Department of Dermatology, Hôpital Saint-André, CHU de Bordeaux, Bordeaux, France.
Department of Rheumatology, Hôpital Pellegrin, CHU de Bordeaux, Bordeaux, France.
Br J Dermatol. 2018 Mar;178(3):650-658. doi: 10.1111/bjd.15993. Epub 2018 Feb 1.
We aimed to assess the efficacy of autologous haematopoietic stem cell transplantation (HSCT) for skin sclerosis (SSc) and lung function in SSc. We performed a systematic literature review in the PubMed and Scopus databases from the earliest records to March 2016. We assessed study quality using the Cochrane tool for randomized studies, the Newcastle-Ottawa Scale for controlled cohort studies and an 18-item quality-appraisal checklist for case series. The primary outcome was the improvement of skin thickening using the modified Rodnan Skin Score (mRSS). The secondary outcome was efficacy on lung function, using diffusing capacity of the lungs for carbon monoxide and forced vital capacity (FVC). The safety of the procedure was evaluated. The literature search identified 431 citations. There were 38 studies involving a total of 344 patients who fulfilled our inclusion criteria. No meta-analysis was performed due to a high heterogeneity. There was a significant improvement in mRSS in the majority of the reports (P < 0·05), and the results were sustained for up to 8 years after autologous HSCT. The randomized studies and the four cohort studies each showed a slight but statistically significant improvement in FVC at 1 or 2 years. The treatment-related mortality calculated by pooling patients of 35 studies (336 patients with a follow-up up to 146 months) was 8·3% after autologous HSCT and 1% in cyclophosphamide-treated groups. Despite heterogeneity among the studies, we determined that autologous HSCT significantly improved cutaneous fibrosis and slightly improved FVC. Safety of autologous HSCT is acceptable given the severity of the disease. This systematic review was registered on PROSPERO, number CRD42016027951.
我们旨在评估自体造血干细胞移植（HSCT）治疗皮肤硬化症（SSc）和 SSc 患者肺功能的疗效。我们在 PubMed 和 Scopus 数据库中进行了系统文献回顾，检索时间从最早记录至 2016 年 3 月。我们使用 Cochrane 随机研究工具、纽卡斯尔-渥太华量表评估队列研究和 18 项病例系列质量评估检查表来评估研究质量。主要结局是使用改良 Rodnan 皮肤评分（mRSS）评估皮肤增厚的改善情况。次要结局是使用一氧化碳弥散量和用力肺活量（FVC）评估肺功能的疗效。评估了该程序的安全性。文献检索共确定了 431 条引文。有 38 项研究共纳入了 344 名符合纳入标准的患者。由于高度异质性，未进行荟萃分析。大多数报告显示 mRSS 显著改善（P<0.05），且自自体 HSCT 后长达 8 年仍保持持续改善。随机研究和四项队列研究均显示 FVC 在 1 或 2 年内略有但有统计学意义的改善。通过汇总 35 项研究的患者数据（336 例患者的随访时间长达 146 个月）计算的治疗相关死亡率为自体 HSCT 后 8.3%，环磷酰胺治疗组为 1%。尽管研究间存在异质性，但我们确定自体 HSCT 显著改善了皮肤纤维化，且略微改善了 FVC。考虑到疾病的严重程度，自体 HSCT 的安全性是可以接受的。本系统评价已在 PROSPERO 上注册，编号为 CRD42016027951。
