Miyamoto Toshihiro, Kikushige Yoshikane, Yoshimoto And Goichi
Medicine and Biosystemic Science, Kyushu University Graduate School of Medical Science.
Rinsho Ketsueki. 2017;58(10):1884-1894. doi: 10.11406/rinketsu.58.1884.
Despite recent progress in diagnosis and leukemogenesis based on genomic landscapes in acute myelogenous leukemia (AML), advances in AML treatment lag behind. Over the past four decades, combination chemotherapy with anthracycline and cytarabine remains the standard induction therapy. Subsequent post-remission consolidation therapy stratifies patients into favorable-risk, intermediate-risk, and unfavorable-risk groups to assign post-remission therapies based on cytogenetic abnormalities and molecular mutations. Allogeneic stem-cell transplant decreases the risk of AML recurrence compared to standard chemotherapy, but it also raises the risk of serious complications. Recent large collections of matched genomic and clinical data may assist in selecting the best individualized therapy for each AML patient. Emerging evidence indicates that molecularly targeted therapies such as FLT3 and IDH inhibitors may be effective in distinct AML subtypes, providing further rationale for a personalized medicine approach. An umbrella trial, such as "BEAT AML Master Trial," designed to offer novel targeted therapy to AML patients based on their genetic characteristics, will be launching worldwide in the near future.
尽管近期在基于急性髓系白血病（AML）基因组图谱的诊断和白血病发生机制方面取得了进展，但AML治疗的进展仍较为滞后。在过去的四十年里，蒽环类药物和阿糖胞苷联合化疗仍然是标准的诱导治疗方法。随后的缓解后巩固治疗根据细胞遗传学异常和分子突变将患者分为低危、中危和高危组，以分配缓解后治疗方案。与标准化疗相比，异基因干细胞移植降低了AML复发的风险，但同时也增加了严重并发症的风险。近期大量匹配的基因组和临床数据可能有助于为每位AML患者选择最佳的个体化治疗方案。新出现的证据表明，如FLT3和IDH抑制剂等分子靶向疗法可能对不同的AML亚型有效，这为个性化医疗方法提供了进一步的理论依据。一项伞式试验，如“BEAT AML主试验”，旨在根据AML患者的基因特征为其提供新型靶向治疗，将于近期在全球范围内开展。
