title: BRAF inhibitor therapy in HCL.
abstract: Targeted treatment approaches are transforming the therapeutic landscape of cancer care. The discovery of the BRAF V600E mutation in most cases of classical hairy cell leukemia opens up unique opportunities for tumor specific treatment of HCL targeting the MEK/ERK signaling pathway. The discovery and biological implications of BRAF V600E in HCL are summarized to form a basis for our current understanding of the potential for clinical exploitation. There is overwhelming clinical evidence for activity of inhibitors of BRAF in the disease. The review will review current trial activity as well as discuss novel trial concepts exploiting targeted treatment focusing on BRAF inhibition in HCL.
authors.affiliation: Department of Translational Oncology, National Center for Tumor Diseases (NCT) and German Cancer Research Center (DKFZ), Im Neuenheimer Feld 460, 69120 Heidelberg, Germany; Department of Medicine V, Heidelberg University Medical Center, Heidelberg, Germany; Genome Biology Unit, European Molecular Biology Laboratory, Heidelberg, Germany.
authors.forename: Sascha
authors.initials: S
authors.lastname: Dietrich
authors.affiliation: Department of Translational Oncology, National Center for Tumor Diseases (NCT) and German Cancer Research Center (DKFZ), Im Neuenheimer Feld 460, 69120 Heidelberg, Germany; Department of Medicine V, Heidelberg University Medical Center, Heidelberg, Germany. Electronic address: thorsten.zenz@nct-heidelberg.de.
authors.forename: Thorsten
authors.initials: T
authors.lastname: Zenz
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D015973:Gene Expression Regulation, Leukemic; D006801:Humans; D007093:Imidazoles / Q000627:therapeutic use*; D007211:Indoles / Q000627:therapeutic use*; D007943:Leukemia, Hairy Cell / Q000188:drug therapy* / Q000235:genetics / Q000401:mortality / Q000473:pathology; D020935:MAP Kinase Signaling System / Q000187:drug effects; D058990:Molecular Targeted Therapy; D009154:Mutation; D018365:Neoplasm, Residual; D010091:Oximes / Q000627:therapeutic use*; D048493:Proto-Oncogene Proteins B-raf / Q000037:antagonists & inhibitors* / Q000235:genetics / Q000378:metabolism; D012074:Remission Induction; D013449:Sulfonamides / Q000627:therapeutic use*; D016019:Survival Analysis; D000077484:Vemurafenib
chemical_list: D000970:Antineoplastic Agents; D007093:Imidazoles; D007211:Indoles; D010091:Oximes; D013449:Sulfonamides; D000077484:Vemurafenib; C482119:BRAF protein, human; D048493:Proto-Oncogene Proteins B-raf; C561627:dabrafenib
keywords: BRAF V600E; BRAF inhibitor; Dabrafenib; Hairy cell leukemia; MEK; Vemurafenib