Sally Dunaway Young1,2, Jacqueline Montes3,4, Samantha S Kramer5, Jonathan Marra3, Rachel Salazar3, Rosangel Cruz3, Claudia A Chiriboga3, Carol Ewing Garber5, Darryl C De Vivo3. 1. Department of Neurology, Columbia University Medical Center, New York, New York, USA. sld2120@columbia.edu. 2. Department of Rehabilitation and Regenerative Medicine, Columbia University Medical Center, New York, New York, USA. sld2120@columbia.edu. 3. Department of Neurology, Columbia University Medical Center, New York, New York, USA. 4. Department of Rehabilitation and Regenerative Medicine, Columbia University Medical Center, New York, New York, USA. 5. Department of Biobehavioral Sciences, Teachers College, Columbia University, New York, New York, USA.
Abstract
INTRODUCTION: The Six-Minute Walk Test (6MWT) was adopted as a clinical outcome measure for ambulatory spinal muscular atrophy (SMA). However, a systematic review of measurement properties reported significant variation among chronic pediatric conditions. Our purpose was to assess the reliability/validity of the 6MWT in SMA. METHODS: Thirty participants performed assessments, including the 6MWT, strength, and function. Reproducibility was evaluated by intraclass correlation coefficients. Criterion/convergent validity were determined using Pearson correlation coefficients. RESULTS: Test-retest reliability was excellent. The 6MWT was associated positively with peak oxygen uptake, Hammersmith Functional Motor Scale Expanded (HFMSE), lower extremity manual muscle testing, knee flexion hand-held dynamometry, and inversely with 10-m walk/run. The 6MWT discriminates between disease severity, unlike the HFMSE. CONCLUSIONS: This study documents measurement properties of reproducibility, positive criterion validity, and convergent validity with established clinical assessments and reaffirms the value of the 6MWT as a pivotal outcome measure in SMA clinical trials. Muscle Nerve 54: 836-842, 2016.
INTRODUCTION: The Six-Minute Walk Test (6MWT) was adopted as a clinical outcome measure for ambulatory spinal muscular atrophy (SMA). However, a systematic review of measurement properties reported significant variation among chronic pediatric conditions. Our purpose was to assess the reliability/validity of the 6MWT in SMA. METHODS: Thirty participants performed assessments, including the 6MWT, strength, and function. Reproducibility was evaluated by intraclass correlation coefficients. Criterion/convergent validity were determined using Pearson correlation coefficients. RESULTS: Test-retest reliability was excellent. The 6MWT was associated positively with peak oxygen uptake, Hammersmith Functional Motor Scale Expanded (HFMSE), lower extremity manual muscle testing, knee flexion hand-held dynamometry, and inversely with 10-m walk/run. The 6MWT discriminates between disease severity, unlike the HFMSE. CONCLUSIONS: This study documents measurement properties of reproducibility, positive criterion validity, and convergent validity with established clinical assessments and reaffirms the value of the 6MWT as a pivotal outcome measure in SMA clinical trials. Muscle Nerve 54: 836-842, 2016.
Authors: Ashley M Goodwin; Kayla M D Cornett; Marnee J McKay; Joshua Burns; Carol Ewing Garber; Darryl C De Vivo; Jacqueline Montes Journal: Muscle Nerve Date: 2020-01-16 Impact factor: 3.217
Authors: Jacqueline Montes; Ashley M Goodwin; Michael P McDermott; David Uher; Feliz Marie Hernandez; Kayla Coutts; Julia Cocchi; Margarethe Hauschildt; Kayla M Cornett; Ashwini K Rao; Umrao R Monani; Carol Ewing Garber; Darryl C De Vivo Journal: Ann Clin Transl Neurol Date: 2021-03-31 Impact factor: 5.430
Authors: Rafael Rodriguez-Torres; Julia Fabiano; Ashley Goodwin; Ashwini K Rao; Stacy Kinirons; Darryl De Vivo; Jacqueline Montes Journal: J Neuromuscul Dis Date: 2020
Authors: Maria Carmela Pera; Giorgia Coratti; Francesca Bovis; Marika Pane; Amy Pasternak; Jacqueline Montes; Valeria A Sansone; Sally Dunaway Young; Tina Duong; Sonia Messina; Irene Mizzoni; Adele D'Amico; Matthew Civitello; Allan M Glanzman; Claudio Bruno; Francesca Salmin; Simone Morando; Roberto De Sanctis; Maria Sframeli; Laura Antonaci; Anna Lia Frongia; Annemarie Rohwer; Mariacristina Scoto; Darryl C De Vivo; Basil T Darras; John Day; William Martens; Katia A Patanella; Enrico Bertini; Francesco Muntoni; Richard Finkel; Eugenio Mercuri Journal: Ann Clin Transl Neurol Date: 2021-06-24 Impact factor: 4.511