NEJM:治疗白血病,全反式维甲酸联合砷剂疗效优于联合化疗
丁香园 · 2016/05/19
近20年间,全反式维甲酸(ATRA)和三氧化二砷应用于初治急性早幼粒细胞白血病疗效显著。英国国立肿瘤研究所的结果明显表明:非高风险的急性早幼粒细胞性白血病患者,全反式维甲酸—三氧化二砷治疗效果优于标准的全反式维甲酸—化疗方案。


近 20 年间,全反式维甲酸(ATRA)和三氧化二砷应用于初治急性早幼粒细胞白血病疗效显著。全反式维甲酸联合化疗的治愈率约 80%,然而此疗法导致重度感染及二次白血病的风险极高;全反式维甲酸联合三氧化二砷有效性佳且毒性反应可控,如 QT 间期延长及肝酶水平增高,但该疗法的远期疗效仍有待观察。

意大利罗马第二大学 Lo‑Coco 医师等在 NEJM 上发表了一篇文章,将全反式维甲酸联合砷剂或化疗两方案在一个随机临床试验中进行对比。受试者为非高危急性早幼粒细胞性白血病患者(白细胞计数 ≤ 10 × 109/ L)。中位随访时间 34 月,结果示:接受全反式维甲酸 - 砷剂治疗的患者 2 年无进展生存率明显高于接受全反式维甲酸 - 化疗的患者。

在此意向治疗分析中,中位随访时间 53 月, 156 例患者在第 50 个月时,全反式维甲酸 - 三氧化二砷组无进展生存率为 96 %,全反式维甲酸 - 化疗组为 81 % ,总生存率分别 99 % 和 88 % ,全反式维甲酸 - 化疗组缓解后有 6 名复发、 5 例死亡(其中一名为继发性白血病),全反式维甲酸 - 三氧化二砷组 2 例复发,1 例死亡。

另一相似的由英国国立肿瘤研究所急性髓系白血病组成员报道的随机多中心 3 期临床试验同样表明,接受全反式维甲酸 - 三氧化二砷治疗的急性早幼粒细胞白血病患者疗效显著好于全反式维甲酸 - 化疗的患者,在接受抗 - CD 33 抗体 - 卡里奇霉素联合吉妥珠单抗奥唑米星治疗的高风险患者亚组,全反式维甲酸 - 三氧化二砷治疗的优势同样明显。

总之,该研究的长期数据和英国国立肿瘤研究所的结果明显表明:非高风险的急性早幼粒细胞性白血病患者,全反式维甲酸——三氧化二砷治疗效果优于标准的全反式维甲酸——化疗方案。英国国立肿瘤研究所的高风险患者人数较少,因此研究人员认为,需要一随机临床试验对比此类患者中这两种治疗方案的疗效。

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