Assessing the benefits of FEIBA prophylaxis in haemophilia patients with inhibitors.
Prophylaxis with concentrates of factor VIII has become the standard of care for patients with severe haemophilia A because of its ability to prevent joint and other bleeding events. Recent evidence suggests that the prophylactic use of bypassing therapy--activated prothrombin complex concentrate (aPCC) and recombinant activated factor VII (rFVIIa)--provides similar benefits to haemophilia patients with inhibitors. To assess the efficacy and safety of prophylaxis with the aPCC FEIBA, a meta-analysis was conducted of six studies and a total of 34 inhibitor patients. The mean prophylactic dose was 78.5 U kg(-1), typically infused three to four times weekly. A total of 31 of 33 patients (94%) for whom bleeding data were available prior to prophylaxis experienced a decrease in the rate of haemorrhage, albeit minor in some patients, and, regardless of the type of haemorrhage measured, had on average a 63.9% reduction in bleeding episodes during FEIBA prophylaxis. In the three studies that specifically assessed all joint bleeding, the 18 patients evaluated experienced an average reduction in annual joint bleeding of 74% while on prophylactic regimens. Among the four patients in this group who received concurrent immune tolerance induction and the 14 patients treated with prophylaxis only, annual joint bleeding decreased by an average of 79% and 78%, respectively. Anamnesis following FEIBA exposure was observed in some patients but had no impact on prophylactic efficacy. No thrombotic or other complications were reported. These results suggest that the prophylactic administration of FEIBA can be an effective and safe method for reducing bleeding events in patients with haemophilia A and inhibitors.
Valentino
L A
LA
Rush Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, IL 60612-3833, USA. lvalentino@rush.edu
D000293:Adolescent; D000328:Adult; D019774:Blood Coagulation Factor Inhibitors / Q000097:blood*; D001779:Blood Coagulation Factors / Q000008:administration & dosage / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D006395:Hemarthrosis / Q000517:prevention & control*; D006467:Hemophilia A / Q000188:drug therapy*; D002836:Hemophilia B / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control*; D006801:Humans; D011292:Premedication; D055815:Young Adult
D019774:Blood Coagulation Factor Inhibitors; D001779:Blood Coagulation Factors; C025667:prothrombin complex concentrates