Ruxolitinib Prior to Transplantation Linked to Better Survival in Myelofibrosis
Ruxolitinib was initially studied in patients with myelofibrosis (MF) who were not considered candidates for transplantation. However, its ability to reduce splenomegaly and improve symptoms has led to increased eligibility for hematopoietic cell transplantation (HCT) in some patients. As the role of JAK inhibitors as a routine pre-HCT therapy remains uncertain, researchers sought to investigate its potential benefits in transplant-eligible patients in a recent phase 2 study.
The researchers suggest that pre-transplant use of ruxolitinib may provide survival benefits for patients with MF undergoing HCT. The study was conducted at a single center between 2014 and 2020, and enrolled 61 adult patients with MF (median age, 57 years) who received ruxolitinib for a median of 7 months before undergoing HCT.
The results showed that patients who received ruxolitinib prior to HCT had improved overall survival (OS) and lower nonrelapse mortality (NRM) compared to historical controls. At a median follow up of 5.2 years, OS was 79% (95% CI, 69-90) vs 60% at 2 years, and 74% (95% CI, 64-86) vs 57% at 5 years, respectively. Additionally, NRM at 1 year was 13%, significantly lower than the 26% observed in controls.
While ruxolitinib pre-treatment appeared to reduce NRM and improve survival, the incidence of graft-vs-host disease (GVHD) remained high, with acute grade II-IV GVHD occurring in 71% of patients. Interestingly, longer pre-HCT JAK inhibitor use was associated with a lower risk of acute GVHD.
The findings suggest that pre-HCT JAK inhibition may offer meaningful benefits to patients with MF who are eligible for transplant, though the relationship between JAK inhibition, GVHD, and disease risk stratification requires further investigation.
Reference
Salit RB, Fan X, Ratsamee N, et al. Phase II prospective study of intentional pre-treatment of hematopoietic cell transplant eligible myelofibrosis patients with ruxolitinib. Presented at Tandem Meetings 2025. February 12-15, 2025. Honolulu, HI. Abstract 271.
