A pilot randomized trial of adjuvant rituximab or placebo for nonsplenectomized patients with immune thrombocytopenia
Open Access
- 9 February 2012
- journal article
- research article
- Published by American Society of Hematology in Blood
- Vol. 119 (6), 1356-1362
- https://doi.org/10.1182/blood-2011-08-374777
Abstract
The benefit of adding rituximab to standard treatment in nonsplenectomized patients with primary immune thrombocytopenia (ITP) is uncertain. We performed a pilot randomized trial to determine the feasibility of recruitment, protocol adherence, and blinding of a larger trial of rituximab versus placebo; and to evaluate the potential efficacy of adjuvant rituximab in ITP. Nonsplenectomized adults with newly diagnosed or relapsed ITP who were receiving standard ITP therapy for a platelet count below 30 × 109/L were randomly allocated to receive 4 weekly infusions of 375 mg/m2 rituximab or saline placebo. Sixty patients were recruited over 46 months, which was slower than anticipated. Protocol adherence and follow-up targets were achieved, and blinding was successful for research staff but not for patients. After 6 months, there was no difference between rituximab and placebo groups for the composite outcome of any platelet count below 50 × 109/L, significant bleeding or rescue treatment once standard treatment was stopped (21/32 [65.6%] vs 21/26 [80.8%]; relative risk = 0.81, 95% confidence intervals, 0.59%-1.11%). Timely accrual poses a challenge to the conduct of a large randomized trial of rituximab for presplenectomy ITP. No difference in the frequency of the composite outcome was observed in this pilot trial (registered at www.clinicaltrials.gov NCT00372892).Keywords
This publication has 28 references indexed in Scilit:
- Progressive multifocal leukoencephalopathy after rituximab therapy in HIV-negative patients: a report of 57 cases from the Research on Adverse Drug Events and Reports projectBlood, 2009
- Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working groupBlood, 2009
- Improved quality of life for romiplostim‐treated patients with chronic immune thrombocytopenic purpura: results from two randomized, placebo‐controlled trialsBritish Journal of Haematology, 2009
- The design and interpretation of pilot trials in clinical research in critical careCritical Care Medicine, 2009
- Severe hemorrhage in children with newly diagnosed immune thrombocytopenic purpuraBlood, 2008
- The immune thrombocytopenic purpura (ITP) bleeding score: assessment of bleeding in patients with ITPBritish Journal of Haematology, 2007
- Mapping helper T-cell epitopes on platelet membrane glycoprotein IIIa in chronic autoimmune thrombocytopenic purpuraBlood, 2007
- The efficacy and safety of B‐cell depletion with anti‐CD20 monoclonal antibody in adults with chronic immune thrombocytopenic purpuraBritish Journal of Haematology, 2004
- CTCAE v3.0: development of a comprehensive grading system for the adverse effects of cancer treatmentSeminars in Radiation Oncology, 2003
- The MOS 36-Item Short-Form Health Survey (SF-36)Medical Care, 1993