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EBMT 2023 | A novel clinical trial endpoint for acute GvHD

James Ferrara, MD, DSc, Icahn School of Medicine at Mount Sinai, New York City, NY, comments on a novel early endpoint for acute graft-versus-host disease (GvHD) clinical trials combining the MAGIC algorithm probability (MAP) with the clinical response at week two, highlighting some of the issues associated with the currently used primary endpoint. Using this novel method, it is possible to distinguish three types of responses to treatment – complete, partial, and non-responses. This interview took place at the 49th Annual Meeting of the European Group for Blood and Marrow Transplantation (EBMT) held in Paris, France.

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Transcript (edited for clarity)

So the standard endpoint for acute GvHD treatment trials is the overall response rate at week four. That’s been the clinical endpoint for most trials, but there are a couple of problems with that endpoint. The first is that four weeks is a long time to wait to see if we can get a response. The second is that a response that goes from grade IV GvHD to grade III is the same as one that goes from grade II to grade I and those outcomes can actually be very different for patients...

So the standard endpoint for acute GvHD treatment trials is the overall response rate at week four. That’s been the clinical endpoint for most trials, but there are a couple of problems with that endpoint. The first is that four weeks is a long time to wait to see if we can get a response. The second is that a response that goes from grade IV GvHD to grade III is the same as one that goes from grade II to grade I and those outcomes can actually be very different for patients. So, we wanted to see whether we could design a faster and better clinical trial endpoint. The first way we did that was to look at the onset overall grade and then the change in grade at week two. What we found was that we could find an endpoint that works as well as the overall response grade at week four. So that’s the first part of our abstract. The second part of our abstract actually looked at the serum biomarkers, the map, the MAGIC algorithm probability, which is the combination of two biomarkers ST2 and REG3α. That blood test, when added to the clinical response at week two, actually gives a far superior risk stratification and actually gives us three different groups of patients: complete responses, intermediate responses or partial responses, and non-responses. So, this is now a both faster and better clinical trial endpoint, that if we can validate it in other data sets, [it] could become the new primary endpoint for GvHD clinical trials.

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Disclosures

US patent for serum biomarkers of GVHD