BackgroundPerianal fistulas of Crohn's disease (CD) create a significant burden on patients' lives. However, the efficacy and safety of adipose‐derived mesenchymal stem cell treatment are contradicting, and real‐world evidence is lacking.AimsTo examine the usability of darvadstrocel therapy in managing perianal CD.MethodsWe enrolled patients with CD and perianal fistulas in this retrospective multicenter study. The primary outcome was perianal clinical remission (defined as all treated fistulas closed) at weeks 26 and 52. Secondary outcomes were clinical response rates (≥ 1 fistulas closed), perianal activity (PDAI), patient satisfaction, and adverse events. Data were recorded at baseline and weeks 12, 26 and 52. Prediction of primary outcomes was performed by logistic regression.ResultsOverall, among 223 patients (male/female ratio: 0.48), perianal clinical remission was achieved in 78.2% and 62.3% until weeks 26 and 52. Baseline PDAI score (OR 0.75), number of fistulas (OR 0.28) and the number of weeks after preparation for surgery (OR 0.98) were associated with treatment failure. The clinical response rates were 84.8% and 79.8% at weeks 26 and 52. Improvement of subjective perianal symptoms was achieved in 77.8% and 78.4% of patients, respectively. Adverse events occurred in 13.5% of patients; perianal abscesses and proctalgia were the most frequently reported.ConclusionEffectiveness data were higher than in clinical trials. The safety profile was reassuring, and patients' satisfaction was high. Appropriate patient selection, fistula preparation and expertise may help to achieve treatment success.

中文翻译：

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间充质干细胞治疗瘘管性克罗恩病的有效性和安全性评价：一项国际现实生活回顾性多中心队列研究


背景 克罗恩病 （CD） 的肛周瘘对患者的生活造成了沉重的负担。然而，脂肪来源的间充质干细胞治疗的有效性和安全性是相互矛盾的，并且缺乏真实世界的证据。目的检查 darvadstrocel 疗法在治疗肛周 CD 中的可用性。方法我们在这项回顾性多中心研究中招募了 CD 和肛周瘘患者。主要结局是第 26 周和第 52 周的肛周临床缓解 （定义为所有治疗过的瘘管闭合）。次要结局是临床缓解率 （≥ 1 个瘘管闭合）、肛周活动 （PDAI） 、患者满意度和不良事件。在基线和第 12 、 26 和 52 周记录数据。通过 logistic 回归预测主要结局。结果总体而言，在 223 例患者 （男女比例： 0.48） 中，直到第 26 周和第 52 周，肛周临床缓解率分别为 78.2% 和 62.3%。基线 PDAI 评分 （OR 0.75） 、瘘管数量 （OR 0.28） 和手术准备后周数 （OR 0.98） 与治疗失败相关。第 26 周和第 52 周的临床缓解率分别为 84.8% 和 79.8%。77.8% 和 78.4% 的患者主观肛周症状得到改善。不良事件发生于 13.5% 的患者;肛周脓肿和直肠痛是最常报告的。结论有效性数据高于临床试验。安全性令人放心，患者的满意度很高。适当的患者选择、瘘管准备和专业知识可能有助于实现治疗成功。