Targeted inhibition of the MAPK pathway: emerging salvage option for progressive life-threatening multisystem LCH

This is the authors' abstract. We don't add key points for this paper.

Key Points Single-agent vemurafenib leads to a rapid and sustained clinical response in severe multisystem LCH but does not eradicate the disease. Longitudinal assessment of BRAF V600E during treatment shows that clinical remission can occur despite significant amounts of mutated BRAF.