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Targeted inhibition of the MAPK pathway: emerging salvage option for progressive life-threatening multisystem LCH.
Blood Advances 2017 Februrary 15
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.
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