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Blood, 15 November 2007, Vol. 110, No. 10, pp. 3526-3531. Prepublished online as a Blood First Edition Paper on August 13, 2007; DOI 10.1182/blood-2007-01-065763.
CLINICAL TRIALS AND OBSERVATIONS Multiagent induction and maintenance therapy for patients with refractory immune thrombocytopenic purpura (ITP)1 The Brookdale University Hospital and Medical Center Department of Pediatrics, Hematology Oncology, Brooklyn, NY; 2 The Preston Robert Tisch Brain Tumor Center and the Departments of Pediatrics and Surgery, Duke University Medical Center, Durham, NC; 3 Children's National Medical Center, Department of Hematology-Oncology, Washington, DC; and 4 Weill Medical College of Cornell University Division of Pediatric Hematology Oncology, New York, NY Patients with severe immune thrombocytopenic purpura (ITP) may require an acute increase in the platelet count for surgery or ongoing hemorrhage as well as long-term maintenance treatment. Certain of these patients may be refractory to steroids, intravenous anti-D, intravenous immunoglobulin (IVIG), and splenectomy. Therefore, acute platelet increases were studied in 35 patients completely unresponsive to IVIG or high-dose steroid treatment. Because of their lack of response to either or both single agents, these patients were administered a 3- or 4-drug combination including IVIG 1 g/kg, intravenous methylprednisolone 30 mg/kg, Vinca alkaloids (VCR 0.03 mg/kg), and/or intravenous anti-D (50-75 µg/kg). Subsequent maintenance therapy with the oral combination of danazol (10-15 mg/kg) and azathioprine (2 mg/kg) was given to 18 of the 35 patients. Seventy-one percent of the patients responded to the intravenous combination treatment with acute platelet increases of at least 20x109/L to a level greater than 30x109/L. Two thirds of the patients given maintenance therapy achieved stable platelet counts greater than 50x109/L without other treatments. One patient developed an ileus, but otherwise there was little toxicity of combination treatment. Combination chemotherapy is a useful approach for patients with ITP refractory to conventional treatments both for acute induction and for long-term maintenance therapy.
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