Sanz Miguel A, Grimwade David, Tallman Martin S, Lowenberg Bob, Fenaux Pierre, Estey Elihu H, Naoe Tomoki, Lengfelder Eva, Büchner Thomas, Döhner Hartmut, Burnett Alan K, Lo-Coco Francesco
University Hospital La Fe, Valencia, Spain.
Blood. 2009 Feb 26;113(9):1875-91. doi: 10.1182/blood-2008-04-150250. Epub 2008 Sep 23.
The introduction of all-trans retinoic acid (ATRA) and, more recently, arsenic trioxide (ATO) into the therapy of acute promyelocytic leukemia (APL) has revolutionized the management and outcome of this disease. Several treatment strategies using these agents, usually in combination with chemotherapy, but also without or with minimal use of cytotoxic agents, have provided excellent therapeutic results. Cure of APL patients, however, is also dependent on peculiar aspects related to the management and supportive measures that are crucial to counteract life-threatening complications associated with the disease biology and molecularly targeted treatment. The European LeukemiaNet recently appointed an international panel of experts to develop evidence- and expert opinion-based guidelines on the diagnosis and management of APL. Together with providing current indications on genetic diagnosis, modern risk-adapted front-line therapy and salvage treatment, the review contains specific recommendations for the identification and management of most important complications such as the bleeding disorder, APL differentiation syndrome, QT prolongation and other ATRA- and ATO-related toxicities, as well as for molecular assessment of response to treatment. Finally, the approach to special situations is also discussed, including management of APL in children, elderly patients, and pregnant women.
全反式维甲酸（ATRA）以及最近三氧化二砷（ATO）被引入急性早幼粒细胞白血病（APL）的治疗，彻底改变了这种疾病的管理方式和治疗结果。使用这些药物的几种治疗策略，通常与化疗联合使用，但也有无化疗或极少使用细胞毒性药物的情况，都取得了出色的治疗效果。然而，APL患者的治愈还取决于与管理和支持措施相关的特殊方面，这些对于对抗与疾病生物学和分子靶向治疗相关的危及生命的并发症至关重要。欧洲白血病网最近任命了一个国际专家小组，以制定基于证据和专家意见的APL诊断和管理指南。该综述除了提供有关基因诊断、现代风险适应性一线治疗和挽救治疗的当前指征外，还包含针对识别和管理最重要并发症的具体建议，如出血性疾病、APL分化综合征、QT延长以及其他与ATRA和ATO相关的毒性，以及治疗反应的分子评估。最后，还讨论了特殊情况的处理方法，包括儿童、老年患者和孕妇的APL管理。
