Development of a Rasch-Built Amyotrophic Lateral Sclerosis Impairment Multidomain Scale to Measure Disease Progression in ALS.

Autor:	de Jongh AD; From the Department of Neurology (A.D.d.J., R.P.A.v.E., L.A.B., T.M.B., M.A.v.E., J.H.V., L.H.v.d.B.), UMC Utrecht Brain Center, University Medical Center Utrecht; Department of Neurology (A.D.d.J.), Tergooi Hospital, Blaricum; Biostatistics & Research Support (R.P.A.v.E.), Julius Center for Health Sciences and Primary Care, Department of Rehabilitation, Physical Therapy Science and Sports (L.A.B., A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, and Center of Excellence for Rehabilitation Medicine (A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, University Medical Center Utrecht, and De Hoogstraat Rehabilitation; and ALS Patients Connected (C.v.d.M.), Bilthoven, the Netherlands., van Eijk RPA; From the Department of Neurology (A.D.d.J., R.P.A.v.E., L.A.B., T.M.B., M.A.v.E., J.H.V., L.H.v.d.B.), UMC Utrecht Brain Center, University Medical Center Utrecht; Department of Neurology (A.D.d.J.), Tergooi Hospital, Blaricum; Biostatistics & Research Support (R.P.A.v.E.), Julius Center for Health Sciences and Primary Care, Department of Rehabilitation, Physical Therapy Science and Sports (L.A.B., A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, and Center of Excellence for Rehabilitation Medicine (A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, University Medical Center Utrecht, and De Hoogstraat Rehabilitation; and ALS Patients Connected (C.v.d.M.), Bilthoven, the Netherlands., Bakker LA; From the Department of Neurology (A.D.d.J., R.P.A.v.E., L.A.B., T.M.B., M.A.v.E., J.H.V., L.H.v.d.B.), UMC Utrecht Brain Center, University Medical Center Utrecht; Department of Neurology (A.D.d.J.), Tergooi Hospital, Blaricum; Biostatistics & Research Support (R.P.A.v.E.), Julius Center for Health Sciences and Primary Care, Department of Rehabilitation, Physical Therapy Science and Sports (L.A.B., A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, and Center of Excellence for Rehabilitation Medicine (A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, University Medical Center Utrecht, and De Hoogstraat Rehabilitation; and ALS Patients Connected (C.v.d.M.), Bilthoven, the Netherlands., Bunte TM; From the Department of Neurology (A.D.d.J., R.P.A.v.E., L.A.B., T.M.B., M.A.v.E., J.H.V., L.H.v.d.B.), UMC Utrecht Brain Center, University Medical Center Utrecht; Department of Neurology (A.D.d.J.), Tergooi Hospital, Blaricum; Biostatistics & Research Support (R.P.A.v.E.), Julius Center for Health Sciences and Primary Care, Department of Rehabilitation, Physical Therapy Science and Sports (L.A.B., A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, and Center of Excellence for Rehabilitation Medicine (A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, University Medical Center Utrecht, and De Hoogstraat Rehabilitation; and ALS Patients Connected (C.v.d.M.), Bilthoven, the Netherlands., Beelen A; From the Department of Neurology (A.D.d.J., R.P.A.v.E., L.A.B., T.M.B., M.A.v.E., J.H.V., L.H.v.d.B.), UMC Utrecht Brain Center, University Medical Center Utrecht; Department of Neurology (A.D.d.J.), Tergooi Hospital, Blaricum; Biostatistics & Research Support (R.P.A.v.E.), Julius Center for Health Sciences and Primary Care, Department of Rehabilitation, Physical Therapy Science and Sports (L.A.B., A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, and Center of Excellence for Rehabilitation Medicine (A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, University Medical Center Utrecht, and De Hoogstraat Rehabilitation; and ALS Patients Connected (C.v.d.M.), Bilthoven, the Netherlands., van der Meijden C; From the Department of Neurology (A.D.d.J., R.P.A.v.E., L.A.B., T.M.B., M.A.v.E., J.H.V., L.H.v.d.B.), UMC Utrecht Brain Center, University Medical Center Utrecht; Department of Neurology (A.D.d.J.), Tergooi Hospital, Blaricum; Biostatistics & Research Support (R.P.A.v.E.), Julius Center for Health Sciences and Primary Care, Department of Rehabilitation, Physical Therapy Science and Sports (L.A.B., A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, and Center of Excellence for Rehabilitation Medicine (A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, University Medical Center Utrecht, and De Hoogstraat Rehabilitation; and ALS Patients Connected (C.v.d.M.), Bilthoven, the Netherlands., van Es MA; From the Department of Neurology (A.D.d.J., R.P.A.v.E., L.A.B., T.M.B., M.A.v.E., J.H.V., L.H.v.d.B.), UMC Utrecht Brain Center, University Medical Center Utrecht; Department of Neurology (A.D.d.J.), Tergooi Hospital, Blaricum; Biostatistics & Research Support (R.P.A.v.E.), Julius Center for Health Sciences and Primary Care, Department of Rehabilitation, Physical Therapy Science and Sports (L.A.B., A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, and Center of Excellence for Rehabilitation Medicine (A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, University Medical Center Utrecht, and De Hoogstraat Rehabilitation; and ALS Patients Connected (C.v.d.M.), Bilthoven, the Netherlands., Visser-Meily JMA; From the Department of Neurology (A.D.d.J., R.P.A.v.E., L.A.B., T.M.B., M.A.v.E., J.H.V., L.H.v.d.B.), UMC Utrecht Brain Center, University Medical Center Utrecht; Department of Neurology (A.D.d.J.), Tergooi Hospital, Blaricum; Biostatistics & Research Support (R.P.A.v.E.), Julius Center for Health Sciences and Primary Care, Department of Rehabilitation, Physical Therapy Science and Sports (L.A.B., A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, and Center of Excellence for Rehabilitation Medicine (A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, University Medical Center Utrecht, and De Hoogstraat Rehabilitation; and ALS Patients Connected (C.v.d.M.), Bilthoven, the Netherlands., Kruitwagen ET; From the Department of Neurology (A.D.d.J., R.P.A.v.E., L.A.B., T.M.B., M.A.v.E., J.H.V., L.H.v.d.B.), UMC Utrecht Brain Center, University Medical Center Utrecht; Department of Neurology (A.D.d.J.), Tergooi Hospital, Blaricum; Biostatistics & Research Support (R.P.A.v.E.), Julius Center for Health Sciences and Primary Care, Department of Rehabilitation, Physical Therapy Science and Sports (L.A.B., A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, and Center of Excellence for Rehabilitation Medicine (A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, University Medical Center Utrecht, and De Hoogstraat Rehabilitation; and ALS Patients Connected (C.v.d.M.), Bilthoven, the Netherlands., Veldink JH; From the Department of Neurology (A.D.d.J., R.P.A.v.E., L.A.B., T.M.B., M.A.v.E., J.H.V., L.H.v.d.B.), UMC Utrecht Brain Center, University Medical Center Utrecht; Department of Neurology (A.D.d.J.), Tergooi Hospital, Blaricum; Biostatistics & Research Support (R.P.A.v.E.), Julius Center for Health Sciences and Primary Care, Department of Rehabilitation, Physical Therapy Science and Sports (L.A.B., A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, and Center of Excellence for Rehabilitation Medicine (A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, University Medical Center Utrecht, and De Hoogstraat Rehabilitation; and ALS Patients Connected (C.v.d.M.), Bilthoven, the Netherlands., van den Berg LH; From the Department of Neurology (A.D.d.J., R.P.A.v.E., L.A.B., T.M.B., M.A.v.E., J.H.V., L.H.v.d.B.), UMC Utrecht Brain Center, University Medical Center Utrecht; Department of Neurology (A.D.d.J.), Tergooi Hospital, Blaricum; Biostatistics & Research Support (R.P.A.v.E.), Julius Center for Health Sciences and Primary Care, Department of Rehabilitation, Physical Therapy Science and Sports (L.A.B., A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, and Center of Excellence for Rehabilitation Medicine (A.B., J.M.A.V.-M., E.T.K.), UMC Utrecht Brain Center, University Medical Center Utrecht, and De Hoogstraat Rehabilitation; and ALS Patients Connected (C.v.d.M.), Bilthoven, the Netherlands. lberg@umcutrecht.nl.
Jazyk:	angličtina
Zdroj:	Neurology [Neurology] 2023 Aug 08; Vol. 101 (6), pp. e602-e612. Date of Electronic Publication: 2023 Jun 13.
DOI:	10.1212/WNL.0000000000207483
Abstrakt:	Background and Objectives: Current scales used in amyotrophic lateral sclerosis (ALS) attempt to summarize different functional domains or "dimensions" into 1 overall score, which may not accurately characterize the individual patient's disease severity or prognosis. The use of composite score risks declaring treatments ineffective if not all dimensions of ALS disease progression are affected equally. We aimed to develop the ALS Impairment Multidomain Scale (AIMS) to comprehensively characterize disease progression and increase the likelihood of identifying effective treatments. Methods: The Revised ALS Functional Rating Scale (ALSFRS-R) and a preliminary questionnaire, based on literature review and patient input, were completed online by patients from the Netherlands ALS registry at bimonthly intervals over a period of 12 months. A 2-week test-retest, factor analysis, Rasch analysis, and a signal-to-noise optimization strategy were performed to create a multidomain scale. Reliability, longitudinal decline, and associations with survival were evaluated. The sample size required to detect a 35% reduction in progression rate over 6 or 12 months was assessed for a clinical trial that defines the ALSFRS-R or AIMS subscales as a primary endpoint family. Results: The preliminary questionnaire, consisting of 110 questions, was completed by 367 patients. Three unidimensional subscales were identified, and a multidomain scale was constructed with 7 bulbar, 11 motor, and 5 respiratory questions. Subscales fulfilled Rasch model requirements, with excellent test-retest reliability of 0.91-0.94 and a strong relationship with survival ( p < 0.001). Compared with the ALSFRS-R, signal-to-noise ratios were higher as patients declined more uniformly per subscale. Consequently, the estimated sample size reductions achieved with the AIMS compared with those achieved with the ALSFRS-R were 16.3% and 25.9% for 6-month and 12-month clinical trials, respectively. Discussion: We developed the AIMS, consisting of unidimensional bulbar, motor, and respiratory subscales, which may characterize disease severity better than a total score. AIMS subscales have high test-retest reliability, are optimized to measure disease progression, and are strongly related to survival time. The AIMS can be easily administered and may increase the likelihood of identifying effective treatments in ALS clinical trials. (Copyright © 2023 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology.)
Databáze:	MEDLINE
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