Garcia Jacqueline S, Stone Richard M
Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, 450 Brookline Avenue, Dana 2058, Boston, MA 02215-5450, USA.
Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, 450 Brookline Avenue, Dana 2058, Boston, MA 02215-5450, USA.
Hematol Oncol Clin North Am. 2017 Aug;31(4):663-680. doi: 10.1016/j.hoc.2017.03.002. Epub 2017 May 17.
FLT3 mutations, generally associated with a poor prognosis, are found in approximately one-third of patients with acute myeloid leukemia (AML) and represent an attractive therapeutic target. FLT3 inhibitors undergoing clinical evaluation include first-generation relatively non-specific small molecules and second-generation compounds with higher potency and selectivity against mutant FLT3. Recently presented results from a prospective randomized clinical trial will likely lead to a change in the standard of care for younger patients with FLT3-mutated AML: addition of the multi-targeted FLT3 inhibitor midostaurin to standard induction and consolidation chemotherapy. Thus, personalized therapies for this subset of AML will soon be possible.
FLT3突变通常与预后不良相关，约三分之一的急性髓系白血病（AML）患者存在该突变，它是一个有吸引力的治疗靶点。正在进行临床评估的FLT3抑制剂包括第一代相对非特异性的小分子抑制剂以及对突变型FLT3具有更高效力和选择性的第二代化合物。一项前瞻性随机临床试验最近公布的结果可能会改变FLT3突变型AML年轻患者的治疗标准：在标准诱导和巩固化疗中加入多靶点FLT3抑制剂米哚妥林。因此，针对这一亚组AML患者的个性化治疗将很快成为可能。
