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Vemurafenib for Refractory Multisystem Langerhans Cell Histiocytosis in Children: An International Observational Study

PURPOSE: Off-label use of vemurafenib (VMF) to treat BRAF(V600E) 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: live...

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Detalles Bibliográficos
Autores principales: Donadieu, Jean, Larabi, Islam Amine, Tardieu, Mathilde, Visser, Johannes, Hutter, Caroline, Sieni, Elena, Kabbara, Nabil, Barkaoui, Mohamed, Miron, Jean, Chalard, François, Milne, Paul, Haroche, Julien, Cohen, Fleur, Hélias-Rodzewicz, Zofia, Simon, Nicolas, Jehanne, Mathilde, Kolenova, Alexandra, Pagnier, Anne, Aladjidi, Nathalie, Schneider, Pascale, Plat, Geneviève, Lutun, Anne, Sonntagbauer, Anne, Lehrnbecher, Thomas, Ferster, Alina, Efremova, Viktoria, Ahlmann, Martina, Blanc, Laurence, Nicholson, James, Lambilliote, Anne, Boudiaf, Houda, Lissat, Andrej, Svojgr, Karel, Bernard, Fanette, Elitzur, Sarah, Golan, Michal, Evseev, Dmitriy, Maschan, Michael, Idbaih, Ahmed, Slater, Olga, Minkov, Milen, Taly, Valerie, Collin, Matthew, Alvarez, Jean-Claude, Emile, Jean-François, Héritier, Sébastien
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Clinical Oncology 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6823889/
https://www.ncbi.nlm.nih.gov/pubmed/31513482
http://dx.doi.org/10.1200/JCO.19.00456