EBMT 2026 | Investigating peri-transplant ruxolitinib as GvHD prophylaxis in patients with myelofibrosis

So, the rationale for the peritransplant RUX study originated from an initial study I did open in 2014 with pre-transplant only RUX. In the past, the non-relapse mortality for patients with myelofibrosis, at least at our center, had been around 30%. And with the approval of JAK inhibitors, we hypothesized that patients who had spleen response and symptom response would have improved response post-transplant because of improved performance status and engraftment. So we, in that study, administered ruxolitinib for at least eight weeks prior to transplant and improved survival by 20% and decreased non-relapsed mortality down from 30% to about 12%. But patients still had 71% incidence of acute graft-versus-host disease and 55% chronic with 44% of that being moderate to severe. And that was published in the TCT journal. 

So the next step I thought after the approval of ruxolitinib for the treatment of acute and chronic graft-versus-host disease was to continue it through transplant for prevention. And so in this study, we required patients to receive at least eight weeks but continue through transplant and then receive ruxolitinib for at least nine months, so at least two months after stopping tacrolimus. And we did note in the study almost identical non-relapse mortality, though the one year was only 2.5%, and the graft-versus-host disease incidence was only 27% acute with about 10% chronic and almost no moderate to severe, about 5%. So we’ve seen great improvements with the peritransplant RUX platform at our center. And I wouldn’t say it’s the standard of care for us at this point, but we have a follow-up study now looking at patients with mismatched donors, antigen mismatched donors, and overlap syndromes that’s going to further expand that platform.

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