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Targeted inhibition of the MAPK pathway: emerging salvage option for progressive life-threatening multisystem LCH
Alexandra Kolenová1,2, Raphaela Schwentner3, Gunhild Jug3
1Department of Pediatric Hematology and Oncology, Comenius University Medical School, Bratislava, Slovakia.
Blood Advances
|January 4, 2018
Abstract:
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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