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

Research output: Contribution to journalArticlepeer-review

188 Scopus citations


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 × 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.

Original languageEnglish (US)
Pages (from-to)2639-2642
Number of pages4
Issue number7
StatePublished - Apr 1 2006

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology


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