Loading
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.
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.
Erdheim-Chester disease with a BRAF V600E mutation is treated first with a BRAF inhibitor; the first targeted approval in any histiocytosis.
Shares Erdheim-Chester disease, Vemurafenib.
Shares Erdheim-Chester disease, Vemurafenib.
Shares Erdheim-Chester disease, Vemurafenib.
Shares Erdheim-Chester disease, Vemurafenib.
Shares Erdheim-Chester disease, Vemurafenib.