Division of Haematology and Stem Cell Transplantation Unit, Naples, Italy.
Lancet. 2013 Feb 9;381(9865):484-95. doi: 10.1016/S0140-6736(12)61727-9.
The outlook for patients with acute myeloid leukaemia has improved in the past 30 years. Unlike other cancers, much of this progress is attributable to refinement of supportive treatment, rather than the introduction of new drugs. New antibacterial and antifungal agents, antiemetics, and improved transfusion support have decreased the rate of early death, and morbidity and mortality from allogeneic stem cell transplantation has been substantially reduced. However, more than half of young adult patients and about 90% of older patients still die from their disease. Refractoriness to initial induction treatment and, more frequently, relapse after complete remission, are still the main obstacles to cure. Accordingly, new treatment approaches with mechanisms of action different from those of conventional chemotherapy are needed. Our knowledge of the various chromosomal and molecular abnormalities implicated in the pathogenesis of the many subtypes of the disease has greatly expanded; as a result, clinical research is moving towards the investigation of new non-cytotoxic agents in combination with chemotherapy. The goal is to target the molecular abnormalities identified at diagnosis; however, several aberrations can coexist in subclones of acute myeloid leukaemia, making the disease less likely to be inhibited by a single agent.
在过去的 30 年中，急性髓系白血病患者的预后有所改善。与其他癌症不同，这一进展在很大程度上归因于支持性治疗的精细化，而不是新药的引入。新型抗菌和抗真菌药物、止吐药以及更好的输血支持降低了早期死亡率，异基因造血干细胞移植的发病率和死亡率也大幅降低。然而，超过一半的年轻成年患者和约 90%的老年患者仍死于该病。对初始诱导治疗的耐药性，以及更常见的完全缓解后复发，仍然是治愈的主要障碍。因此，需要采用与传统化疗不同作用机制的新治疗方法。我们对导致该病多种亚型发生的各种染色体和分子异常的认识已经大大扩展；因此，临床研究正在转向联合化疗研究新型非细胞毒性药物。目标是针对诊断时确定的分子异常；然而，急性髓系白血病的亚克隆中可能同时存在几种异常，使得单一药物更难以抑制疾病。
