Anthony Stein, MD, associate director, Gehr Family Center for Leukemia Research, and professor, Division of Leukemia, Department of Hematology & Hematopoietic Cell Transplantation, City of Hope discusses updated findings on the use of tagraxofusp (TAG) in younger patients diagnosed with blastic plasmacytoid dendritic cell neoplasm (BPDCN). TAG remains the only FDA-approved therapy for BPDCN and is increasingly being used as frontline treatment in transplant-eligible patients.
Dr. Stein reviews a subgroup analysis of 10 patients, aged 22 to 45, from a larger pivotal trial. Seven patients achieved a complete response following treatment with TAG, allowing all to proceed directly to hematopoietic stem cell transplant without the need for aggressive combination chemotherapy. The three patients who did not initially respond were subsequently treated with other regimens and also reached transplant.
Dr. Stein highlights that TAG demonstrated a favorable safety profile in this younger cohort. No patients developed capillary leak syndrome, and most toxicities occurred only during the first cycle. Importantly, treatment did not lead to increased risk of graft-versus-host disease or sinusoidal obstructive syndrome following transplant.
Avoiding multi-agent chemotherapy mitigated risks of severe cytopenia, infections, and treatment-related delays. According to Dr. Stein, these findings support a shift in the treatment paradigm, positioning TAG as the preferred initial therapy in eligible patients with BPDCN, especially those under age 50. TAG allows for efficient bridging to transplant with fewer complications, contributing to better long-term outcomes.
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