Evaluator Training and Reliability for SMA Global Nusinersen Trials1.

Content Provider	Europe PMC
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
Copyright Year	2018
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.Conclusions:Rigorous evaluator training ensures reliability of assessment of subjects with spinal muscular atrophy (SMA) in multicenter international trials.
Related Links	https://europepmc.org/backend/ptpmcrender.fcgi?accid=PMC6030668&blobtype=pdf
ISSN	22143599
Journal	Journal of Neuromuscular Diseases [J Neuromuscul Dis]
Volume Number	5
PubMed Central reference number	PMC6030668
Issue Number	2
PubMed reference number	29865090
e-ISSN	22143602
DOI	10.3233/jnd-180301
Language	English
Publisher	IOS Press
Publisher Date	2018-01-01
Publisher Place	Nieuwe Hemweg 6B, 1013 BG Amsterdam, The Netherlands
Access Restriction	Open
Rights License	This is an open access article distributed under the terms of the Creative Commons Attribution Non-Commercial (CC BY-NC 4.0) License, which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. © 2018 – IOS Press and the authors. All rights reserved
Subject Keyword	SMA spinal muscular atrophy nusinersen outcome measures outcome assessment reliability of results multicenter studies
Content Type	Text
Resource Type	Article
Subject	Neurology Neurology (clinical)