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Psychometric Validation of the Growth Hormone Deficiency–Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency–Parent Treatment Burden Measure (GHD-PTB)
PURPOSE: The aim was to evaluate the measurement properties of the Growth Hormone Deficiency–Child Treatment Burden Measure–Child (GHD-CTB–Child), a patient-reported outcome (PRO) for children aged 9 to < 13 years; the Growth Hormone Deficiency–Child Treatment Burden Measure–Observer (GHD-CTB–Obs...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Springer International Publishing
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9929004/ https://www.ncbi.nlm.nih.gov/pubmed/36255609 http://dx.doi.org/10.1007/s41669-022-00373-z |
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author | Brod, Meryl Rasmussen, Michael Højby Alolga, Suzanne Beck, Jane F. Bushnell, Donald M. Lee, Kai Wai Maniatis, Aristides |
author_facet | Brod, Meryl Rasmussen, Michael Højby Alolga, Suzanne Beck, Jane F. Bushnell, Donald M. Lee, Kai Wai Maniatis, Aristides |
author_sort | Brod, Meryl |
collection | PubMed |
description | PURPOSE: The aim was to evaluate the measurement properties of the Growth Hormone Deficiency–Child Treatment Burden Measure–Child (GHD-CTB–Child), a patient-reported outcome (PRO) for children aged 9 to < 13 years; the Growth Hormone Deficiency–Child Treatment Burden Measure–Observer (GHD-CTB–Observer), an observer-reported outcome (ObsRO) version completed by parents/guardians of children with growth hormone deficiency (GHD) aged 4 to < 9 years; and the Growth Hormone Deficiency–Parent Treatment Burden Measure (GHD-PTB), a PRO that assesses the treatment burden of parents/guardians living with children with GHD aged 4 to < 13 years. METHODS: A non-interventional, multi-center, clinic-based study across 30 private practice and large institutional sites in the United States and the United Kingdom was conducted. The sample consisted of 145 pre-pubertal children aged 9 to < 13 years at enrollment with a physician confirmed GHD diagnosis as well as 98 parents/guardians of pre-pubertal younger children aged 4 to < 9 years at enrollment with a physician confirmed GHD diagnosis. The child sample consisted of 59 treatment-naïve children (no prior exposure to growth hormone [GH] therapy; were starting GH treatment at study start per standard of care) and 184 children already maintained on treatment for at least 6 months. At baseline, all study participants completed a paper validation battery including all measures needed to conduct the validation analyses. Follow-up assessments with children in the maintenance group and their caregiver/parent were conducted approximately 2 weeks post-baseline to evaluate test–retest reproducibility. To evaluate sensitivity to change and meaningful change thresholds, treatment-naïve participants in both child and parent/guardian populations were assessed within 1 week of report of minimal improvement between week 3 and week 11 and at week 12. Psychometric analyses were implemented following an a priori statistical analysis plan. RESULTS: Factor analyses confirmed the a priori conceptual domains and Overall score for each measure (GHD-CTB–Child and GHD-CTB–Observer domains: Physical, Emotional Well-being, and Interference; GHD-PTB domains: Emotional Well-being and Interference). Internal consistency was acceptable for all measures (Cronbach’s alpha > 0.70). Test–retest reliability was acceptable for the Physical, Emotional, and Overall domains of the GHD-CTB versions, and the Emotional and Overall domains of the GHD-PTB (intraclass correlation coefficient above 0.70). All but one of the convergent validity hypotheses for the GHD-CTB versions and all hypotheses for the GHD-PTB were proven (r > 0.40). Known-groups validity hypotheses were significant for length of time to administer the injections in the GHD-CTB versions (p < 0.001 for Physical, Emotional, and Overall, and p < 0.01 for Interference) and whether parents/guardians versus child gave the injections more often for the Emotional domain of the GHD-PTB (p < 0.05). Associated effect sizes ranged from −0.27 to −0.57 for GHD-CTB versions and from −0.74 to −0.69 for the GHD-PTB, indicating that the measures are sensitive to change. Anchor-based patient and parent/guardian ratings of severity suggest preliminary meaningful change thresholds (GHD-CTB: 6 points for Physical score, 9 for Emotional, and 6 for Interference; GHD-PTB: 10 points for Emotional and 6 for Interference scores). CONCLUSIONS: The psychometric properties of the GHD-CTB–Child, GHD-CTB–Observer, and GHD-PTB support the validity of their use as PRO and ObsRO measures to capture the experiences associated with treatment burden for children with GHD and their parents/guardians in both clinical and research settings. The Clinicaltrials.gov registration number NCT02580032 was first posted October 20, 2015. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s41669-022-00373-z. |
format | Online Article Text |
id | pubmed-9929004 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | Springer International Publishing |
record_format | MEDLINE/PubMed |
spelling | pubmed-99290042023-02-16 Psychometric Validation of the Growth Hormone Deficiency–Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency–Parent Treatment Burden Measure (GHD-PTB) Brod, Meryl Rasmussen, Michael Højby Alolga, Suzanne Beck, Jane F. Bushnell, Donald M. Lee, Kai Wai Maniatis, Aristides Pharmacoecon Open Original Research Article PURPOSE: The aim was to evaluate the measurement properties of the Growth Hormone Deficiency–Child Treatment Burden Measure–Child (GHD-CTB–Child), a patient-reported outcome (PRO) for children aged 9 to < 13 years; the Growth Hormone Deficiency–Child Treatment Burden Measure–Observer (GHD-CTB–Observer), an observer-reported outcome (ObsRO) version completed by parents/guardians of children with growth hormone deficiency (GHD) aged 4 to < 9 years; and the Growth Hormone Deficiency–Parent Treatment Burden Measure (GHD-PTB), a PRO that assesses the treatment burden of parents/guardians living with children with GHD aged 4 to < 13 years. METHODS: A non-interventional, multi-center, clinic-based study across 30 private practice and large institutional sites in the United States and the United Kingdom was conducted. The sample consisted of 145 pre-pubertal children aged 9 to < 13 years at enrollment with a physician confirmed GHD diagnosis as well as 98 parents/guardians of pre-pubertal younger children aged 4 to < 9 years at enrollment with a physician confirmed GHD diagnosis. The child sample consisted of 59 treatment-naïve children (no prior exposure to growth hormone [GH] therapy; were starting GH treatment at study start per standard of care) and 184 children already maintained on treatment for at least 6 months. At baseline, all study participants completed a paper validation battery including all measures needed to conduct the validation analyses. Follow-up assessments with children in the maintenance group and their caregiver/parent were conducted approximately 2 weeks post-baseline to evaluate test–retest reproducibility. To evaluate sensitivity to change and meaningful change thresholds, treatment-naïve participants in both child and parent/guardian populations were assessed within 1 week of report of minimal improvement between week 3 and week 11 and at week 12. Psychometric analyses were implemented following an a priori statistical analysis plan. RESULTS: Factor analyses confirmed the a priori conceptual domains and Overall score for each measure (GHD-CTB–Child and GHD-CTB–Observer domains: Physical, Emotional Well-being, and Interference; GHD-PTB domains: Emotional Well-being and Interference). Internal consistency was acceptable for all measures (Cronbach’s alpha > 0.70). Test–retest reliability was acceptable for the Physical, Emotional, and Overall domains of the GHD-CTB versions, and the Emotional and Overall domains of the GHD-PTB (intraclass correlation coefficient above 0.70). All but one of the convergent validity hypotheses for the GHD-CTB versions and all hypotheses for the GHD-PTB were proven (r > 0.40). Known-groups validity hypotheses were significant for length of time to administer the injections in the GHD-CTB versions (p < 0.001 for Physical, Emotional, and Overall, and p < 0.01 for Interference) and whether parents/guardians versus child gave the injections more often for the Emotional domain of the GHD-PTB (p < 0.05). Associated effect sizes ranged from −0.27 to −0.57 for GHD-CTB versions and from −0.74 to −0.69 for the GHD-PTB, indicating that the measures are sensitive to change. Anchor-based patient and parent/guardian ratings of severity suggest preliminary meaningful change thresholds (GHD-CTB: 6 points for Physical score, 9 for Emotional, and 6 for Interference; GHD-PTB: 10 points for Emotional and 6 for Interference scores). CONCLUSIONS: The psychometric properties of the GHD-CTB–Child, GHD-CTB–Observer, and GHD-PTB support the validity of their use as PRO and ObsRO measures to capture the experiences associated with treatment burden for children with GHD and their parents/guardians in both clinical and research settings. The Clinicaltrials.gov registration number NCT02580032 was first posted October 20, 2015. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s41669-022-00373-z. Springer International Publishing 2022-10-18 /pmc/articles/PMC9929004/ /pubmed/36255609 http://dx.doi.org/10.1007/s41669-022-00373-z Text en © The Author(s) 2022 https://creativecommons.org/licenses/by-nc/4.0/Open AccessThis article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) . |
spellingShingle | Original Research Article Brod, Meryl Rasmussen, Michael Højby Alolga, Suzanne Beck, Jane F. Bushnell, Donald M. Lee, Kai Wai Maniatis, Aristides Psychometric Validation of the Growth Hormone Deficiency–Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency–Parent Treatment Burden Measure (GHD-PTB) |
title | Psychometric Validation of the Growth Hormone Deficiency–Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency–Parent Treatment Burden Measure (GHD-PTB) |
title_full | Psychometric Validation of the Growth Hormone Deficiency–Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency–Parent Treatment Burden Measure (GHD-PTB) |
title_fullStr | Psychometric Validation of the Growth Hormone Deficiency–Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency–Parent Treatment Burden Measure (GHD-PTB) |
title_full_unstemmed | Psychometric Validation of the Growth Hormone Deficiency–Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency–Parent Treatment Burden Measure (GHD-PTB) |
title_short | Psychometric Validation of the Growth Hormone Deficiency–Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency–Parent Treatment Burden Measure (GHD-PTB) |
title_sort | psychometric validation of the growth hormone deficiency–child treatment burden measure (ghd-ctb) and the growth hormone deficiency–parent treatment burden measure (ghd-ptb) |
topic | Original Research Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9929004/ https://www.ncbi.nlm.nih.gov/pubmed/36255609 http://dx.doi.org/10.1007/s41669-022-00373-z |
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