Department of hematology, First affiliated hospital of Xi'an Jiaotong University, Yanta West Road, Xi'an, 710061, Shaanxi, China.
Department of hematology, Tangdu Hospital, Fourth Military Medical University of Chinese PLA, No. 1 Xinsi Road, Xi'an, 710038, Shaanxi, China.
Trials. 2020 Jan 2;21(1):7. doi: 10.1186/s13063-019-3983-2.
Acute promyelocytic leukemia (APL) is a highly curable disease when treated with all-trans retinoid acid (ATRA) and arsenic trioxide (ATO). The combination of ATO and ATRA has become the standard therapeutic protocol for induction therapy in non-high-risk APL. An oral arsenic realgar-indigo naturalis formula (RIF) has also showed high efficacy and it has a more convenient route of administration than the standard intravenous regimen. Unlike in previous trials, the arsenical agent was used simultaneously with ATRA during post-remission therapy in this trial.
This study was designed as a multicenter, randomized controlled trial. The trial has a non-inferiority design with superiority being explored if non-inferiority is identified. All patients receive ATRA-ATO during the induction therapy. After achieving hematologic complete remission (HCR), patients were randomly assigned (1:1) to receive treatment with ATRA-RIF (experimental group) or ATRA-ATO (control group) as the consolidation therapy. During the consolidation therapy, the two groups receive ATRA plus RIF or intravenous ATO 2 weeks on and 2 to ~ 4 weeks off until molecular complete remission (MCR), then ATRA and oral RIF 2 weeks on and 2 to ~ 4 weeks off giving a total of six courses.
This trial aims to compare the efficacy of ATRA-ATO versus ATRA-RIF in non-high-risk patients with APL, to demonstrate that oral RIF application reduces the total hospitalization days and medical costs. The simple schedule was studied in this trial.
ClinicalTrials.gov, NCT02899169. Registered on 14 September 2016.
全反式维甲酸（ATRA）联合三氧化二砷（ATO）治疗急性早幼粒细胞白血病（APL）可获得极高的缓解率。ATO 联合 ATRA 已成为非高危 APL 诱导治疗的标准治疗方案。口服砷剂雄黄-青黛复方（RIF）也显示出较高的疗效，且给药途径较标准静脉方案更为方便。与既往试验不同，在该试验中，砷剂在缓解后治疗期间与 ATRA 同时使用。
本研究设计为多中心、随机对照试验。该试验采用非劣效性设计，如果确定非劣效性，则探索其优越性。所有患者在诱导治疗期间接受 ATRA-ATO 治疗。达到血液学完全缓解（HCR）后，患者按 1:1 随机分配接受 ATRA-RIF（实验组）或 ATRA-ATO（对照组）作为巩固治疗。在巩固治疗期间，两组均接受 ATRA 联合 RIF 或静脉 ATO（2 周用药，2 至 4 周停药），直至分子完全缓解（MCR），然后 ATRA 和口服 RIF（2 周用药，2 至 4 周停药），共 6 个疗程。
本试验旨在比较 ATRA-ATO 与 ATRA-RIF 在非高危 APL 患者中的疗效，证明口服 RIF 应用可减少总住院天数和医疗费用。本试验研究了简单的方案。
ClinicalTrials.gov，NCT02899169。于 2016 年 9 月 14 日注册。
