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

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

doi:10.1182/blood.2024028032

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 due to adverse events. Additionally, patients experienced persistently poor health-related quality of life.

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2025

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

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

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