Clinical Trial Investigation: Where to Go From Here



Zachariah DeFilipp, MD: The treatment options for patients with graft-versus-host disease [GvHD] are still limited. Therefore, future trials need to continue to investigate additional therapeutic agents. The investigation of well tolerated agents that address GvHD pathophysiology by different mechanisms is an essential need.

I also believe that the individualization of GvHD treatment is immensely important. Blood biomarkers for graft-versus-host disease, mainly ST2 and REG3 alpha, have been shown by the MAGIC consortium to be able to risk stratify graft-versus-host disease patients at the time of diagnosis and also into their treatment.

In patients with high-risk biomarkers, this may indicate that we should escalate their GvHD treatment. In patients with low-risk biomarkers, we may be able to de-escalate their therapy and still have successful outcomes.

Corey Cutler, MD: For future clinical trials, we need to learn how to tailor therapy to the individual patient. There are certainly some patients who we over treat with large doses of steroids, and then there are others who are destined to fail even before they receive their first dose of corticosteroids. Determining, either based on clinical or biomarker data, which patients we are going to over treat and under treat is an important research question. Minimizing toxicity from corticosteroids, either by the additive use of alternative agents with steroids or the alternative use to corticosteroids, is probably another important advancement that our field needs to take. But overall, we’ve made some tremendous strides, both in acute and chronic graft-versus-host disease in the last few years, and I suspect it’s only going to get better in the coming years.

Transcript Edited for Clarity

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