Systematic Review: Efficacy and Safety of Rituximab for Adults with Idiopathic Thrombocytopenic Purpura

January 3, 2007

BlankSystematic Review: Efficacy and Safety of Rituximab for Adults with

Idiopathic Thrombocytopenic Purpura

M. Arnold, MD, MSc; Francesco Dentali, MD; Mark A. Crowther, MD, MSc;

Ralph M. Meyer, MD; J. Cook, PhD; Sigouin, MSc; Graeme A.

Fraser, MD; Lim, MD, MSc; and G. Kelton, MD

2 January 2007 | Volume 146 Issue 1 | Pages 25-33

Background: Rituximab, a monoclonal anti-CD20 antibody, is increasingly used to

treat idiopathic thrombocytopenic purpura (ITP).

Purpose: To systematically review the literature on the efficacy and safety of

rituximab for the treatment of adults with ITP.

Data Sources: MEDLINE, EMBASE, the Cochrane Library, abstracts from the American

Societies of Hematology and Clinical Oncology annual meetings, and

bibliographies of relevant articles and reviews were searched in duplicate until

April 2006.

Study Selection: Descriptive and comparative studies in any language that met

predefined inclusion criteria were eligible. Efficacy analysis was restricted to

studies enrolling 5 or more patients.

Data Extraction: Platelet count response, toxicities, dose, previous treatments,

baseline platelet count, duration of ITP, study design, and sources of funding

were extracted in duplicate.

Data Synthesis: We identified 19 eligible reports on efficacy (313 patients) and

29 on safety (306 patients). Weighted means for complete response (platelet

count > 150 x 109 cells/L) and overall response (platelet count > 50 x 109

cells/L) with rituximab were 43.6% (95% CI, 29.5% to 57.7%) and 62.5% (CI, 52.6%

to 72.5%), respectively. Responses lasted from 2 to 48 months. Nearly all

patients had received corticosteroids, and 53.8% had undergone splenectomy. Nine

patients (2.9%) died.

Limitations: There were no controlled studies, and no studies met all criteria

for study quality. Reported deaths could not necessarily be attributed to

rituximab. Overall, the number of rituximab-treated patients with ITP reported

in the literature is small.

Conclusions: Rituximab resulted in an overall platelet count response in 62.5%

of adults with ITP. However, this finding derives from uncontrolled studies that

also reported significant toxicities, including death in 2.9% of cases. These

data suggest that providers should avoid indiscriminate use of rituximab and

that randomized, controlled trials of rituximab for ITP are urgently needed.

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