New advances in the therapeutic and laboratory management of patients with haemophilia and inhibitors

Abstract

The management of haemophilia patients with high inhibitor titres remains a major clinical challenge. In this manuscript, we present the new developments in the treatment and laboratory monitoring of these patients. First, we discuss a general treatment algorithm to control severe bleeding episodes in these patients, established by an international panel of haemophilia specialists. The main features of this algorithm concern (i) the optimal timing of clinical and therapeutic decisions and (ii) the appropriate use of recombinant activated factor VII (rFVIIa) or activated prothrombin complex concentrates. However, until the clinical value of this algorithm is validated in real-world practice, the use of single high doses of rFVIIa should be considered as a valuable therapeutic option. Secondly, we present four laboratory assays, potential surrogate markers for the efficacy of bypassing agents used in haemophilia patients with high titre inhibitors. Preliminary data suggest that some of these tests, notably thromboelastography and the thrombin generation test, may be helpful for predicting the individual bleeding risk and for providing individually tailored treatment regimens. Overall, it may be hoped that these new developments will lead to a marked improvement in the clinical management of haemophilia patients with inhibitors in the near future.