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Autologous stem cell transplant for severe, progressive juvenile systemic sclerosis
Shaikha Alqahtani1, Fabiana Cacace1,2, Andrea Bauchat1
1Pediatric Transplant and Cellular Therapy, Duke University, Durham, NC 27705, United States.
Stem Cells Translational Medicine
|March 23, 2026
Summary
Autologous stem cell transplantation shows promise for treating juvenile systemic sclerosis (jSSc), a rare autoimmune condition in children. This approach led to clinical improvement in a patient with difficult-to-treat jSSc.
Area of Science:
- Pediatric Rheumatology
- Autoimmune Diseases
- Stem Cell Transplantation
Background:
- Juvenile systemic sclerosis (jSSc) is a rare, chronic autoimmune condition affecting children and adolescents.
- jSSc presents with significant morbidity, including skin thickening (scleroderma), organ toxicity, and limited treatment options.
Purpose of the Study:
- To report the outcomes of autologous stem cell transplantation in a patient with refractory juvenile systemic sclerosis.
Main Methods:
- A case report detailing the treatment of a 17-year-old patient with refractory jSSc.
- Administration of autologous stem cell transplantation.
Main Results:
- The patient experienced notable clinical improvement following the transplantation.
Conclusions:
- Autologous stem cell transplantation may be a viable therapeutic option for refractory juvenile systemic sclerosis.
- Further research is warranted to evaluate the efficacy and safety of this treatment in a larger cohort.
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