"Primary prophylaxis" with rFVIIa in a patient with severe haemophilia A and inhibitor

Abstract

The development of antibodies that inhibit or neutralize replacement therapy with factor VIII or factor IX is today the most serious complication of haemophilia and its treatment. Inhibitor patients have more severe joint morbidity than patients without inhibitors, and older adults experience significant orthopaedic disabilities. Because of the serious and disabling consequences of persistent inhibitors, there is considerable clinical and research interest in establishing effective bypassing agent regimens to prevent bleeding in inhibitor patients in much the same way as prophylaxis procedure works in noninhibitor patients. In the majority of these patients, the bypass agent was used as a secondary prophylactic. In this report, the use of recombinant factor VIIa prior to any clinically evident joint bleed in a patient with haemophilia A and high-titre inhibitor is described.