Real-world experience with ruxolitinib therapy for steroid-refractory acute graft versus host disease.
Alistair MurraySwe Mar LinnBenoit YuIgor Novitzky-BassoJonas MattssonMichael KennahMohamed ElemaryJennifer WhiteChristopher LemieuxKareem JamaniDennis Dong Hwan KimPublished in: Bone marrow transplantation (2024)
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.
Keyphrases
- end stage renal disease
- ejection fraction
- clinical trial
- prognostic factors
- peritoneal dialysis
- randomized controlled trial
- liver failure
- intensive care unit
- low dose
- palliative care
- patient reported outcomes
- cross sectional
- big data
- cell death
- open label
- cell proliferation
- high dose
- pain management
- chronic pain
- patient reported
- hepatitis b virus
- smoking cessation