This episode was funded by Stemline Therapeutics, Inc., a Menarini Company Group, and is intended for United States healthcare professionals only.
The opinions expressed here are solely those of the named speaker and do not represent those of his affiliated institution. His participation in this discussion does not constitute, and should not be construed as, endorsement, recommendation, or promotion of any product, service, or organization.
In this podcast, we shine a light on blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and highly aggressive hematologic malignancy.
Before 2018, there were no approved treatments for BPDCN, and oncologists typically used chemotherapy regimens commonly used for acute leukemias and lymphomas that were associated with limited durability and high rates of induction mortality.
The landscape changed when the FDA approved tagraxofusp, a CD123-directed fusion protein, as the first therapy approved for adult and pediatric patients 2 years and older with BPDCN.
Listen to Thomas LeBlanc explore the safety and efficacy of tagraxofusp from STML-401-0114 and real-world evidence, as well as how he approaches optimizing clinical management and interdisciplinary care.
Topics covered:
- How BPDCN presents and what steps are needed to make a correct diagnosis
- The safety and efficacy outcomes from STML-401-0114, including patient subgroups based on patients’ age, fitness, or baseline skin burden
- How real-world evidence complements the findings from the pivotal STML-401-0114 study
- How the ‘Prepare, Look, Apply, and Next Steps’ (PLAN) management framework can help your team monitor and manage adverse events
Speaker
Thomas LeBlanc1
1. Division of Hematologic Malignancies and Cellular Therapy, Duke University School of Medicine, Durham, North Carolina, USA
INDICATION
- Tagraoxfusp is a CD123-directed cytotoxin indicated for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients 2 years and older
IMPORTANT SAFETY INFORMATION
Boxed WARNING: CAPILLARY LEAK SYNDROME
- Capillary Leak Syndrome (CLS), which may be life-threatening or fatal, can occur in patients receiving tagraxofusp. Monitor for signs and symptoms of CLS and take actions as recommended.
WARNINGS AND PRECAUTIONS
Capillary Leak Syndrome
- Capillary leak syndrome (CLS), including life-threatening and fatal cases, has been reported among patients treated with tagraxofusp. In patients receiving tagraxofusp in clinical trials, the overall incidence of CLS was 53% (65/122), including Grade 1 or 2 in 43% (52/122) of patients, Grade 3 in 7% (8/122) of patients, Grade 4 in 1% (1/122) of patients, and four fatalities (3%). The median time to onset was 4 days (range-1 to 46 days), and all but 5 patients experienced an event in Cycle 1.
- Before initiating therapy with tagraxofusp, ensure that the patient has adequate cardiac function and serum albumin is greater than or equal to 3.2 g/dL. During treatment with tagraxofusp, monitor serum albumin levels prior to the initiation of each dose of tagraxofusp and as indicated clinically thereafter, and assess patients for other signs or symptoms of CLS, including weight gain, new onset or worsening edema, including pulmonary edema, hypotension or hemodynamic instability.
Hypersensitivity Reactions
- Tagraxofusp can cause severe hypersensitivity reactions. In patients receiving tagraxofusp in clinical trials, hypersensitivity reactions were reported in 43% (53/122) of patients treated with tagraxofusp and were Grade ≥ 3 in 7% (9/122). Manifestations of hypersensitivity reported in ≥ 5% of patients include rash, pruritus, and stomatitis. Monitor patients for hypersensitivity reactions during treatment with tagraxofusp. Interrupt tagraxofusp infusion and provide supportive care as needed if a hypersensitivity reaction should occur.
Hepatotoxicity
- Treatment with tagraxofusp was associated with elevations in liver enzymes. In patients receiving tagraxofuspin clinical trials, elevations in ALT occurred in 79% (96/122) and elevations in AST occurred in 76% (93/122). Grade 3 ALT elevations were reported in 26% (32/122) of patients. Grade 3 AST elevations were reported in 30% (36/122) and Grade 4 AST elevations were reported in 3% (4/122) of patients. Elevated liver enzymes occurred in the majority of patients in Cycle 1 and were reversible following dose interruption.
- Monitor alanine aminotransferase (ALT) and aspartate aminotransferase (AST) prior to each infusion with tagraxofusp. Withhold tagraxofusp temporarily if the transaminases rise to greater than 5 times the upper limit of normal and resume treatment upon normalization or when resolved.
ADVERSE REACTIONS:
Most common adverse reactions (incidence ≥ 30%) are capillary leak syndrome, nausea, fatigue, pyrexia, peripheral edema, and weight increase. Most common laboratory abnormalities (incidence ≥ 50%) are decreases in albumin, platelets, hemoglobin, calcium, and sodium, and increases in glucose, ALT and AST.
Please see Full Prescribing Information, including Boxed WARNING.
To report SUSPECTED ADVERSE REACTIONS, contact Stemline Therapeutics, Inc. at 1-877-332-7961 or contact the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
MED-05440 / © 2026 Stemline Therapeutics, Inc. All rights reserved. 07/2026
