Long-term evaluation of the effectiveness and safety of autologous haematopoietic stem-cell transplantation for systemic sclerosis (NISSC1-LTE): a cohort study

Why clinicians should know about it

• Picked for Immunology and Allergy (paper of the day, 1 November 2026): Long-term effectiveness and safety of HSCT in systemic sclerosis

Abstract

BACKGROUND: Systemic sclerosis is a rare and heterogeneous chronic autoimmune disease. It has the highest mortality of the rheumatic diseases and few treatment options exist. Autologous haematopoietic stem-cell transplantation (HSCT) is an effective and established therapeutic option in selected patients with early severe systemic sclerosis. This study aimed to evaluate the long-term safety and effectiveness of autologous HSCT in patients with systemic sclerosis in real-life clinical practice. METHODS: This cohort study (NISSC1-LTE) followed up patients with systemic sclerosis included in the prospective European Society for Blood and Marrow Transplantation NISSC1 study who had undergone their first autologous HSCT for severe progressive systemic sclerosis. Follow-up data were collected from 13 participating centres in France, Germany, Netherlands, Italy, Spain, Switzerland, and Brazil. Participating centres completed a yearly case report form for each patient alive after 2 years and up to 10 years after autologous HSCT. The primary endpoint was progression-free survival, defined as survival from baseline without evidence of systemic sclerosis progression. Late complications after autologous HSCT, including malignancies, secondary autoimmune diseases, infections, and major adverse cardiovascular events were also analysed. People with lived experience of systemic sclerosis were involved in the design and conduct of this study. The NISSC1 study is registered with ClinicalTrials.gov (NCT02516124) and is complete. FINDINGS: All 80 patients with systemic sclerosis who had an autologous HSCT between Dec 4, 2012, and Jan 19, 2016, were included in this long-term extension study. 12 patients were lost to follow-up, including five patients with a follow-up less than 5 years. Median age at autologous HSCT was 43 years (IQR 33-51), 57 (71%) of 80 patients were female, 23 (29%) were male, and median follow-up was 9·2 years (IQR 8·9-9·6) after autologous HSCT. Progression-free survival was 81% (95% CI 71-88) at 2 years, 72% (61-81) at 5 years, and 60% (47-71) at 10 years. 18 patients died during the study; causes of death were autologous HSCT-related in five (6%) of 80 patients, disease progression in nine (11%) patients, post-autologous HSCT malignancy in two (3%) patients, and late serious infection in two (3%) patients. 34 (43%) of 80 patients maintained remission without immunosuppressive treatment over 10 years. INTERPRETATION: This study provides long-term follow-up data after autologous HSCT in patients with systemic sclerosis allowing robust assessment of response rate, late complications, and sustained treatment benefit. The extended timeframe might serve as an important comparator for all upcoming, cellular treatment options. FUNDING: None.

Abstract as published, via PubMed.