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.

ABSTRACT:

Importance

The histiocytic neoplasms Erdheim-Chester disease (ECD) and Langerhans cell histiocytosis (LCH) are highly enriched for BRAF V600 mutations and have been previously shown to be responsive to treatment with vemurafenib, an inhibitor of the BRAF V600 kinase. However, the long-term efficacy and safety of prolonged vemurafenib use in these patients are not defined. Here we analyze the final efficacy and safety data for vemurafenib in patients with ECD and LCH enrolled in the VE-BASKET study.

Objective

To determine the efficacy and safety of vemurafenib in adults with ECD or LCH enrolled in the VE-BASKET study.

Design, setting, and participants

The VE-BASKET study was an open-label, nonrandomized, multicohort study for patients with nonmelanoma cancers harboring

SUBMITTER: Diamond EL 

PROVIDER: S-EPMC5844839 | biostudies-literature | 2018 Mar

REPOSITORIES: biostudies-literature