Jeon Faith Hyun Kyung, Griffin Michelle, Denton Christopher Paul, Butler Peter Edward Michael
Division of Surgery and Interventional Sciences, University College London, London, UK.
Charles Wolfson Centre for Reconstructive Surgery, Royal Free Hospital, London, UK.
Int J Surg Protoc. 2020 Jul 18;23:6-10. doi: 10.1016/j.isjp.2020.07.002. eCollection 2020.
Oro-facial fibrosis is a common and disabling manifestation of systemic sclerosis (SSc), causing a plethora of functional, aesthetic and social compromise, yet is without effective treatment. Autologous lipotransfer is an established minimally invasive surgical procedure that is postulated to exert anti-fibrotic effects by adipose-derived stem cells, and presents a novel method in the treatment of fibrotic conditions. This study aims to assess the safety and efficacy of autologous lipotransfer for facial involvement in SSc.
This is the first randomised controlled study with an open label design to assess autologous lipotransfer for oro-facial involvement in systemic sclerosis. The goals of this study are to assess the feasibility of using a range of quantitative and qualitative outcome measures to effectively measure disease severity and treatment outcome, and to assess patient acceptability for future multi-centre trials. A total of 50 participants will be randomised to a treatment or control group. The treatment group will receive autologous fat transfer to the peri-oral region by a single surgeon. Dermal fibroblasts and adipose-derived stem cells will be isolated from tissue samples. All outcome measures will be taken at baseline, then at 6 weeks, 3 months and 6 months from the time of intervention in the treatment arm, or from baseline in the control arm.
The study has ethical approval (REC reference 19/LO/0718). Results will be available to patients, patient user groups, clinicians and the public through presentations at national and international rheumatology conferences and published in peer reviewed journals.
Registered on ISRCTN registry (ISRCTN17793055).
口面部纤维化是系统性硬化症（SSc）常见且导致功能障碍的表现，会造成大量功能、美观和社交方面的损害，但尚无有效治疗方法。自体脂肪移植是一种既定的微创手术，据推测可通过脂肪来源干细胞发挥抗纤维化作用，为纤维化疾病的治疗提供了一种新方法。本研究旨在评估自体脂肪移植治疗SSc面部受累的安全性和有效性。
这是第一项采用开放标签设计的随机对照研究，旨在评估自体脂肪移植治疗系统性硬化症口面部受累的情况。本研究的目的是评估使用一系列定量和定性结局指标有效测量疾病严重程度和治疗效果的可行性，以及评估患者对未来多中心试验的可接受性。总共50名参与者将被随机分为治疗组或对照组。治疗组将由一名外科医生对口周区域进行自体脂肪移植。将从组织样本中分离真皮成纤维细胞和脂肪来源干细胞。所有结局指标将在基线时采集，然后在治疗组干预后6周、3个月和6个月时采集，对照组则从基线开始采集。
本研究已获得伦理批准（研究伦理委员会参考编号19/LO/0718）。研究结果将通过在国内和国际风湿病学会议上的报告，向患者、患者用户群体、临床医生和公众公开，并发表在同行评审期刊上。
已在国际标准随机对照试验编号注册库（ISRCTN17793055）注册。
