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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.
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CLINICAL TRIALS AND OBSERVATIONS Brief report
Prospective phase 1/2 study of rituximab in childhood and adolescent chronic immune thrombocytopenic purpura
Carolyn M. Bennett,
Zora R. Rogers,
Daniel D. Kinnamon,
James B. Bussel,
Donald H. Mahoney,
Thomas C. Abshire,
Hadi Sawaf,
Theodore B. Moore,
Mignon L. Loh,
Bertil E. Glader,
Maggie C. McCarthy,
Brigitta U. Mueller,
Thomas A. Olson,
Adonis N. Lorenzana,
William C. Mentzer,
George R. Buchanan,
Henry A. Feldman,
Ellis J. Neufeld, the Pediatric Rituximab/ITP Study Group and the Glaser Pediatric Research Network
From 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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