Anthony Stein, MD, co-director of the Leukemia Program at City of Hope, Duarte, California, discusses outcomes from a study evaluating tagraxofusp (TAG) as frontline therapy for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). The conversation highlights shifts in the treatment landscape and implications for stem cell transplant eligibility, particularly in patients aged younger than 50 years.
Prior to the approval of tagraxofusp, BPDCN was managed with intensive, multi-agent chemotherapy regimens, which were associated with substantial toxicity. These adverse effects frequently prevented patients from proceeding to potentially curative stem cell transplant.
Dr. Stein explains that TAG has significantly altered the treatment approach. In the study cohort, TAG was associated with a favorable safety profile. No patients experienced capillary leak syndrome, and toxicities were largely limited to the first cycle of treatment. The majority of patients required minimal transfusional support, and no deaths occurred due to treatment-related toxicity.
Importantly, the use of TAG as a bridge to transplant was not associated with increased incidence of graft-versus-host disease or sinusoidal obstructive syndrome—adverse events that are occasionally observed with other therapies. According to Dr. Stein, these findings support the use of TAG as the standard initial treatment for eligible patients with BPDCN, allowing for safer transition to transplant.
Given the aggressive nature of BPDCN and the limited clinical data available for younger populations, the study contributes valuable insight into treatment optimization. TAG may reduce the need for cytotoxic chemotherapy and its associated complications, such as profound cytopenias and infectious risk, thereby improving transplant eligibility and outcomes in this patient population.