Stem cell transplant in systemic sclerosis: An Indian experience.

Area of Science:

• Immunology
• Rheumatology
• Hematology

Background:

• Systemic Sclerosis (SSc) is a chronic autoimmune disease with limited treatment options.
• Autologous Hematopoietic Stem Cell Transplant (HSCT) is an emerging therapy for severe SSc.
• Long-term outcomes of HSCT in SSc require further evaluation.

Purpose of the Study:

• To prospectively assess the long-term outcomes of Systemic Sclerosis patients treated with autologous HSCT.
• To evaluate the impact of HSCT on disease manifestations and quality of life in SSc.

Main Methods:

• Prospective observational study of four SSc patients undergoing HSCT.
• Patients received G-CSF for stem cell mobilization, followed by conditioning (fludarabine, cyclophosphamide, ATG) and autologous stem cell re-infusion.
• Follow-up duration averaged 7 years.

Main Results:

• Significant, sustained improvement in skin scores, vasculopathy, and gastrointestinal symptoms observed.
• No deterioration in mild interstitial lung disease (ILD) noted.
• Marked improvement in quality of life indices with no transplant-related complications.

Conclusions:

• Autologous HSCT demonstrates significant potential for managing both cutaneous and internal organ manifestations of SSc.
• HSCT offers a promising therapeutic strategy in the absence of effective pharmacotherapy for SSc.
• The study highlights the long-term efficacy and safety of HSCT for severe SSc.