Stone Richard M
Adult Acute Leukemia Program, Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA, USA.
CA Cancer J Clin. 2002 Nov-Dec;52(6):363-71. doi: 10.3322/canjclin.52.6.363.
Acute myeloid leukemia (AML) in older adults is a biologically and clinically distinct entity. Based on analysis of cytogenetic and molecular data, it is known that leukemic cells in older patients are intrinsically resistant to standard chemotherapy. Due to comorbid disease and impaired bone marrow stem cell reserve, older adults tolerate myelosuppressive chemotherapy poorly, with a treatment-related mortality rate of 25 percent. About 35 percent of adults under age 40 are cured, but the complete remission rate (likelihood of temporary disease eradication) is 45 percent in those over age 60, considerably lower than the 75% rate among younger patients, and the possibility of long-term disease free survival is 20 percent in those achieving remission or less than 10 percent overall. Standard allogeneic bone marrow transplantation is too dangerous to be considered as a means to eradicate minimal residual disease after remission is obtained and myelointensive chemotherapy is not a beneficial post-remission strategy in this age cohort. These disappointing results call for more effective and less toxic therapeutic options. Advances in our understanding of the pathophysiology of AML and promising early clinical data suggest that the era of truly targeted therapy in this difficult disease may soon be a reality.
老年急性髓系白血病（AML）在生物学和临床上是一个独特的实体。基于细胞遗传学和分子数据的分析，已知老年患者的白血病细胞对标准化疗具有内在抗性。由于合并症和骨髓干细胞储备受损，老年人对骨髓抑制性化疗的耐受性较差，治疗相关死亡率为25%。40岁以下的成年人约有35%被治愈，但60岁以上患者的完全缓解率（暂时消除疾病的可能性）为45%，远低于年轻患者的75%，实现缓解的患者长期无病生存的可能性为20%，总体上低于10%。标准的异基因骨髓移植过于危险，不能被视为缓解后消除微小残留病的手段，而强化疗在这个年龄组中并不是一种有益的缓解后策略。这些令人失望的结果需要更有效且毒性更小的治疗选择。我们对AML病理生理学认识的进展以及有前景的早期临床数据表明，在这种难治性疾病中真正的靶向治疗时代可能很快成为现实。
