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Hoffmann, R. A wiki for the life sciences where authorship matters. Nature Genetics (2008)

Response of resistant idiopathic thrombocytopenic purpura to pulsed high-dose dexamethasone therapy.

BACKGROUND. Most patients with chronic idiopathic thrombocytopenic purpura have a response to corticosteroids or intravenous immune globulin, but improvement is often transitory. Splenectomy may provide only a short-term benefit. Because pulsed high-dose therapy with potent synthetic corticosteroids is inexpensive, well tolerated, and effective in patients with secretory B-cell neoplasms, a similar regimen was examined for its efficacy in patients with chronic idiopathic thrombocytopenic purpura that was resistant to other treatments. METHODS. Ten consecutively referred patients who had persistent symptomatic idiopathic thrombocytopenic purpura after undergoing at least two standard therapies were treated with six cycles of dexamethasone (40 mg per day for 4 sequential days every 28 days). RESULTS. All patients had increased platelet counts (mean [+/- SD] count before treatment, 12,000 +/- 8200 per cubic millimeter; after treatment, 248,000 +/- 130,000 per cubic millimeter). The platelet counts remained above 100,000 per cubic millimeter for at least six months after the last cycle of treatment. There were no serious side effects. Features of hyperadrenocorticism due to prior corticosteroid therapy resolved during treatment. The cost of the drug was approximately $100 per patient. CONCLUSIONS. Although the possibility of spontaneous remission and a delayed benefit from prior therapy cannot be excluded in this small group of patients, pulsed high-dose treatment with dexamethasone may provide a low-cost therapeutic option with minimal side effects in patients with refractory idiopathic thrombocytopenic purpura.[1]


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