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

  


Authors: Goyal, G.; Reiner, A. S.; Bossert, D.; Sigler, A. M.; Lacouture, M. E.; Rotemberg, V.; Francis, J. H.; Go, R. S.; Rampal, R. K.; Diamond, E. L.
Title: Long-term outcomes with single-agent BRAF inhibitor therapy in Erdheim-Chester disease
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. © 2025 American Society of Hematology
Keywords: immunohistochemistry; adult; controlled study; event free survival; aged; major clinical study; overall survival; fatigue; drug dose reduction; drug withdrawal; side effect; skin toxicity; comparative study; positron emission tomography; follow up; low drug dose; progression free survival; prevalence; retrospective study; central nervous system; arthralgia; death; fluorodeoxyglucose f 18; b raf kinase; disease exacerbation; photosensitivity; musculoskeletal disease; hair loss; molecularly targeted therapy; functional assessment of cancer therapy; abnormal laboratory result; clinical outcome; overall response rate; vemurafenib; mitogen activated protein kinase kinase inhibitor; dabrafenib; long term survival; high throughput sequencing; human; male; female; article; erdheim chester disease; functional assessment of cancer therapy general; physical well-being; emotional well-being
Journal Title: Blood
Volume: 145
Issue: 18
ISSN: 0006-4971
Publisher: American Society of Hematology  
Date Published: 2025-05-01
Start Page: 2100
End Page: 2103
Language: English
DOI: 10.1182/blood.2024028032
PUBMED: 40053897
PROVIDER: scopus
DOI/URL:

https://www.scopus.com/inward/record.uri?eid=2-s2.0-105001137796&doi=10.1182%2fblood.2024028032&partnerID=40&md5=692190c9feed7b97698fae5ca3e8cdea
Notes: Article -- MSK Cancer Center Support Grant (P30 CA008748) acknowledged in PDF -- MSK corresponding author is Eli Diamond -- Source: Scopus
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MSK Authors
  1. Anne S Reiner
    248 Reiner
  2. Mario E Lacouture
    457 Lacouture
  3. Jasmine Helen Francis
    256 Francis
  4. Raajit Kumar Rampal
    338 Rampal
  5. Dana F Bossert
    13 Bossert
  6. Eli Louis Diamond
    202 Diamond
  7. Veronica Miriam Rotemberg
    92 Rotemberg
  8. Allison Marie Sigler
    36 Sigler
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