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Evaluator Training and Reliability for SMA Global Nusinersen Trials(1)
BACKGROUND: Training methodology was established to optimize reliability of outcome measures in the nusinersen clinical trials. The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), Hammersmith Functional Motor Scale Expanded (HFMSE), and Revised Upper Limb (R...
Autores principales: | , , , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
IOS Press
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6030668/ https://www.ncbi.nlm.nih.gov/pubmed/29865090 http://dx.doi.org/10.3233/JND-180301 |
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author | Glanzman, Allan M. Mazzone, Elena S. Young, Sally Dunaway Gee, Richard Rose, Kristy Mayhew, Anna Nelson, Leslie Yun, Chris Alexander, Katie Darras, Basil T. Zolkipli-Cunningham, Zarazuela Tennekoon, Gihan Day, John W. Finkel, Richard S. Mercuri, Eugenio De Vivo, Darryl C. Baldwin, Ron Bishop, Kathie M. Montes, Jacqueline |
author_facet | Glanzman, Allan M. Mazzone, Elena S. Young, Sally Dunaway Gee, Richard Rose, Kristy Mayhew, Anna Nelson, Leslie Yun, Chris Alexander, Katie Darras, Basil T. Zolkipli-Cunningham, Zarazuela Tennekoon, Gihan Day, John W. Finkel, Richard S. Mercuri, Eugenio De Vivo, Darryl C. Baldwin, Ron Bishop, Kathie M. Montes, Jacqueline |
author_sort | Glanzman, Allan M. |
collection | PubMed |
description | BACKGROUND: Training methodology was established to optimize reliability of outcome measures in the nusinersen clinical trials. The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), Hammersmith Functional Motor Scale Expanded (HFMSE), and Revised Upper Limb (RULM) were primary or secondary outcomes. METHODS: Video review, quarterly conference calls, and item scoring checks supported evaluator competence. Baseline and screening along with video review established intra and inter-rater reliability. RESULTS: Inter and intra-rater reliability were both excellent. Intraclass correlation coefficients (ICC) ranged between 0.906–0.994 across initial training meetings and 0.824–0.996 across annual retraining meetings. This was similar for CHOP INTEND (ICC = 0.824–0.951), HFMSE (ICC = 0.981–0.996), and RULM (ICC = 0.966–0.990). Intra-rater reliability for the CHOP INTEND, HFMSE, and RULM were ICC = 0.895 (95% CI: 0.852–0.926; n = 116), ICC = 0.959 (95% CI: 0.942–0.971; n = 125), and ICC = 0.948 (95% CI: 0.927–0.963; n = 126) respectively. CONCLUSIONS: Rigorous evaluator training ensures reliability of assessment of subjects with spinal muscular atrophy (SMA) in multicenter international trials. |
format | Online Article Text |
id | pubmed-6030668 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2018 |
publisher | IOS Press |
record_format | MEDLINE/PubMed |
spelling | pubmed-60306682018-07-05 Evaluator Training and Reliability for SMA Global Nusinersen Trials(1) Glanzman, Allan M. Mazzone, Elena S. Young, Sally Dunaway Gee, Richard Rose, Kristy Mayhew, Anna Nelson, Leslie Yun, Chris Alexander, Katie Darras, Basil T. Zolkipli-Cunningham, Zarazuela Tennekoon, Gihan Day, John W. Finkel, Richard S. Mercuri, Eugenio De Vivo, Darryl C. Baldwin, Ron Bishop, Kathie M. Montes, Jacqueline J Neuromuscul Dis Research Report BACKGROUND: Training methodology was established to optimize reliability of outcome measures in the nusinersen clinical trials. The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), Hammersmith Functional Motor Scale Expanded (HFMSE), and Revised Upper Limb (RULM) were primary or secondary outcomes. METHODS: Video review, quarterly conference calls, and item scoring checks supported evaluator competence. Baseline and screening along with video review established intra and inter-rater reliability. RESULTS: Inter and intra-rater reliability were both excellent. Intraclass correlation coefficients (ICC) ranged between 0.906–0.994 across initial training meetings and 0.824–0.996 across annual retraining meetings. This was similar for CHOP INTEND (ICC = 0.824–0.951), HFMSE (ICC = 0.981–0.996), and RULM (ICC = 0.966–0.990). Intra-rater reliability for the CHOP INTEND, HFMSE, and RULM were ICC = 0.895 (95% CI: 0.852–0.926; n = 116), ICC = 0.959 (95% CI: 0.942–0.971; n = 125), and ICC = 0.948 (95% CI: 0.927–0.963; n = 126) respectively. CONCLUSIONS: Rigorous evaluator training ensures reliability of assessment of subjects with spinal muscular atrophy (SMA) in multicenter international trials. IOS Press 2018-05-29 /pmc/articles/PMC6030668/ /pubmed/29865090 http://dx.doi.org/10.3233/JND-180301 Text en © 2018 – IOS Press and the authors. All rights reserved https://creativecommons.org/licenses/by-nc/4.0/ This is an open access article distributed under the terms of the Creative Commons Attribution Non-Commercial (CC BY-NC 4.0) License (https://creativecommons.org/licenses/by-nc/4.0/) , which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Research Report Glanzman, Allan M. Mazzone, Elena S. Young, Sally Dunaway Gee, Richard Rose, Kristy Mayhew, Anna Nelson, Leslie Yun, Chris Alexander, Katie Darras, Basil T. Zolkipli-Cunningham, Zarazuela Tennekoon, Gihan Day, John W. Finkel, Richard S. Mercuri, Eugenio De Vivo, Darryl C. Baldwin, Ron Bishop, Kathie M. Montes, Jacqueline Evaluator Training and Reliability for SMA Global Nusinersen Trials(1) |
title | Evaluator Training and Reliability for SMA Global Nusinersen Trials(1) |
title_full | Evaluator Training and Reliability for SMA Global Nusinersen Trials(1) |
title_fullStr | Evaluator Training and Reliability for SMA Global Nusinersen Trials(1) |
title_full_unstemmed | Evaluator Training and Reliability for SMA Global Nusinersen Trials(1) |
title_short | Evaluator Training and Reliability for SMA Global Nusinersen Trials(1) |
title_sort | evaluator training and reliability for sma global nusinersen trials(1) |
topic | Research Report |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6030668/ https://www.ncbi.nlm.nih.gov/pubmed/29865090 http://dx.doi.org/10.3233/JND-180301 |
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