Evidence map›Paper›PMID 37311649›Full record

ArticleNeurology2023

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

Adriaan D de Jongh, Ruben P A van Eijk, Leonhard A Bakker, Tommy M Bunte, Anita Beelen, Conny van der Meijden, Michael A van Es, Johanna M A Visser-Meily, Esther T Kruitwagen, Jan H Veldink and 1 more

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In one paragraph

Article in Neurology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
2.9field-weighted citation impact, top 9% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed, 14 citations in OpenAlex.

  1. Review
  2. Estimating the minimum important difference in the ALSFRS-R-instrument in people living with MND.Amyotrophic lateral sclerosis & frontotemporal degeneration · 2025
    Observational
  3. Article
  4. Review
  5. Article
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  7. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

11 authors at 1 institution in 3 countries.

Adriaan D de JonghFrom 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.ORCID 0000-0002-3658-3397
Ruben P A van EijkFrom 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.ORCID 0000-0002-7132-5967
Leonhard A BakkerFrom 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.ORCID 0000-0001-5221-5095
Tommy M BunteFrom 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.
Anita BeelenFrom 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.
Conny van der MeijdenFrom 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.
Michael A van EsFrom 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.
Johanna M A Visser-MeilyFrom 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.ORCID 0000-0002-5955-8012
Esther T KruitwagenFrom 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.
Jan H VeldinkFrom 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.ORCID 0000-0001-5572-9657
Leonard H van den BergFrom 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.
University Medical Center · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

BACKGROUND AND

objectivesCurrent 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.

methodsThe 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.

resultsThe 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 ( 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.

Indexed as

Amyotrophic Lateral SclerosisDisease ProgressionHumansProbabilityPrognosisReproducibility of Results

Identifiers

PMID37311649
PMCPMC10424842
OpenAlexW4380446690

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.