Systemic sclerosis & Raynaud's
Long-term evaluation of the effectiveness and safety of autologous haematopoietic stem-cell transplantation for systemic sclerosis (NISSC1-LTE): a cohort study
Authors' conclusion
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.
Study context
- Population
- Patients with systemic sclerosis included in the prospective NISSC1 study who had undergone their first autologous haematopoietic stem-cell transplantation (HSCT) for severe progressive systemic sclerosis, centres in France, Germany, Netherlands, Italy, Spain, Switzerland, and Brazilall patients who had an autologous HSCT between Dec 4, 2012, and Jan 19, 2016, 12 lost to follow-up
- Participants
- 80 patients
- Outcome
- Progression-free survival, defined as survival from baseline without evidence of systemic sclerosis progression
- Follow-up
- 2, 5 and 10 years
Methodological detailslong-term extension of the prospective European Society for Blood and Marrow Transplantation NISSC1 study, 13 participating centres
- Long-term extension of the prospective European Society for Blood and Marrow Transplantation NISSC1 study
- 13 participating centres
Main finding
As reported in the abstract
Progression-free survival at 2 years
81%
95% CI 71–88
Progression-free survival at 5 years
72%
61–81
Progression-free survival at 10 years
60%
47–71
Observational data: associations, not effects.
Further findings
Remission maintained without immunosuppressive treatment · over 10 years
- Patients maintaining remission without immunosuppressive treatment
- 34 (43%) of 80
Baseline characteristics and follow-up
- Median age at autologous HSCT
- 43 years (IQR 33-51)
- Female
- 57 (71%) of 80
- Male
- 23 (29%)
- Median follow-up after autologous HSCT
- 9.2 years (IQR 8.9-9.6)
- Lost to follow-up
- 12
- Lost to follow-up with a follow-up less than 5 years
- five
Safety
Deaths and causes of death
- Patients who died during the study
- 18
- Autologous HSCT-related deaths
- five (6%) of 80
- Deaths from disease progression
- nine (11%)
- Deaths from post-autologous HSCT malignancy
- two (3%)
- Deaths from late serious infection
- two (3%)
Original publication
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