Langfus Joshua A, Youngstrom Eric A, Daniel David, Busner Joan, Findling Robert L
University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.
Nationwide Children's Hospital and The Ohio State University, Columbus, Ohio, USA.
J Child Adolesc Psychopharmacol. 2024 Dec;34(10):447-457. doi: 10.1089/cap.2024.0078. Epub 2024 Oct 17.
The Positive and Negative Syndrome Scale (PANSS) is a widely accepted outcome measure for pediatric schizophrenia trials; however, it has notable limitations. Psychometric investigations have shown a multifactorial structure and some items have limited utility assessing symptom severity in children. To address these issues, we developed and evaluated optimized 10- and 20-item PANSS short-forms (PANSS10 and PANSS20) using patient-level clinical trial data. This study further assesses these optimized forms using independent clinical trial data. We examined patient-level data from a randomized pediatric schizophrenia trial comparing paliperidone ER to aripiprazole. Data were accessed through the Yale Open Data Access (YODA) secure platform. Analyses included confirmatory factor analyses, graded response models, ω score reliability, internal consistency, sensitivity to change, and criterion validity versus the Clinical Global Impressions of Severity (CGI-S). Bland-Altman analyses examined score calibration versus the 30-item PANSS and inclusion cut scores. Participants ( = 288) were ages 12 to 17 years ( = 15.3, SD = 1.46; 66% male). Total scores for the PANSS10 and PANSS20 showed strong correlations with the 30-item PANSS (0.90 and 0.97, respectively). Average inter-item correlations were 0.10 and 0.14 and ω reliabilities were 0.74 and 0.85. Both PANSS10 and PANSS20 scores showed reliability >0.80-2.3 to 4.5 SD and -3.0 to 6.0 SD about mean symptom severity, respectively. Sensitivity to treatment was also similar (partial squared 0.23 and 0.22), as was correlation with CGI-S at baseline (0.45 and 0.48; not significantly different). The mean item-average discrepancy with the 30-item PANSS was 0.095 for PANSS10 and 0.033 for PANSS20. The optimized PANSS forms continue to show impressive reliability, validity, and calibration compared with the 30-item PANSS. Researchers should consider replacing the 30-item PANSS with the PANSS10 as a clinical outcome and screening measure due to its length and psychometric performance.
阳性和阴性症状量表（PANSS）是儿科精神分裂症试验中广泛接受的疗效评估指标；然而，它有明显的局限性。心理测量学研究表明其具有多因素结构，且一些条目在评估儿童症状严重程度方面效用有限。为解决这些问题，我们利用患者层面的临床试验数据开发并评估了优化后的10项和20项PANSS简表（PANSS10和PANSS20）。本研究使用独立的临床试验数据进一步评估这些优化后的量表。我们检查了一项比较帕利哌酮缓释片与阿立哌唑的随机儿科精神分裂症试验中的患者层面数据。数据通过耶鲁开放数据访问（YODA）安全平台获取。分析包括验证性因子分析、等级反应模型、ω分数信度、内部一致性、对变化的敏感性以及与临床总体印象严重程度（CGI-S）的效标效度。Bland-Altman分析检验了与30项PANSS的分数校准以及纳入截点。参与者（n = 288）年龄在12至17岁之间（M = 15.3，标准差 = 1.46；66%为男性）。PANSS10和PANSS20的总分与30项PANSS显示出强相关性（分别为0.90和0.97）。平均项目间相关性分别为0.10和0.14，ω信度分别为0.74和0.85。PANSS10和PANSS20的分数在平均症状严重程度上下2.3至4.5个标准差和 -3.0至6.0个标准差范围内的信度均>0.80。对治疗的敏感性也相似（偏η²分别为0.23和0.22），与基线时CGI-S的相关性也相似（分别为0.45和0.48；无显著差异）。PANSS10与30项PANSS的平均项目平均差异为0.095，PANSS20为0.033。与30项PANSS相比，优化后的PANSS量表在信度、效度和校准方面继续表现出色。由于其长度和心理测量性能，研究人员应考虑用PANSS10替代30项PANSS作为临床疗效和筛查指标。
