Thompson Philip A, Kantarjian Hagop M, Cortes Jorge E
Department of Leukemia, The University of Texas, MD Anderson Cancer Center, Houston.
Department of Leukemia, The University of Texas, MD Anderson Cancer Center, Houston.
Mayo Clin Proc. 2015 Oct;90(10):1440-54. doi: 10.1016/j.mayocp.2015.08.010.
Few neoplastic diseases have undergone a transformation in a relatively short period like chronic myeloid leukemia (CML) has in the last few years. In 1960, CML was the first cancer in which a unique chromosomal abnormality was identified and a pathophysiologic correlation suggested. Landmark work followed, recognizing the underlying translocation between chromosomes 9 and 22 that gave rise to this abnormality and, shortly afterward, the specific genes involved and the pathophysiologic implications of this novel rearrangement. Fast forward a few years and this knowledge has given us the most remarkable example of a specific therapy that targets the dysregulated kinase activity represented by this molecular change. The broad use of tyrosine kinase inhibitors has resulted in an improvement in the overall survival to the point where the life expectancy of patients today is nearly equal to that of the general population. Still, there are challenges and unanswered questions that define the reasons why the progress still escapes many patients, and the details that separate patients from ultimate cure. In this article, we review our current understanding of CML in 2015, present recommendations for optimal management, and discuss the unanswered questions and what could be done to answer them in the near future.
很少有肿瘤性疾病能像慢性髓系白血病（CML）在过去几年那样在相对较短的时间内发生转变。1960年，CML是首个被发现存在独特染色体异常并提示有病理生理相关性的癌症。随后具有里程碑意义的研究相继出现，确认了导致这种异常的9号和22号染色体之间的潜在易位，不久之后，又明确了相关的特定基因以及这种新型重排的病理生理意义。几年后，基于这些知识，我们有了针对由这种分子变化所代表的失调激酶活性的最显著的特异性治疗范例。酪氨酸激酶抑制剂的广泛应用使总体生存率得到了改善，如今患者的预期寿命几乎与普通人群相当。然而，仍然存在一些挑战和未解决的问题，这些问题解释了为何许多患者仍未取得进展，以及将患者与最终治愈区分开来的细节。在本文中，我们回顾了2015年我们对CML的当前认识，提出了最佳管理建议，并讨论了未解决的问题以及在不久的将来可以采取哪些措施来回答这些问题。
