Efficacy and safety of rituximab for minors with immune thrombocytopenia: a systematic review and meta-analysis.

Objective: We reviewed relevant research on rituximab (RTX) treatment for pediatric immune thrombocytopenia (ITP) to elucidate the efficacy and safety of RTX.

Methods: Prospective clinical trials of RTX for the treatment of pediatric ITP were collected by searching the PubMed, Cochrane Library, Web of Science, and OVID: EMBASE databases and ClinicalTrials.gov. We examined rates of overall response (OR), complete response (CR), partial response (PR), sustained response (SR), relapse (R), and adverse drug reaction (ADR). The Methodological Index for Nonrandomized Studies scale was used, and sensitivity analyses were performed.

Results: For five studies, including 100 patients, the pooled OR, CR, PR, SR, R, and ADR rates were 52% (95% CI: 0.36-0.77,  = 78%), 52% (95% CI: 0.41-0.67,  = 45%), 18% (95% CI: 0.10-0.33,  = 33%), 43% (95% CI: 0.29-0.63,  = 0%), 25% (95% CI: 0.06-0.96,  = 52%), and 30% (95% CI: 0.15-0.58,  = 64%), respectively.

Conclusion: There is evidence, albeit low quality, that RTX may be a better second-line therapy than splenectomy for children with ITP; however, its efficacy and safety need to be validated by further high-quality clinical trials, such as randomized controlled trials.