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    PURPOSE Off-label use of vemurafenib (VMF) to treat BRAFV600E mutation–positive, refractory, childhood Langerhans cell histiocytosis (LCH) was evaluated. PATIENTS AND METHODS Fifty-four patients from 12 countries took VMF 20 mg/kg/d. They were classified according to risk organ involvement: liver, spleen, and/or blood cytopenia. The main evaluation criteria were adverse events (Common Terminology…

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    • #Vemurafenib for #Refractory Multisystem Langerhans Cell Histiocytosis #LCH in Children: An International Observational Study 38/54 achieved CR The response to VMF persisted as long as the patient remained on treatment 24/30 relapsed after stopping https://t.co/gNskE5HDrW

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    JNCCN 360 - 4 year(s) ago

    By: Lauren Harrison, MS Posted: Tuesday, May 26, 2020 In cancers with BRAF V600 mutations, single-agent treatment BRAF inhibition with vemurafenib may prove to have broader clinical activity than was previously recognized, with responses reported in 13 different types of cancer. Vivek Subbiah, MD, of The…

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    • #Vemurafenib is effective in 13 cancer types, including #thyroidcancer: https://t.co/B9BNkKNXNg #thycsm #JNCCN360 https://t.co/OWr6FZ21Hh