EHA 2026 | Advances in BPDCN research: novel agents, combination approaches & increased disease awareness

Turning our attention to blastic plasmacytoid dendritic cell neoplasm, or BPDCN, there is a lot of excitement in our field, and a lot of it is taking place internationally, and so I’m excited to be here in Stockholm for the EHA meeting. I would say there are three areas in the BPDCN field that have become quite exciting for our research area in this year of 2026. One is the new approval, so a brand new agent, second now in the field, pivekimab sunirine, or PVEK, another CD123 targeted drug, which sort of catapults on the attention that was drawn by tagraxofusp, which was the first CD123. So a second CD123 drug, now US FDA approved. This was published in JCO earlier this year by myself and my colleagues. And what it tells us is that even in a rare disease area, we are able to get not only drug approvals, but pioneer a new target, CD123. And the excitement there is, can that be brought to other areas beyond BPDCN, such as AML? So we’ll keep our eyes on that growing CD123 field. Number two in BPDCN is that of combinatorial therapy. These two approvals have been monotherapy. So single agent, broad approvals, either frontline or relapsed refractory, and they have revolutionized the field. But we’re still going for cures and we’re hoping to prevent relapse from ever happening so let’s keep our eye on those combinations the two approaches so far have been combining CD123 with HMA and BCL2 that can be for perhaps older unfit patients or all patients and then a second approach is combining CD123 with chemotherapy and BCL2 inhibition and let’s keep our eyes on that. And still a third development in our BPDCN field is identifying that it’s a bit more common than we thought. When we first entered the field, the teaching was that it was older male predominance skin disease, and that certainly is the prototype classical presentation. But we’re starting to see now with more diagnostics, education females younger patients pediatric adolescent young adults those without skin involvement, CNS involvement so there are so many more ways to diagnose BPDCN in this multi-compartmental disease and so it emphasizes programs such as this one and others that we need to continue to educate the new generation of doctors, including dermatologists, so skin doctors, pathologists, stem cell transplant doctors, oncologists, hematologists. It’s a disease that affects quite a few compartments. And so I’d like to just say in this program that this is why awareness, education, and dissemination of information is so important.

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