- Design
- Long-term extension of a prospective multicentre registry cohort
- Population
- 80 adults after autologous HSCT for severe progressive systemic sclerosis
- Primary outcome
- Progression-free survival up to 10 years
- Effect
- 81% at 2 years, 72% at 5, 60% at 10; transplant-related death 6%
This study followed all 80 patients from a prospective European registry who had autologous haematopoietic stem-cell transplantation for severe progressive systemic sclerosis at 13 centres between 2012 and 2016. Median age was 43 and median follow-up 9.2 years.
Progression-free survival was 81% at 2 years, 72% at 5 and 60% at 10. Of 80 patients, 34 (43%) stayed in remission without immunosuppression throughout. Eighteen died: five (6%) from transplant-related causes, nine from disease progression, two from later cancer and two from late serious infection.
These are real-world results in carefully selected patients, and they set a benchmark that CAR-T and other cellular therapies will now be measured against. The upfront mortality remains the central trade-off, so patient selection and referral to experienced centres are what matter for clinicians.
- Consider early referral to a transplant-experienced centre for severe, rapidly progressive diffuse systemic sclerosis
- Discuss transplant-related mortality of about 6% alongside long-term benefit
- Screen for cardiac involvement before referral, as it drives transplant risk
- Plan long-term surveillance for late infection and malignancy after transplant
Why it matters
It gives patients and clinicians a realistic ten-year picture of a high-risk, high-reward treatment.
Don't overread it
A single-arm cohort without a comparison group; it does not show transplant is better than modern drug therapy.
The statistics, in plain English
60% progression-free at 10 years means six in ten patients had not worsened. Twelve patients were lost to follow-up, which may make results look better or worse than they are.
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