Granel Brigitte, Daumas Aurélie, Jouve Elisabeth, Harlé Jean-Robert, Nguyen Pierre-Sébastien, Chabannon Christian, Colavolpe Nathalie, Reynier Jean-Charles, Truillet Romain, Mallet Stéphanie, Baiada Antoine, Casanova Dominique, Giraudo Laurent, Arnaud Laurent, Veran Julie, Sabatier Florence, Magalon Guy
Internal Medicine Department, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France Vascular Research Center Marseille, INSERM UMRS-1076, Aix-Marseilles University, Marseilles, France.
Internal Medicine Department, Assistance Publique Hôpitaux de Marseilles (AP-HM), Aix-Marseilles University, Marseilles, France.
Ann Rheum Dis. 2015 Dec;74(12):2175-82. doi: 10.1136/annrheumdis-2014-205681. Epub 2014 Aug 11.
In patients with systemic sclerosis (scleroderma, SSc), impaired hand function greatly contributes to disability and reduced quality of life, and is insufficiently relieved by currently available therapies. Adipose tissue-derived stromal vascular fraction (SVF) is increasingly recognised as an easily accessible source of regenerative cells with therapeutic potential in ischaemic or autoimmune diseases. We aimed to measure for the first time the safety, tolerability and potential efficacy of autologous SVF cells local injections in patients with SSc with hand disability.
We did an open-label, single arm, at one study site with 6-month follow-up among 12 female SSc patients with Cochin Hand Function Scale score >20/90. Autologous SVF was obtained from lipoaspirates, using an automated processing system, and subsequently injected into the subcutaneous tissue of each finger in contact with neurovascular pedicles. Primary outcome was the number and the severity of adverse events related to SVF-based therapy. Secondary endpoints were changes in hand disability and fibrosis, vascular manifestations, pain and quality of life from baseline to 2 and 6 months after cell therapy.
All enrolled patients had surgery, and there were no dropouts or patients lost to follow-up. No severe adverse events occurred during the procedure and follow-up. Four minor adverse events were reported and resolved spontaneously. A significant improvement in hand disability and pain, Raynaud's phenomenon, finger oedema and quality of life was observed.
This study outlines the safety of the autologous SVF cells injection in the hands of patients with SSc. Preliminary assessments at 6 months suggest potential efficacy needing confirmation in a randomised placebo-controlled trial on a larger population.
GFRS (Groupe Francophone de Recherche sur la Sclérodermie).
NCT01813279.
在系统性硬化症（硬皮病，SSc）患者中，手部功能受损极大地导致了残疾并降低了生活质量，而目前可用的治疗方法对此缓解不足。脂肪组织来源的基质血管成分（SVF）越来越被认为是一种易于获取的再生细胞来源，在缺血性或自身免疫性疾病中具有治疗潜力。我们旨在首次测量自体SVF细胞局部注射对患有手部残疾的SSc患者的安全性、耐受性和潜在疗效。
我们在一个研究地点进行了一项开放标签、单臂研究，对12名科钦手部功能量表评分>20/90的女性SSc患者进行了6个月的随访。使用自动处理系统从抽脂物中获取自体SVF，随后将其注射到每个手指与神经血管蒂接触的皮下组织中。主要结局是与基于SVF的治疗相关的不良事件的数量和严重程度。次要终点是从基线到细胞治疗后2个月和6个月手部残疾和纤维化、血管表现、疼痛和生活质量的变化。
所有入组患者均接受了手术，没有退出者或失访患者。在手术和随访期间未发生严重不良事件。报告了4例轻微不良事件，均自发缓解。观察到手部残疾、疼痛、雷诺现象、手指水肿和生活质量有显著改善。
本研究概述了自体SVF细胞注射到SSc患者手部的安全性。6个月时的初步评估表明其潜在疗效需要在更大规模的随机安慰剂对照试验中得到证实。
GFRS（法语硬皮病研究小组）。
NCT01813279。
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