Kathie M Bishop1, Jacqueline Montes2, Richard S Finkel3. 1. Ionis Pharmaceuticals, Inc., Carlsbad, California, USA. 2. Departments of Rehabilitation and Regenerative Medicine and Neurology, Columbia University Medical Center, New York, New York, USA. 3. Division of Neurology, Department of Pediatrics, Nemours Children's Hospital, Orlando, Florida, USA.
Abstract
INTRODUCTION: In this study we examined the feasibility of assessing motor milestone performance of infants with spinal muscular atrophy (SMA) using the Hammersmith Infant Neurological Exam-Part 2 (HINE-2) in a phase 2 study of nusinersen. METHODS: Nineteen SMA infants were assessed using the HINE-2 at baseline (≤7 months of age), and periodically up to 39 months of age. We evaluated whether the HINE-2 was feasible, reliable, and sensitive to change. RESULTS: Motor milestone assessments in SMA infants were feasible using the HINE-2. Baseline test-retest reliability was excellent (R = 0.987; P < 0.0001). SMA infants were extremely low functioning at baseline and the HINE-2 was able to detect changes over time in 16 of 19 infants within all 8 domains. HINE-2 improvements were correlated with changes in other neuromuscular outcome measures. CONCLUSION: Results support the use of the HINE-2 motor milestone assessment in clinical trials of SMA infants. Muscle Nerve 57: 143-146, 2017.
INTRODUCTION: In this study we examined the feasibility of assessing motor milestone performance of infants with spinal muscular atrophy (SMA) using the Hammersmith Infant Neurological Exam-Part 2 (HINE-2) in a phase 2 study of nusinersen. METHODS: Nineteen SMA infants were assessed using the HINE-2 at baseline (≤7 months of age), and periodically up to 39 months of age. We evaluated whether the HINE-2 was feasible, reliable, and sensitive to change. RESULTS: Motor milestone assessments in SMA infants were feasible using the HINE-2. Baseline test-retest reliability was excellent (R = 0.987; P < 0.0001). SMA infants were extremely low functioning at baseline and the HINE-2 was able to detect changes over time in 16 of 19 infants within all 8 domains. HINE-2 improvements were correlated with changes in other neuromuscular outcome measures. CONCLUSION: Results support the use of the HINE-2 motor milestone assessment in clinical trials of SMA infants. Muscle Nerve 57: 143-146, 2017.
Authors: Bo Hoon Lee; Stella Deng; Claudia A Chiriboga; Denise M Kay; Obehioya Irumudomon; Emma Laureta; Leslie Delfiner; Simona O Treidler; Yaacov Anziska; Ai Sakonju; Chelsea Kois; Osman Farooq; Kristin Engelstad; Alexandra Laurenzano; Katherine Hogan; Michele Caggana; Carlos A Saavedra-Matiz; Colleen F Stevens; Emma Ciafaloni Journal: Neurology Date: 2022-07-14 Impact factor: 11.800
Authors: Kristin J Krosschell; Michael Bosch; Leslie Nelson; Tina Duong; Linda P Lowes; Lindsay N Alfano; Danielle Benjamin; Terri B Carry; Ginger Devine; Carolyn Kelley; Rebecca Gadekan; Elizabeth C Malkus; Amy Pasternak; Stephanie Provance-Orr; Lynne Roemeiser-Logan; Alina Nicorici; Donata Trussell; Sally Dunaway Young; Jennifer R Fetterman; Jacqueline Montes; Penny J Powers; Rebecca Quinones; Janet Quigley; Christopher S Coffey; Jon W Yankey; Amy Bartlett; John T Kissel; Stephen J Kolb Journal: J Neuromuscul Dis Date: 2018