title: Refractory thrombotic thrombocytopenic purpura in a 16-year-old girl: successful treatment with bortezomib.
abstract: We present a case of a 16-year-old girl with autoimmune thrombotic thrombocytopenic purpura (TTP), refractory to plasma exchange and high-dose prednisone. Despite the additional treatment with rituximab, she developed renal and neurological complications with ongoing hemolysis and thrombocytopenia. Bortezomib, a proteasome inhibitor and thereby blocking plasma cells, was added, and our patient recovered. We suggest that bortezomib can be of additional value in severe immunologically mediated TTP in adolescents. Its use may prevent the necessity of other invasive therapies, such as splenectomy, with significant side effects.
authors.affiliation: Department of Pediatric Nephrology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands.
authors.forename: Tessa
authors.initials: T
authors.lastname: van Balen
authors.forename: Michiel F
authors.initials: MF
authors.lastname: Schreuder
authors.forename: Huib
authors.initials: H
authors.lastname: de Jong
authors.forename: Nicole C A J
authors.initials: NC
authors.lastname: van de Kar
mesh_terms: D051722:ADAM Proteins / Q000378:metabolism; D000071120:ADAMTS13 Protein; D000293:Adolescent; D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D001897:Boronic Acids / Q000008:administration & dosage / Q000627:therapeutic use; D000069286:Bortezomib; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D010951:Plasma Exchange; D011697:Purpura, Thrombotic Thrombocytopenic / Q000175:diagnosis / Q000188:drug therapy* / Q000628:therapy; D011719:Pyrazines / Q000008:administration & dosage / Q000627:therapeutic use; D012008:Recurrence; D012074:Remission Induction; D016896:Treatment Outcome
chemical_list: D000911:Antibodies, Monoclonal; D001897:Boronic Acids; D007166:Immunosuppressive Agents; D011719:Pyrazines; D000069286:Bortezomib; D051722:ADAM Proteins; D000071120:ADAMTS13 Protein; C099604:ADAMTS13 protein, human
keywords: ADAMTS13 autoantibodies; bortezomib; plasma exchange; rituximab; thrombotic thrombocytopenic purpura