Marko Barešić, Ljiljana Smiljanić Tomičević, Igor Aurer, Mislav Cerovec, Branimir Anić
{"title":"Autologous hematopoietic stem-cell transplantation in diffuse cutaneous systemic sclerosis: a single-center experience.","authors":"Marko Barešić, Ljiljana Smiljanić Tomičević, Igor Aurer, Mislav Cerovec, Branimir Anić","doi":"10.4081/reumatismo.2025.1822","DOIUrl":null,"url":null,"abstract":"<p><p>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 SSc (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.</p>","PeriodicalId":21222,"journal":{"name":"Reumatismo","volume":" ","pages":""},"PeriodicalIF":1.0000,"publicationDate":"2025-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Reumatismo","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.4081/reumatismo.2025.1822","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/5/28 0:00:00","PubModel":"Epub","JCR":"Q4","JCRName":"RHEUMATOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
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 SSc (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.
期刊介绍:
Reumatismo is the official Journal of the Italian Society of Rheumatology (SIR). It publishes Abstracts and Proceedings of Italian Congresses and original papers concerning rheumatology. Reumatismo is published quarterly and is sent free of charge to the Members of the SIR who regularly pay the annual fee. Those who are not Members of the SIR as well as Corporations and Institutions may also subscribe to the Journal.