Ruxolitinib as second-line therapy for bronchiolitis obliterans syndrome after hematopoietic cell transplantation: a retrospective multicenter study

Chronic graft-versus-host disease (cGVHD) is a major complication for long-term survivors after hematopoietic cell transplantation (HCT). Bronchiolitis obliterans syndrome (BOS) is the crucial manifestation of lung cGVHD. Ruxolitinib has been approved by the Food and Drug Administration (FDA) for cGVHD treatment, but the response for BOS has not been sufficiently evaluated. We conducted a multicenter study involving 125 patients diagnosed with BOS post-HCT who received second-line therapies, including 77 patients treated with ruxolitinib (ruxolitinib group) and 48 patients with non-ruxolitinib therapies (control group). The primary endpoint was progression of BOS. By the third month, 18.2% of patients in the ruxolitinib group progressed, versus 43.8% in the control group (p = 0.001). The 2-year overall survival (OS) was 73.2% (95%CI 63.5%-84.5%) for the ruxolitinib group versus 60.2% (95%CI 47.4%-76.6%) for the control group (p = 0.012). The BOS-progression-free survival (BOS-PFS) was 69.0% (95%CI 59.1%-80.5%) versus 51.2% (95%CI 38.7%-67.7%, p = 0.011). Early switch to second-line therapy for BOS independently favored lower progression incidence (OR = 0.203, [95%CI 0.070-0.585], p = 0.003). Additionally, we observed that ruxolitinib was well-tolerated during the treatment of BOS. In conclusion, our findings indicated that ruxolitinib is an effective and safe second-line option for BOS.

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Publication Details

Journal
Annals of Hematology
Published
2025-11-21
DOI
https://doi.org/10.1007/s00277-025-06726-y
Primary Topic
Hematopoietic Stem Cell Transplantation
Type
article
Field-Weighted Citation Impact
0.00

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article

Ruxolitinib as second-line therapy for bronchiolitis obliterans syndrome after hematopoietic cell transplantation: a retrospective multicenter study

Yinghao Lu, Yue Han, Lingyi Yang, Yuqing Tu et al.
Annals of Hematology
Hematopoietic Stem Cell Transplantation
article

Ruxolitinib as second-line therapy for bronchiolitis obliterans syndrome after hematopoietic cell transplantation: a retrospective multicenter study

Yinghao Lu, Yue Han, Lingyi Yang, Yuqing Tu, Yuhua Ru, Xiaowen Tang, Ye Zhao, Xiaojin Wu, Mengqi Xiang, Shushu Xu, Mimi Xu, Yulian Zheng, Fei Li, Feng Chen, Xiaoli Li, Jia Chen, Yanming Zhang, Depei Wu, Yang Xu, Miao Miao, Yi Fan, Xiaoyu Zhu, Yaxin Cheng, Huiying Qiu
article en

Abstract

Chronic graft-versus-host disease (cGVHD) is a major complication for long-term survivors after hematopoietic cell transplantation (HCT). Bronchiolitis obliterans syndrome (BOS) is the crucial manifestation of lung cGVHD. Ruxolitinib has been approved by the Food and Drug Administration (FDA) for cGVHD treatment, but the response for BOS has not been sufficiently evaluated. We conducted a multicenter study involving 125 patients diagnosed with BOS post-HCT who received second-line therapies, including 77 patients treated with ruxolitinib (ruxolitinib group) and 48 patients with non-ruxolitinib therapies (control group). The primary endpoint was progression of BOS. By the third month, 18.2% of patients in the ruxolitinib group progressed, versus 43.8% in the control group (p = 0.001). The 2-year overall survival (OS) was 73.2% (95%CI 63.5%-84.5%) for the ruxolitinib group versus 60.2% (95%CI 47.4%-76.6%) for the control group (p = 0.012). The BOS-progression-free survival (BOS-PFS) was 69.0% (95%CI 59.1%-80.5%) versus 51.2% (95%CI 38.7%-67.7%, p = 0.011). Early switch to second-line therapy for BOS independently favored lower progression incidence (OR = 0.203, [95%CI 0.070-0.585], p = 0.003). Additionally, we observed that ruxolitinib was well-tolerated during the treatment of BOS. In conclusion, our findings indicated that ruxolitinib is an effective and safe second-line option for BOS.

Annals of HematologyVol. 104(12)
University of Science and Technology of China (CN), Guiyang Medical University (CN), Xuzhou Medical College (CN), Soochow University (CN), Affiliated Hospital of Guizhou Medical University (CN), Second Affiliated Hospital of Soochow University (CN), Second People’s Hospital of Huai’an (CN), First Affiliated Hospital of Soochow University (CN)
National Natural Science Foundation of China
Openalex Percentile: Top 54%
Hematopoietic Stem Cell Transplantation
DOIOpen Access PDFOpenAlex
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