# VE-BASKET: vemurafenib for BRAF V600-mutant Erdheim-Chester disease and Langerhans cell histiocytosis

Source: https://onco.cc/key-papers/paper-ve-basket-vemurafenib-erdheim-chester-lch-jama-oncol-2018/  
OnCo record `paper-ve-basket-vemurafenib-erdheim-chester-lch-jama-oncol-2018` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

The 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.

## Fields

- Kind: Key paper
- Last checked: 2026-09-18
- Journal: JAMA Oncology
- Year: 2018
- DOI: 10.1001/jamaoncol.2017.5029
- Authors: Diamond EL, Subbiah V, Lockhart AC, et al.
- 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.
- What it means: 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.

## Sources

- JAMA Oncol 2018: https://doi.org/10.1001/jamaoncol.2017.5029
- PubMed: https://pubmed.ncbi.nlm.nih.gov/29188284/

## Connected records

- cancers: [Erdheim-Chester disease](https://onco.cc/cancers/erdheim-chester-disease/)
- drugs: [Vemurafenib](https://onco.cc/drugs/vemurafenib/)
- journals: [JAMA Oncology](https://onco.cc/journals/jama-oncology/)

---
JSON: https://onco.cc/api/v1/entities/paper-ve-basket-vemurafenib-erdheim-chester-lch-jama-oncol-2018.json