title: The use of intermediate purity factor VIII concentrate BPL 8Y as prophylaxis and treatment in congenital thrombotic thrombocytopenic purpura.
abstract: This report presents seven children with congenital thrombotic thrombocytopenic purpura (TTP). Six had a history of severe neonatal unconjugated hyperbilirubinaemia and thrombocytopenia. The seventh child had no neonatal problems but has suffered three episodes of acute TTP. The subsequent clinical course of the children varied. Five had a relapsing-remitting course and one had chronic microangiopathic haemolytic anaemia. The five oldest children initially received plasma infusions but, because of viral safety issues and easier administration, they now receive intermediate purity US-sourced plasma-derived factor VIII concentrate: BPL 8Y. Effective prophylaxis and treatment is possible in congenital TTP using BPL 8Y.
authors.affiliation: Haemostasis Research Unit, UCL, London, UK.
authors.forename: Marie
authors.initials: M
authors.lastname: Scully
authors.forename: Michael
authors.initials: M
authors.lastname: Gattens
authors.forename: Kate
authors.initials: K
authors.lastname: Khair
authors.forename: Ri
authors.initials: R
authors.lastname: Liesner
mesh_terms: D005169:Factor VIII / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D006932:Hyperbilirubinemia / Q000209:etiology; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D010976:Platelet Count; D011697:Purpura, Thrombotic Thrombocytopenic / Q000150:complications / Q000151:congenital* / Q000175:diagnosis / Q000628:therapy*; D012008:Recurrence; D016896:Treatment Outcome
chemical_list: D005169:Factor VIII