Efficacy and Safety of Mesenchymal Stromal Cells for Steroid-Refractory Acute Graft-versus-Host Disease: An Updated Meta-Analysis of Randomized Controlled Trials.

IF 4.5 3区医学 Q2 CELL & TISSUE ENGINEERING

Stem Cell Reviews and Reports Pub Date : 2025-07-22 DOI:10.1007/s12015-025-10947-z

Iftikhar Khan, Umama Rehman, Fatima Aslam, Saad Khan, Umna Bhatti, Hussain Ramzan, Fatima Naveed, Javeria Nawaz, Syeda Malaika Raza, Kiran Inam, Faiza Rajput, Syed Muhammad Seyab, Muhammad Riyyan, Ayesha Imran Butt, Hira Habib, Ehsanullah Alokozay

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引用次数: 0

Abstract

Background: Steroid-refractory acute graft-versus-host disease (SR-aGVHD) is a life-threatening complication of allogeneic hematopoietic stem cell transplantation with limited effective second-line therapies. Mesenchymal stromal cells (MSCs) have emerged as a promising therapeutic option due to their immunomodulatory properties; however, their clinical efficacy and safety remain under debate.

Methods: A systematic review and meta-analysis were conducted in accordance with the PRISMA 2020 guidelines, including four randomized controlled trials (RCTs) comprising 650 patients. Primary outcomes included overall response rate (ORR), complete response (CR), and overall survival (OS). Secondary outcomes assessed organ-specific responses, adverse events (AEs), serious adverse events (SAEs), and failure-free survival.

Results: MSC therapy significantly improved overall response rate (ORR) (RR = 1.13; 95% CI: 1.04-1.23; P = 0.005) and complete response (CR) rate (RR = 1.60; 95% CI: 1.35-1.90; P < 0.00001), particularly in patients with grade III-IV acute graft-versus-host disease (aGVHD) and those with gut and skin involvement. There was no significant improvement in OS (RR = 1.05; 95% CI: 0.90-1.23; P = 0.52), AEs (RR = 1.00; P = 0.96), or SAEs (RR = 1.01; P = 0.79). MSCs showed significant benefit in reducing multi-organ aGVHD (RR = 1.25; P = 0.007), but not for liver involvement or grade II-IV disease. Heterogeneity across studies was generally low to moderate.

Conclusion: MSCs demonstrate significant efficacy in improving short-term clinical responses in SR-aGVHD, without increasing adverse events, particularly in cases of severe and multi-organ disease. However, they do not confer a survival benefit. Further large-scale, standardized, randomized controlled trials (RCTs) with long-term follow-up are warranted to validate these findings and inform clinical implementation.

Clinical trial number: Not applicable.

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间充质基质细胞治疗类固醇难治性急性移植物抗宿主病的疗效和安全性：一项随机对照试验的最新meta分析

背景：类固醇难治性急性移植物抗宿主病（SR-aGVHD）是异基因造血干细胞移植的一种危及生命的并发症，其有效的二线治疗方法有限。间充质基质细胞（MSCs）由于其免疫调节特性而成为一种有希望的治疗选择；然而，它们的临床疗效和安全性仍存在争议。方法：根据PRISMA 2020指南进行系统评价和荟萃分析，包括4项随机对照试验（rct），共650例患者。主要结局包括总缓解率（ORR）、完全缓解（CR）和总生存期（OS）。次要结局评估了器官特异性反应、不良事件（ae）、严重不良事件（sae）和无衰竭生存期。结果：MSC治疗显著提高总有效率（ORR） (RR = 1.13；95% ci: 1.04-1.23；P = 0.005)和完全缓解（CR）率(RR = 1.60；95% ci: 1.35-1.90；结论：MSCs在改善SR-aGVHD的短期临床反应方面表现出显著的疗效，没有增加不良事件，特别是在严重和多器官疾病的情况下。然而，它们并不能给生存带来好处。需要进一步的大规模、标准化、随机对照试验（rct）和长期随访来验证这些发现并为临床实施提供信息。临床试验号：不适用。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Stem Cell Reviews and Reports 医学-细胞生物学

CiteScore

9.30

自引率

4.20%

发文量

审稿时长

3 months

期刊介绍： The purpose of Stem Cell Reviews and Reports is to cover contemporary and emerging areas in stem cell research and regenerative medicine. The journal will consider for publication: i) solicited or unsolicited reviews of topical areas of stem cell biology that highlight, critique and synthesize recent important findings in the field. ii) full length and short reports presenting original experimental work. iii) translational stem cell studies describing results of clinical trials using stem cells as therapeutics. iv) papers focused on diseases of stem cells. v) hypothesis and commentary articles as opinion-based pieces in which authors can propose a new theory, interpretation of a controversial area in stem cell biology, or a stem cell biology question or paradigm. These articles contain more speculation than reviews, but they should be based on solid rationale. vi) protocols as peer-reviewed procedures that provide step-by-step descriptions, outlined in sufficient detail, so that both experts and novices can apply them to their own research. vii) letters to the editor and correspondence. In order to facilitate this exchange of scientific information and exciting novel ideas, the journal has created five thematic sections, focusing on: i) the role of adult stem cells in tissue regeneration; ii) progress in research on induced pluripotent stem cells, embryonic stem cells and mechanism governing embryogenesis and tissue development; iii) the role of microenvironment and extracellular microvesicles in directing the fate of stem cells; iv) mechanisms of stem cell trafficking, stem cell mobilization and homing with special emphasis on hematopoiesis; v) the role of stem cells in aging processes and cancerogenesis.