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. 2025 May 1;145(18):2100-2103.
doi: 10.1182/blood.2024028032.

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

Gaurav Goyal  1 , Anne S Reiner  2 , Dana Bossert  3 , Allison M Sigler  3 , Mario E Lacouture  4 , Veronica Rotemberg  4 , Jasmine H Francis  5 , Ronald S Go  6 , Raajit K Rampal  7 , Eli L Diamond  3
Affiliations

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

Gaurav Goyal et al. Blood. .

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.

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Conflict of interest statement

Conflict-of-interest disclosure: G.G. has served on an advisory board for Opna Bio, Seagen, and Sobi and received consulting fees from Recordati. E.L.D. discloses unpaid editorial support from Pfizer Inc and has served on an advisory board for Opna Bio, both outside the submitted work. The remaining authors declare no competing financial interests.

References

    1. Goyal G, Heaney ML, Collin M, et al. Erdheim-Chester disease: consensus recommendations for evaluation, diagnosis, and treatment in the molecular era. Blood. 2020;135(22):1929–1945. - PubMed
    1. Haroche J, Charlotte F, Arnaud L, et al. High prevalence of BRAF V600E mutations in Erdheim-Chester disease but not in other non-Langerhans cell histiocytoses. Blood. 2012;120(13):2700–2703. - PubMed
    1. Diamond EL, Subbiah V, Lockhart AC, et al. Vemurafenib for BRAF V600-mutant Erdheim-Chester disease and Langerhans cell histiocytosis: analysis of data from the histology-independent, phase 2, open-label VE-BASKET study. JAMA Oncol. 2018;4(3):384–388. - PMC - PubMed
    1. Reiner AS, Durham BH, Yabe M, et al. Outcomes after interruption of targeted therapy in patients with histiocytic neoplasms. Br J Haematol. 2023;203(3):389–394. - PMC - PubMed
    1. Goyal G, Shah MV, Call TG, Litzow MR, Hogan WJ, Go RS. Clinical and radiologic responses to cladribine for the treatment of Erdheim-Chester disease. JAMA Oncol. 2017;3(9):1253–1256. - PMC - PubMed