Dietrich Sascha, Zenz Thorsten
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.
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.
Best Pract Res Clin Haematol. 2015 Dec;28(4):246-52. doi: 10.1016/j.beha.2015.10.001. Epub 2015 Oct 20.
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.
靶向治疗方法正在改变癌症治疗的格局。大多数经典毛细胞白血病病例中BRAF V600E突变的发现为靶向MEK/ERK信号通路的毛细胞白血病肿瘤特异性治疗带来了独特机遇。总结BRAF V600E在毛细胞白血病中的发现及其生物学意义，为我们目前对临床应用潜力的理解奠定基础。有大量临床证据表明BRAF抑制剂在该疾病中具有活性。本综述将回顾当前的试验活动，并讨论利用靶向治疗的新试验概念，重点是毛细胞白血病中的BRAF抑制。
