ASH 2025 | Advances in GvHD management presented at ASH 2025

Despite all advances in terms of targeted therapies, small molecules, we still need to perform allogeneic stem cell transplantation in many patients where we don’t have such effective treatments. And interestingly, in myeloid malignancies, MDS, high-risk AML, myelofibrosis, allotransplant is likely here to stay for the next few years. While the results of transplant are clearly and significantly improving, the morbidity and mortality is decreasing,  GvHD remains a matter of concern, although the number of patients being confronted is likely decreasing thanks to more and more powerful GvHD prophylaxis regimens. Nevertheless, when it comes to acute GvHD, for instance, especially for those patients who are steroid-refractory, a few years ago, we celebrated at ASH the advent of ruxolitinib. And this is now a standard of care for those who are steroid-refractory with acute GvHD. 

But the problem we’re facing and we’ve been facing in the last few years was about, actually, the ruxolitinib-refractory patient. No options are available for these patients. And during this ASH 2025 in Orlando, we have the release of the ARES phase III trial, which has tested a fecal microbiota product, namely MaaT013, now with a nice name, Xervyteg, in those patients who are ruxolitinib refractory with acute GvHD. The results are amazing – a very high response rate, namely CRs and VGPRs, almost 60%. And most importantly, this is translating into a clear benefit in terms of survival at one year. Because we know very well that, in the last two or three decades, many drugs were developed and proved to be effective, at least in the early days when you treat acute GvHD, but they failed at the end because the response didn’t translate into a survival benefit. So the FMT story with this Xervyteg product is clearly proving to be very exciting. 

Of course, we have also chronic GVHD. And we are seeing also some nice advances, critical improvements when it comes to management. We have a similar scenario, corticosteroids, ruxolitinib is also approved. And now we’re lucky, we have belumosudil and a ROCK2 inhibitor. And during this ASH annual meeting in Orlando, we had communications looking with a lot of granularity about the kinetics of responses, the features of responses in the different organs. And it is clear now that belumosudil works very well, but you need to give it time to work. So you need to stick to belumosudil before moving onto another line of therapy. 

We have also new exciting data with axatilimab, which is a monoclonal antibody targeting the colony-stimulating factor receptor. But also now it’s being combined with other agents. So you can see lots of advances, lots of excitement. And I think this is important because chronic GvHD has a significant impact in the long term on the quality of life of patients. And this is why developing new agents, but also early interventions, I think early intervention is a key word when it comes to management of chronic GvHD, is something crucial, and I’m very pleased to see that this field, after 30 or 40 years of stagnation, has moved very nicely in a positive way.

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