DOI: 10.1182/blood.2024028032 ISSN: 0006-4971
Long-term outcomes with single-agent BRAF inhibitor therapy in Erdheim-Chester disease
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 DiamondAmong 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 due to adverse events. Additionally, patients experienced persistently poor health-related quality of life.