ArticleJournal of Huntington's disease2018
Disease Progression in Huntington Disease: An Analysis of Multiple Longitudinal Outcomes.
Article in Journal of Huntington's disease, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
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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.
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.
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Who cites it
2 citing papers in PubMed, 4 citations in OpenAlex.
- Movement Disorder Society Task Force Viewpoint: Huntington's Disease Diagnostic Categories.Movement disorders clinical practice · 2019Article
- Predicting the Risk of Huntington's Disease with Multiple Longitudinal Biomarkers.Journal of Huntington's disease · 2019Observational
Corrections and comments
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Authors and funding
5 authors at 5 institutions in 1 country.
Funding
Abstract
backgroundCritical to discovering targeted therapies for Huntington disease (HD) are validated methods that more precisely predict when clinical outcomes occur for different patient profiles.
objectiveTo more precisely predict the probability of when motor diagnosis (diagnostic confidence level 4) on the Unified Huntington's Disease Rating Scale (UHDRS), cognitive impairment (two or more neuropsychological scores on the UHDRS were 1.5 standard deviations below normative means) and Stage II Total Functional Capacity (TFC) first occur by accounting for dependencies between these outcomes.
methodsAdult premanifest participants with ≥36 CAG repeats were selected from multi-center, longitudinal, observational studies: Prospective Huntington At Risk Observational Study (PHAROS, n = 346), Neurobiological Predictors of Huntington Disease (PREDICT, n = 909); and Cooperative Huntington Observational Research Trial (COHORT, n = 430). Probabilities were estimated for each study, and pooled using the Joint Progression of Risk Assessment Tool (JPRAT) which accounts for dependencies between outcomes.
resultsAll studies had similar probabilities of when motor diagnosis, cognitive impairment, and Stage II TFC first occurred. Probability estimates from JPRAT were 43% less variable than from models that ignored dependencies between outcomes. The probability of experiencing motor-diagnosis, cognitive impairment, and Stage II TFC within 5 years was 10%, 18%, and 7%, respectively for 45-year-olds with 42 CAG repeats, and was 4%, 10% and 5%, respectively, for 40 year olds with 42 CAG repeats.
conclusionsImproved predictions from JPRAT may benefit treatment studies of rare diseases and is an alternative to composite outcomes when the objective is interpreting individual outcomes within the same model.
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Registered trials
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.