VE-BASKET: vemurafenib for BRAF V600-mutant Erdheim-Chester disease and Langerhans cell histiocytosis
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.
Overview
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.
- 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.
Erdheim-Chester disease with a BRAF V600E mutation is treated first with a BRAF inhibitor; the first targeted approval in any histiocytosis.
- 26 patients, single-arm.
- Disease usually returns when the drug is stopped, so treatment is long term.
Similar pages
not linked directly; found by shared links- Key paperErdheim-Chester disease: consensus recommendations for evaluation, diagnosis and treatment in the molecular era
Shares Erdheim-Chester disease, Vemurafenib.
- TermBRAF V600E mutation
Shares Erdheim-Chester disease, Vemurafenib.
- CancerMultisystem Langerhans cell histiocytosis (with or without risk-organ involvement)
Shares Erdheim-Chester disease, Vemurafenib.
- CancerErdheim-Chester disease, Rosai-Dorfman disease and other histiocytic neoplasms
Shares Erdheim-Chester disease, Vemurafenib.
- TreatmentCobimetinib
Shares Erdheim-Chester disease, Vemurafenib.