Department of Surgery and New Therapy Laboratory, Health Research Institute Fundación Jiménez Díaz (FIIS-FJD), Universidad Autónoma de Madrid (UAM), Madrid, Spain.
Department of Colorectal Surgery, "Lozano Blesa" University Hospital, Aragon Health Sciences Institute, Zaragoza, Spain.
Stem Cells Transl Med. 2020 Mar;9(3):295-301. doi: 10.1002/sctm.19-0271. Epub 2019 Dec 30.
The aim of this clinical trial (ID Number NCT01803347) was to determine the safety and efficacy of autologous adipose-derived stem cells (ASCs) for treatment of cryptoglandular fistula. This research was conducted following an analysis of the mistakes of a same previous phase III clinical trial. We designed a multicenter, randomized, single-blind clinical trial, recruiting 57 patients. Forty-four patients were categorized as belonging to the intent-to-treat group. Of these, 23 patients received 100 million ASCs plus intralesional fibrin glue (group A) and 21 received intralesional fibrin glue (group B), both after a deeper curettage of tracks and closure of internal openings. Fistula healing was defined as complete re-epithelialization of external openings. Those patients in whom the fistula had not healed after 16 weeks were eligible for retreatment. Patients were evaluated at 1, 4, 16, 36, and 52 weeks and 2 years after treatment. Results were assessed by an evaluator blinded to the type of treatment. After 16 weeks, the healing rate was 30.4% in group A and 42.8% in group B, rising to 55.0% and 63.1%, respectively, at 52 weeks. At the end of the study (2 years after treatment), the healing rate remained at 50.0% in group A and had reduced to 26.3% in group B. The safety of the cellular treatment was confirmed and no impact on fecal continence was detected. The main conclusion was that autologous ASCs for the treatment of cryptoglandular perianal fistula is safe and can favor long-term and sustained fistula healing.
本临床试验（注册号 NCT01803347）旨在确定自体脂肪来源干细胞（ASCs）治疗隐窝性肛痿的安全性和有效性。本研究是在分析同一项前期 III 期临床试验错误的基础上进行的。我们设计了一项多中心、随机、单盲临床试验，招募了 57 名患者。44 名患者被归类为意向治疗组。其中，23 名患者接受了 1 亿个 ASC 加局部纤维蛋白胶（A 组），21 名患者接受了局部纤维蛋白胶（B 组），两者均在对轨道进行更深层次的刮除和内口闭合后进行。瘘管愈合定义为外部开口的完全再上皮化。在 16 周后瘘管未愈合的患者有资格进行再治疗。患者在治疗后 1、4、16、36 和 52 周以及 2 年时进行评估。结果由一名对治疗类型不知情的评估者进行评估。在 16 周时，A 组的愈合率为 30.4%，B 组为 42.8%，在 52 周时分别上升至 55.0%和 63.1%。在研究结束时（治疗后 2 年），A 组的愈合率保持在 50.0%，B 组降至 26.3%。细胞治疗的安全性得到了证实，并且没有发现对粪便节制的影响。主要结论是，自体 ASC 治疗隐窝性肛周瘘是安全的，可以促进长期和持续的瘘管愈合。
