Although patients with ECD and LCH frequently have BRAFV600 mutations and respond to BRAFV600 inhibitor therapy, there was previously a lack of data describing long-term efficacy and safety.
Indications for vemurafenib are expanded to include BRAF V600 mutation-positive Erdheim-Chester disease, an extremely rare non-Langerhans cell histiocytosis. FDA approval is based on results from the phase 2 VE-BASKET study.
This is the first-ever drug approval for the blood cancer, which, although rare, carries a poor prognosis.
A rare case of Langerhans cell histiocytosis (LCH) in a patient with idiopathic thrombocytopenic purpura (ITP) highlights the difficulty in diagnosing LCH.
BRAF other MAPK pathway gene mutations associated with Erdheim-Chester disease reveal that ECD is a neoplastic disease.
