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VJHemOnc Podcast
The treatment landscape and challenges in rare diseases: BPDCN, CMML, MPN-AP/BP, and VEXAS
Nov 6, 2024
Marina Konopleva is the Director of Leukemia at Montefiore Cancer Center, while Emma Groarke is an Associate Research Physician at the NIH. They delve into the landscape of rare myeloid diseases like BPDCN, CMML, and VEXAS, discussing innovative therapies including Tagrexofaspa and IDH inhibitors. The conversation highlights the complexities in treatment due to patient variability and the significance of risk stratification in decision-making. They also emphasize the importance of collaborative research to enhance outcomes in these challenging conditions.
22:39
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Quick takeaways
- The podcast discusses the promising advancements in targeted therapies for BPDCN, yielding a 70% response rate but limited overall survival.
- Various treatment challenges in CMML are highlighted, emphasizing the need for targeted therapies due to its genetic and clinical diversity.
Deep dives
Innovations in Treating BPDCN
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive disease with a low incidence rate. Recent advancements have led to the approval of targeted therapies like Tagraxofus and PIVAC, which target CD123, a receptor overexpressed in BPDCN cells. A significant study demonstrated a high response rate of around 70% in newly diagnosed patients, although overall survival was still limited to about 15 months. The goal remains to achieve remission and then transition patients to allogeneic stem cell transplantation, enhancing their chances of long-term survival.
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Discover
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Listen to the best highlights from the podcasts you love and dive into the full episode