{"entity":{"id":"paper-ve-basket-vemurafenib-erdheim-chester-lch-jama-oncol-2018","kind":"paper","name":"VE-BASKET: vemurafenib for BRAF V600-mutant Erdheim-Chester disease and Langerhans cell histiocytosis","aka":[],"tldr":"The BRAF inhibitor vemurafenib shrank disease in most adults with BRAF-mutant Erdheim-Chester disease, and the responses lasted, leading to the first drug approval for this histiocytosis.","summary":"Analysis of the histiocytosis cohort of the histology-independent phase 2 VE-BASKET study: 26 patients (22 with Erdheim-Chester disease, 4 with Langerhans cell histiocytosis) with BRAF V600 mutations treated with vemurafenib.\n\nThe objective response rate was 61.5 percent, no patient progressed on treatment and median progression-free survival was not reached after about two years of follow-up; skin toxicity, arthralgia and secondary skin cancers were common. The FDA approved vemurafenib for Erdheim-Chester disease in 2017 on these data.","asOf":"2026-09-18","links":[{"label":"JAMA Oncol 2018","url":"https://doi.org/10.1001/jamaoncol.2017.5029"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/29188284/"}],"tags":[],"related":[],"cancers":["erdheim-chester-disease"],"sections":[],"technologies":[],"targets":[],"drugs":["vemurafenib"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["jama-oncology"],"dependsOn":[],"notes":[],"journal":"JAMA Oncology","year":2018,"doi":"10.1001/jamaoncol.2017.5029","pmid":"29188284","authors":"Diamond EL, Subbiah V, Lockhart AC, et al.","paperType":"observational","findings":["Objective response 61.5 percent; no progression on treatment at a median follow-up of about two years.","Frequent skin toxicity and secondary cutaneous squamous cell carcinomas."],"whatItMeans":"Erdheim-Chester disease with a BRAF V600E mutation is treated first with a BRAF inhibitor; the first targeted approval in any histiocytosis.","caveats":["26 patients, single-arm.","Disease usually returns when the drug is stopped, so treatment is long term."],"changedPractice":true,"participants":26},"route":"/key-papers/paper-ve-basket-vemurafenib-erdheim-chester-lch-jama-oncol-2018/","neighbours":{"cancer":[{"id":"erdheim-chester-disease","kind":"cancer","name":"Erdheim-Chester disease","route":"/cancers/erdheim-chester-disease/"}],"drug":[{"id":"vemurafenib","kind":"drug","name":"Vemurafenib","route":"/drugs/vemurafenib/"}],"journal":[{"id":"jama-oncology","kind":"journal","name":"JAMA Oncology","route":"/journals/jama-oncology/"}]}}