https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9668796/ Off label...

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9668796/

Off label use for Adult SR-RR-aGvHD Grade lll-lV would be, imo, immediate- look at the gobblegook re an already approved approved therapy

Patients and methods

This retrospective study included 18 patients treated with RUX as a salvage therapy for GIII–IV SR-aGVHD.

Steroid-refractory acute graft-versus-host disease (SR-aGVHD) remains a major cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Ruxolitinib (RUX), an oral JAK1 and JAK2 inhibitor, has recently been approved for patients with SR-aGVHD. The aim of this study was to evaluate RUX efficacy and toxicity in a real-world setting. Eighteen patients received RUX at 5 mg or 10 mg twice a day after a median 3 lines of prior unsuccessful immunosuppressive therapy. Median time on RUX therapy was 28 days (range 7–129). Five patients (28%) responded to RUX, including 4 complete responses and 1 partial response. Response to RUX was irrespective of aGVHD grade and the number of involved organs. One-year overall survival (OS) was 60% for RUX-responders versus 31% for non-responders (p = ns). Treatment duration greater than 29.5 days was found to have a positive impact on OS (p < 0.007). Major adverse events during RUX treatment were grade 3–4 thrombocytopenia (61% of patients) and cytomegalovirus reactivation (50%). After median follow-up of 55 days (range 29–706), 14 patients (78%) died, mainly due to further progression of GVHD. RUX may represent a valuable therapeutic option for some patients with advanced SR-aGVHD, but more studies are warranted.

Valuable option?????????????????????????? almost all have left this earth in just 55 days....................

@stanjupiter Hi SJ, hopefully we can get a positive result this time around and a major shift in how our most vulnerable are treated. Thank-you for your ongoing , thoroughly researched posts on this forum.


Reg