Realgar-Indigo Naturalis Formula (RIF) is an oral form of arsenic that is effective against acute promyelocytic leukemia (APL). This multicenter, randomized, controlled trial compared the efficacy of all-trans retinoic acid (ATRA) plus RIF with ATRA plus arsenic trioxide (ATO) in a simplified regimen for non-high-risk APL. Following induction therapy with ATRA and ATO, participants were randomly assigned to receive either ATRA plus ATO or ATRA plus RIF both in a 2-week on 2-week off schedule for consolidation therapy. Once achieving molecular complete remission, the regimen was administered for a total of 6 cycles. All of 108 eligible patients achieved hematological complete remission after induction therapy. The median follow-up time was 29 months. The primary endpoint of two-year disease-free survival was 97% in the ATRA-RIF arm and 98% in the ATRA-ATO arm, respectively. (The ATRA-RIF arm was found to be non-inferior to the ATRA-ATO arm, (P < .01), with a percentage difference of -1% (95%CI, -4.8 to 6.9). No deaths have been observed. Most adverse events were moderate. This study confirms the noninferiority of RIF to ATO for non-high-risk APL, while also offering a more favorable regimen schedule for post-remission therapy. (ClinicalTrials gov. Identifier: as NCT02899169).

中文翻译：

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三氧化二砷与雄黄-青靛配方在非高危急性早幼粒细胞白血病中的疗效：一项多中心随机试验。


雄黄-青蓝配方 （RIF） 是一种口服形式的砷，对急性早幼粒细胞白血病 （APL） 有效。这项多中心、随机、对照试验比较了全反式维甲酸 （ATRA） 加 RIF 与 ATRA 加三氧化二砷 （ATO） 在简化方案中对非高危 APL 的疗效。在使用 ATRA 和 ATO 诱导治疗后，参与者被随机分配接受 ATRA 加 ATO 或 ATRA 加 RIF，两者均在 2 周到 2 周的停药时间表中接受巩固治疗。一旦达到分子完全缓解，该方案总共给药 6 个周期。108 例符合条件的患者在诱导治疗后均达到血液学完全缓解。中位随访时间为 29 个月。ATRA-RIF 组的两年无病生存率主要终点分别为 97% 和 ATRA-ATO 组的 98%。（发现 ATRA-RIF 组不劣于 ATRA-ATO 组 （P < .01），百分比差异为 -1% （95%CI，-4.8 至 6.9）。尚未观察到死亡病例。大多数不良事件为中度。本研究证实了 RIF 在非高危 APL 方面优于 ATO 的非劣效性，同时也为缓解后治疗提供了更有利的方案方案。（ClinicalTrials gov. 标识符：as NCT02899169）。