Six-minute walk test is reliable and valid in spinal muscular atrophy

Young, Sally Dunaway; Montes, Jacqueline; Kramer, Samantha; Marra, Jonathan; Salazar, Rachel; Cruz, Rosángel; Chiriboga, Claudia A.; Garber, Carol Ewing; Vivo, Darryl C. De

doi:10.1002/mus.25120

Cited by 116 publications

(84 citation statements)

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“…Ambulatory children and adolescents treated with nusinersen in the CS2 multiple‐dose and CS12 extension studies demonstrated improvements in ambulatory function (6MWT), with clinically meaningful increases in walking distance and modest decreases or stabilization in fatigue. Although there is no precedent for improvements like these in individuals with SMA, changes of ≥30 meters in 6MWT distance are considered clinically meaningful and to impact everyday activities in other pediatric neuromuscular disorders …”

Section: Discussionmentioning

confidence: 99%

“…Tools assessing walking ability and fatigue are clinically relevant in individuals with milder forms of SMA . The 6‐minute walk test (6MWT) is a valid and clinically meaningful measure of ambulatory function and has demonstrated sensitivity to fatigue‐related changes in individuals with SMA . Fatigue measured during the 6MWT may represent underlying disease‐specific mechanisms, and improvements may represent treatment benefit.…”

Section: Introductionmentioning

confidence: 99%

“…Nusinersen is the first drug approved for the treatment of SMA, and has demonstrated significant and clinically meaningful efficacy by increasing event‐free survival, motor function, and achievement of motor milestones in infantile‐onset SMA, and improving/maintaining motor function in later‐onset SMA . Nusinersen has resulted in clinically meaningful improvements in walking ability (defined as ≥30‐meter increase from baseline in 6MWT distance) in children/adolescents with later‐onset SMA; however, the effects of nusinersen treatment on fatigability are unknown. In this post hoc analysis we further examined 6MWT performance and the relationship between distance walked and fatigue in the subgroup of nusinersen‐treated children and adolescents with SMA who were ambulatory during the CS2 dose‐escalation or CS12 extension studies and completed the 6MWT.…”

Section: Introductionmentioning

confidence: 99%

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Nusinersen improves walking distance and reduces fatigue in later‐onset spinal muscular atrophy

et al. 2019

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Introduction Ambulatory individuals with spinal muscular atrophy (SMA) experience muscle weakness, gait impairments, and fatigue that affect their walking ability. Improvements have been observed in motor function in children treated with nusinersen, but its impact on fatigue has not been studied. Methods Post hoc analyses were used to examine changes in 6‐minute walk test (6MWT) distance and fatigue in children and adolescents with SMA type II and III who received their first dose of nusinersen in the phase Ib/IIa, open‐label CS2 study and were ambulatory during CS2 or the extension study, CS12. Results Fourteen children performed the 6MWT. Median (25th, 75th percentile) distance walked increased over time by 98.0 (62.0, 135.0) meters at day 1050, whereas median fatigue changed by −3.8% (−19.7%, 1.4%). Discussion These results support previous studies demonstrating clinically meaningful effects of nusinersen on motor function in children and adolescents with later‐onset SMA.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Nusinersen improves walking distance and reduces fatigue in later‐onset spinal muscular atrophy

et al. 2019

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“…The 6‐minute walk test (6MWT) measures the maximum distance a person can walk in 6 minutes over a 25‐meter linear course. It has been shown to be a valid and reliable assessment of exercise capacity and functional walking ability, and it can capture physiological fatigue in patients with SMA . Distance walked over the entire 6‐minute time period, distance covered each minute, and the time to complete each 25‐meter interval were recorded.…”

Section: Methodsmentioning

confidence: 99%

Gait assessment with solesound instrumented footwear in spinal muscular atrophy

et al. 2017

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“…The 6‐Minute Walk Test (6MWT) is a self‐paced walking test that measures the distance an individual is able to walk on a hard, flat surface for 6 min . Originally developed for assessment of aerobic activity in patients with respiratory disease, the 6MWT is now validated in numerous other patient populations, including those with musculoskeletal diseases (eg, Duchenne/Becker muscular dystrophy, facioscapulohumeral muscular dystrophy, spinal muscle atrophy), and has been used as an efficacy measure in clinical studies of patients with muscular and metabolic disorders. The 6MWT has also been used as an outcome measure in clinical studies of patients with other rare diseases, such as Pompe disease, Hunter syndrome, and Morquio A syndrome .…”

Section: Introductionmentioning

confidence: 99%

Reliability and Validity of the 6‐Minute Walk Test in Hypophosphatasia

et al. 2019

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This investigation evaluated the reliability and validity of the 6‐Minute Walk Test (6MWT) in patients with pediatric hypophosphatasia (HPP). Children (aged 6 to 12 years; n = 11), adolescents (13 to 17 years; n = 4), and adults (18 to 65 years; n = 9) completed the 6MWT at screening and baseline in two clinical studies of asfotase alfa. Test‐retest reliability of the 6MWT, evaluated with Pearson's correlation coefficients ( r ) for screening versus baseline, was high for children ( r = 0.95; p < 0.0001), adolescents ( r = 0.81; p = 0.125), and adults ( r = 0.94; p = 0.0001). The most conservative minimal clinically important differences, estimated using distribution‐based methods, were 31 m (children and adults) and 43 m (adolescents). In children, the 6MWT correlated significantly with scores on measures of skeletal disease, which included the Radiographic Global Impression of Change scale ( r = 0.50; p < 0.0001) and the Rickets Severity Scale ( r = −0.78; p < 0.0001), such that distance walked increased as the severity of skeletal disease decreased. Significant ( p < 0.0001) correlations with the 6MWT distance walked were also observed for children with scores on parent‐reported measures of disability ( r = −0.67), ability to function in activities of daily living ( r = 0.71 to 0.77), and parent‐reported measures of pain ( r = −0.39). In adolescents and adults, 6MWT distance walked correlated significantly ( p < 0.05) with measures of lower extremity function ( r = 0.83 and 0.60, respectively), total pain severity ( r = −0.41 and −0.36, respectively), and total pain interference ( r = −0.41 and −0.49, respectively). Collectively, these data indicate that the 6MWT is a reliable, valid measure of physical functioning in patients with pediatric HPP. © 2018 The Authors. JBMR Plus Published by Wiley Periodicals, Inc. on behalf of the American Society for Bone and Mineral Research.

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Six-minute walk test is reliable and valid in spinal muscular atrophy

Abstract: 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.

Cited by 116 publications

References 54 publications

Nusinersen improves walking distance and reduces fatigue in later‐onset spinal muscular atrophy

Nusinersen improves walking distance and reduces fatigue in later‐onset spinal muscular atrophy

Gait assessment with solesound instrumented footwear in spinal muscular atrophy

Reliability and Validity of the 6‐Minute Walk Test in Hypophosphatasia

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