Article ; Online: Real-world experience with ruxolitinib therapy for steroid-refractory acute graft versus host disease.
2024
Abstract: Acute graft versus host disease (aGVHD) is a complication of allogeneic hematopoietic stem cell transplant (HCT) and is associated with significant morbidity and mortality. Steroid refractory aGVHD (SR-aGVHD) carries a particularly grim prognosis. ... ...
Abstract | Acute graft versus host disease (aGVHD) is a complication of allogeneic hematopoietic stem cell transplant (HCT) and is associated with significant morbidity and mortality. Steroid refractory aGVHD (SR-aGVHD) carries a particularly grim prognosis. Ruxolitinib has shown promise for treatment of SR-aGVHD in a phase 3 trial; however, safety and efficacy data outside of the clinical trial setting is lacking. We performed a multicenter retrospective study to examine the response to ruxolitinib and its efficacy in patients with SR-aGVHD. We included 59 patients treated with ruxolitinib for SR-aGVHD between 2015 and 2022. Of these 59 patients, 36 patients (61.0%) achieved a complete (CR) or partial response (PR) at 28 days, while 31 patients (52.5%) obtained a CR/PR at day 56. Patients that achieved a CR or PR at day 28 had a higher rate of overall survival (OS; 69.2%), compared with patients that did not (31.6%; p = 0.037). OS at 12 months was 41.5%, with a median OS duration of 5.3 months. Failure free survival (FFS) at 12 months was 29.1%, with a median FFS of 2.6 months. Overall, this real-world experience data support ruxolitinib as the standard of care for SR-aGVHD in a non-controlled trial population. |
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Language | English |
Publishing date | 2024-02-24 |
Publishing country | England |
Document type | Journal Article |
ZDB-ID | 632854-4 |
ISSN | 1476-5365 ; 0268-3369 ; 0951-3078 |
ISSN (online) | 1476-5365 |
ISSN | 0268-3369 ; 0951-3078 |
DOI | 10.1038/s41409-024-02249-8 |
Database | MEDical Literature Analysis and Retrieval System OnLINE |
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