title: Ten years of prophylactic treatment with fresh-frozen plasma in a child with chronic relapsing thrombotic thrombocytopenic purpura as a result of a congenital deficiency of von Willebrand factor-cleaving protease.
abstract: We report the results of 10 years of prophylactic fresh-frozen plasma (FFP) infusion therapy in a 14-year-old girl with chronic relapsing thrombotic thrombocytopenic purpura (TTP), in whom a severe congenital von Willebrand factor (VWF)-cleaving protease deficiency has been documented. Severe haemolytic crises triggered by infections were prevented and her present renal and neurological functions have been preserved. Sequential measurements of protease activity and platelet count after FFP infusion led us to conclude tentatively that 5% may be sufficient to degrade very large and adhesive VWF multimers.
authors.affiliation: Serviço de Hematologia do Hospital de Crianças Maria Pia, Porto, Portugal. hematologica@hmaria.min-saude.pt
authors.forename: J
authors.initials: J
authors.lastname: Barbot
authors.forename: E
authors.initials: E
authors.lastname: Costa
authors.forename: M
authors.initials: M
authors.lastname: Guerra
authors.forename: M S
authors.initials: MS
authors.lastname: Barreirinho
authors.forename: P
authors.initials: P
authors.lastname: Isvarlal
authors.forename: R
authors.initials: R
authors.lastname: Robles
authors.forename: H E
authors.initials: HE
authors.lastname: Gerritsen
authors.forename: B
authors.initials: B
authors.lastname: Lämmle
authors.forename: M
authors.initials: M
authors.lastname: Furlan
mesh_terms: D051722:ADAM Proteins; D000071120:ADAMTS13 Protein; D000293:Adolescent; D002908:Chronic Disease; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008666:Metalloendopeptidases / Q000172:deficiency* / Q000378:metabolism; D010375:Pedigree; D010949:Plasma; D010976:Platelet Count; D011697:Purpura, Thrombotic Thrombocytopenic / Q000097:blood / Q000235:genetics / Q000517:prevention & control*; D012008:Recurrence
chemical_list: D051722:ADAM Proteins; D008666:Metalloendopeptidases; D000071120:ADAMTS13 Protein; C099604:ADAMTS13 protein, human