Clinical efficacy and recovery levels of recombinant FVIIa (NovoSeven) in the treatment of intracranial haemorrhage in severe neonatal FVII deficiency

The use of replacement FVII is critical to the successful treatment of life -threatening bleeds in newborns and infants with severe FVII deficiency (<1 %). However, the clinical efficacy, optimum dosage and pharmacologic recove ry of rFVIIa in such children has not been studied systematically. This rep ort is a case of an infant with severe FVII deficiency (FVII:C at 0%) and m assive intracranial haemorrhage in which successful use of rFVIIa (NovoSeve n) was carefully monitored. The drug was administered by intravenous bolus through a central line every 4 h at each of three dose levels: 15 mu g kg(- 1), 22 mu g kg(-1) and 30 mu g kg(-1). FVII:C was >100% between 30 and 180 min after each infusion with mean trough levels above 25% for all three dos e levels. There was no evidence of hyper-coagulation as indicated by measur ements of the platelet count, D-dimer, plasma protamine paracoagulant and f ibrinogen levels in spite of high FVII:C concentration. In this infant, rFVIIa was well-tolerated, maintained effective haemostasis with good clinical outcome, and produced consistent therapeutic mean troug h levels above 25% FVII:C even at 15 mu g kg(-1) every 4 h.