Biostatistics Core, Centre for Addiction and Mental Health, 1001 Queen Street West, Toronto, ON, Canada.
Center for Complex Interventions, Centre for Addiction and Mental Health, Toronto, ON, Canada.
BMC Psychiatry. 2023 Mar 23;23(1):192. doi: 10.1186/s12888-023-04676-1.
Randomized Controlled Trial is the most rigorous study design to test the efficacy and effectiveness of an intervention. Patient preference may negatively affect patient performance and decrease the generalizability of a trial to clinical population. Patient preference trial have particular implications in the field of mental health and addiction since mental health interventions are generally complex, blinding of intervention is often difficult or impossible, patients may have strong preference, and outcome measures are often subjective patient self-report which may be greatly influenced if patient's preference did not match with the intervention received.
In this review, we have surveyed the application of two-stage randomized preference trial with focus on studies in the field of mental health and addiction. The study selection followed the guideline provided by Preferred Reporting Items for Systematic reviews and Meta-Analyses extension for Scoping Reviews.
Six two-stage randomized preference trials (ten publications) have been identified in the field of mental health field and addiction. In these trials, the pooled dropout rates were 18.3% for the preference arm, and 28.7% for the random arm, with a pooled RR of 0.70 (95% CI, 0.56-0.88; P = 0.010) indicating lower risk of dropout in the preference arm. The standardized preference effects varied widely from 0.07 to 0.57, and could be as large as the treatment effect in some of the trials.
This scoping review has shown that two-stage randomized preference trials are not as popular as expected in mental health research. The results indicated that two-stage randomized preference trials in mental health would be beneficial in retaining patients to expand the generalizability of the trial.
随机对照试验是测试干预措施疗效和效果最严格的研究设计。患者偏好可能会对患者的表现产生负面影响，并降低试验对临床人群的普遍性。在心理健康和成瘾领域，患者偏好试验具有特殊意义，因为心理健康干预措施通常比较复杂，干预措施的盲法往往比较困难或不可能，患者可能有强烈的偏好，而且结果测量通常是主观的患者自我报告，如果患者的偏好与接受的干预措施不匹配，可能会对结果产生很大影响。
在本次综述中，我们调查了两阶段随机偏好试验的应用，重点是心理健康和成瘾领域的研究。研究选择遵循了系统评价和荟萃分析扩展的首选报告项目提供的指南。
在心理健康领域和成瘾领域共确定了六项两阶段随机偏好试验（十篇文献）。在这些试验中，偏好组的累积失访率为 18.3%，随机组为 28.7%，合并 RR 为 0.70（95%CI，0.56-0.88；P=0.010），表明偏好组的失访风险较低。标准化偏好效应的范围从 0.07 到 0.57 不等，在某些试验中，其大小可与治疗效果相当。
本次范围综述表明，两阶段随机偏好试验在心理健康研究中并不像预期的那么受欢迎。结果表明，在心理健康领域进行两阶段随机偏好试验有助于保留患者，从而扩大试验的普遍性。
