ISAL 2025 | Ongoing research and unmet needs in BPDCN
Cristina Papayannidis, MD, PhD, University of Bologna, Bologna, Italy, discusses ongoing research and unmet needs in blastic plasmacytoid dendritic cell neoplasm (BPDCN). Dr Papayannidis highlights the promising results of the approved drug tagraxofusp, and ongoing research investigating its combination. She emphasizes the importance of multidisciplinary teams and better diagnostic tools to address the significant challenges in managing this rare disease. This interview took place at the 19th International Symposium on Acute Leukemias (ISAL XIX) in Munich, Germany.
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Transcript
BPDCN is a very rare disease. The most difficult topic is to diagnose this neoplasia because the clinical presentation and also the multidisciplinary involvement which is required is still a challenge. But the good news is that at the moment we have a new drug which has been approved for the first-line treatment in these patients which is tagraxofusp which is an anti-CD123 antibody and the rate of response with tag published in the registrative trial in the New England Journal of Medicine is very very promising...
BPDCN is a very rare disease. The most difficult topic is to diagnose this neoplasia because the clinical presentation and also the multidisciplinary involvement which is required is still a challenge. But the good news is that at the moment we have a new drug which has been approved for the first-line treatment in these patients which is tagraxofusp which is an anti-CD123 antibody and the rate of response with tag published in the registrative trial in the New England Journal of Medicine is very very promising. I think that for the future this cannot be considered enough we need to combine this drug with other compounds there are clinical trials ongoing trying to put together tag with the hypomethylating agents, venetoclax and other molecules in order to improve the outcome. The biggest unmet need is the diagnosis. It’s very, very difficult to find these patients and perhaps when we say that this is a rare disease, we are underestimating the real incidence of BPDCN. The other point is the need for creating multidisciplinary teams because the dermatologist alone is not able to make the right diagnosis. It’s necessary to work together with hematologists. The role of the pathologist and the expert in flow is relevant. But in the real clinical daily practice, it’s not so easy to have so many healthcare professionals devoted to such a rare disease. So I think this is the main point.
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