Lemons R S, Keller S, Gietzen D, Dufner J, Rebentisch M, Feusner J, Eilender D
Department of Pediatrics, University of Utah, Salt Lake City 84112, USA.
J Pediatr Hematol Oncol. 1995 Aug;17(3):198-210. doi: 10.1097/00043426-199508000-00002.
Significant advances have occurred in the diagnosis, treatment, and long-term outcome of patients with acute promyelocytic leukemia (APL). The purpose of this review is to describe the molecular genetics of this disease, the use of all-trans retinoic acid (ATRA) in clinical trials of APL, and the clinical and basic research questions for future investigation. Findings of clinical studies in mainland China using ATRA as induction therapy for patients with APL concurrent with laboratory characterization of the molecular changes in APL have led to worldwide clinical trials of ATRA in the treatment of patients with APL. Major advances in understanding the molecular biology and genetics of APL have occurred over the past 5 years. These findings have been translated into novel treatment strategies using all-trans retinoic acid as a differentiation agent in the induction phase of therapy resulting in improved long-term outcome, reduced morbidity, and lower costs for patients with APL. Advanced molecular techniques are being employed for diagnosis and for monitoring of patient response to treatment.
急性早幼粒细胞白血病（APL）患者在诊断、治疗及长期预后方面已取得显著进展。本综述旨在描述该疾病的分子遗传学、全反式维甲酸（ATRA）在APL临床试验中的应用，以及未来研究的临床和基础研究问题。中国大陆使用ATRA作为APL患者诱导治疗的临床研究结果，以及APL分子变化的实验室特征，促使了全球范围内ATRA治疗APL患者的临床试验。在过去5年里，对APL分子生物学和遗传学的理解取得了重大进展。这些发现已转化为新的治疗策略，即在治疗诱导阶段使用全反式维甲酸作为分化剂，从而改善了APL患者的长期预后，降低了发病率，并降低了治疗成本。先进的分子技术正被用于诊断和监测患者对治疗的反应。
