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Postural Body Sway as Surrogate Outcome for Myelopathy in Adrenoleukodystrophy.

ABSTRACT: Background:Myelopathy is the core clinical manifestation of adrenoleukodystrophy (ALD), which is the most common peroxisomal disorder. Development of therapies requires sensitive and clinically relevant outcome measures. Together with spastic paraparesis, balance disturbance is the main cause of disability from myelopathy in ALD. In this cross-sectional study, we evaluated whether postural body sway - a measure of balance - could serve as a surrogate outcome in clinical trials. Methods:Forty-eight male ALD patients and 49 age-matched healthy male controls were included in this study. We compared sway amplitude and sway path of ALD patients to controls. We then correlated the body sway parameters showing the largest between-group differences with clinical measures of severity of myelopathy. To correct for age, we performed multiple linear regression analysis with age and severity of myelopathy as independent variables. Results:All body sway parameters were significantly higher in patients than in controls, with medium to large effect sizes (r = 0.43-0.66, p < 0.001). In the subgroup of asymptomatic patients, body sway amplitude was also higher, but the difference with controls was smaller than for symptomatic patients (effect size r = 0.38-0.46). We found moderate to strong correlations between body sway amplitude and clinical severity of myelopathy (r = 0.40-0.79, p < 0.005). After correction for age, severity of myelopathy was a significant predictor of body sway amplitude in all regression models. Conclusions:These results indicate that postural body sway may serve as a surrogate outcome for myelopathy in ALD. Such outcomes are important to evaluate new therapies in clinical trials. Further longitudinal studies are needed and ongoing in this cohort.

SUBMITTER: van Ballegoij WJC

PROVIDER: S-EPMC7379508 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature

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Postural Body Sway as Surrogate Outcome for Myelopathy in Adrenoleukodystrophy.

van Ballegoij Wouter J C WJC van de Stadt Stephanie I W SIW Huffnagel Irene C IC Kemp Stephan S van der Knaap Marjo S MS Engelen Marc M

Frontiers in physiology 20200717

<h4>Background</h4>Myelopathy is the core clinical manifestation of adrenoleukodystrophy (ALD), which is the most common peroxisomal disorder. Development of therapies requires sensitive and clinically relevant outcome measures. Together with spastic paraparesis, balance disturbance is the main cause of disability from myelopathy in ALD. In this cross-sectional study, we evaluated whether postural body sway - a measure of balance - could serve as a surrogate outcome in clinical trials.<h4>Method ...[more]

PMID: 32765293

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Project description:ObjectiveAre people with a characteristically large physiological sway rendered particularly unstable when standing on a moving surface? Is postural sway in standing individuals idiosyncratic? In this study, we examine postural sway in individuals standing normally, and when subtle continuous sinusoidal disturbances are applied to their support platform. We calculate consistency between conditions to verify if sway can be considered characteristic of each individual. We also correlate two different aspects of participants' responses to disturbance; their sway velocity and their regulation of body orientation.MethodsNineteen healthy adults (age 29.2 ± 3.2 years) stood freely on footplates coaxially aligned with their ankles and attached to a motorized platform. They had their eyes closed, and hips and knees locked with a light wooden board attached to their body. Participants either stood quietly on a fixed platform or on a slowly tilting platform (0.1 Hz sinusoid; 0.2 and 0.4 deg). Postural sway size was separated into two entities: (1) the spontaneous sway velocity component (natural random relatively rapid postural adjustments, RMS body angular velocity) and (2) the evoked tilt gain component (much slower 0.1 Hz synchronous tilt induced by the movement of the platform, measured as peak-to-peak (p-p) gain, ratio of body angle to applied footplate rotation).ResultsThere was no correlation between the velocity of an individual's sway and their evoked tilt gain (r = 0.34, p = 0.15 and r = 0.30, p = 0.22). However, when considered separately, each of the two measurements showed fair to good absolute agreement within conditions. Spontaneous sway velocity consistently increased as participants were subjected to increasing disturbance. Participants who swayed more (or less) did so across all conditions [ICC(3,k) = 0.95]. Evoked tilt gain also showed consistency between conditions [ICC(3,k) = 0.79], but decreased from least to most disturbed conditions.ConclusionThe two measurements remain consistent between conditions. Consistency between conditions of two very distinct unrelated measurements reflects the idiosyncratic nature of postural sway. However, sway velocity and tilt gain are not related, which supports the idea that the short-term regulation of stability and the longer-term regulation of orientation are controlled by different processes.

Dataset Information

Postural Body Sway as Surrogate Outcome for Myelopathy in Adrenoleukodystrophy.

Publications

Postural Body Sway as Surrogate Outcome for Myelopathy in Adrenoleukodystrophy.

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