Comparison of percentage of syllables stuttered with parent-reported severity ratings as a primary outcome measure in clinical trials of early stuttering treatment

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

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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.

Original languageEnglish
Pages (from-to)811-819
Number of pages9
JournalJournal of Speech, Language, and Hearing Research
Volume61
Issue number4
DOIs
Publication statusPublished - 17 Apr 2018
Externally publishedYes

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Stuttering
parents
rating
Outcome Assessment (Health Care)
Clinical Trials
Therapeutics
preschool child
confidence
Rating
Stuttering Treatment
Preschool Children
Randomized Controlled Trials
Confidence Intervals
Group

Cite this

Onslow, Mark ; Jones, Mark ; O’Brian, Sue ; Packman, Ann ; Menzies, Ross ; Lowe, Robyn ; Arnott, Simone ; Bridgman, Kate ; de Sonneville, Caroline ; Franken, Marie Christine. / Comparison of percentage of syllables stuttered with parent-reported severity ratings as a primary outcome measure in clinical trials of early stuttering treatment. In: Journal of Speech, Language, and Hearing Research. 2018 ; Vol. 61, No. 4. pp. 811-819.
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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.",
author = "Mark Onslow and Mark Jones and Sue O’Brian and Ann Packman and Ross Menzies and Robyn Lowe and Simone Arnott and Kate Bridgman and {de Sonneville}, Caroline and Franken, {Marie Christine}",
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Comparison of percentage of syllables stuttered with parent-reported severity ratings as a primary outcome measure in clinical trials of early stuttering treatment. / Onslow, Mark; Jones, Mark; O’Brian, Sue; Packman, Ann; Menzies, Ross; Lowe, Robyn; Arnott, Simone; Bridgman, Kate; de Sonneville, Caroline; Franken, Marie Christine.

In: Journal of Speech, Language, and Hearing Research, Vol. 61, No. 4, 17.04.2018, p. 811-819.

Research output: Contribution to journalArticleResearchpeer-review

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AU - Menzies, Ross

AU - Lowe, Robyn

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N2 - 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.

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