Evaluator Training and Reliability for SMA Global Nusinersen Trials

• DOI: 10.3233/jnd-180301
• Publication Date: 2018-05-29T15:23:55Z
• Authors: Allan M. Glanzman; Elena S. Mazzone; Sally Dunaway Young; Richard Gee; Kristy Rose; Anna Mayhew; Leslie Nelson; Chris Yun; Katie Alexander; Basil T. Darras; Zarazuela Zolkipl...; Gihan Tennekoon; John W. Day; Richard S. Finkel; Eugenio Mercuri; Darryl C. De Vivo; Ron Baldwin; Kathie M. Bishop; Jacqueline Montes
• Keywords: Research Report; Observer Variation; 0301 basic medicine; Clinical Trials as Topic; Teaching; Oligonucleotides; Infant; Reproducibility of Results; Oligonucleotides, Antisense; Spinal Muscular Atrophies of Childhood; 3. Good health; Physical Therapists; 03 medical and health sciences; Humans; SMA; Clinical Competence

Background: Training methodology was established to optimize reliability of outcome measures in the nusinersen clinical trials.The Children's Hospital Philadelphia Infant Test 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, item scoring checks supported evaluator competence.Baseline screening along with video review intra inter-rater reliability.Results: Inter intra-rater both excellent.Intraclass correlation coefficients (ICC) ranged between 0.906-0.994across initial training meetings 0.824-0.996across annual retraining meetings.This similar for CHOP INTEND (ICC = 0.824-0.951),HFMSE 0.981-0.996),and RULM 0.966-0.990).Intra-rater INTEND, HFMSE, ICC 0.895 (95%CI: 0.852-0.926;n 116), 0.959 0.942-0.971;n 125), 0.948 0.927-0.963;n 126) respectively. 1We confirm that we have read Journal's position on issues involved ethical publication affirm this report is consistent those guidelines.