Future Directions for the Treatment of GVHD

— Doris Ponce, MD, discusses the interest around designing customized clinical trials

MedicalToday

Graft-versus-host disease (GVHD) remains one of the most common complications of allogeneic stem cell transplantation (ASCT).

In this exclusive video, , of Memorial Sloan Kettering Cancer Center in New York City, shares her insights on the future treatment landscape for patients with GVHD.

Following is a transcript of her remarks:

I think I'm seeing more customized treatment and interest in designing clinical trials that are customized. For example, if a patient has graft-versus-host disease, we're looking now into putting that patient into a box if they are a standard risk or high risk, and then in their clinical trials ... taking that into consideration.

Like we have the BMT CTN study for alpha-1 antitrypsin designed for high-risk patients. We also have a BMT CTN study designed for patients with a standard risk, trying to minimize the steroid exposure.

So, I see more now ... trying to customize treatment according to the patient's need. I also see in graft-versus-host disease of trying to reduce exposure to steroids. And we have newer drugs that had been recently approved. So I see clinicians more comfortable using treatment.

More patients, for example, are receiving ruxolitinib [Jakafi] for steroid-refractory GVH. Because this is now standard of care, we're looking into protocol designs that are incorporating ruxolitinib as part of their treatment. For example, combination of the study drug plus ruxolitinib. And I think part of this era is trying to improve outcomes that we already observed. So we know ruxolitinib provides better treatment response than our previous standard of care, but now we want to do better and improve outcomes even further with the use of ruxolitinib in steroid-refractory GVHD.

So that's how I see things coming up. Like microbiome, I feel it's a field that could be quite customized for the GI track. This is a setting for prevention for study design, but it could also expand into treatment. So, it is not applicable to the study, but it's possible in the future that as we see with FMT [fecal microbiota transplantation], that microbiome could be used in the setting of patients that have GI track involvement alone, and this could be another therapeutic option for them.

  • author['full_name']

    Greg Laub is the Senior Director of Video and currently leads the video and podcast production teams.