Autologous adipose tissue-derived stem cells for the treatment of complex perianal fistulas not associated with Crohn’s disease: a phase II clinical trial for safety and efficacy
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Injection of adipose tissue-derived stem cells (ASCs) is a novel method for the treatment of complex perianal fistulas. We aimed to evaluate the safety and efficacy of ASCs in the treatment of complex anal fistulas not associated with Crohn’s disease.
A phase II clinical trial was performed comparing two different doses of ASCs (group 1: 1 × 107 cells/mL and group 2: 2 × 107 cells/mL). Eligible patients were administered an amount of ASCs proportional to the length of the fistula by injection into the submucosal layer surrounding the internal opening and inside of the fistula tract. ASCs at twice the initial concentration were administered if complete closure was not achieved within 8 weeks. The efficacy endpoint was the complete closure of fistulas 8 weeks after injection. Patients demonstrating complete closure at week 8 were subjected to follow-up for 6 months.
Fifteen patients were injected with ASCs; thirteen completed the study. Complete closure was observed in 69.2% (9/13) of patients at 8 weeks. Three of five patients in group 1, and six of eight in group 2 displayed complete closure; no significant differences were observed between the groups. Six of nine patients who showed complete closure participated in additional follow-up; five (83.3%) showed persistent response at 6 months. No grade 3 or 4 adverse events (AEs) were observed; observed AEs were not related to ASC treatment.
ASCs might be a good option for the treatment of complex perianal fistulas are not healed by conventional operative procedures.
KeywordsStem cells Adipose tissue-derived Autologous Perianal fistula Complex
Sang-ji Choi, and Seung-Bum Ryoo contributed equally to this study. This research was sponsored by the Grant of Clinical Research Institute, Seoul National University Hospital (Grant No: 0620102730), from Anterogen Co., Ltd.
Compliance with ethical standards
Conflict of interest
The authors declare that they have no conflict of interest.
The protocol was approved by the institutional review boards at each of the four study sites for compliance with ethical standards and by the Ministry of Food and Drug Safety (IND number: 10844).
Written informed consent was obtained from all patients.
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