Work

Long-term outcomes with single-agent BRAF inhibitor therapy in Erdheim-Chester disease

Year: 2025

Type: article

Abstract: ABSTRACT: Among 64 patients with Erdheim-Chester disease treated with a BRAF inhibitor (median follow-up 4 years), we found high response rates (85%) but frequent discontinuations (61%), primarily because of adverse events. Additionally, patients experienced poor health-related quality of life and high symptom burden.

Source: Blood

DOI: 10.1182/blood.2024028032

Authors: Gaurav Goyal, Anne S. Reiner, Dana Bossert, Allison M. Sigler, Mario E. Lacouture, Veronica Rotemberg, Jasmine H. Francis, Ronald S. Go, Raajit K. Rampal, Eli L. Diamond

Institutions: University of Alabama at Birmingham, UAB Medicine, Memorial Sloan Kettering Cancer Center, NYU Langone Health, New York University, Mayo Clinic

Language: English

Topic: Histiocytic Disorders and Treatments

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: green

Funder: National Cancer Institute

Awards: G4523948649, Cancer Center Support Grant

OpenAlex ID: W4408214707

FWCI: 4.249

Cites: 11

Cited by: 8

Related to: 10