Prescrire Int. 2004 Aug;13(72):135-7.
(1) Acute promyelocytic leukaemia is a rare disease. There is a high remission rate after combination treatment with tretinoin and anthracycline, but there is no established treatment for refractory or relapsed disease. Further treatment with tretinoin, combined with intensive cytotoxic chemotherapy, seems to give the best results in patients who qualify for this treatment, but assessment is limited. (2) Arsenic trioxide has now been approved for induction of remission and consolidation in patients with refractory or relapsed acute promyelocytic leukaemia. (3) The clinical evaluation dossier that supported the application contains data from two non comparative trials including 12 and 40 patients. A complete haematological response was obtained in 45 (87%) of the 52 patients, and the survival rate among patients in first relapse was 77% after a median follow-up of two years. These results are similar to those previously obtained with tretinoin plus intensive cytotoxic chemotherapy. (4) All the patients treated with arsenic trioxide experienced adverse events. This was to be expected given the acute and chronic toxicity of arsenicals. Most events included fatigue, gastrointestinal disturbances, peripheral neuropathies, prolongation of the QT interval; and biochemical disturbances (hypokalaemia, hyperglycaemia, elevated transaminase activity). (5) Like tretinoin, arsenic trioxide can provoke a potentially severe leukocyte activation syndrome. (6) In practice, these encouraging data justify further assessment of arsenic trioxide. This drug is already an option for patients with refractory or relapsed disease who cannot receive tretinoin plus intensive chemotherapy.
（1）急性早幼粒细胞白血病是一种罕见疾病。维甲酸与蒽环类药物联合治疗后缓解率较高，但对于难治性或复发性疾病尚无既定治疗方法。对于符合该治疗条件的患者，维甲酸联合强化细胞毒性化疗的进一步治疗似乎能取得最佳效果，但评估有限。（2）三氧化二砷现已被批准用于难治性或复发性急性早幼粒细胞白血病患者的诱导缓解和巩固治疗。（3）支持该申请的临床评估档案包含来自两项非对照试验的数据，分别有12名和40名患者。52例患者中有45例（87%）获得了完全血液学缓解，首次复发患者的生存率在中位随访两年后为77%。这些结果与之前维甲酸加强化细胞毒性化疗所取得的结果相似。（4）所有接受三氧化二砷治疗的患者均出现了不良事件。鉴于砷剂的急性和慢性毒性，出现这种情况在意料之中。大多数事件包括疲劳、胃肠道紊乱、周围神经病变、QT间期延长以及生化紊乱（低钾血症、高血糖症、转氨酶活性升高）。（5）与维甲酸一样，三氧化二砷可引发潜在的严重白细胞活化综合征。（6）在实际应用中，这些令人鼓舞的数据证明对三氧化二砷进行进一步评估是合理的。对于无法接受维甲酸加强化化疗的难治性或复发性疾病患者，这种药物已经是一种选择。
