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