Northwestern University Feinberg School of Medicine, Robert H. Lurie Comprehensive Cancer Center, Chicago, IL 60611, USA.
Blood. 2009 Dec 10;114(25):5126-35. doi: 10.1182/blood-2009-07-216457.
Acute promyelocytic leukemia is the first malignant disease highly curable with targeted therapy directed at a unique molecular abnormality. The characteristic bleeding diathesis is the most notorious manifestation of the disease, which historically has accounted for a high mortality rate during induction. Acute promyelocytic leukemia is one of the few hematologic diseases that must be recognized under the microscope by the practicing hematologist because early institution of all-trans retinoic acid (ATRA) at the first suspicion of the disease before confirmation of the diagnosis and aggressive blood product support are critical to reduce early mortality. ATRA plus anthracycline-based chemotherapy for induction and consolidation followed by maintenance ATRA with low-dose chemotherapy is currently the standard of care. However, the combination of ATRA and arsenic trioxide, with minimal chemotherapy to control leukocytosis, is very effective therapy for newly diagnosed patients. This combination may replace conventional approaches for most, if not all, patients in the very near future. Acute promyelocytic leukemia should be considered in any patient with newly diagnosed acute myeloid leukemia because the treatment is urgent and different from all other subtypes.
急性早幼粒细胞白血病是第一种可通过针对独特分子异常的靶向治疗高度治愈的恶性疾病。特征性的出血倾向是该疾病最臭名昭著的表现，在诱导治疗期间，该疾病的死亡率历来很高。急性早幼粒细胞白血病是为数不多的必须由执业血液学家在显微镜下识别的血液疾病之一，因为在确诊前，只要怀疑患有该疾病，就应立即开始使用全反式维甲酸（ATRA）治疗，并积极支持血制品，这对于降低早期死亡率至关重要。目前，诱导和巩固阶段采用 ATRA 联合蒽环类药物化疗，随后采用 ATRA 联合低剂量化疗维持治疗，这是标准的治疗方案。然而，ATRA 联合三氧化二砷，辅以控制白细胞增多的最小剂量化疗，对于新诊断的患者是非常有效的治疗方法。在不久的将来，这种联合治疗方法可能会替代大多数（如果不是全部）患者的常规治疗方法。对于新诊断的急性髓系白血病患者，都应考虑急性早幼粒细胞白血病，因为其治疗是紧急的，与所有其他亚型不同。
