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Research Article

Progress on the efficacy and potential mechanisms of rapamycin in the treatment of immune thrombocytopenia

, , , &
Pages 1282-1289 | Received 03 Aug 2022, Accepted 20 Nov 2022, Published online: 05 Dec 2022
 

ABSTRACT

Objective

The complex pathogenesis of relapsed and refractory (R/R) immune thrombocytopenia (ITP) contributes to the varied efficacy and tolerability of current treatment regimens. Rapamycin, an immunomodulatory agent, was originally used in the prevention of organ rejection after organ transplantation. Additional evidence now shows that rapamycin can successfully treat R/R ITP. Here, we summarize recent clinical progress on the role and potential mechanism of rapamycin in the treatment of ITP.

Methods

PubMed, Web of Science and CNKI database were searched to identify eligible studies, and the clinical data and preclinical studies on the use of mTOR inhibitors in ITP treatment were reviewed. The key results (efficacy and safety) of the most recent clinical reports were summarized.

Results summarized

Case series provide evidence of the effectiveness and tolerable safety profile of rapamycin in ITP, including primary and some secondary ITP. Mechanistic explorations indicate that rapamycin can regulate immune cell subsets (Th1, Th2, Th17, Treg, Breg, MDSC, etc.), modulate cytokine secretion (IL-6, IL-10, TGF-β, BAFF, etc.) and promote platelet autophagy.

Conclusions

Emerging clinical data and basic studies suggest that rapamycin, as a multifaceted regulator, could provide a new promising option for the therapy of ITP. Additional research is needed to identify those patients which may benefit the most, as well as therapeutic regimens with which rapamycin may be combined.

Disclosure statement

No potential conflict of interest was reported by the author(s).

Additional information

Funding

This work was supported by The Projects of International Cooperation and Exchanges NSFC [grant number 82020108004], Natural Science Foundation of Chongqing [grant numbers cstc2019jcyjmsxmX0273 and cstc2020jcyjmsxmX1086], and National Center for Clinical Medicine Research on Blood System Diseases 2020 Open Project (Key Project) [grant number 2020ZKZC02].