What is the role of HSCT in the treatment of scleroderma?

Updated: Jun 24, 2020
  • Author: Sergio A Jimenez, MD, MACR, FACP, FRCP(UK Hon); Chief Editor: Herbert S Diamond, MD  more...
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Hematopoietic stem cell transplantation (HSCT) has been shown to be effective. However, it is associated with a high rate of procedure-related complications. [159, 160, 161, 162]  

In the prospective Autologous Stem Cell Transplantation International Scleroderma (ASTIS) trial, a phase 3 comparison of autologous  HSCT with 12 successive monthly intravenous pulses of cyclophosphamide in 156 patients with early diffuse cutaneous systemic sclerosis, HCST was associated with higher treatment-related mortality than in the first year after treatment. However, HCST conferred a significant long-term survival benefit. [163]

In ASTIS during the first year, 13 events, including 8 treatment-related deaths, occurred in the HSCT group and 8 events with no treatment-related deaths occurred in the control group (16.5% vs. 10.4%, respectively). At 4 years, however, 15 events had occurred cumulatively in the HSCT group compared with 20 events in the control group (19% vs. 26%, respectively). [163]

In the Scleroderma: Cyclophosphamide Or Transplantation (SCOT) trial, a randomized, open-label, phase 2 trial in 75 patients with  severe scleroderma, autologous HSCT improved event-free and overall survival, at a cost of increased expected toxicity, compared with 12 monthly infusions of cyclophosphamide. Rates of treatment-related death and post-transplantation use of disease-modifying antirheumatic drugs (DMARDs) were lower than those in previous reports of nonmyeloablative transplantation. [164]

In SCOT participants who received a transplant or completed 9 or more doses of cyclophosphamide, event-free survival was 79% in the transplantation group and 50% in the cyclophosphamide group (P = 0.02) at 54 months and 74% vs 47%, respectively, at 72 months (P = 0.03), By 54 months, 9% of the participants in the transplantation group had initiated DMARDs, as compared with 44% of those in the cyclophosphamide group (P = 0.001). Treatment-related mortality in the transplantation group was 3% at 54 months and 6% at 72 months; no treatment-related deaths occurred in the cyclophosphamide group. [164]

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