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World J Stem Cells. Jul 26, 2026; 18(7): 122048
Published online Jul 26, 2026. doi: 10.4252/wjsc.122048
Efficacy and safety of cell-based regenerative therapies for fistula-related diseases: An umbrella review of systematic reviews
Yun-Long Duan, Yong-Hao Wen, Si-Ai Chen, Ru Nie, Ming-Quan Pang, Man-Jun Deng, Zhi-Xin Wang, Hai-Ning Fan
Yun-Long Duan, Yong-Hao Wen, Man-Jun Deng, Hai-Ning Fan, Stem Cell and Regenerative Medicine Laboratory, Affiliated Hospital of Qinghai University, Xining 810001, Qinghai Province, China
Yun-Long Duan, Yong-Hao Wen, Ru Nie, School of Clinical Medicine, Affiliated Hospital of Qinghai University, Xining 810001, Qinghai Province, China
Si-Ai Chen, Department of Basic Medical Sciences, Qinghai University Medical College, Xining 810001, Qinghai Province, China
Ru Nie, Stem Cell and Regenerative Medicine Laboratory, Qinghai University Medical College, Xining 810001, Qinghai Province, China
Ming-Quan Pang, Zhi-Xin Wang, Hai-Ning Fan, Department of Hepatopancreatobiliary Surgery, Affiliated Hospital of Qinghai University, Xining 810001, Qinghai Province, China
Co-corresponding authors: Zhi-Xin Wang and Hai-Ning Fan.
Author contributions: Duan YL and Wen YH contributed to the conception and design of the study; Duan YL, Wen YH, Chen SA, and Nie R contributed to the literature search and data collection and analysis; Duan YL interpreted the findings and prepared the first draft of the manuscript; Pang MQ, Deng MJ, Wang ZX, and Fan HN contributed to the protocol development and reviewed the manuscript; Deng MJ, Wang ZX, and Fan HN had full access to all study data and took final responsibility for the decision to submit the manuscript for publication. Wang ZX and Fan HN are co-corresponding authors and contributed equally to this work. Wang ZX was primarily responsible for the academic guidance, data analysis, manuscript revision. Fan HN has taken on the primary role in the study conception, experimental design, and project administration. Both authors were involved in supervision, review & editing of the manuscript, and have approved the final version. They agreed to be jointly accountable for all aspects of the work.
AI contribution statement: AI tools (specifically ChatGPT) were used only for language editing and formatting support. It was not used to generate research data, analyze data, interpret results, or draw conclusions. All output was critically reviewed and revised by the authors.
Supported by the Fourth “Qinghai Scholar” Program, No. 2025-qhxz-fhn.
Conflict-of-interest statement: All the authors report no relevant conflicts of interest for this article.
PRISMA 2009 Checklist statement: The authors have read the PRISMA 2009 Checklist, and the manuscript was prepared and revised according to the PRISMA 2009 Checklist.
Corresponding author: Hai-Ning Fan, PhD, Chief Physician, Full Professor, Department of Hepatopancreatobiliary Surgery, Affiliated Hospital of Qinghai University, No. 29 Tongren Road, Xining 810001, Qinghai Province, China. fanhaining@medmail.com.cn
Received: April 8, 2026
Revised: May 13, 2026
Accepted: June 18, 2026
Published online: July 26, 2026
Processing time: 107 Days and 22.3 Hours
Abstract
BACKGROUND

Fistula-related diseases are characterized by a chronic course, high recurrence rates, and limited therapeutic benefits. Conventional treatments are associated with suboptimal healing rates and substantial complication risks. Cell-based regenerative therapies have attracted increasing attention due to their combined immunomodulatory and tissue-repair properties. However, evidence regarding their overall efficacy and safety remains fragmented and heterogeneous.

AIM

To determine the overall efficacy and safety of cell-based regenerative therapies for fistula-related diseases through a systematic review and meta-analysis.

METHODS

PubMed, EMBASE, Web of Science, and the Cochrane Library were searched from database inception through December 15, 2025 to identify systematic reviews and meta-analyses examining regenerative therapies for fistula-related diseases. Review quality was appraised with A Measurement Tool to Assess Systematic Reviews 2, certainty of evidence was evaluated according to Grading of Recommendations, Assessment, Development and Evaluation, overlap among primary studies was measured using the corrected covered area, and visual evidence maps were generated.

RESULTS

Thirty-five eligible systematic reviews and meta-analyses were included, and most of the evidence concerned perianal fistulas (PF) and fistulizing Crohn’s disease (FCD). Mesenchymal stem cells (MSCs) were the most frequently evaluated intervention, whereas evidence for platelet-rich plasma (PRP), MSC-derived exosomes, and hematopoietic stem cells (HSCs) was limited. Across PF and FCD, MSC-based therapy was generally associated with higher rates of combined remission, clinical and/or radiological healing, clinical remission, and clinical response than comparator treatments, although the strength and consistency of evidence varied across outcomes and reviews. Some findings were supported by moderate-certainty evidence, but most included reviews were rated as low or critically low confidence. Treatment effects appeared to differ according to cell source and delivery strategy, with adipose-derived, allogeneic, and locally injected MSC more often associated with favorable outcomes. MSC therapy did not increase the overall risk of adverse events (AE), and low-certainty evidence suggested that it may be associated with a reduced risk of serious AEs. Evidence for PRP, exosomes, and HSCs remained sparse and insufficient for firm conclusions.

CONCLUSION

Thirty-five eligible systematic reviews and meta-analyses were included, and most of the evidence concerned PF and FCD. MSCs were the most frequently evaluated intervention, whereas evidence for PRP, MSC-derived exosomes, and HSCs was limited. Across PF and FCD, MSC-based therapy was generally associated with higher rates of combined remission, clinical and/or radiological healing, clinical remission, and clinical response than comparator treatments, although the strength and consistency of evidence varied across outcomes and reviews. Some findings were supported by moderate-certainty evidence, but most included reviews were rated as low or critically low confidence. Treatment effects appeared to differ according to cell source and delivery strategy, with adipose-derived, allogeneic, and locally injected MSC more often associated with favorable outcomes. MSC therapy did not increase the overall risk of AEs, and low-certainty evidence suggested that it may be associated with a reduced risk of serious adverse events. Evidence for PRP, exosomes, and HSCs remained sparse and insufficient for firm conclusions.

Keywords: Regenerative therapy; Mesenchymal stem cells; Platelet-rich plasma; Perianal fistulas; Fistulizing Crohn’s disease

Core Tip: This umbrella review demonstrates that mesenchymal stem cells (MSCs) based regenerative therapy is associated with meaningful improvements in healing and remission in perianal fistulas and fistulizing Crohn’s disease. The most consistent benefits were observed with adipose-derived, allogeneic, and locally injected MSC. MSC therapy was generally well tolerated, with no increase in overall adverse events and potential reduction in serious adverse events. Among currently studied regenerative approaches, MSC appears to be the most promising option for fistula-related diseases.

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