Evaluator Training and Reliability for SMA Global Nusinersen Trials

Allan M. Glanzmana; Elena S. Mazzone; Sally Dunaway Young; Richard Gee; Kristy Rose; Anna Mayhew; Leslie Nelson; Chris Yun; Katie Alexander; Basil T. Darras; Zarazuela Zolkipli-Cunningham; Gihan Tennekoon; John W. Day; Richard S. Finkel; Eugenio Mercuri; Darryl C. De Vivo; Ron Baldwin; Kathie M. Bishop; Jacqueline Montes

doi:10.3233/JND-180301

Evaluator Training and Reliability for SMA Global Nusinersen Trials

Allan M. Glanzmana, Elena S. Mazzone, Sally Dunaway Young, Richard Gee, Kristy Rose, Anna Mayhew, Leslie Nelson, Chris Yun, Katie Alexander, Basil T. Darras, Zarazuela Zolkipli-Cunningham, Gihan Tennekoon, John W. Day, Richard S. Finkel, Eugenio Mercuri, Darryl C. De Vivo, Ron Baldwin, Kathie M. Bishop, Jacqueline Montes

Physical Therapy

Research output: Contribution to journal › Article › peer-review

33 Scopus citations

Abstract

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.

Original language	English (US)
Pages (from-to)	159-166
Number of pages	8
Journal	Journal of Neuromuscular Diseases
Volume	5
Issue number	2
DOIs	https://doi.org/10.3233/JND-180301
State	Published - 2018

ASJC Scopus subject areas

Neurology
Clinical Neurology

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Glanzmana, A. M., Mazzone, E. S., Young, S. D., Gee, R., Rose, K., Mayhew, A., Nelson, L., Yun, C., Alexander, K., Darras, B. T., Zolkipli-Cunningham, Z., Tennekoon, G., Day, J. W., Finkel, R. S., Mercuri, E., De Vivo, D. C., Baldwin, R., Bishop, K. M., & Montes, J. (2018). Evaluator Training and Reliability for SMA Global Nusinersen Trials. Journal of Neuromuscular Diseases, 5(2), 159-166. https://doi.org/10.3233/JND-180301

Glanzmana, AM, Mazzone, ES, Young, SD, Gee, R, Rose, K, Mayhew, A, Nelson, L, Yun, C, Alexander, K, Darras, BT, Zolkipli-Cunningham, Z, Tennekoon, G, Day, JW, Finkel, RS, Mercuri, E, De Vivo, DC, Baldwin, R, Bishop, KM & Montes, J 2018, 'Evaluator Training and Reliability for SMA Global Nusinersen Trials', Journal of Neuromuscular Diseases, vol. 5, no. 2, pp. 159-166. https://doi.org/10.3233/JND-180301

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title = "Evaluator Training and Reliability for SMA Global Nusinersen Trials",

abstract = "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.",

author = "Glanzmana, {Allan M.} and Mazzone, {Elena S.} and Young, {Sally Dunaway} and Richard Gee and Kristy Rose and Anna Mayhew and Leslie Nelson and Chris Yun and Katie Alexander and Darras, {Basil T.} and Zarazuela Zolkipli-Cunningham and Gihan Tennekoon and Day, {John W.} and Finkel, {Richard S.} and Eugenio Mercuri and {De Vivo}, {Darryl C.} and Ron Baldwin and Bishop, {Kathie M.} and Jacqueline Montes",

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doi = "10.3233/JND-180301",

language = "English (US)",

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T1 - Evaluator Training and Reliability for SMA Global Nusinersen Trials

AU - Glanzmana, Allan M.

AU - Mazzone, Elena S.

AU - Young, Sally Dunaway

AU - Gee, Richard

AU - Rose, Kristy

AU - Mayhew, Anna

AU - Nelson, Leslie

AU - Yun, Chris

AU - Alexander, Katie

AU - Darras, Basil T.

AU - Zolkipli-Cunningham, Zarazuela

AU - Tennekoon, Gihan

AU - Day, John W.

AU - Finkel, Richard S.

AU - Mercuri, Eugenio

AU - De Vivo, Darryl C.

AU - Baldwin, Ron

AU - Bishop, Kathie M.

AU - Montes, Jacqueline

PY - 2018

Y1 - 2018

N2 - 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.

AB - 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.

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Evaluator Training and Reliability for SMA Global Nusinersen Trials

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