Long-term follow-up after autologous adipose-derived stromal vascular fraction injection into fingers in systemic sclerosis patients
Long-term follow-up after autologous adipose-derived stromal vascular fraction injection into fingers in systemic sclerosis patients
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DOI：
10.1016/j.retram.2016.10.006
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发表时间：
2017-01-01
影响因子：
4.1
通讯作者：
Granel, B.
中科院分区：
文献类型：
--
作者：
Daumas, A.;Magalon, J.;Granel, B.
导论. - 系统性硬化症（SSc）患者的手部受累使其在工作和日常活动中受到严重障碍。自体脂肪来源的基质血管成分是一种容易获得的具有再生作用的细胞来源。我们之前进行了一项I期开放标签临床试验（NTC 01813279），评估皮下注射自体脂肪源性基质血管组分的安全性。报告了6个月和12个月的数据。由于患者在我们的医疗中心进行了随访，我们报告了他们在试验结束后的长期结局。- 12名女性，平均年龄54.5 +/- 10.3岁，最初参加了临床试验，在治疗后22至30个月的预定医疗护理期间进行了评估。- 与术前评估相比，多个患者报告的结局显示持续改善：Cochin手功能量表为62.5%，硬皮病健康评估问卷为51.1%，手部疼痛为33.1%，雷诺状况评分为88.3%。观察到手指溃疡数量减少。手部的活动性、力量和纤维化也有所改善。8例既往输注伊洛前列素的患者均不需要重新输注。- 尽管开放标签研究存在局限性，但数据支持脂肪源性基质血管组分注射的长期安全性。两项关于这种治疗药物的随机双盲、安慰剂对照试验正在美国（NCT 02396238）和法国（NCT 02558543）进行，将有助于确定这种创新疗法在SSc患者中的地位。(C)2016 Elsevier Masson SAS。All rights reserved.
Introduction. - Hand involvement confers a substantial handicap in work and daily activities in patients with Systemic sclerosis (SSc). Autologous adipose-derived stromal vascular fraction is as an easily accessible source of cells with regenerative effects. We previously performed a phase I open-label clinical trial (NTC01813279) assessing the safety of subcutaneous injection of autologous adipose-derived stromal vascular fraction. Six and 12-month data have been reported. As patients were followed in our medical centre, we report their longer-term outcome beyond the end of the trial.Patients and method. - Twelve females, mean age 54.5 +/- 10.3 years, initially enrolled in the clinical trial were assessed during a scheduled medical care, which took place between 22 and 30 months after treatment.Results. - Multiple patient-reported outcomes showed sustained improvement, in comparison with the assessment performed just before surgery: 62.5% in the Cochin Hand Function Scale, 51.1% in the Scleroderma Health Assessment Questionnaire, 33.1% in hand pain, and 88.3% in the Raynaud Condition Score. A decrease in the number of digital ulcers number was noted. Mobility, strength and fibrosis of the hand also showed improvement. None of the 8 patients who had previously received iloprost infusion required new infusion.Conclusion. - Despite the limits of an open label study, the data are in favour of the long-term safety of the adipose-derived stromal vascular fraction injection. Two randomized double blind, placebo-controlled trials of this therapeutic agent are ongoing in the USA (NCT02396238) and in France (NCT02558543) and will help determine the place of this innovative therapy for SSc patients. (C) 2016 Elsevier Masson SAS. All rights reserved.