RESEARCHERS have found that many patients with systemic sclerosis have remained progression-free for up to 10 years after autologous haematopoietic stem-cell transplantation (HSCT), according to the longest cohort follow-up reported to date.
Systemic sclerosis is a rare autoimmune disease with the highest mortality of the rheumatic diseases, and few treatment options exist. Autologous HSCT has been recommended as a standard option since 2018, supported by Level 1 evidence from three randomised controlled trials. However, long-term efficacy and safety data for stem-cell transplantation in systemic sclerosis were scarce.
The NISSC1-LTE cohort study followed up with patients from the prospective European Society for Blood and Marrow Transplantation (EBMT) NISSC1 study who had undergone their first autologous HSCT for severe progressive disease.
Data were collected from 13 centres in seven countries, using yearly case report forms for patients alive from 2 to 10 years after transplantation. All 80 patients transplanted between 4 December 2012 and 19 January 2016 were included; median age was 43 years (interquartile range: 33–51), 57 (71%) were female, and 12 were lost to follow-up.
Median follow-up was 9.2 years (interquartile range: 8.9–9.6). Progression-free survival was 81% at 2 years, 72% at 5 years, and 60% at 10 years, with 10-year overall survival of 71% and non-relapse mortality of 13%. Notably, 34 (43%) patients maintained remission without immunosuppressive treatment over 10 years. Of 18 deaths, five (6%) were transplantation-related, nine (11%) were due to progression, two (3%) followed malignancy, and two (3%) followed late serious infection. Additionally, 10-year cumulative incidences were 8% for malignancies, 14% for secondary autoimmune diseases, 20% for late serious infections, and 5% for major adverse cardiovascular events.
These data provide the longest reported follow-up of patients with systemic sclerosis after autologous HSCT, although early treatment-related mortality remains a matter of debate. The long-lasting outcomes may help foster earlier discussions about transplantation in experienced centres and may serve as a comparator for upcoming cellular treatments. Further randomised controlled studies are needed to identify subgroups most likely to benefit.
Reference
Henes J et al. Long-term evaluation of the effectiveness and safety of autologous haematopoietic stem-cell transplantation for systemic sclerosis (NISSC1-LTE): a cohort study. The Lancet Rheumatology. 2026;DOI:10.1016/S2665-9913(26)00227-4.
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