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使用儿科克罗恩病活动指数评估疾病活动度:我们能否仅使用主观或客观参数?

Assessing disease activity using the pediatric Crohn's disease activity index: Can we use subjective or objective parameters alone?

机构信息

Division of Pediatrics, IWK Health, Halifax, NS B3K6R8, Canada.

Division of Research, Connecticut Children's Medical Center, Hartford, CT 06106, United States.

出版信息

World J Gastroenterol. 2021 Aug 14;27(30):5100-5111. doi: 10.3748/wjg.v27.i30.5100.

Abstract

BACKGROUND

The pediatric Crohn's disease activity index (PCDAI) is used as a standard tool to assess disease activity in clinical trials for pediatric Crohn's disease.

AIM

To examine which items on the PCDAI drive assessment of disease activity, and how subgroups of subjective and objective items reflect change in disease state over time.

METHODS

Selective raw data from three prospectively collected datasets were combined, including 703 children with full PCDAI data at baseline, at 3-mo (Q1, = 670), and 1-year (Q4, = 474). Change in individual PCDAI scores from baseline to Q1 and to Q4 were examined using the non-weighted PCDAI.

RESULTS

Abdominal pain, well-being, weight, and stooling had the highest change scores over time. Objective indicators including albumin, abdominal exam, and height velocity followed. Change scores for well-being and abdominal exam did not explain significant variance at Q1 but were significant predictors at Q4 ( < 0.001 and < 0.05). Subjective and objective subgroups of items predicted less variance (18% and 22%) on total PCDAI scores at Q1 and Q4 compared to the full PCDAI, or a composite scale (both 32%) containing significant predictors.

CONCLUSION

Although subjective items on the PCDAI change the most over time, the full PCDAI or a smaller composite of items including a combination of subjective and objective components classifies disease activity better than a subgroup of either subjective or objective items alone. Reliance on subjective or objective items as stand-alone proxies for disease activity measurement could result in misclassification of disease state.

摘要

背景

儿科克罗恩病活动指数(PCDAI)被用作评估儿科克罗恩病临床试验中疾病活动的标准工具。

目的

研究 PCDAI 中的哪些项目可用于评估疾病活动,以及主观和客观项目的亚组如何反映疾病状态随时间的变化。

方法

选择三个前瞻性数据集的部分原始数据进行组合,包括基线时、3 个月时(Q1,n = 670)和 1 年时(Q4,n = 474)有完整 PCDAI 数据的 703 名儿童。使用非加权 PCDAI 检查从基线到 Q1 和 Q4 时 PCDAI 评分的个体变化。

结果

腹痛、舒适度、体重和排便的变化评分随时间推移最高。随后是客观指标,包括白蛋白、腹部检查和身高增速。舒适度和腹部检查的变化评分在 Q1 时没有解释显著的方差,但在 Q4 时是显著的预测因子(<0.001 和<0.05)。主观和客观项目亚组在 Q1 和 Q4 时对总 PCDAI 评分的预测方差(18%和 22%)低于全 PCDAI 或包含显著预测因子的综合评分(两者均为 32%)。

结论

尽管 PCDAI 中的主观项目随时间变化最大,但全 PCDAI 或包含主观和客观成分组合的较小综合评分比仅包含主观或客观项目的亚组能更好地分类疾病活动。仅依赖主观或客观项目作为疾病活动测量的独立替代指标可能导致疾病状态的错误分类。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c1d0/8384732/e60664ce81c1/WJG-27-5100-g001.jpg

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