Summary

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• Tagraxofusp-erzs (Elzonris) is indicated for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and pediatric patients aged 2 years and older. It targets CD123+ cells characteristic of BPDCN, providing a more tailored approach to managing this aggressive disease.
• Two studies focused on Elzonris' application in managing BPDCN emphasized its role as a landmark development due to its targeted nature and precision oncology approach.
• Before the introduction of Elzonris, management of BPDCN mainly involved remission induction therapy followed by stem cell transplant, a process often hampered by the difficulty in achieving sufficient remission with conventional chemotherapy. Elzonris, in contrast, presents a targeted therapy option specifically for this condition.
• Despite BPDCN occurring most frequently in patients over 60, it also affects children and adults of various ages. The introduction of Elzonris has been significant across all affected age groups, considering the historically complex diagnosis process, which often overlaps with other hematologic malignancies.
• Even though Elzonris is associated with a unique toxicity profile, predominately capillary leak syndrome (CLS), these adverse events can be managed with adequate patient selection and monitoring, making it an important option in the treatment landscape despite potential risks.
• Detailed clinical case studies have led to approval, demonstrating effectiveness against CD123+ cells. This marks a progression towards precision oncology, addressing unmet needs within this patient population without direct comparison to other specific drugs in controlled trials being available from the studies reviewed.