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EBMT 2026 | Key findings from a European survey on allogeneic transplantation for myelofibrosis

Alex Rampotas, MD, PhD, University College London, London, UK, discusses the results of a European survey on allogeneic transplantation for myelofibrosis, conducted on behalf of the Chronic Malignancies Working Party of the EBMT. Dr Rampotas notes the benefits of using novel agents for symptom management, but highlights that most institutions still aim to proceed with allogeneic transplantation in eligible patients. This interview took place at the 52nd Annual Meeting of the EBMT in Madrid, Spain.

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Transcript

Yeah, thanks so much. Yeah, so in myelofibrosis, the only curative treatment we have available at the moment is an allogeneic stem cell transplant. However, that has a few limitations. One limitation is that it’s only available for younger and fit patients, which means that probably 10 to 20% of all patients with myelofibrosis will ever receive a transplant. But secondly, there is significant treatment-related toxicity that can reach 30 to 40%...

Yeah, thanks so much. Yeah, so in myelofibrosis, the only curative treatment we have available at the moment is an allogeneic stem cell transplant. However, that has a few limitations. One limitation is that it’s only available for younger and fit patients, which means that probably 10 to 20% of all patients with myelofibrosis will ever receive a transplant. But secondly, there is significant treatment-related toxicity that can reach 30 to 40%. Now, the problem also is that there have been a few… well, it’s not a problem…the opportunity for myelofibrosis, there have been quite a few novel agents recently, specifically targeting the JAK-STAT pathway, like ruxolitinib, momelotinib, or fedratinib. Now, those agents are not curative, but can improve symptoms. Patients can be on them for years, potentially delaying the progression of the disease. However, the question is when do you take those patients into a transplant so we want to see how transplant centers manage those patients in the era of those novel agents and maybe some centers would delay a transplant or maybe the situations would have changed because of the use of these agents. 

So the first important conclusion we have described in our papers is that there is significant heterogeneity. Centers do things differently as expected. However, most centers would still proceed to a stem cell transplant. They will use those agents to manage the symptoms initially, but at the moment the patient is in a situation where they can go ahead with the transplant, they will do so. So that highlights that those agents are important in the management of myelofibrosis, but they won’t change the treatment algorithm as of yet, with the caveat that there is significant heterogeneity. I think there are even more agents coming for myelofibrosis, so this question, how do you sequence all these treatments, will become even more challenging. And obviously for patients, a transplant can seem like the goal in a way, because they want to cure their disease, but transplant carries many, many toxicities. So if we find treatments to delay the disease, to delay the progression, manage their symptoms, that could be a very valid alternative, but hopefully without hampering their chances of having a successful transplant.

 

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