Autologous hematopoietic stem-cell transplantation in diffuse cutaneous systemic sclerosis: a single-center experience
Systemic sclerosis (SSc) is a chronic autoimmune disorder characterized by multisystem involvement. Patients can be stratified into an indolent or rapidly progressive disease course. A progressive course warrants early and more aggressive treatment to prevent irreversible organ damage. Therapeutic...
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| Format: | Article |
| Language: | English |
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PAGEPress Publications
2025-05-01
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| Series: | Reumatismo |
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| Online Access: | https://www.reumatismo.org/reuma/article/view/1822 |
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| author | Marko Barešić Ljiljana Smiljanić Tomičević Igor Aurer Mislav Cerovec Branimir Anić |
| author_facet | Marko Barešić Ljiljana Smiljanić Tomičević Igor Aurer Mislav Cerovec Branimir Anić |
| author_sort | Marko Barešić |
| collection | DOAJ |
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Systemic sclerosis (SSc) is a chronic autoimmune disorder characterized by multisystem involvement. Patients can be stratified into an indolent or rapidly progressive disease course. A progressive course warrants early and more aggressive treatment to prevent irreversible organ damage. Therapeutic strategies should be tailored to the presenting symptoms and organ involvement. Autologous hematopoietic stem cell transplantation (AHSCT) has proven to be an effective treatment modality for specific phenotypes of SSc, especially progressive diffuse cutaneous SSc. However, the optimal timing for the transplantation remains unknown. We present two cases of rapidly progressive diffuse cutaneous systemic sclerosis (dcSSc) treated with AHSCT following inadequate response to conventional immunosuppressive therapy. While both patients experienced significant cutaneous improvement post-AHSCT, internal organ involvement progressed in one case, ultimately resulting in a fatal outcome due to severe sepsis, whereas the second patient remained clinically stable and without immunosuppressive therapy during long-term follow-up. This report contributes to the growing body of evidence supporting AHSCT as a therapeutic option in carefully selected cases of progressive dcSSc. To our knowledge, our cases are the first successful experiences with this treatment modality in Croatia and among the Slavic populations of the Balkan Peninsula, promoting the need for earlier interventions in patients who develop a progressive disease course, particularly with skin involvement.
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| format | Article |
| id | doaj-art-0c39cf49bc3b475b8f4eebac4eec6ef6 |
| institution | OA Journals |
| issn | 0048-7449 2240-2683 |
| language | English |
| publishDate | 2025-05-01 |
| publisher | PAGEPress Publications |
| record_format | Article |
| series | Reumatismo |
| spelling | doaj-art-0c39cf49bc3b475b8f4eebac4eec6ef62025-08-20T02:39:02ZengPAGEPress PublicationsReumatismo0048-74492240-26832025-05-0110.4081/reumatismo.2025.1822Autologous hematopoietic stem-cell transplantation in diffuse cutaneous systemic sclerosis: a single-center experienceMarko Barešić0Ljiljana Smiljanić Tomičević1https://orcid.org/0000-0003-2807-7440Igor Aurer2Mislav Cerovec3Branimir Anić4Division of Clinical Immunology and Rheumatology, Department of Internal Medicine, School of Medicine, University Hospital Center Zagreb, University of ZagrebDivision of Clinical Immunology and Rheumatology, Department of Internal Medicine, School of Medicine, University Hospital Center Zagreb, University of ZagrebDivision of Hematology, Department of Internal Medicine, School of Medicine, University Hospital Center Zagreb, University of ZagrebDivision of Clinical Immunology and Rheumatology, Department of Internal Medicine, School of Medicine, University Hospital Center Zagreb, University of ZagrebDivision of Clinical Immunology and Rheumatology, Department of Internal Medicine, School of Medicine, University Hospital Center Zagreb, University of Zagreb Systemic sclerosis (SSc) is a chronic autoimmune disorder characterized by multisystem involvement. Patients can be stratified into an indolent or rapidly progressive disease course. A progressive course warrants early and more aggressive treatment to prevent irreversible organ damage. Therapeutic strategies should be tailored to the presenting symptoms and organ involvement. Autologous hematopoietic stem cell transplantation (AHSCT) has proven to be an effective treatment modality for specific phenotypes of SSc, especially progressive diffuse cutaneous SSc. However, the optimal timing for the transplantation remains unknown. We present two cases of rapidly progressive diffuse cutaneous systemic sclerosis (dcSSc) treated with AHSCT following inadequate response to conventional immunosuppressive therapy. While both patients experienced significant cutaneous improvement post-AHSCT, internal organ involvement progressed in one case, ultimately resulting in a fatal outcome due to severe sepsis, whereas the second patient remained clinically stable and without immunosuppressive therapy during long-term follow-up. This report contributes to the growing body of evidence supporting AHSCT as a therapeutic option in carefully selected cases of progressive dcSSc. To our knowledge, our cases are the first successful experiences with this treatment modality in Croatia and among the Slavic populations of the Balkan Peninsula, promoting the need for earlier interventions in patients who develop a progressive disease course, particularly with skin involvement. https://www.reumatismo.org/reuma/article/view/1822Systemic sclerosisautologous stem cell transplantationmodified Rodnan skin scoreCroatia |
| spellingShingle | Marko Barešić Ljiljana Smiljanić Tomičević Igor Aurer Mislav Cerovec Branimir Anić Autologous hematopoietic stem-cell transplantation in diffuse cutaneous systemic sclerosis: a single-center experience Reumatismo Systemic sclerosis autologous stem cell transplantation modified Rodnan skin score Croatia |
| title | Autologous hematopoietic stem-cell transplantation in diffuse cutaneous systemic sclerosis: a single-center experience |
| title_full | Autologous hematopoietic stem-cell transplantation in diffuse cutaneous systemic sclerosis: a single-center experience |
| title_fullStr | Autologous hematopoietic stem-cell transplantation in diffuse cutaneous systemic sclerosis: a single-center experience |
| title_full_unstemmed | Autologous hematopoietic stem-cell transplantation in diffuse cutaneous systemic sclerosis: a single-center experience |
| title_short | Autologous hematopoietic stem-cell transplantation in diffuse cutaneous systemic sclerosis: a single-center experience |
| title_sort | autologous hematopoietic stem cell transplantation in diffuse cutaneous systemic sclerosis a single center experience |
| topic | Systemic sclerosis autologous stem cell transplantation modified Rodnan skin score Croatia |
| url | https://www.reumatismo.org/reuma/article/view/1822 |
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