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EBMT 2026 | Expert recommendations on the management of ruxolitinib-refractory GvHD

Guillermo Ortí, MD, PhD, Vall d’Hebron Institute of Oncology, Barcelona, Spain, provides insight into managing patients with ruxolitinib-refractory graft-versus-host disease (GvHD), noting that over 50% of patients with GvHD fall into this group. Dr Ortí highlights the need to refine and consolidate the definition of ruxolitinib-refractory status and to expand the evidence base on the best therapeutic approach in this setting. This interview took place at the 52nd Annual Meeting of the EBMT in Madrid, Spain.

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Transcript

So we know that in both acute graft-versus-host disease and chronic graft-versus-host disease, the standard of care is steroids and then ruxolitinib, it’s now accepted as the standard of therapy. I would say that failure, which combines patients that are dependent, intolerant, or that they are refractory in acute graft-versus-host disease, as per what we’ve seen in the most recent trials, it’s more than 50%...

So we know that in both acute graft-versus-host disease and chronic graft-versus-host disease, the standard of care is steroids and then ruxolitinib, it’s now accepted as the standard of therapy. I would say that failure, which combines patients that are dependent, intolerant, or that they are refractory in acute graft-versus-host disease, as per what we’ve seen in the most recent trials, it’s more than 50%. And in chronic graft-versus-host disease, this also applies to more than 50% of patients with a long follow-up of around three years. So I would say it’s not an uncommon scenario in this setting, and still an unmet need there. 

In these expert recommendations, we try to address some of the open issues that we have currently in this setting. For instance, how we define ruxolitinib refractoriness after chronic graft-versus-host disease. We know that there has been efforts and there is published work, on what to consider ruxolitinib refractory graft versus host disease. But in chronic graft versus host disease, I think that that remains a little bit more open. So we try to work on defining when should we consider patients to be ruxolitinib refractory or failing to ruxolitinib, and then try to recommend to do another therapeutic approach in this setting. And in terms of what to do, we know that the evidence in this setting is limited. We, for the time being, don’t have published phase three randomized trials there. So we try to analyze what’s the data there and try to do some recommendations but with being humble in terms of that as there is no great evidence there we don’t know where to place one or another approach.

 

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Disclosures

Honoraria: Chiesi, Jazz, MSD, Novartis, Pfizer, Sanofi, Therakos; Travel support: Incyte, Jazz, Novartis, Pfizer, Sanofi. Research support: Incyte (institutional).