,2

Affiliations: ¹Unite d'Hemostase Clinique, Hopital Edouard Herriot, Lyon, France and ²EA 4174, Unite de recherche sur l'hemophilie, Universite Lyon 1, France


ABSTRACT

Since the 1950s, prophylaxis has been used in patients with severe hemophilia in order to prevent joint bleeds and life-threatening bleeding episodes. Several studies have demonstrated the importance of starting prophylactic therapy during the first years of life, before the start of joint damage. However, the regimen can be individualized and adjusted according to the bleeding phenotype of individual patients. In general, the current strategies used for prophylaxis can be divided into those that aim to maintain residual plasma FVIII/FIX activity >1 IU/dL and those that are guided by the clinical bleeding patterns of individual patients. Laboratory markers to predict the phenotype are warranted. Pharmacokinetics have been reported as a useful laboratory tool for factor dosing, but clinical bleeding patterns may be significantly different in patients having similar coagulation factor activity. Therefore, the optimal prophylactic regimen is still under debate and many unanswered questions still exist. Recently, global hemostasis assays, such as the thrombin generation test, have been extensively studied and several groups reported a statistically significant correlation between the thrombin-generating capacity of patients and their bleeding symptoms. Recent data suggest that thrombin generation measurement may be useful for individually tailoring a prophylactic regimen to the needs of each patient with hemophilia. Optimizing prophylactic therapy to patient's phenotype with no loss of clinical effectiveness would be highly relevant and can significantly improve patients' quality of life.

Keywords: hemophilia, prophylaxis, thrombin generation assay, ETP, individualization

Correspondence: Yesim Dargaud, Unite d'Hemostase Clinique, Hopital Edouard Herriot, 5 place d'Arsonval, 69003 Lyon, France. Tel: +33 4 72117370; Fax: +33 4 72117312; e-mail: ydargaud@univ-lyon1.fr