[Autologous stem cell transplantation in a patient with diffuse systemic sclerosis]

Area of Science:

• Immunology
• Hematology
• Rheumatology

Background:

• Systemic Sclerosis (SSc) is a fibrotic disease with limited treatment options, particularly for diffuse, rapidly progressing forms.
• Diffuse SSc often involves critical organs like lungs and kidneys, leading to high morbidity and mortality.
• Standard therapies are often ineffective against the severe fibrotic manifestations of diffuse SSc.

Observation:

• A young woman with rapidly progressing diffuse SSc underwent high-dose chemotherapy with fludarabine, cyclophosphamide, and anti-thymoglobulins, followed by autologous stem cell transplantation.
• The conditioning regimen utilized mobilized peripheral blood progenitor cells to counteract myeloablation.
• This case represents a recent application of a therapeutic approach typically used for hematological diseases.

Findings:

• Two years post-transplantation, the patient exhibited significant clinical and instrumental improvements, indicating effective disease control.
• A decrease in fibrosis extent was instrumentally detected.
• The only residual sign of disease activity was a slow progression of skin involvement.

Implications:

• Autologous stem cell transplantation may be a viable therapeutic option for refractory, rapidly progressive systemic sclerosis.
• This treatment strategy offers potential for halting or reversing fibrotic organ damage in SSc.
• Further research with larger cohorts is warranted to confirm the long-term efficacy and safety of stem cell transplantation in SSc.