Optimizing capillary leak syndrome prevention and management in patients receiving tagraxofusp for blastic plasmacytoid dendritic cell neoplasm
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive, orphan hematologic malignancy characterized by cells expressing CD123 and other markers. The only drug approved for BPDCN is tagraxofusp, a first-in-class CD123-targeted therapy. Tagraxofusp has a boxed warning for capillary leak syndrome (CLS) in the US prescribing information. CLS symptoms include hypoalbuminemia, weight gain, edema, and in severe cases, hypotension and hemodynamic instability. In the tagraxofusp registrational trial, 21% of patients receiving 12 µg/kg/day developed CLS. Most events were grade 2; higher grade CLS events including 2% grade 3, 2% grade 4, and two deaths occurred. During tagraxofusp treatment, strict monitoring for early recognition of CLS symptoms and directed intervention is essential for managing CLS; with this approach CLS is typically mild and occurs mostly in cycle 1, without recurrence. We discuss patient selection and optimization, monitoring, and early intervention for successful identification and optimized management of CLS in real-world practice.
Authors
- Yasir M. Nagarwala
- Pau Montesinos (ORCID: https://orcid.org/0000-0002-3275-5593)
- James McCloskey
- Adolfo de la Fuente (ORCID: https://orcid.org/0000-0002-1324-5555)
- Marco Herling (ORCID: https://orcid.org/0000-0001-7473-7476)
- Gary J. Schiller (ORCID: https://orcid.org/0000-0002-7508-3467)
- Cristina Papayannidis (ORCID: https://orcid.org/0000-0001-8705-8333)
- Andrew A. Lane (ORCID: https://orcid.org/0000-0001-7380-0226)
- Emanuele Angelucci (ORCID: https://orcid.org/0000-0002-6512-6080)
- Gabriel N. Mannis
- Naveen Pemmaraju (ORCID: https://orcid.org/0000-0002-1670-6513)
Institutions
- Hackensack University Medical Center (US)
- The University of Texas MD Anderson Cancer Center (US)
- Harvard University (US)
- University of California, Los Angeles (US)
- Hospital Universitari i Politècnic La Fe (ES)
- Menarini Group (Italy) (IT)
- Dana-Farber Cancer Institute (US)
- Istituto di Ematologia di Bologna (IT)
- Ospedale Policlinico San Martino (IT)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- MD Anderson Cancer Center Madrid (ES)
- Leipzig University (DE)
- Stanford University (US)
Publication Details
- Journal
- Leukemia & lymphoma/Leukemia and lymphoma
- Published
- 2025-10-24
- DOI
- https://doi.org/10.1080/10428194.2025.2557505
- Primary Topic
- Muscle and Compartmental Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00