New Treatment Options in Chronic GVHD - Episode 6

The Evolving Treatment Landscape of Chronic GVHD

Closing out his discussion on chronic GVHD, Corey Cutler, MD, MPH, FRCPC, discusses belumosudil and how all 3 newly approved agents fit into the evolving treatment landscape.


Corey Cutler, MD, MPH, FRCPC: There are other agents available in chronic graft versus host disease [GVHD], and most recently, belumosudil was approved for therapy in individuals with steroid-refractory disease who had progressed despite 2 lines of therapy. It was based on the ROCKstar study, which enrolled approximately 132 subjects and randomized them to KD025, or belumosudil, at a dose of 200 mg once daily or 200 mg twice daily. There was no active control in this study. So this was a randomized phase 2 trial. Here, we measured the best overall response rate, which for the entire group, was approximately 75%, without important differences between the 200 mg once-daily and 200 mg twice-daily arms. Also very importantly, in this trial we were able to demonstrate a very high response rate in subjects who had previously received ruxolitinib or ibrutinib; 46 subjects had previously received ibrutinib and 38 had received ruxolitinib, and the response rates in those patients were between 68% and 74%. On the basis of this trial, belumosudil, at a dose of 200 mg once daily, or 200 mg twice daily for subjects on proton pump inhibitors, was approved for steroid-resistant disease as well.

I think the impact of these novel agents and their approval is really going to help our patients. Having 3 active drugs, whereas only 4 years ago we had none, really is going to change how we view chronic graft versus host disease. I think we’re going to clearly start using these compounds with demonstrated efficacy earlier rather than waiting until subjects have 2 or 3 lines of prior failed therapies. I think you’re going to see this gradual progression of using these agents, somewhat off-label, and earlier in the disease course. We’re of course going to try novel prophylaxis strategies. We are really looking at a whole new world of chronic GVHD with the recent uptake in post-transplant cyclophosphamide, because it is true that this regimen does lead to lower incidence rates of chronic GVHD. With lower chronic GVHD overall, there’s certainly going to be less steroid-refractory chronic GVHD for us to have to deal with. Overall, having new agents, and having strategies that actually reduce the rates of chronic GVHD, is really going to be groundbreaking for our patients.

While we’re making tremendous strides in the therapy of chronic graft vs host disease, the way we continue to move the field forward is with continued clinical research. I would encourage referral back to a transplant center for participation in clinical trials for those patients who are having steroid-resistant chronic GVHD who are being treated in the community. I think it’s really the way we move the field forward. Most of us at academic or university centers are really happy to be a resource to our community partners who are treating chronic GVHD in the community.

Transcript edited for clarity.