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Autologous hematopoietic stem-cell transplantation in diffuse cutaneous systemic sclerosis: a single-center
Marko Barešić1, Ljiljana Smiljanić Tomičević1, Igor Aurer2
1Division of Clinical Immunology and Rheumatology, Department of Internal Medicine, School of Medicine, University Hospital Center Zagreb, University of Zagreb.
Reumatismo
|May 29, 2025
Summary
Autologous hematopoietic stem cell transplantation (AHSCT) shows promise for severe systemic sclerosis (SSc). While one patient improved, another experienced disease progression, highlighting the need for careful patient selection and timing for this intensive treatment.
Area of Science:
- Immunology
- Rheumatology
- Hematology
Background:
- Systemic sclerosis (SSc) is a complex autoimmune disease affecting multiple organs.
- Disease progression in SSc can lead to irreversible damage, necessitating aggressive treatment.
- Autologous hematopoietic stem cell transplantation (AHSCT) is an emerging therapy for severe SSc phenotypes.
Purpose of the Study:
- To evaluate the efficacy and outcomes of AHSCT in two patients with rapidly progressive diffuse cutaneous SSc (dcSSc).
- To contribute to the understanding of AHSCT's role in managing refractory SSc.
- To highlight the importance of early intervention in progressive SSc.
Main Methods:
- Case report of two patients with rapidly progressive dcSSc.
- Treatment with AHSCT after failure of conventional immunosuppressive therapies.
- Long-term follow-up assessing clinical response, cutaneous and internal organ involvement.
Main Results:
- Both patients achieved significant improvement in skin manifestations post-AHSCT.
- One patient experienced progression of internal organ disease, leading to a fatal outcome from sepsis.
- The second patient remained stable long-term without further immunosuppressive therapy.
Conclusions:
- AHSCT can be an effective treatment for carefully selected patients with progressive dcSSc.
- Optimal timing for AHSCT in SSc remains an important consideration.
- These cases represent the first successful AHSCT experiences in Croatia and the Balkan region for SSc.
