Long-term outcomes with single-agent BRAF inhibitor therapy in Erdheim-Chester disease
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.
Article Details
Authors (10)
Gaurav Goyal
1Division of Hematology and Oncology, University of Alabama at Birmingham, Birmingham, AL
Anne S. Reiner
2Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY
Dana Bossert
3Department of neurology, New York, United States
Allison M. Sigler
3Department of Neurology, Memorial Sloan Kettering Cancer Center, New York, NY
Mario E. Lacouture
4Dermatology Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY
Veronica Rotemberg
4Dermatology Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY
Jasmine H. Francis
Memorial Sloan Kettering Cancer Center, New York
Ronald S. Go
9Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN
Raajit K. Rampal
11Leukemia Service, Memorial Sloan Kettering Cancer Center, New York, NY
Eli L. Diamond
4Department of Neurology, Memorial Sloan Kettering Cancer Center, New York, NY