EBMT 2023 | Frontline treatment in BPDCN & the best route to allogeneic transplantation
Marco Herling, MD, University of Leipzig, Leipzig, Germany, discusses how to select a frontline treatment for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), commenting on the best strategy to get a patient to allogeneic transplantation. Dr Herling explains that it is first important to determine whether a patient is fit to receive systemic therapy, and argues that milder regimens like single-agent tagraxofusp might be preferable prior to allogeneic transplantation. 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)
The answer in a patient population that is on average of a median age, 67 to 70 years old, it’s the question how do I get a patient actually towards systemic treatment? These patients are often frail and have disease burden and therefore the first question that a clinical treating physician has to answer is, is my patient fit enough for systemic treatment? And if so, and we are talking about 80% of these patients, then you apply the systemic treatment...
The answer in a patient population that is on average of a median age, 67 to 70 years old, it’s the question how do I get a patient actually towards systemic treatment? These patients are often frail and have disease burden and therefore the first question that a clinical treating physician has to answer is, is my patient fit enough for systemic treatment? And if so, and we are talking about 80% of these patients, then you apply the systemic treatment. The disease is to be understood as an acute leukemia, therefore is systemic and not the local skin restricted treatment is advised and there historically, we have used protocols that range from lymphoma, acute myeloid leukemia, or ALL protocols. It turns out that these ALL type protocols have been until recently, the most efficient. Since 2018 in the US, and since 2021 in the EU, we have a new approved and the first and only approved substance in BPDCN and that is called tagraxofusp (elzonris) and it is at least equally as effective and you need to consider this as the standard of care in these patients. You might want to call it a double standard because you have both therapies available, and you need to consider what is your patient able to handle. The remission rates according to the historic data, retrospective analysis, are about 90% in very intensive ALL-like protocols, very toxic. But you get about half of your patients in this first CR and get them to allogeneic transplantation. Whereas we have prospective data for tagraxofusp in 60/70 patients and this indicates that you accomplish a CR in an older population of about 50 to 60% and you get about the same amount of patients to allogeneic transplantation. Now, as a transplanter, one wonders what is the better way to get to an allogeneic transplantation with a means with a strategy that is toxic, has a cumulative toxicity with the dropout rate of about 15 to 20% of patients not getting there due to chemo-toxicity or with the regimen, mono agent, single agent tagraxofusp is much less toxic, has new toxicities that people had to get used to. But I would argue that it matters how to get to the allogeneic stem cell transplantation, preferably with the milder regimen.
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