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Updated: Jun 19, 2026

Development of Stem Cell-derived Antigen-specific Regulatory T Cells Against Autoimmunity

Development of Stem Cell-derived Antigen-specific Regulatory T Cells Against Autoimmunity

Published on: November 8, 2016

Autologous stem cell transplantation (ASCT) for Refractory juvenile-onset systemic sclerosis (JSSc).

Kathryn S Torok¹, Paulina Horvei², Jonathan Li³

¹Division of Paediatric Rheumatology, University of Pittsburgh Medical Center (UPMC) Children's Hospital of Pittsburgh, Pittsburgh, PA, USA; UPMC & University of Pittsburgh Scleroderma Center, Pittsburgh, USA.

Annals of the Rheumatic Diseases

|June 17, 2026

Summary

Autologous stem cell transplantation (ASCT) offers a safe and effective treatment for juvenile-onset systemic sclerosis (JSSc), significantly improving multisystem outcomes in refractory cases. This approach shows promise for patients with progressive, treatment-resistant disease.

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Development of Stem Cell-derived Antigen-specific Regulatory T Cells Against Autoimmunity

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Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome

Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome

Published on: October 3, 2018

Area of Science:

Rheumatology
Immunology
Hematology

Background:

Juvenile-onset systemic sclerosis (JSSc) is a rare, severe autoimmune disease causing vasculopathy and fibrosis.
Current treatments for JSSc have limitations, and data on advanced therapies are scarce.
Autologous stem cell transplantation (ASCT) is effective in adult systemic sclerosis but understudied in pediatric populations.

Purpose of the Study:

To evaluate the safety and efficacy of autologous stem cell transplantation (ASCT) in patients with treatment-refractory juvenile-onset systemic sclerosis (JSSc).
To assess the impact of ASCT on multisystemic manifestations and long-term outcomes in pediatric patients with severe JSSc.

Main Methods:

Eight consecutive patients with moderate-to-severe, refractory JSSc received CD34+-selected ASCT.
Conditioning regimens included alemtuzumab, rituximab, antithymocyte globulin, total body irradiation, and thiotepa.
Clinical outcomes were monitored for up to 48 months post-transplantation.

Main Results:

Successful engraftment was achieved in all patients with no treatment-related mortality or organ toxicity.
Event-free survival was 100% at a median follow-up of 30 months.
Significant improvements were observed in skin scores, functional capacity, pulmonary hypertension, gastrointestinal symptoms, and quality of life.

Conclusions:

ASCT is a safe and well-tolerated procedure for refractory JSSc, leading to durable, multisystemic improvements.
These findings suggest that ASCT should be considered earlier for pediatric patients with progressive, treatment-resistant JSSc.