Ruxolitinib Improves Survival but Increases Infections in Acute GVHD

Chelsea Peterson, DO

While ruxolitinib (Jakafi) has been an FDA-approved treatment for steroid-refractory acute graft-vs-host disease (GVHD) since 2019, real-world data on its usage is sparse. Researchers at Allegheny Health Network Cancer Institute, including Chelsea Peterson, DO, sought to look at real-world usage and potential complications that can arise with ruxolitinib treatment.

In a study that Peterson presented at the 2024 Transplantation and Cellular Therapy Tandem Meetings, researchers compared outcomes of patients who received ruxolitinib as a second-line option (after corticosteroids) to those who did not require further treatment.

It was observed that overall survival was similar between both groups at around 16 to 18 months; however, ruxolitinib demonstrated a significant improvement over historic survival rates for patients with steroid-refractory acute GVHD, which was about 6.5 months. Patients on ruxolitinib had a higher rate of viral infections (74% vs 52%) and cytomegalovirus (CMV) reactivation (30% vs 22%).

“I think 1 of the big takeaways is that ruxolitinib, now that it has been approved for steroid-refractory acute GVHD, should be the standard of care, and we should be using it. I think that as oncologists and community oncologists, too, we have to just be cognizant of the fact that we are possibly putting patients at increased risk for infectious complications [and] cytopenias,” Peterson, hematology/oncology fellow at Allegheny Health Network Cancer Institute in Pittsburgh, Pennsylvania, said in an interview with Targeted Oncology^TM.

In the interview, Peterson discussed the background and findings of the study, as well as unmet needs in the GVHD treatment space.

Targeted Oncology: What was the rationale or inspiration for the study?

Peterson: The inspiration for the study was [that] ruxolitinib has become more widely used over the past few years for treatment of acute and chronic GVHD. Initially, when the REACH trials [NCT02953678; NCT02913261] came out, they showed promising response rates for GVHD, but we thought it was important to look at real-life experiences and outcomes with our patients.

What are some of the unmet needs in the GVHD space?

Treatment of GVHD has come a long way. But along with these treatments that we have discovered, we are starting to notice that there are some complications associated with the treatments that we're using for GVHD, such as cytopenias [and] infections. Finding ways to mitigate some of those complications while still being able to effectively treat GVHD is where we are at now.

What were some of the goals of the study?

Our first goal was to look at overall survival in patients in our institution that were treated with ruxolitinib for acute GVHD. We compared that with patients that did not require second-line treatment for acute GVHD. Our other objective was to look at rates of CMV reactivation.

Could you summarize your findings?

We found that overall survival in our patients that were treated relative for steroid-refractory GVHD was about 16.6 months. This is compared with 18.8 months in patients that did not require second-line treatment for GVHD. This is pretty big, because traditionally, patients that were treated in the past prior to the approval of ruxolitinib for steroid-refractory GVHD, overall survival was about 6.5 months. One of the other things that we found in this study was increased rates of CMV reactivation in patients that are treated with ruxolitinib.

Are there any takeaways for a community oncologist?

I think 1 of the big takeaways is that ruxolitinib, now that it has been approved for steroid-refractory acute GVHD, should be the standard of care, and we should be using it. I think that as oncologists and community oncologists, too, we have to just be cognizant of the fact that we are possibly putting patients at increased risk for infectious complications [and] cytopenias. So close monitoring of CMV PCR [and] considering longer term prophylaxis for CMV may be important for these patients.

REFERENCE:

Peterson, C, Patrus G, Sadashiv S, et al. Outcomes of patients initiated on ruxolitinib as second line treatment of aGVHD: a single institution experience. Presented at: 2024 Transplantation and Cellular Therapy Tandem Meetings. February 21-24, 2024. San Antonio, TX. Poster 383.