Efficacy and safety of autologous haematopoietic stem cell transplantation in systemic sclerosis: a systematic review of the literature

摘要

Summary We aimed to assess the efficacy of autologous haematopoietic stem cell transplantation (HSCT ) for skin sclerosis (SS c) and lung function inSS c. 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 NewcastleOttawa Scale for controlled cohort studies and an 18item qualityappraisal 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 metaanalysis was performed due to a high heterogeneity. There was a significant improvement inmRSS in the majority of the reports (P 0?5), and the results were sustained for up to 8 years after autologousHSCT . The randomized studies and the four cohort studies each showed a slight but statistically significant improvement inFVC at 1 or 2 years. The treatmentrelated mortality calculated by pooling patients of 35 studies (336 patients with a followup up to 146 months) was 8?% after autologousHSCT and 1% in cyclophosphamidetreated groups. Despite heterogeneity among the studies, we determined that autologousHSCT significantly improved cutaneous fibrosis and slightly improvedFVC . Safety of autologousHSCT is acceptable given the severity of the disease. This systematic review was registered onPROSPERO , numberCRD 42016027951.