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Psychometric validation of patient-reported outcome measures assessing chronic constipation

BACKGROUND: Measures assessing treatment outcomes in previous CC clinical trials have not met the requirements described in the US Food and Drug Administration’s guidance on patient-reported outcomes. AIM: Psychometric analyses using data from one Phase IIb study and two Phase III trials of linaclot...

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Autores principales: Nelson, Lauren M, Williams, Valerie SL, Fehnel, Sheri E, Carson, Robyn T, MacDougall, James, Baird, Mollie J, Tourkodimitris, Stavros, Kurtz, Caroline B, Johnston, Jeffrey M
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove Medical Press 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4186570/
https://www.ncbi.nlm.nih.gov/pubmed/25298737
http://dx.doi.org/10.2147/CEG.S64713
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author Nelson, Lauren M
Williams, Valerie SL
Fehnel, Sheri E
Carson, Robyn T
MacDougall, James
Baird, Mollie J
Tourkodimitris, Stavros
Kurtz, Caroline B
Johnston, Jeffrey M
author_facet Nelson, Lauren M
Williams, Valerie SL
Fehnel, Sheri E
Carson, Robyn T
MacDougall, James
Baird, Mollie J
Tourkodimitris, Stavros
Kurtz, Caroline B
Johnston, Jeffrey M
author_sort Nelson, Lauren M
collection PubMed
description BACKGROUND: Measures assessing treatment outcomes in previous CC clinical trials have not met the requirements described in the US Food and Drug Administration’s guidance on patient-reported outcomes. AIM: Psychometric analyses using data from one Phase IIb study and two Phase III trials of linaclotide for the treatment of chronic constipation (CC) were conducted to document the measurement properties of patient-reported CC Symptom Severity Measures. STUDY METHODS: Each study had a multicenter, randomized, double-blind, placebo-controlled, parallel-group design, comparing placebo to four doses of oral linaclotide taken once daily for 4 weeks in the Phase IIb dose-ranging study (n=307) and to two doses of linaclotide taken once daily for 12 weeks in the Phase III trials (n=1,272). The CC Symptom Severity Measures addressing bowel function (Bowel Movement Frequency, Stool Consistency, Straining) and abdominal symptoms (Bloating, Abdominal Discomfort, Abdominal Pain) were administered daily using interactive voice-response system technology. Intraclass correlations, Pearson correlations, factor analyses, F-tests, and effect sizes were computed. RESULTS: The CC Symptom Severity Measures demonstrated satisfactory test–retest reliability and construct validity. Factor analyses indicated one factor for abdominal symptoms and another for bowel symptoms. Known-groups F-tests substantiated the discriminating ability of the CC Symptom Severity Measures. Responsiveness statistics were moderate to strong, indicating that these measures are capable of detecting change. CONCLUSION: In large studies of CC patients, linaclotide significantly improved abdominal and bowel symptoms. These psychometric analyses support the reliability, validity, discriminating ability, and responsiveness of the CC Symptom Severity Measures for evaluating treatment outcomes in the linaclotide clinical studies.
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spelling pubmed-41865702014-10-08 Psychometric validation of patient-reported outcome measures assessing chronic constipation Nelson, Lauren M Williams, Valerie SL Fehnel, Sheri E Carson, Robyn T MacDougall, James Baird, Mollie J Tourkodimitris, Stavros Kurtz, Caroline B Johnston, Jeffrey M Clin Exp Gastroenterol Original Research BACKGROUND: Measures assessing treatment outcomes in previous CC clinical trials have not met the requirements described in the US Food and Drug Administration’s guidance on patient-reported outcomes. AIM: Psychometric analyses using data from one Phase IIb study and two Phase III trials of linaclotide for the treatment of chronic constipation (CC) were conducted to document the measurement properties of patient-reported CC Symptom Severity Measures. STUDY METHODS: Each study had a multicenter, randomized, double-blind, placebo-controlled, parallel-group design, comparing placebo to four doses of oral linaclotide taken once daily for 4 weeks in the Phase IIb dose-ranging study (n=307) and to two doses of linaclotide taken once daily for 12 weeks in the Phase III trials (n=1,272). The CC Symptom Severity Measures addressing bowel function (Bowel Movement Frequency, Stool Consistency, Straining) and abdominal symptoms (Bloating, Abdominal Discomfort, Abdominal Pain) were administered daily using interactive voice-response system technology. Intraclass correlations, Pearson correlations, factor analyses, F-tests, and effect sizes were computed. RESULTS: The CC Symptom Severity Measures demonstrated satisfactory test–retest reliability and construct validity. Factor analyses indicated one factor for abdominal symptoms and another for bowel symptoms. Known-groups F-tests substantiated the discriminating ability of the CC Symptom Severity Measures. Responsiveness statistics were moderate to strong, indicating that these measures are capable of detecting change. CONCLUSION: In large studies of CC patients, linaclotide significantly improved abdominal and bowel symptoms. These psychometric analyses support the reliability, validity, discriminating ability, and responsiveness of the CC Symptom Severity Measures for evaluating treatment outcomes in the linaclotide clinical studies. Dove Medical Press 2014-09-26 /pmc/articles/PMC4186570/ /pubmed/25298737 http://dx.doi.org/10.2147/CEG.S64713 Text en © 2014 Nelson et al. This work is published by Dove Medical Press Limited, and licensed under Creative Commons Attribution – Non Commercial (unported, v3.0) License The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed.
spellingShingle Original Research
Nelson, Lauren M
Williams, Valerie SL
Fehnel, Sheri E
Carson, Robyn T
MacDougall, James
Baird, Mollie J
Tourkodimitris, Stavros
Kurtz, Caroline B
Johnston, Jeffrey M
Psychometric validation of patient-reported outcome measures assessing chronic constipation
title Psychometric validation of patient-reported outcome measures assessing chronic constipation
title_full Psychometric validation of patient-reported outcome measures assessing chronic constipation
title_fullStr Psychometric validation of patient-reported outcome measures assessing chronic constipation
title_full_unstemmed Psychometric validation of patient-reported outcome measures assessing chronic constipation
title_short Psychometric validation of patient-reported outcome measures assessing chronic constipation
title_sort psychometric validation of patient-reported outcome measures assessing chronic constipation
topic Original Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4186570/
https://www.ncbi.nlm.nih.gov/pubmed/25298737
http://dx.doi.org/10.2147/CEG.S64713
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