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Open Access 20-11-2023 | Mycophenolate Mofetil | REVIEW ARTICLE

Efficacy and safety of mycophenolate mofetil in patients with immune thrombocytopenic purpura: a systematic review and meta-analysis

Authors: Omar Ahmed Abdelwahab, Ahmed Mechi, Shereen Gahlan, Fatima-Elzahraa Hamadein, Hallas Kadhim, Doaa Ismail, Youssef Soliman, Mohamed El‑Samahy

Published in: Clinical Rheumatology

Abstract

Background

Immune thrombocytopenic purpura (ITP) is a challenging disease in its presentation and management as it may cause life-threatening hemorrhaging in vital organs and may resist several lines of treatment. This systematic review and meta-analysis aimed to evaluate the safety and efficacy of mycophenolate mofetil (MMF) in treating patients with ITP.

Methods

We systematically searched four electronic databases (PubMed, Scopus, Web of Science, and Cochrane Central Register of Controlled Trials) from inception until 10 October 2022. We included all clinical trials, either controlled or single arm, and prospective and retrospective observational studies that evaluate the efficacy and safety of MMF in patients with ITP. We assessed the risk of bias using three tools (ROBINS-I, Cochrane ROB-2, and NIH), each for eligible study design.

Results

Nine studies were included in this meta-analysis, with a total of 411 patients with ITP. We found that MMF demonstrated an overall response rate of (62.09%; 95% CI = [43.29 to 77.84]) and the complete response rate was (46.75%; 95% CI = [24.84 to 69.99]). The overall proportion of adverse events was (12%; 95% CI = [6 to 24]). After the sensitivity analysis, the overall response rate became 50%; 95% CI = [38 to 63]) and the complete response rate became (32%; 95% CI = [24 to 42]). However, MMF did not appear to affect white blood cell counts or hemoglobin levels significantly.

Conclusion

This systematic review and meta-analysis demonstrate that MMF appears to be an effective and relatively safe treatment option for patients with ITP when combined with steroids and even in those who have not responded to standard therapies (steroid-resistant cases). Further research with well-designed studies is warranted to better understand the factors influencing treatment response and to refine the use of MMF in the management of ITP. An interactive version of our analysis can be accessed from here: https://databoard.shinyapps.io/mycophenolate_meta/

Graphical abstract

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Title: Efficacy and safety of mycophenolate mofetil in patients with immune thrombocytopenic purpura: a systematic review and meta-analysis
Authors: Omar Ahmed Abdelwahab
Ahmed Mechi
Shereen Gahlan
Fatima-Elzahraa Hamadein
Hallas Kadhim
Doaa Ismail
Youssef Soliman
Mohamed El‑Samahy
Publication date: 20-11-2023
Publisher: Springer International Publishing
Keyword: Mycophenolate Mofetil
Published in: Clinical Rheumatology
Print ISSN: 0770-3198
Electronic ISSN: 1434-9949
DOI: https://doi.org/10.1007/s10067-023-06820-4

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