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Blood, 1 April 2006, Vol. 107, No. 7, pp. 2639-2642. Prepublished online as a Blood First Edition Paper on December 13, 2005; DOI 10.1182/blood-2005-08-3518.
CLINICAL TRIALS AND OBSERVATIONS Prospective phase 1/2 study of rituximab in childhood and adolescent chronic immune thrombocytopenic purpuraFrom the Division of Hematology/Oncology and the Clinical Research Program, Children's Hospital Boston, Dana-Farber Cancer Institute and Harvard Medical School, MA; the Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas; Weill Medical College at Cornell University, New York, NY; Baylor College of Medicine, Houston, TX; Emory University School of Medicine, Atlanta, GA; Van Eslander Cancer Center, St John Hospital, Detroit, MI; University of California, Los Angeles (UCLA)/Mattel Children's Hospital at UCLA; University of California, San Francisco; and Stanford University, CA.
We assessed safety and efficacy of rituximab in a prospective study of 36 patients, age 2.6 to 18.3 years, with severe chronic immune thrombocytopenic purpura (ITP). The primary outcome of sustained platelets above 50 x 109/L (50 000/mm3) during 4 consecutive weeks, starting in weeks 9 to 12, was achieved by 11 of 36 patients (31%, confidence interval [CI], 16% to 48%). Median response time was 1 week (range, 1 to 7 weeks). Attainment of the primary outcome was not associated with age, prior pharmacologic responses, prior splenectomy, ITP duration, screening platelet count, refractoriness, or IgM reduction. First-dose, infusion-related toxicity was common (47%) despite premedication. Significant drug-related toxicities included third-dose hypotension (n = 1) and serum sickness (n = 2). Peripheral B cells were depleted in all subjects. IgM decreased 3.4% per week, but IgG did not significantly decrease. Rituximab was well tolerated, with manageable infusion-related side effects, but 6% of subjects developed serum sickness. Rituximab is beneficial for some pediatric patients with severe, chronic ITP.
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