Pediatric Gastroenterology Unit, Shaare Zedek Medical Center, Hebrew University of Jerusalem, Israel.
Inflamm Bowel Dis. 2012 Jan;18(1):55-62. doi: 10.1002/ibd.21649. Epub 2011 Feb 23.
The Pediatric Crohn's Disease Activity Index (PCDAI) has become the standard outcome measure in pediatric Crohn's disease (CD) clinical research. Other versions have been proposed but without systematic evaluation. The aim was to assess validity and responsiveness of the abbreviated PCDAI (abbrPCDAI), short PCDAI (shPCDAI), and modified PCDAI (modPCDAI) as measures of disease activity and to compare these with a mathematically weighted version developed here (wPCDAI).
The raw data from four prospectively collected datasets were used, totaling 437 children with CD (including two clinical trials). Discriminant validity utilized physician global assessment of disease activity (PGA), and construct validity the correlation with PGA and laboratory results. Feasibility and face validity were ascertained by a survey of 33 experts in pediatric CD.
The wPCDAI had better performance than the PCDAI in construct validity and responsiveness and it discriminated better between the disease activity categories (area under the receiver operator characteristic [ROC] 0.97; 95% confidence interval [CI]: 0.95-0.99). In comparison to the original PCDAI, the noninvasive versions (abbrPCDAI and shPCDAI) had lower face, construct, and discriminant validity but were judged to be significantly more feasible. The modPCDAI performed well in the construct validation but was consistently inferior in all other parameters. Cutoffs that correspond to remission, response, and gradations of disease activity were determined for each index.
The newly weighted wPCDAI performed better than the original PCDAI and is more feasible. The noninvasive versions (shPCDAI and abbrPCDAI) are inferior to the full PCDAI, but when needed in retrospective studies either may be equally used.
儿科克罗恩病活动指数(PCDAI)已成为儿科克罗恩病(CD)临床研究中的标准结局衡量指标。其他版本也已被提出,但未经系统评估。目的是评估简化版 PCDAI(abbrPCDAI)、短版 PCDAI(shPCDAI)和改良版 PCDAI(modPCDAI)作为疾病活动衡量指标的有效性和反应性,并将其与这里开发的数学加权版(wPCDAI)进行比较。
使用四个前瞻性收集数据集的原始数据,总共有 437 名 CD 儿童(包括两项临床试验)。鉴别效度利用医师对疾病活动的整体评估(PGA),结构效度则与 PGA 和实验室结果的相关性。通过对 33 名儿科 CD 专家进行调查,确定了可行性和表面效度。
wPCDAI 在结构效度和反应性方面的表现优于 PCDAI,并且在疾病活动类别之间的区分更好(接受者操作特征曲线下的面积为 0.97;95%置信区间:0.95-0.99)。与原始 PCDAI 相比,非侵入性版本(abbrPCDAI 和 shPCDAI)的表面、结构和鉴别效度较低,但被认为更可行。改良版 PCDAI 在结构验证方面表现良好,但在所有其他参数方面均始终较差。为每个指数确定了对应于缓解、反应和疾病活动程度的切点。
新加权的 wPCDAI 比原始 PCDAI 表现更好,且更可行。非侵入性版本(shPCDAI 和 abbrPCDAI)劣于完整的 PCDAI,但在回顾性研究中需要时,两者都可以同等使用。