Motor Function Test Reliability During the NeuroNEXT Spinal Muscular Atrophy Infant Biomarker Study

Article type: Research Article

Authors: Krosschell, Kristin J.^{a; b; *} | Bosch, Michael^c | Nelson, Leslie^d | Duong, Tina^e | Lowes, Linda P.^f | Alfano, Lindsay N.^f | Benjamin, Danielle^g | Carry, Terri B.^h | Devine, Gingerⁱ | Kelley, Carolyn^h | Gadekan, Rebecca^j | Malkus, Elizabeth C.^k | Pasternak, Amy^{l; m} | Provance-Orr, Stephanieⁿ | Roemeiser-Logan, Lynne^o | Nicorici, Alina^p | Trussell, Donata^q | Young, Sally Dunaway ^r | Fetterman, Jennifer R.^o | Montes, Jacqueline^r | Powers, Penny J.ⁱ | Quinones, Rebecca^s | Quigley, Janet^{l; m} | Coffey, Christopher S.^c | Yankey, Jon W.^c | Bartlett, Amy^t | Kissel, John T.^t | Kolb, Stephen J.^{t; u} | the NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators

Affiliations: [a] Department of Physical Therapy and Human Movement Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA | [b] Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL, USA | [c] Department of Biostatistics, NeuroNEXT Data Coordinating Center, University of Iowa, Iowa City, IA, USA | [d] Physical Therapy, UT Southwestern Medical Center, Dallas, TX, USA | [e] Department of Neurology, Stanford University, Palo Alto, CA, USA | [f] Neurology, Nationwide Children’s Hospital, Columbus, OH, USA | [g] Physical Therapy, Oregon Health and Science University, Portland, OR, USA | [h] Physical Therapy, Children’s Hospital Colorado, Aurora, CO, USA | [i] Pi Beta Phi Rehabilitation Institute, Vanderbilt University Medical Center, Nashville, TN, USA | [j] Neuromuscular Division, Washington University School of Medicine, St. Louis, MO, USA | [k] Ultragenyx, Novato, CA, USA | [l] The Department of Physical Therapy and Occupational Therapy Services, Boston Children’s Hospital, Boston, MA, USA | [m] The Department of Neurology, Boston Children’s Hospital, Boston, MA, USA | [n] Physical and Occupational Therapy, Children’s Mercy Hospital, Kansas City, MO, USA | [o] SUNY Upstate Medical University, Syracuse, NY, USA | [p] Physical Medicine and Rehabilitation, University of California – Davis, Davis, CA, USA | [q] University of Utah, Salt Lake City, UT, USA | [r] Departments of Neurology and Rehabilitation and Regenerative Medicine, Columbia University Medical Center, New York, NY, USA | [s] Physical and Occupational Therapy, Duke Health, Durham, NC, USA | [t] Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, USA | [u] Department of Biological Chemistry and Pharmacology, The Ohio State University Wexner Medical Center, Columbus, OH, USA

Correspondence: [*] Correspondence to: Kristin J. Krosschell, PT, DPT, MA, PCS, Associate Professor, Department of Physical Therapy and Human Movement Sciences and Department of Pediatrics, Suite 1100, 645 N Michigan Ave, Chicago, IL 60611, USA. E-mail: [email protected].

Abstract: Background:The NeuroNEXT SMA Infant Biomarker Study, a two year, longitudinal, multi-center study of infants with SMA type 1 and healthy infants, presented a unique opportunity to assess multi-site rater reliability on three infant motor function tests (MFTs) commonly used to assess infants with SMA type 1. Objective:To determine the effect of prospective MFT rater training and the effect of rater experience on inter-rater and intra-rater reliability for the Test of Infant Motor Performance Screening Items (TIMPSI), the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) and the Alberta Infant Motor Scale (AIMS). Methods:Training was conducted utilizing a novel set of motor function test (MFT) videos to optimize accurate MFT administration and reliability for the study duration. Inter- and intra-rater reliability of scoring for the TIMPSI and inter-rater reliability of scoring for the CHOP INTEND and the AIMS was assessed using intraclass correlation coefficients (ICC). Effect of rater experience on reliability was examined using ICC. Agreement with ‘expert’ consensus scores was examined using Pearson’s correlation coefficients. Results:Inter-rater reliability on all MFTs was good to excellent. Intra-rater reliability for the primary MFT, the TIMPSI, was excellent for the study duration. Agreement with ‘expert’ consensus was within predetermined limits (≥85%) after training. Evaluator experience with SMA and MFTs did not affect reliability. Conclusions:Reliability of scores across evaluators was demonstrated for all three study MFTs and scores were reproducible on repeated administration. Evaluator experience had no effect on reliability.

Keywords: Spinal muscular atrophy, motor function testing, outcome measures, neuromuscular diseases, TIMPSI, CHOP-INTEND, AIMS, clinical evaluator, NeuroNEXT, reliability

DOI: 10.3233/JND-180327

Journal: Journal of Neuromuscular Diseases, vol. 5, no. 4, pp. 509-521, 2018