Vemurafenib use in an infant for high-risk Langerhans cell histiocytosis
S Héritier, M Jehanne, G Leverger, JF Emile… - JAMA …, 2015 - jamanetwork.com
Methods| Our patient was an 8-month-old girl who was diagnosed as having BRAF V600E–
mutated LCH with skin, bone, gut, node, and spleen involvement and hematological
dysfunction (DAS, 5). After the failure of 2 vinblastine-steroid inductions followed by 1 course
of cladribine, her DAS was 10. Off-label treatment with the BRAF inhibitor vemurafenib was
started at an initial dose of 120 mg twice daily (33.8 mg/kg/d) for 60 days after written
informed consent was obtained from the parents. The tablets were split, crushed, and …
mutated LCH with skin, bone, gut, node, and spleen involvement and hematological
dysfunction (DAS, 5). After the failure of 2 vinblastine-steroid inductions followed by 1 course
of cladribine, her DAS was 10. Off-label treatment with the BRAF inhibitor vemurafenib was
started at an initial dose of 120 mg twice daily (33.8 mg/kg/d) for 60 days after written
informed consent was obtained from the parents. The tablets were split, crushed, and …