Heiat Asefeh, Gross Cary P, Krumholz Harlan M
Department of Internal Medicine/Preventive Medicine, Griffin Hospital, Derby, Conn, USA.
Arch Intern Med. 2002;162(15):1682-8. doi: 10.1001/archinte.162.15.1682.
Appropriate representation of specific groups of patients in randomized controlled trials (RCTs) and generalizability of evidence obtained have been questioned. We sought to compare the characteristics of patients in heart failure (HF) RCTs with those of patients with HF in the community, and to document these trends over time.
MEDLINE was searched from 1985 through December 1999 for all HF RCTs. Additional trials were obtained through bibliographies of the reviewed articles, previous meta-analyses, and overviews of the HF trials. Original reports of RCTs of interventions for chronic HF were selected if they were published in the English language and included 50 or more participants. Publication year, sources of support, location of principal investigator, sample size, type of intervention, inclusion and exclusion criteria, characteristics of participants, and the use of invasive diagnostic tests were extracted.
Participants in the 59 HF RCTs we reviewed were markedly different from the patients with HF in the community. Patients in RCTs were younger, more often male, more likely to have subnormal systolic ejection fraction, and were most commonly white. We did not find a significant improvement in the representation of the trials, with respect to these characteristics, among those published recently compared with those from the late 1980s and early 1990s.
Clinical trials are focusing on a relatively small segment of the HF population. The consequences of underrepresenting minorities, women, and elderly are unknown but may be particularly important for HF. Future clinical trials should adequately include populations that carry the burden of the disease.
随机对照试验（RCT）中特定患者群体的恰当代表性以及所获证据的可推广性受到了质疑。我们试图比较心力衰竭（HF）RCT中的患者特征与社区中HF患者的特征，并记录这些特征随时间的变化趋势。
检索MEDLINE数据库1985年至1999年12月期间所有的HF RCT。通过已审阅文章的参考文献、先前的荟萃分析以及HF试验综述获取其他试验。入选以英文发表且纳入50名或更多参与者的慢性HF干预措施RCT的原始报告。提取发表年份、资助来源、主要研究者所在地点、样本量、干预类型、纳入和排除标准、参与者特征以及侵入性诊断检查的使用情况。
我们所审阅的59项HF RCT中的参与者与社区中的HF患者明显不同。RCT中的患者更年轻，男性更多，收缩期射血分数低于正常的可能性更大，且最常见为白人。与20世纪80年代末和90年代初的试验相比，我们未发现近期发表的试验在这些特征的代表性方面有显著改善。
临床试验关注的是HF人群中相对较小的一部分。少数族裔、女性和老年人代表性不足的后果尚不清楚，但对HF可能尤为重要。未来的临床试验应充分纳入承担疾病负担的人群。
