title: Late response to cyclosporine in refractory thrombotic thrombocytopenic purpura.
abstract: Plasma exchange (PEX) with fresh frozen plasma, usually in association with steroid therapy, has been shown to be the first-line treatment of thrombotic thrombocytopenic purpura. It works by removing ultralarge von Willebrand factor (vWF) multimers and inhibitory antibody and by supplying normal protease. For patients with disease refractory to PEX, there is no standardized treatment. Limited and sporadic success with different therapies (vincristine sulfate, prostacyclin, intravenous immunoglobulins, splenectomy) has been described. We report the case of a woman who developed refractory disease after an initial response to PEX and despite a very high number of PEX procedures performed. Low activity (< 5%) of serum vWF-cleaving protease and a low level of protease inhibitor were documented. The patient had a slow but sustained response when oral cyclosporine was administered concomitantly with PEX, which was slowly tapered. The activity of serum vWF-cleaving protease normalized. At relapse, treatment with cyclosporine, added after the failure of steroids and PEX, led to a lasting response. It is possible that in some cases cyclosporine therapy must be of particularly long duration before being considered ineffective.
authors.affiliation: Department of Oncology and Hematology, Niguarda Cà Granda Hospital, Milan, Italy. miccaimi@tin.it
authors.forename: Annamaria
authors.initials: A
authors.lastname: Nosari
authors.forename: Patrizia
authors.initials: P
authors.lastname: Bernuzzi
authors.forename: Roberto
authors.initials: R
authors.lastname: Corneo
authors.forename: Ester
authors.initials: E
authors.lastname: Pungolino
authors.forename: Giuliana
authors.initials: G
authors.lastname: Muti
authors.forename: Valentina
authors.initials: V
authors.lastname: Rossi
authors.forename: Enrica
authors.initials: E
authors.lastname: Morra
mesh_terms: D000328:Adult; D016572:Cyclosporine / Q000494:pharmacology* / Q000627:therapeutic use; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000494:pharmacology* / Q000627:therapeutic use; D010951:Plasma Exchange; D011697:Purpura, Thrombotic Thrombocytopenic / Q000188:drug therapy* / Q000473:pathology; D012008:Recurrence; D013256:Steroids / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents; D013256:Steroids; D016572:Cyclosporine