title: Recombinant FVIIa in the management of intracerebral haemorrhage in severe thrombocytopenia unresponsive to platelet-enhancing treatment.
abstract: Intracranial haemorrhage (ICH) is a dramatic and potentially life-threatening presentation of children with thrombocytopenia. Management is limited to supportive care. Recent evidence suggests that ongoing bleeding following the initial ICH may result in greater neurological morbidity and mortality. Haemostatic agents, including recombinant factor VIIa (rFVIIa), a product licensed for use in patients with haemophilia and inhibitors, may be helpful in reducing bleeding in children with refractory thrombocytopenia. We present the case of a 16-year-old girl with severe refractory immune thrombocytopenia, who presented with a major ICH and responded to treatment that included rFVIIa and platelet transfusions. The dose of rFVIIa was empirically chosen and based on reported cases in the literature. The case highlights a number of issues regarding off-label use of rFVIIa and demonstrates the need to prospectively collect accurate information on the off-label use of this new potentially useful medication.
authors.affiliation: Division of Haematology/Oncology, Department of Paediatrics, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
authors.forename: C
authors.initials: C
authors.lastname: Barnes
authors.forename: V
authors.initials: V
authors.lastname: Blanchette
authors.forename: P
authors.initials: P
authors.lastname: Canning
authors.forename: M
authors.initials: M
authors.lastname: Carcao
mesh_terms: D000293:Adolescent; D002543:Cerebral Hemorrhage / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000209:etiology; D005167:Factor VII / Q000008:administration & dosage*; D015942:Factor VIIa; D005260:Female; D006801:Humans; D017713:Platelet Transfusion; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000150:complications* / Q000000981:diagnostic imaging / Q000628:therapy; D011859:Radiography; D011994:Recombinant Proteins / Q000008:administration & dosage
chemical_list: D011994:Recombinant Proteins; D005167:Factor VII; C103587:recombinant FVIIa; D015942:Factor VIIa