Benefits and risks of haematopoietic stem cell transplantation for systemic sclerosis: A systematic review and meta-analysis

Mod Rheumatol. 2023 Mar 2;33(2):330-337. doi: 10.1093/mr/roac026.


Objectives: We aimed to evaluate the efficacy and safety of haematopoietic stem cell transplantation (HSCT) in patients with systemic sclerosis.

Methods: A systematic literature review and meta-analysis were carried out. We compared survival outcomes using the Kaplan-Meier method with patient-level data between HSCT and intravenous pulse cyclophosphamide. Additionally, the incidence rate of treatment-related deaths with HSCT was pooled using a random-effect model.

Results: Of the 2091 articles screened, 22 were included: 3 randomized controlled trials and 19 observational studies. HSCT studies showed significant improvement in the skin thickness score and lung function. Despite treatment-related deaths being higher in HSCT than in intravenous pulse cyclophosphamide, the Kaplan-Meier analysis showed a high survival rate of 2 years post-transplant (log-rank, P = 0.004). The pooled frequency of transplant-related death from 700 systemic sclerosis patients was 6.30% (95% confidence interval 4.21-8.38). However, the estimated frequency of treatment-related deaths has been reducing over the last decade.

Conclusions: HSCT is an effective treatment for systemic sclerosis, but the optimal indications must be carefully determined by balancing the risks.

Keywords: Systemic sclerosis; long-term outcomes; meta-analysis; stem cell transplantation.

Publication types

  • Meta-Analysis
  • Systematic Review

MeSH terms

  • Cyclophosphamide
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Humans
  • Risk Assessment
  • Scleroderma, Systemic* / surgery
  • Transplantation, Autologous


  • Cyclophosphamide