EBMT 2026 | Clinical outcomes among patients with chronic GvHD treated with axatilimab

So this study is the clinical outcomes among patients with chronic graft-versus-host disease treated with axatilimab, and it’s an analysis of an expanded access program. So basically the objective of the study was to evaluate the safety and clinical benefit of axatilimab amongst patients with chronic GVHD enrolled in the U.S. on an expanded access program. The treatment was basically given every two weeks, either until the patient could not tolerate the treatment or their GVHD improved. So the key eligibility criteria, patients had to have refractory or recurrent active chronic GVHD per the 2014 NIH consensus criteria, and they had to fail greater than or equal to two prior lines of systemic therapy. So approximately 112 patients were enrolled on the study and about 104 patients were included in the analysis. So once the drug was approved in the U.S. for commercial use, patients were then transitioned off the expanded access program to receive commercial supply. 

So at the end of the study, 67% of the patients were still receiving axatilimab and were able to move on and receive commercial supply of the drug. So the drug was very well tolerated. I mean, the safety profile was very good. So unfortunately, one of the pitfalls of the poster is that it doesn’t, because this was patients who transitioned from the expanded access protocol to commercial supply. There’s no real follow-up data to show exactly how patients are currently doing and exactly how the chronic GVHD has responded to the treatment. So all you can really say is we can assume that if patients are continued on the therapy, that either the chronic GVHD is stable or improving with the treatment. And as far as safety-wise, the drug is well-tolerated. It’s given every two weeks with very little toxicity.

This transcript is AI-generated. While we strive for accuracy, please verify this copy with the video.