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Refractory thrombotic thrombocytopenic purpura in a 16‐year‐old girl: successful treatment with bortezomib
Authors:Tessa van Balen  Michiel F. Schreuder  Huib de Jong  Nicole C. A. J. van de Kar
Affiliation:Department of Pediatric Nephrology, Radboud University Nijmegen Medical Centre, , Nijmegen, The Netherlands
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.
Keywords:thrombotic thrombocytopenic purpura  bortezomib  rituximab  plasma exchange  ADAMTS13 autoantibodies
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