Autologous stem cell transplantation (ASCT) for Refractory juvenile-onset systemic sclerosis (JSSc).
Kathryn S Torok, Paulina Horvei, Jonathan Li et al.
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In 30 seconds
This single-centre phase II clinical trial reported outcomes for 8 patients with moderate-to-severe juvenile-onset systemic sclerosis refractory to at least 2 disease-modifying antirheumatic drugs who underwent CD34+-selected autologous stem cell transplantation. All engrafted, with no treatment-related deaths or organ toxicities. At a median follow-up of 30 months, event-free survival—defined as no death, cardiopulmonary failure, or renal crisis—was 100%. The cohort also showed multisystem improvements, including a median 93% reduction in modified Rodnan skin score; most patients discontinued immunosuppression for at least 18 months.
Key findings
- All 8 patients engrafted successfully; median neutrophil and platelet engraftment occurred at 13 and 22 days, respectively.
- Event-free survival was 100% at a median follow-up of 30 months, with no treatment-related deaths or organ toxicities.
- Modified Rodnan skin scores improved by a median of 93%, and 6-minute walk distance improved by 13%.
- All patients stopped immunosuppression for at least 18 months after transplantation; 1 later restarted it for a disease flare.
Why it matters
Juvenile-onset systemic sclerosis can cause substantial multiorgan morbidity, while evidence for transplantation in this population has been limited. These results describe durable, multisystem improvement alongside successful engraftment in a small group with treatment-refractory disease. They add clinical data relevant to weighing this intensive approach for progressive disease, while the uncontrolled, single-centre experience cannot establish comparative benefit.
What to keep in mind
The study included only 8 patients at a single centre and had no comparator group, limiting the certainty and generalisability of the findings.