Long-term outcomes with single-agent BRAF inhibitor therapy in Erdheim-Chester disease
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.
Authors
- Jasmine H. Francis (ORCID: https://orcid.org/0000-0002-7637-3108)
- Mario E. Lacouture (ORCID: https://orcid.org/0000-0002-4818-3710)
- Dana Bossert
- Ronald S. Go (ORCID: https://orcid.org/0000-0002-8284-3495)
- Anne S. Reiner (ORCID: https://orcid.org/0000-0002-1258-6112)
- Gaurav Goyal (ORCID: https://orcid.org/0000-0001-6148-5177)
- Allison Sigler (ORCID: https://orcid.org/0000-0002-1519-2119)
- Raajit K. Rampal (ORCID: https://orcid.org/0000-0002-0355-6368)
- Eli L. Diamond (ORCID: https://orcid.org/0000-0001-5456-5961)
- Veronica Rotemberg (ORCID: https://orcid.org/0000-0003-0639-2677)
Institutions
- Mayo Clinic (US)
- Memorial Sloan Kettering Cancer Center (US)
- WinnMed (US)
- University of Alabama at Birmingham (US)
Publication Details
- Journal
- Blood
- Published
- 2025-03-07
- DOI
- https://doi.org/10.1182/blood.2024028032
- Citations
- 4
- Primary Topic
- Histiocytic Disorders and Treatments
- Type
- article
- Field-Weighted Citation Impact
- 4.66