急性早幼粒细胞白血病的成功故事：通过靶向治疗治愈白血病。
The acute promyelocytic leukaemia success story: curing leukaemia through targeted therapies.
机构信息
Université Paris Diderot, Sorbonne Paris Cité, Hôpital St. Louis, Paris Cedex, France; INSERM UMR 944, Equipe labellisée par la Ligue Nationale contre le Cancer, Institut Universitaire d'Hématologie, Hôpital St. Louis, Paris Cedex, France; CNRS UMR 7212, Hôpital St. Louis, Paris Cedex, France.
出版信息
J Intern Med. 2014 Jul;276(1):61-70. doi: 10.1111/joim.12208.
The recent finding that almost all patients with acute promyelocytic leukaemia (APL) may be cured using a combination of retinoic acid (RA) and arsenic trioxide (As(2)O(3)) (N Engl J Med, 369, 2013 and 111) highlights the progress made in our understanding of APL pathogenesis and therapeutic approaches over the past 25 years. The study of APL has revealed many important lessons related to transcriptional control, nuclear organization, epigenetics and the role of proteolysis in biological control. Even more important has been the clinical demonstration that molecularly targeted therapy can eradicate disease.
最近的一项发现表明，几乎所有急性早幼粒细胞白血病（APL）患者都可以通过使用维甲酸（RA）和三氧化二砷（As(2)O(3)）（《新英格兰医学杂志》，369，2013 年和 111）联合治疗来治愈。这突显了过去 25 年来我们在 APL 发病机制和治疗方法方面取得的进展。对 APL 的研究揭示了许多与转录控制、核组织、表观遗传学以及蛋白水解在生物控制中的作用相关的重要知识。更重要的是，临床研究表明，分子靶向治疗可以根除疾病。
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