Optimizing capillary leak syndrome prevention and management in patients receiving tagraxofusp for blastic plasmacytoid dendritic cell neoplasm

Fecha de publicación: 01/01/2025

Autores de IIS La Fe

Pau Montesinos Fernández

Autor

Participantes ajenos a IIS La Fe

Lane A.A.
Papayannidis C.
Angelucci E.
McCloskey J.
Schiller G.J.
de la Fuente Burguera A.
Nagarwala Y.
Herling M.
Mannis G.
Pemmaraju N.

Grupos

Hematología y Hemoterapia

Abstract

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. © 2025 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.

Datos de la publicación

ISSN/ISSNe:: 1042-8194, 1029-2403
Tipo:: Review
Páginas:: 2569-2577
DOI:: 10.1080/10428194.2025.2557505
PubMed:: 41134616
Enlace a otro recurso:: www.scopus.com
Factor de Impacto:: 0,741 SCImago ℠
Cuartil:: Q2 SCImago ℠

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Filiaciones

Lane A.A.:: Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, United States
Papayannidis C.:: IRCCS Azienda Ospedaliero-Universitaria di Bologna, Istituto di Ematologia “Seràgnoli”, Bologna, Italy
Angelucci E.:: Hematology and Cellular Therapy Center, IRCCS Ospedale Policlinico San Martino, Genova, Italy
McCloskey J.:: Hackensack University Medical Center, Hackensack, NJ, United States
Schiller G.J.:: David Geffen School of Medicine, UCLA, Los Angeles, CA, United States
de la Fuente Burguera A.:: Hematology and Hemotherapy Department, MD Anderson Cancer Center Madrid Hospiten, Madrid, Spain
Montesinos P.:: Department of Hematology, Hospital Universitari i Politècnic La Fe, Valencia, Spain
Nagarwala Y.:: Menarini Group, New York, NY, United States
Herling M.:: Department of Hematology, Cellular Therapy and Hemostaseology, University of Leipzig and Cancer Center Central Germany (CCCG), Jena, Leipzig, Germany
Mannis G.:: Division of Hematology, Stanford University, Stanford, CA, United States
Pemmaraju N.:: Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX, United States

Keywords

Blastic Plasmacytoid Dendritic Cell Neoplasm; Capillary Leak Syndrome; Dendritic Cells; Disease Management; Hematologic Neoplasms; Humans; Recombinant Fusion Proteins; tagraxofusp; fusion protein; tagraxofusp; adverse drug reaction; blastic plasmacytoid dendritic cell neoplasm; body weight gain; boxed warning; capillary leak syndrome; early intervention; edema; hemodynamics; human; hypoalbuminemia; hypotension; male; patient selection; prevention; review; side effect; blastic plasmacytoid dendritic cell neoplasm; complication; dendritic cell; diagnosis; disease management; drug effect; drug therapy; etiology; hematologic disease; pathology; prevention and control

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