title: Long-term treatment of refractory thrombocytopenia in a patient with Wiskott-Aldrich syndrome with vincristine, immunoglobulin, and methylprednisolone.
abstract: We report a child with Wiskott-Aldrich syndrome with severe, refractory, symptomatic thrombocytopenia who achieved an excellent response to combination therapy with vincristine 1.5 mg/m(2) x 1 day, intravenous immunoglobulin 1 g/kg x 3 days, and methylprednisolone 25 mg/kg x 3 days (VIM) for 7 years after failing multiple treatments. He did not have a histocompatible donor for bone marrow transplantation. When the patient ceased to respond to this regimen, he was rescued with pulse dexamethasone. Vincristine, immunoglobulin, and methylprednisolone might serve as a novel treatment option for the patient with refractory thrombocytopenia. Our patient had a sustained remission of symptomatic thrombocytopenia without toxicity. Furthermore, pulse dexamethasone might be an alternative treatment option to which patients with Wiskott-Aldrich syndrome may respond.
authors.affiliation: Department of Pediatrics, Division of Hematology/Oncology, Loma Linda University, Loma Linda, California 92350, USA. gyoung@ahs.llumc.edu
authors.forename: G
authors.initials: G
authors.lastname: Young
authors.forename: A L
authors.initials: AL
authors.lastname: Angiolillo
mesh_terms: D000972:Antineoplastic Agents, Phytogenic / Q000627:therapeutic use*; D002648:Child; D003907:Dexamethasone / Q000627:therapeutic use; D004359:Drug Therapy, Combination; D005938:Glucocorticoids / Q000627:therapeutic use*; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D008775:Methylprednisolone / Q000627:therapeutic use*; D011696:Purpura, Thrombocytopenic / Q000188:drug therapy* / Q000209:etiology; D013156:Splenectomy; D014750:Vincristine / Q000627:therapeutic use*; D014923:Wiskott-Aldrich Syndrome / Q000150:complications / Q000188:drug therapy*
chemical_list: D000972:Antineoplastic Agents, Phytogenic; D005938:Glucocorticoids; D016756:Immunoglobulins, Intravenous; D014750:Vincristine; D003907:Dexamethasone; D008775:Methylprednisolone