Safety and long-term efficacy of autologous hematopoietic cell transplantation for patients with systemic sclerosis
Autologous hematopoietic cell transplantation (HCT) has been introduced for patients with severe systemic sclerosis (SSc). We aimed to assess the safety and long-term efficacy of HCT modality for severe SSc, refractory to conventional therapy, in 17 patients who were referred to our – The Joint Accr...
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Frontiers Media S.A.
2025-07-01
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| author | Eleni Gavriilaki Eleni Gavriilaki Despina Mallouri Ioannis Batsis Zoi Bousiou Anna Vardi Nikolaos Spyridis Georgios Karavalakis Alkistis kyra Panteliadou Panagiotis Dolgyras Christos Varelas Vlasios I. Alevizopoulos Panagiotis G. Asteris Panayiotis Vlachoyiannopoulos Damianos Sotiropoulos Petros P. Sfikakis Ioanna Sakellari |
| author_facet | Eleni Gavriilaki Eleni Gavriilaki Despina Mallouri Ioannis Batsis Zoi Bousiou Anna Vardi Nikolaos Spyridis Georgios Karavalakis Alkistis kyra Panteliadou Panagiotis Dolgyras Christos Varelas Vlasios I. Alevizopoulos Panagiotis G. Asteris Panayiotis Vlachoyiannopoulos Damianos Sotiropoulos Petros P. Sfikakis Ioanna Sakellari |
| author_sort | Eleni Gavriilaki |
| collection | DOAJ |
| description | Autologous hematopoietic cell transplantation (HCT) has been introduced for patients with severe systemic sclerosis (SSc). We aimed to assess the safety and long-term efficacy of HCT modality for severe SSc, refractory to conventional therapy, in 17 patients who were referred to our – The Joint Accreditation Committee of the International Society for Cellular Therapy (ISCT) and the European Group for Blood and Marrow Transplantation (EBMT)-accredited Unit from 2005 to 2024. Peripheral blood stem cells were collected using cyclophosphamide and GCSF. An immunoablative conditioning regimen of cyclophosphamide and anti-thymocyte globulin was administered. Disease assessments were done before and after mobilization treatment and post-transplant, focusing on skin sclerosis, pulmonary function, cardiac involvement, gastrointestinal manifestations, the necessity for additional immunosuppressive therapy, and overall patient well-being. Before transplantation, 13/17 (76%) of the patients had diffuse skin involvement with a mean mRSS of 31 (2–49), 2/17 (12%) had pulmonary hypertension, and 14/17 (82%) had gastrointestinal manifestations. The median follow-up period was 9.1 (0. 5–14. 3) years. Improvement of skin sclerosis was observed, with a decrease in mRSS before transplantation from 31 (2–49) to 7 (2–22) post-HCT. Lung function remained stable in 8/15 (53%) patients, improved in 5/15 (33%), and deteriorated in 2/15 (13%). Gastrointestinal manifestations were improved in 12/14 (86%) patients, while all patients (16/16, 100%) reported a great impact on their quality of life. Ten out of the 16 (63%) patients were free of immunosuppressive drugs after the HCT. Overall survival was 16/17 (94.2%). Concerning TRM, there was one (1/17, 5.8%) death early post-transplant. In this specific cohort of selected patients with severe SSc refractory to immunosuppressive medications, autologous HCT led to improvements in the outcomes assessed. |
| format | Article |
| id | doaj-art-e8a4da1cea744eb3ad9419ebf0537b07 |
| institution | DOAJ |
| issn | 2296-858X |
| language | English |
| publishDate | 2025-07-01 |
| publisher | Frontiers Media S.A. |
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| series | Frontiers in Medicine |
| spelling | doaj-art-e8a4da1cea744eb3ad9419ebf0537b072025-08-20T03:12:26ZengFrontiers Media S.A.Frontiers in Medicine2296-858X2025-07-011210.3389/fmed.2025.15277791527779Safety and long-term efficacy of autologous hematopoietic cell transplantation for patients with systemic sclerosisEleni Gavriilaki0Eleni Gavriilaki1Despina Mallouri2Ioannis Batsis3Zoi Bousiou4Anna Vardi5Nikolaos Spyridis6Georgios Karavalakis7Alkistis kyra Panteliadou8Panagiotis Dolgyras9Christos Varelas10Vlasios I. Alevizopoulos11Panagiotis G. Asteris12Panayiotis Vlachoyiannopoulos13Damianos Sotiropoulos14Petros P. Sfikakis15Ioanna Sakellari16Hematology Department and Bone Marrow Transplant (BMT) Unit, G. Papanicolaou Hospital, Thessaloniki, Greece2nd Propedeutic Department of Internal Medicine, Hippocration Hospital, Aristotle University of Thessaloniki, Thessaloniki, GreeceGerman Oncology Center, Limassol, CyprusHematology Department and Bone Marrow Transplant (BMT) Unit, G. Papanicolaou Hospital, Thessaloniki, GreeceHematology Department and Bone Marrow Transplant (BMT) Unit, G. Papanicolaou Hospital, Thessaloniki, GreeceHematology Department and Bone Marrow Transplant (BMT) Unit, G. Papanicolaou Hospital, Thessaloniki, GreeceHematology Department and Bone Marrow Transplant (BMT) Unit, G. Papanicolaou Hospital, Thessaloniki, GreeceHematology Department and Bone Marrow Transplant (BMT) Unit, G. Papanicolaou Hospital, Thessaloniki, GreeceHematology Department and Bone Marrow Transplant (BMT) Unit, G. Papanicolaou Hospital, Thessaloniki, GreeceHematology Department and Bone Marrow Transplant (BMT) Unit, G. Papanicolaou Hospital, Thessaloniki, GreeceHematology Department and Bone Marrow Transplant (BMT) Unit, G. Papanicolaou Hospital, Thessaloniki, Greece2nd Propedeutic Department of Internal Medicine, Hippocration Hospital, Aristotle University of Thessaloniki, Thessaloniki, GreeceComputational Mechanics Laboratory, School of Pedagogical and Technological Education, Athens, GreeceDepartment of Pathophysiology, School of Medicine, National and Kapodistrian University of Athens, Greece; Institute for Autoimmune Systemic and Neurologic Diseases, Athens, GreeceHematology Department and Bone Marrow Transplant (BMT) Unit, G. Papanicolaou Hospital, Thessaloniki, Greece1st Department of Propaedeutic and Internal Medicine, Medical School, Joint Academic Rheumatology Program, National and Kapodistrian University of Athens, Athens, GreeceHematology Department and Bone Marrow Transplant (BMT) Unit, G. Papanicolaou Hospital, Thessaloniki, GreeceAutologous hematopoietic cell transplantation (HCT) has been introduced for patients with severe systemic sclerosis (SSc). We aimed to assess the safety and long-term efficacy of HCT modality for severe SSc, refractory to conventional therapy, in 17 patients who were referred to our – The Joint Accreditation Committee of the International Society for Cellular Therapy (ISCT) and the European Group for Blood and Marrow Transplantation (EBMT)-accredited Unit from 2005 to 2024. Peripheral blood stem cells were collected using cyclophosphamide and GCSF. An immunoablative conditioning regimen of cyclophosphamide and anti-thymocyte globulin was administered. Disease assessments were done before and after mobilization treatment and post-transplant, focusing on skin sclerosis, pulmonary function, cardiac involvement, gastrointestinal manifestations, the necessity for additional immunosuppressive therapy, and overall patient well-being. Before transplantation, 13/17 (76%) of the patients had diffuse skin involvement with a mean mRSS of 31 (2–49), 2/17 (12%) had pulmonary hypertension, and 14/17 (82%) had gastrointestinal manifestations. The median follow-up period was 9.1 (0. 5–14. 3) years. Improvement of skin sclerosis was observed, with a decrease in mRSS before transplantation from 31 (2–49) to 7 (2–22) post-HCT. Lung function remained stable in 8/15 (53%) patients, improved in 5/15 (33%), and deteriorated in 2/15 (13%). Gastrointestinal manifestations were improved in 12/14 (86%) patients, while all patients (16/16, 100%) reported a great impact on their quality of life. Ten out of the 16 (63%) patients were free of immunosuppressive drugs after the HCT. Overall survival was 16/17 (94.2%). Concerning TRM, there was one (1/17, 5.8%) death early post-transplant. In this specific cohort of selected patients with severe SSc refractory to immunosuppressive medications, autologous HCT led to improvements in the outcomes assessed.https://www.frontiersin.org/articles/10.3389/fmed.2025.1527779/fullcell therapyhematopoietic cell transplantationsystemic sclerosissclerodermatreatment-related mortality |
| spellingShingle | Eleni Gavriilaki Eleni Gavriilaki Despina Mallouri Ioannis Batsis Zoi Bousiou Anna Vardi Nikolaos Spyridis Georgios Karavalakis Alkistis kyra Panteliadou Panagiotis Dolgyras Christos Varelas Vlasios I. Alevizopoulos Panagiotis G. Asteris Panayiotis Vlachoyiannopoulos Damianos Sotiropoulos Petros P. Sfikakis Ioanna Sakellari Safety and long-term efficacy of autologous hematopoietic cell transplantation for patients with systemic sclerosis Frontiers in Medicine cell therapy hematopoietic cell transplantation systemic sclerosis scleroderma treatment-related mortality |
| title | Safety and long-term efficacy of autologous hematopoietic cell transplantation for patients with systemic sclerosis |
| title_full | Safety and long-term efficacy of autologous hematopoietic cell transplantation for patients with systemic sclerosis |
| title_fullStr | Safety and long-term efficacy of autologous hematopoietic cell transplantation for patients with systemic sclerosis |
| title_full_unstemmed | Safety and long-term efficacy of autologous hematopoietic cell transplantation for patients with systemic sclerosis |
| title_short | Safety and long-term efficacy of autologous hematopoietic cell transplantation for patients with systemic sclerosis |
| title_sort | safety and long term efficacy of autologous hematopoietic cell transplantation for patients with systemic sclerosis |
| topic | cell therapy hematopoietic cell transplantation systemic sclerosis scleroderma treatment-related mortality |
| url | https://www.frontiersin.org/articles/10.3389/fmed.2025.1527779/full |
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