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知是成功的一半:精神分裂症研究中实现高效招募代表性样本的影响因素

Knowing is Half the Battle: The Factors Leading to Efficient Recruitment of Representative Samples in Schizophrenia Research.

作者信息

Yoon Joohyun, Mayer Megan R, Berro Tala, Brazis Stephanie, Kantrowitz Joshua T

机构信息

New York State Psychiatric Institute, 1051 Riverside Drive, New York, NY, 10032, USA.

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.

出版信息

Pharmaceut Med. 2025 Jan;39(1):29-38. doi: 10.1007/s40290-024-00545-8. Epub 2025 Jan 11.

Abstract

BACKGROUND AND OBJECTIVES

Drug development in schizophrenia is limited by the differential scaling of the active treatment and placebo arms of a study, such that, as the number of sites increases, the magnitude of placebo response disproportionately increases. The objective of this article was to identify factors conducive to efficient recruitment as a step towards trial designs allowing recruitment of more participants per site, leading to reduced variability, and potentially a smaller placebo effect.

PATIENTS AND METHODS

Using the information of 554 individuals, we calculated the percentage of individuals who were screened, consented, and retained in our research, along with rationale for nonconsent. Independent t tests and Chi-squared tests were performed to compare participant characteristics.

RESULTS

Out of the 273 individuals who were fully screened, 84 did not progress to the consented stage owing to various reasons, leading to a total of 189 individuals who were screened and consented and a total of 365 individuals who were either incompletely screened or not consented into a study. Individuals with an externally validated medical history showed the highest yield in being consented and retained in research as new participants. In particular, chart reviews from clinics were highly efficient (25.8%) in facilitating new participants' enrollment, even when these individuals were not actively/prospectively seeking research. The most common reason for nonconsent was difficulty in telephone or email contact. Consenting and nonconsenting participants were similar in demographics, and recruitment sources only differed in their reported substance use.

CONCLUSIONS

Referrals and chart reviews from known clinics were the most efficient method in retaining new participants, highlighting the importance of external validation and communication between research and clinical staff within a system. Consenting participants showed no significant differences from the database as a whole, demonstrating that the study samples were representative. Future studies should aim to confirm the present findings at other academic and commercial research centers.

摘要

背景与目的

精神分裂症药物研发受到研究中活性治疗组与安慰剂组不同程度变化的限制,即随着研究地点数量的增加,安慰剂反应的幅度会不成比例地增加。本文的目的是确定有助于高效招募的因素,作为迈向试验设计的一步,该试验设计允许每个地点招募更多参与者,从而减少变异性,并可能减小安慰剂效应。

患者与方法

利用554名个体的信息,我们计算了被筛选、同意参与并留在我们研究中的个体百分比,以及不同意参与的理由。进行独立t检验和卡方检验以比较参与者特征。

结果

在273名被全面筛选的个体中,84名由于各种原因未进入同意参与阶段,导致共有189名个体被筛选并同意参与,以及共有365名个体未被完全筛选或不同意参与研究。具有经外部验证的病史的个体作为新参与者同意参与并留在研究中的比例最高。特别是,来自诊所的病历审查在促进新参与者入组方面效率很高(25.8%),即使这些个体并非积极/前瞻性地寻求参与研究。不同意参与的最常见原因是电话或电子邮件联系困难。同意参与和不同意参与的参与者在人口统计学方面相似,招募来源仅在报告的物质使用方面存在差异。

结论

来自知名诊所的转诊和病历审查是留住新参与者的最有效方法,凸显了系统内研究人员与临床工作人员之间外部验证和沟通的重要性。同意参与的参与者与整个数据库没有显著差异,表明研究样本具有代表性。未来的研究应旨在在其他学术和商业研究中心证实本研究结果。

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