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

Br J Dermatol. 2018 Mar;178(3):650-658. doi: 10.1111/bjd.15993. Epub 2018 Feb 1.

Abstract

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.

Publication types

  • Systematic Review

MeSH terms

  • Adult
  • Blood Gas Analysis
  • Carbon Dioxide / blood
  • Cohort Studies
  • Cyclophosphamide / therapeutic use
  • Female
  • Hematopoietic Stem Cell Transplantation / adverse effects
  • Hematopoietic Stem Cell Transplantation / methods*
  • Hematopoietic Stem Cell Transplantation / mortality
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Lung Diseases / mortality
  • Lung Diseases / physiopathology
  • Male
  • Middle Aged
  • Patient Safety
  • Randomized Controlled Trials as Topic
  • Scleroderma, Systemic / mortality
  • Scleroderma, Systemic / physiopathology
  • Scleroderma, Systemic / therapy*
  • Transplantation, Autologous
  • Treatment Outcome
  • Vital Capacity / physiology

Substances

  • Immunosuppressive Agents
  • Carbon Dioxide
  • Cyclophosphamide