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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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Submitted August 31, 2005
Accepted November 22, 2005
Prospective phase I/II 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*, and
Division of Hematology/Oncology, Children's Hospital Boston, Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA, USA
University of Texas, Southwestern Medical Center, Dallas, TX, USA
Clinical Research Program, Children's Hospital Boston, Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA, USA
Weill Medical College at Cornell University, New York, NY, USA
Baylor College of Medicine, Houston, TX, USA
Emory University School of Medicine, Atlanta, GA, USA
Van Eslander Cancer Center, St. John Hospital, Detroit, MI, USA
University of California, Los Angeles/Mattel Children's Hospital at UCLA, Los Angeles, CA, USA
University of California, San Francisco, CA, USA
Stanford University, Stanford, CA, USA
* Corresponding author; email: ellis.neufeld{at}childrens.harvard.edu.
We assessed safety and efficacy of rituximab in a prospective study of 36 patients, age 2.6-18.3 years, with severe chronic immune thrombocytopenic purpura (ITP). The primary outcome of sustained platelets > 50,000/mm3 during four consecutive weeks, starting in weeks 9 to12, was achieved by 11 of 36 patients (31%, CI 16-48%). Median response time was one week (range 1-7). 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 wk, 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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