Peterson on Second-Line Ruxolitinib in Acute GVHD


Peterson discusses the investigation of ruxolitinib in the second line for patients with acute graft-vs-host disease following allogeneic hematopoietic stem cell transplant.

Graft-versus-host disease (GVHD), a serious complication of stem cell transplants, often requires second-line treatment beyond corticosteroids. This study investigated the outcomes of patients using ruxolitinib (Jakafi), a JAK1/2 inhibitor, as a second-line treatment for acute GVHD (aGVHD).

Researchers compared the survival and viral infection rates of 46 patients who received ruxolitinib after initial treatment failed to a group of 23 patients who did not require additional treatment. The two groups showed similar overall survival rates, with a median of 16.6 months for the ruxolitinib group and 18.8 months for the non-ruxolitinib group.

However, the ruxolitinib group saw a higher rate of viral infections (74% vs 52%) and cytomegalovirus (CMV) reactivation (30% vs 22%). This raises concerns about potential side effects of ruxolitinib, particularly for immunocompromised patients.

Chelsea Peterson, DO, who presented the poster at the 2024 Transplantation and Cellular Therapy Tandem Meetings, emphasized the need for close monitoring for infections and suggest further studies to explore the effectiveness of CMV prophylaxis in patients receiving ruxolitinib for aGVHD.

While ruxolitinib appears to be a viable second-line treatment option for aGVHD with similar survival rates to alternative approaches, its potential impact on the risk of viral infections warrants further investigation and careful patient management.

Here Peterson, hematologist/oncologist at Allegheny Health Network, discusses these findings.


0:05 | Ruxolitinib has become more widely used over the past few years for treatment of acute and chronic GVHD. And initially, when the REACH trials came out, they showed very promising response rates for GVHD, but we thought it was important to kind of look at real-life experiences and outcomes with our patients.

0:26 | GVHD, treatment of GVHD has actually come a long way. But along with these treatments that we've discovered, we're starting to notice that there are some complications associated with the treatments that we're using for GVHD, such as cytopenias, infections. So kind of finding ways to mitigate some of those complications while still being able to effectively treat GVHD is kind of where we're at now.

Our first goal was to look at overall survival in patients in our institution that were treated with oxidative or acute GVHD. And we compare that with patients that did not require second line treatment for acute GVHD.

1:07 | And then our other objective was to look at rates of CMV reactivation. We actually found that overall survival in our patients that were treated relative for steroid-refractory GVHD was about 16.6 months. And this is compared to 18.8 months in patients that did not require second-line treatment for GVHD. So this is pretty big, because traditionally, patients that were treated in the past with priority, approval of ruxolitinib for steroid-refractory GVHD, overall survival was about 6.5 months. So this is pretty big. One of the other things that we found in this study was increased rates of CMV reactivation in patients that are treated with ruxolitinib.

1:51 | So I think one of the big takeaways is that ruxolitinib, now that it's 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, you know, possibly putting patients at increased risk for infectious complications, cytopenias. So, close monitoring of CMV PCR, considering longer term prophylaxis for CMV, may be important for these patients.

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