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在早期口吃治疗临床试验中,将口吃音节百分比与家长报告的严重程度评分进行比较作为主要结局指标。

Comparison of Percentage of Syllables Stuttered With Parent-Reported Severity Ratings as a Primary Outcome Measure in Clinical Trials of Early Stuttering Treatment.

作者信息

Onslow Mark, Jones Mark, O'Brian Sue, Packman Ann, Menzies Ross, Lowe Robyn, Arnott Simone, Bridgman Kate, de Sonneville Caroline, Franken Marie-Christine

机构信息

The University of Sydney, Australian Stuttering Research Centre, Lidcombe, New South Wales.

The University of Queensland, School of Public Health, Faculty of Medicine, Brisbane, Australia.

出版信息

J Speech Lang Hear Res. 2018 Apr 17;61(4):811-819. doi: 10.1044/2017_JSLHR-S-16-0448.

Abstract

PURPOSE

This report investigates whether parent-reported stuttering severity ratings (SRs) provide similar estimates of effect size as percentage of syllables stuttered (%SS) for randomized trials of early stuttering treatment with preschool children.

METHOD

Data sets from 3 randomized controlled trials of an early stuttering intervention were selected for analyses. Analyses included median changes and 95% confidence intervals per treatment group, Bland-Altman plots, analysis of covariance, and Spearman rho correlations.

RESULTS

Both SRs and %SS showed large effect sizes from pretreatment to follow-up, although correlations between the 2 measures were moderate at best. Absolute agreement between the 2 measures improved as percentage reduction of stuttering frequency and severity increased, probably due to innate measurement limitations for participants with low baseline severity. Analysis of covariance for the 3 trials showed consistent results.

CONCLUSION

There is no statistical reason to favor %SS over parent-reported stuttering SRs as primary outcomes for clinical trials of early stuttering treatment. However, there are logistical reasons to favor parent-reported stuttering SRs. We conclude that parent-reported rating of the child's typical stuttering severity for the week or month prior to each assessment is a justifiable alternative to %SS as a primary outcome measure in clinical trials of early stuttering treatment.

摘要

目的

本报告调查在学龄前儿童早期口吃治疗的随机试验中,家长报告的口吃严重程度评分(SRs)是否能提供与口吃音节百分比(%SS)相似的效应量估计。

方法

选取3项早期口吃干预随机对照试验的数据集进行分析。分析包括每个治疗组的中位数变化和95%置信区间、布兰德-奥特曼图、协方差分析以及斯皮尔曼等级相关分析。

结果

从治疗前到随访,SRs和%SS均显示出较大的效应量,尽管这两种测量方法之间的相关性充其量为中等。随着口吃频率和严重程度降低百分比的增加,这两种测量方法之间的绝对一致性有所改善,这可能是由于基线严重程度较低的参与者存在固有的测量局限性。对这3项试验的协方差分析显示结果一致。

结论

在早期口吃治疗的临床试验中,没有统计学理由支持将%SS作为主要结局指标而不采用家长报告的口吃SRs。然而,从后勤保障角度有理由支持家长报告的口吃SRs。我们得出结论,在早期口吃治疗的临床试验中,家长报告的每次评估前一周或一个月内孩子典型口吃严重程度的评分,是作为主要结局指标的%SS的合理替代指标。

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