Experience of recombinant-activated factor VII to control bleeding in children with Glanzmann’s thrombasthenia

Abstract

Patients with Glanzmann’s thrombasthenia (GT) are at an increased risk of haemorrhage. Recombinant-activated factor VII (rFVIIa) is a new therapeutic option, but experience of its use in GT is limited. We present an analysis of patients ≤16 years with GT receiving rFVIIa, whose details were submitted to an independent, peer-reviewed registry http://www.haemostasis.com. Thirty-five reports of rFVIIa use in ten patients (age range, 2–16 years) with GT were identified. In seven out of ten patients, rFVIIa was administered for the treatment of active bleeding, and in three out of ten, the agent was given prophylactically before an intervention. A median total rFVIIa dose of 270 μg/kg body weight (range, 89.7–635.4 μg/kg) was associated with cessation of bleeding in 23/32 episodes (72%) and absence of significant bleeding in the three patients undergoing invasive procedures. In 14 successful treatment episodes (60.9%), bleeding ceased within 2 h of rFVIIa administration. Fifteen bleeds/potential bleeds were stopped/prevented with a single dose of rFVIIa. No adverse events were reported. These results indicate that rFVIIa is safe and may be effective for the prophylaxis or treatment of bleeding in patients with GT.