Systemic sclerosis & Raynaud's
High-dose chemotherapy and transplantation of autologous CD34+ selected stem cells for progressive systemic sclerosis adjusted to cardiac and lung manifestation: An open-label, non-inferiority phase 2 trial
Authors' conclusion
This adapted regimen showed comparable 3y-OS in patients with advanced organ involvement who would have been excluded from prior trials.
Study context
- Population
- Patients with progressive systemic sclerosis (SSc) (disease duration≤6 years), including ASTIS-ineligible patients, University Hospital Tübingen, Germanytreated 09/2012–07/2022
- Participants
- 35 patients
- Exposure
- Disease-manifestation-adapted high-dose chemotherapy and autologous CD34+ selected stem cell transplantation vs EBMT registry data
- No inflammatory interstitial lung disease (iILD)/cardiac involvementn = 14
- iILD onlyn = 3
- Cardiac onlyn = 12
- Bothn = 6
- Outcome
- Overall survival (non-inferiority vs EBMT registry data)
- Follow-up
- 3 years
Methodological detailsprospective, open-label, monocentric, phase II, single-group, stratified by lung and cardiac involvement, Between 09/2012 and 07/2022
- Prospective
- Open-label
- Monocentric
- Phase II
- Single-group, stratified by lung and cardiac involvement
- Between 09/2012 and 07/2022
Main finding
As reported in the abstract
3-year OS
EBMT registry
80%
- 3-year overall survival (OS), all treated patients
- 77%
Observational data: associations, not effects.
Safety
| Outcome | Values |
|---|---|
| TRM within 100 days | Whole group 11% |
Original publication
High-dose chemotherapy and transplantation of autologous CD34+ selected stem cells for progressive systemic sclerosis adjusted to cardiac and lung manifestation: An open-label, non-inferiority phase 2 trial. Joint Bone Spine. Published 29 January 2026. doi:10.
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