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目的 回顾随机对照试验 (RCT)研究对评估儿科危重症医学预防和治疗措施的益处及对其的争议。结论 RCT设计能控制其他研究方法所不能控制的许多潜在偏差的发生。RCTs结果常可否定那些设计不够严谨的实验结果。在进行RCT前 ,研究者不能预先对所要研究的两种或多种临床治疗有偏见 ,必须有足够数量的合格患儿 ,而且必须对疾病流行病学存在的问题进行很好的分析。在进行高质量的RCTs研究时会遇到很多挑战。对危重病多因素生命支持措施进行研究较对单一药物治疗研究困难得多。由于患儿及临床实践的变异较大 ,且疾病诊断不明 ,致使信息与背景干扰的差异减小。多数危重症的治疗措施可能仅有中度或轻微疗效 ,因此大大增加了所需样本含量。只有很少一部分方法被临床接受 ,用于评估危重患儿的预后 ,如常以死亡率评估罕见事件的预后。处于发展过程中的事件 ,不能予以满意解释和未能成功进行药代动力学研究及安全性研究是儿科研究人员面临的另一些挑战。尽管存在这些限制 ,但一个好的RCT仍然是证明一种治疗有效与否的最好方法。的确 ,RCTs目前是而且会一直是评估一种治疗或预防措施有效与否的金标准
Objective To review the controversy and the benefits of randomized controlled trials (RCTs) in assessing medical preventive and curative measures for pediatric critical care. Conclusion RCT design can control the many potential deviations that other research methods can not control. RCTs often negate the design of those results not rigorous experimental results. Before conducting RCT, researchers can not prejudice two or more clinical treatments to be studied. There must be a sufficient number of eligible children and a good analysis of the epidemiological problems of the disease must be performed. There are many challenges in conducting high quality RCTs. Much more difficult is the study of multi-factor life support measures for critically ill patients than single drug therapy. Due to the large variation in children and clinical practice, and the unknown disease diagnosis, the difference between information and background interference is reduced. Most critically ill treatments may have only moderate or mild efficacy, thus greatly increasing the required sample content. Only a small proportion of the methods are clinically accepted to assess the prognosis of critically ill children, as is the prognosis for rare events, as often as death rates. In-progress events that can not be satisfactorily explained and unsuccessful in pharmacokinetic and safety studies are other challenges for pediatric researchers. Despite these limitations, a good RCT remains the best way to prove that one is effective. Indeed, RCTs are, and will always be, the gold standard for evaluating the effectiveness of a treatment or preventative measure