Citrome Leslie, Mychaskiw Marko A, Cortez Alma, Opler Mark, Sopina Liza, Kotak Sameer
Dr. Citrome is with Department of Psychiatry and Behavioral Sciences, New York Medical College in Valhalla, New York.
Dr. Mychaskiw and Ms. Cortez are with Global Health Economics and Outcomes Research, Teva Branded Pharmaceutical Products R&D, Inc., in West Chester, Pennsylvania.
Innov Clin Neurosci. 2023 Spring;20(4-6):14-33.
The complexity inherent in the treatment of schizophrenia results in a multitude of outcome assessments being employed when conducting clinical trials. Subjective outcome assessments and minimal clinically important differences (MCIDs) to evaluate clinical meaningfulness have gained traction; however, the extent of application in evaluation of treatments for schizophrenia is unknown. A scoping review was conducted to assess the availability of published psychometric evaluations, including MCIDs, for clinical outcome assessments used to evaluate treatments for schizophrenia.
Key databases (PubMed®, Embase®, APA PsycINFO®, International Society for Pharmacoeconomics and Outcomes Research) were searched for studies on schizophrenia published from 2010 to 2020. Secondary sources (ClinicalTrials.gov, PROLABELS™, FDA.gov) were also reviewed. Clinical outcome assessments were organized by type (patient-reported outcomes [PROs], clinician-reported outcomes [ClinROs], observer-reported outcomes [ObsROs]) and further classified by intended use (generic, mental health, schizophrenia). Reliability and internal consistency were evaluated using Cronbach's α. External validity was evaluated by intraclass correlation coefficient (ICC).
Across 140 studies, 66 clinical outcome assessments were identified. MCIDs were reported for eight of the 66 studies. Of these, two were PROs (generic) and six were ClinROs/ObsROs (three mental health-specific, three schizophrenia-specific). Reliability was good across generic, mental health-specific, and schizophrenia-specific categories, whereas external validity was strong mainly for schizophrenia-specific PROs. Overall, ClinROs/ObsROs that focused on mental health had good reliability and strong external validity.
This review provides a comprehensive overview of the clinical outcome assessments used in schizophrenia research during the past ten years. Results highlight the heterogeneity of existing outcomes and a growing interest in PROs for schizophrenia.
精神分裂症治疗本身的复杂性导致在进行临床试验时采用了多种结局评估方法。用于评估临床意义的主观结局评估和最小临床重要差异（MCID）已受到关注；然而，其在精神分裂症治疗评估中的应用程度尚不清楚。进行了一项范围综述，以评估已发表的用于评估精神分裂症治疗的临床结局评估的心理测量学评价（包括MCID）的可获得性。
检索主要数据库（PubMed®、Embase®、美国心理学会心理学文摘数据库®、国际药物经济学与结果研究学会），查找2010年至2020年发表的关于精神分裂症的研究。还查阅了二级来源（ClinicalTrials.gov、PROLABELS™、FDA.gov）。临床结局评估按类型（患者报告结局[PROs]、临床医生报告结局[ClinROs]、观察者报告结局[ObsROs]）进行组织，并进一步按预期用途（通用、心理健康、精神分裂症）分类。使用Cronbach's α评估信度和内部一致性。通过组内相关系数（ICC）评估外部效度。
在140项研究中，共识别出66种临床结局评估。66项研究中有8项报告了MCID。其中，2项是PROs（通用），6项是ClinROs/ObsROs（3项特定于心理健康，3项特定于精神分裂症）。在通用、特定于心理健康和特定于精神分裂症的类别中，信度良好，而外部效度主要在特定于精神分裂症的PROs中较强。总体而言，关注心理健康的ClinROs/ObsROs具有良好的信度和较强的外部效度。
本综述全面概述了过去十年精神分裂症研究中使用的临床结局评估。结果突出了现有结局的异质性以及对精神分裂症PROs的兴趣日益增加。
