背景:急性早幼粒细胞白血病(APML)治疗的最新进展在患者预后方面有了前所未有的改善。然而,这种理解的快速增长往往导致候选治疗方案中优势的不确定性,特别是从流行病学角度进一步审查时。
目的:本系统评价与流行病学分析的目的是确定和比较常用的标准风险APML方案,特别关注完全缓解(CR)。总体/无病生存期(DFS),并报告不良事件。
结果:Medline,Scopus,我们询问了CINAHL,以确定在从头APML治疗中除了使用三氧化二砷(ATO)和/或蒽环类药物如伊达比星(IDA)外还使用全反式维甲酸(ATRA)的研究。经过整理研究,随后进行了流行病学分析,使用获益所需数量(NNB)和损害所需数量(NNH)措施,对方案的相关结局进行比较.17篇文章,描述了12个不同的试验,包括在分析中。这些试验使用了三种独特的方案;ATO/ATRA方案的CR率为94%-100%,ATO/ATRA/蒽环类药物治疗的95%-96%,ATRA/蒽环类药物治疗的比例为89%-94%。流行病学分析表明,CR的NNB为9.09(ATO/ATRA与ATRA/IDA)和20.00(ATO/ATRA与ATO/ATRA/IDA),中性粒细胞减少症的NNH为-3.45(ATO/ATRA与ATRA/IDA),感染的NNH为-3.13(ATO/ATRA与ATRA/IDA)和-1.89(ATO/ATRA与ATO/ATRA/IDA)。
结论:ATO/ATRA方案在诱导缓解和促进APML患者生存方面优于含化疗方案。与建议的替代方案相比,该方案的耐受性更好,不良事件更少。未来的研究机会包括量化APML流行病学和追求口服砷作为简化治疗方案的一种选择。
BACKGROUND: Recent advances in the treatment of acute promyelocytic leukemia (APML) have seen unprecedented improvements in patient outcomes. However, such rapid growth in understanding often leads to uncertainty regarding superiority among candidate treatment regimens, especially when further scrutinized from an epidemiological perspective.
OBJECTIVE: The aim of this systematic
review with epidemiological analysis was to identify and compare commonly utilized protocols for standard-risk APML with a particular focus on complete remission (CR), overall/disease-free survival (DFS), and reported adverse events.
RESULTS: Medline, Scopus, and CINAHL were interrogated to identify studies utilizing all-trans retinoic acid (ATRA) in addition to arsenic trioxide (ATO) and/or anthracyclines such as idarubicin (IDA) in the treatment of de-novo APML. After collation of studies, an epidemiological analysis was subsequently performed to compare protocols with regards to outcomes of interest using number needed to benefit (NNB) and number needed to harm (NNH) measures. Seventeen articles, describing 12 distinct trials, were included in the analysis. These trials made use of three unique protocols; CR rates were 94%-100% for ATO/ATRA regimens, 95%-96% for ATO/ATRA/anthracycline regimens, and 89%-94% for ATRA/anthracycline regimens. Epidemiological analysis demonstrated NNB for CR was 9.09 (ATO/ATRA vs. ATRA/IDA) and 20.00 (ATO/ATRA vs. ATO/ATRA/IDA), NNH for neutropenia was -3.45 (ATO/ATRA vs. ATRA/IDA), and NNH for infection was -3.13 (ATO/ATRA vs. ATRA/IDA) and -1.89 (ATO/ATRA vs. ATO/ATRA/IDA).
CONCLUSIONS: The ATO/ATRA regimen is superior to chemotherapy-containing protocols at inducing remission and promoting survival in patients with APML. The regimen is better tolerated than the proposed alternatives with fewer adverse events. Future research opportunities include quantifying APML epidemiology and pursuing oral arsenic as an option for simplification of therapeutic protocols.