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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.
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Submitted January 3, 2007
Accepted July 24, 2007
Multi-agent induction and maintenance therapy for patients with refractory immune thrombocytopenic purpura (ITP)
Donna M Boruchov, Sri Gururangan, M Catherine Driscoll, and James B Bussel*
Division of Pediatric Hematology and Oncology, The Brookdale University Hospital and Medical Center, Brooklyn, NY, United States
The Preston Robert Tisch Brain Tumor Center, and the Departments of Pediatrics & Surgery, Duke University Medical Center, Durham, NC, United States
Department of Hematology-Oncology, Children's National Medical Center, Washington, DC, United States
Division of Pediatric Hematology Oncology, Weill Medical College of Cornell University, New York, NY, United States
* Corresponding author; email: jbussel{at}med.cornell.edu.
Patients with severe 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, IV anti-D, 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 1gm/kg, IV methylprednisolone 30mg/kg, vinca alkaloids (VCR 0.03mg/kg), and/or IV anti-D (50-75mcg/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 IV combination treatment with acute platelet increases of at least 20,000/µ1 to a level greater than 30,000/µl. Two-thirds of the patients given maintenance therapy achieved stable platelet counts above 50,000/µl 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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