真实世界研究在新生儿医学中的应用

Application of real world study in neonatal medicine

摘要:

真实世界研究（RWS）由于较少涉及临床干预，更加贴近临床实际情况而受到新生儿科医师的重视。通常意义的RWS应该是指在随机对照试验（RCT）验证内部有效性和安全性的基础上，在更加具有代表性的更大样本量基础上，根据患者的实际病情和监护人意愿非随机选择治疗措施，开展长期追踪评估，并随访近期和远期结局变量，以进一步评价干预措施的外部有效性和安全性。目前的临床实践指南大部分是基于RCT，缺乏真实世界数据支撑。加强新生儿医学领域的RWS，有利于指南更加实用化，从而促进新生儿医学发展。

Abstract:

Real world study (RWS) has attracted more and more attention of neonatologists since it involves less clinical intervention and is closer to actual clinical conditions. Generally speaking, RWS means to select treatment measures based on the internal efficacy and safety verified by randomized controlled trials (RCTs), more representative samples, and patients' actual conditions and their guardians' will and conduct follow-up evaluation of short-and long-term outcomes, in order to further evaluate the external efficacy and safety of interventional measures. Most guidelines for clinical practice are based on RCTs and lack the support of real world data. Strengthening of neonatal RWS helps to make these guidelines more practical and thus promotes the development of neonatal medicine.