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Long-term treatment of refractory thrombocytopenia in a patient with wiskott-aldrich syndrome with vincristine, immunoglobulin, and methylprednisolone

✍ Scribed by Young, Guy; Angiolillo, Anne L.

Publisher: John Wiley and Sons
Year: 1999
Tongue: English
Weight: 41 KB
Volume: 62
Category: Article
ISSN: 0361-8609
DOI: 10.1002/(sici)1096-8652(199911)62:3<183::aid-ajh8>3.0.co;2-a

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✦ Synopsis

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 × 1 day, intravenous immunoglobulin 1 g/kg × 3 days, and methylprednisolone 25 mg/kg × 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.

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