Long-term follow-up results of ruxolitinib as salvage therapy for chronic graft-versus-host disease
Introduction: Chronic graft-versus-host disease poses a significant challenge after allogeneic hematopoietic stem cell transplantation with initial treatment often relying on high-dose steroids. However, managing steroid-refractory disease remains daunting. Recent insights into the mechanisms have u...
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Elsevier
2025-07-01
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| Series: | Hematology, Transfusion and Cell Therapy |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2531137925001038 |
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| author | Neslihan Mandaci Sanli Esen Karakuş |
| author_facet | Neslihan Mandaci Sanli Esen Karakuş |
| author_sort | Neslihan Mandaci Sanli |
| collection | DOAJ |
| description | Introduction: Chronic graft-versus-host disease poses a significant challenge after allogeneic hematopoietic stem cell transplantation with initial treatment often relying on high-dose steroids. However, managing steroid-refractory disease remains daunting. Recent insights into the mechanisms have unveiled new treatment targets, with ruxolitinib, a selective JAK1/2 inhibitor, emerging as a promising and safe therapy for chronic graft-versus-host disease patients. Methods: This retrospective study describes the long-term outcomes of 23 chronic graft-versus-host disease patients treated with ruxolitinib. Results: Most patients presented with severe chronic graft-versus-host disease (15/23; 65.2%). The overall response rate was 78.3% (18/23) after a median treatment duration of four weeks, with 55.6% (10/18) achieving complete response. At follow-up, 13 of the 18 responders (72.2%) sustained complete remission. Patients had a median of two previous lines of therapy, with a median follow-up of 14 months (range: 2–46 months) after starting ruxolitinib. Of the patients who were responsive to ruxolitinib, median follow-up extended to 26.5 months. Notably, for the patients who were responsive to ruxolitinib, the 1-year, 2-year, and 3-year overall survival was 83.3% (95% CI: 64.2%-102%), 56.1% (95% CI: 30.1%-80.9%), and 33.3% (95% CI: 9.2%-57.4%), respectively. Malignancy relapse occurred in 17.4% (4/23) of patients, with 34.7% (8/23) experiencing cytopenias, albeit mostly mild. Reactivation rates for cytomegalovirus were nil. Conclusion: The long-term follow-up in this study supports ruxolitinib as an effective salvage therapy for chronic graft-versus-host disease with a 78.3% overall response rate and 55.6% complete remission rate. However, large prospective studies are warranted to validate these findings |
| format | Article |
| id | doaj-art-c81c125de6504b678fa95c1e98757b72 |
| institution | Kabale University |
| issn | 2531-1379 |
| language | English |
| publishDate | 2025-07-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Hematology, Transfusion and Cell Therapy |
| spelling | doaj-art-c81c125de6504b678fa95c1e98757b722025-08-20T03:36:15ZengElsevierHematology, Transfusion and Cell Therapy2531-13792025-07-0147310383510.1016/j.htct.2025.103835Long-term follow-up results of ruxolitinib as salvage therapy for chronic graft-versus-host diseaseNeslihan Mandaci Sanli0Esen Karakuş1Erciyes University Faculty of Medicine, Department of Hematology and Bone Marrow Transplant Center, 38039, Kayseri, Turkey; Corresponding author. Neslihan Mandaci Sanli, Erciyes University Faculty of Medicine, Department of Hematology and Bone Marrow Transplant Center, 38039, Kayseri, Turkey.Erciyes University Faculty of Medicine, 38280 ,Kayseri, TurkeyIntroduction: Chronic graft-versus-host disease poses a significant challenge after allogeneic hematopoietic stem cell transplantation with initial treatment often relying on high-dose steroids. However, managing steroid-refractory disease remains daunting. Recent insights into the mechanisms have unveiled new treatment targets, with ruxolitinib, a selective JAK1/2 inhibitor, emerging as a promising and safe therapy for chronic graft-versus-host disease patients. Methods: This retrospective study describes the long-term outcomes of 23 chronic graft-versus-host disease patients treated with ruxolitinib. Results: Most patients presented with severe chronic graft-versus-host disease (15/23; 65.2%). The overall response rate was 78.3% (18/23) after a median treatment duration of four weeks, with 55.6% (10/18) achieving complete response. At follow-up, 13 of the 18 responders (72.2%) sustained complete remission. Patients had a median of two previous lines of therapy, with a median follow-up of 14 months (range: 2–46 months) after starting ruxolitinib. Of the patients who were responsive to ruxolitinib, median follow-up extended to 26.5 months. Notably, for the patients who were responsive to ruxolitinib, the 1-year, 2-year, and 3-year overall survival was 83.3% (95% CI: 64.2%-102%), 56.1% (95% CI: 30.1%-80.9%), and 33.3% (95% CI: 9.2%-57.4%), respectively. Malignancy relapse occurred in 17.4% (4/23) of patients, with 34.7% (8/23) experiencing cytopenias, albeit mostly mild. Reactivation rates for cytomegalovirus were nil. Conclusion: The long-term follow-up in this study supports ruxolitinib as an effective salvage therapy for chronic graft-versus-host disease with a 78.3% overall response rate and 55.6% complete remission rate. However, large prospective studies are warranted to validate these findingshttp://www.sciencedirect.com/science/article/pii/S2531137925001038Chronic graft-versus-host diseaseRuxolitinibAllogeneic hematopoietic stem cell transplantationStem cell transplantation |
| spellingShingle | Neslihan Mandaci Sanli Esen Karakuş Long-term follow-up results of ruxolitinib as salvage therapy for chronic graft-versus-host disease Hematology, Transfusion and Cell Therapy Chronic graft-versus-host disease Ruxolitinib Allogeneic hematopoietic stem cell transplantation Stem cell transplantation |
| title | Long-term follow-up results of ruxolitinib as salvage therapy for chronic graft-versus-host disease |
| title_full | Long-term follow-up results of ruxolitinib as salvage therapy for chronic graft-versus-host disease |
| title_fullStr | Long-term follow-up results of ruxolitinib as salvage therapy for chronic graft-versus-host disease |
| title_full_unstemmed | Long-term follow-up results of ruxolitinib as salvage therapy for chronic graft-versus-host disease |
| title_short | Long-term follow-up results of ruxolitinib as salvage therapy for chronic graft-versus-host disease |
| title_sort | long term follow up results of ruxolitinib as salvage therapy for chronic graft versus host disease |
| topic | Chronic graft-versus-host disease Ruxolitinib Allogeneic hematopoietic stem cell transplantation Stem cell transplantation |
| url | http://www.sciencedirect.com/science/article/pii/S2531137925001038 |
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