Muscle & nerve · 2026
At-Home Versus in-Clinic Vital Capacity Measurement: Insights From the HEALEY ALS Platform Trial.
СтатьяСравнительное исследованиеНазвание и аннотация: Перевод в очереди
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Macklin Eric A, Berry James D, Harkey Brittney A, Heyd Lindsay, Chase Marianne, Yu Hong, Sherman Alexander V, Dagostino Derek, Kittle Gale, Hall Meghan, Connolly Mariah R, Drake Kristin, Giacomelli Elisa, Scirocco Erica, Sharma Saloni, Babu Suma, Benatar Michael, Simmons Zachary, Young Eufrosina, Cudkowicz Merit E, Shefner Jeremy, Paganoni Sabrina, HEALEY ALS Platform Trial Study Group
Аннотация
INTRODUCTION/AIMS: Respiratory weakness, typically monitored as vital capacity (VC), is a central feature of amyotrophic lateral sclerosis (ALS). VC is increasingly measured remotely in participants' homes, although in-clinic assessment remains the standard. We tested concordance between at-home and in-clinic VC to determine trial eligibility, track progression, and predict survival in a large ALS trial.
METHODS: At-home and in-clinic VC were assessed at baseline and approximately every 8 weeks for a year in the first four regimens of the HEALEY ALS Platform Trial. At-home assessments were coached via live videoconference and centrally reviewed. VC measurements, expressed as percent of predicted normal (%PN), were compared cross-sectionally, longitudinally, and for predicting survival time. Data from 233 participants with 3-8 paired at-home and in-clinic VC assessments completed < 14 days apart were analyzed.
RESULTS: At-home and in-clinic VC were well correlated (Lin's r c = 0.82) with no systematic bias. At-home VC ≥ 60%PN predicted in-clinic VC ≥ 60%PN with a positive predictive value of 91% and a negative predictive value of 65%. VC slopes were moderately correlated (r c = 0.68). At-home VC progressed 28% faster than in-clinic VC with proportionately less variance (at-home [SE] = -1.882 [0.153] %PN/month, in-clinic = -1.476 [0.131] %PN/month). Slopes of at-home and in-clinic VC explained 15% and 17% of variation in future survival time, respectively.
DISCUSSION: At-home and in-clinic VC were well correlated cross-sectionally. At-home VC performed well tracking longitudinal change and predicting survival. The reduced participant burden of assessment and concordance with in-clinic measurement support use of at-home monitoring of VC.
METHODS: At-home and in-clinic VC were assessed at baseline and approximately every 8 weeks for a year in the first four regimens of the HEALEY ALS Platform Trial. At-home assessments were coached via live videoconference and centrally reviewed. VC measurements, expressed as percent of predicted normal (%PN), were compared cross-sectionally, longitudinally, and for predicting survival time. Data from 233 participants with 3-8 paired at-home and in-clinic VC assessments completed < 14 days apart were analyzed.
RESULTS: At-home and in-clinic VC were well correlated (Lin's r c = 0.82) with no systematic bias. At-home VC ≥ 60%PN predicted in-clinic VC ≥ 60%PN with a positive predictive value of 91% and a negative predictive value of 65%. VC slopes were moderately correlated (r c = 0.68). At-home VC progressed 28% faster than in-clinic VC with proportionately less variance (at-home [SE] = -1.882 [0.153] %PN/month, in-clinic = -1.476 [0.131] %PN/month). Slopes of at-home and in-clinic VC explained 15% and 17% of variation in future survival time, respectively.
DISCUSSION: At-home and in-clinic VC were well correlated cross-sectionally. At-home VC performed well tracking longitudinal change and predicting survival. The reduced participant burden of assessment and concordance with in-clinic measurement support use of at-home monitoring of VC.