Prospective phase 1/2 study of rituximab in childhood and adolescent chronic immune thrombocytopenic purpura
| Autor: | George R. Buchanan, Zora R. Rogers, Thomas A. Olson, Brigitta U. Mueller, Donald H. Mahoney, Theodore B. Moore, Bertil Glader, Mignon L. Loh, Maggie McCarthy, Daniel D. Kinnamon, Carolyn M. Bennett, Ellis J. Neufeld, William C. Mentzer, Thomas C. Abshire, James B. Bussel, Adonis Lorenzana, Henry A. Feldman, Hadi Sawaf |
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| Jazyk: | angličtina |
| Rok vydání: | 2006 |
| Předmět: |
medicine.medical_specialty
Adolescent Clinical Trials and Observations medicine.medical_treatment Immunology Splenectomy Biochemistry Gastroenterology Antibodies Monoclonal Murine-Derived Serum Sickness immune system diseases Internal medicine hemic and lymphatic diseases Medicine Humans Immunologic Factors Prospective Studies Prospective cohort study Child Purpura Thrombocytopenic Idiopathic biology business.industry Patient Selection Antibodies Monoclonal Cell Biology Hematology medicine.disease Thrombocytopenic purpura Surgery Treatment Outcome Immunoglobulin M Child Preschool Toxicity Serum sickness biology.protein Rituximab Premedication Hypotension business medicine.drug |
| Popis: | 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 10(9)/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. |
| Databáze: | OpenAIRE |
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