An international study reports that 60 percent of patients with early severe systemic sclerosis remained progression-free ten years after undergoing autologous hematopoietic cell transplantation, offering vital long-term data on durability and ongoing clinical risks.
Systemic sclerosis remains a challenging condition characterized by progressive fibrosis affecting the skin and internal organs. While autologous hematopoietic cell transplantation serves as an established treatment option for carefully selected patients with early diffuse severe disease, concrete evidence tracking outcomes approaching a decade has remained limited.
Patient Survival Reaches 60 Percent After 10 Years
The NISSC1-LTE study followed 80 patients for a median of 9.2 years after their procedures, which were performed between 2012 and 2016 across 13 participating centers in France, Germany, the Netherlands, Italy, Spain, Switzerland, and Brazil. Researchers found that estimated progression-free survival reached 60 percent at the 10-year mark, while overall survival stood at 71 percent.
Clinical measures of disease severity demonstrated notable improvements that persisted over time. The median modified Rodnan skin score, which gauges skin involvement, dropped from 24 points at baseline down to 6 points at five years and 5 points at 10 years. Meanwhile, lung function indicators showed general stability. Median forced vital capacity was 71 percent of the predicted value at 10 years compared to 72 percent before transplantation, and the diffusing capacity of the lungs for carbon monoxide shifted from 59 percent to 61 percent of predicted values.
The project reflects years of collaboration among investigators, clinical teams including hematologists, rheumatologists, and data specialists across expert centers from Europe to Brazil within the EBMT Autoimmune Diseases Working Party network. Study lead authors Joerg Henes, based at University Hospital Tübingen in Germany, and Dominique Farge, based at St-Louis Hospital in Paris, helped coordinate the research, which was previously presented at the 2026 Annual Meeting of EBMT and published in The Lancet Rheumatology.
Procedure Risks Include Early Fatalities and Infections
Despite the sustained benefits observed in surviving participants, the study showed that the risks tied to the procedure remain significant. During the follow-up period, 18 patients died. Five of those deaths were classified as early fatalities directly attributed to the transplantation procedure, representing 6 percent of the total cohort. Nine deaths stemmed from disease progression, two resulted from post-procedure malignancies, and two were caused by severe late infections. Estimated non-relapse mortality at 10 years stood at 13 percent.
At their last recorded follow-up, 34 of the 80 participants—equal to 43 percent of the cohort—were alive and had not required additional immunosuppressive therapy.
These long-term findings help clinicians understand the durability of systemic sclerosis disease control following autologous HCT, while highlighting the risks that require continued monitoring.
Joerg Henes and Dominique Farge, study lead authors
Investigating New Strategies to Enhance Immune Resets
Conditions such as multiple sclerosis, lupus, and rheumatoid arthritis share similar treatment challenges that can require continuous care. Researchers at the Miller School of Medicine, led by Dr. Robert Levy, propose adding post-transplant cyclophosphamide during immune recovery to target autoreactive cells that may survive autologous stem cell transplantation.