ArticleAnnals of neurology2020
Robust Markers and Sample Sizes for Multicenter Trials of Huntington Disease.
Article in Annals of neurology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 1 of them a synthesis that pooled it.
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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
14 citing papers in PubMed, 1 synthesis or guideline pooled it, 29 citations in OpenAlex.
- The presymptomatic phase of amyotrophic lateral sclerosis: are we merely scratching the surface?Journal of neurology · 2021Pooled it
- Brain Age as a New Measure of Disease Stratification in Huntington's Disease.Movement disorders : official journal of the Movement Disorder Society · 2025Article
- Optimizing Screening for Intrastriatal Interventions in Huntington's Disease Using Predictive Models.Movement disorders : official journal of the Movement Disorder Society · 2024Article
- Progression of atypical parkinsonian syndromes: PROSPECT-M-UK study implications for clinical trials.Brain : a journal of neurology · 2023Article
- The temporal event-based model: Learning event timelines in progressive diseases.Imaging neuroscience (Cambridge, Mass.) · 2023Article
- Longitudinal imaging highlights preferential basal ganglia circuit atrophy in Huntington's disease.Brain communications · 2023Article
- Revealing the Timeline of Structural MRI Changes in Premanifest to Manifest Huntington Disease.Neurology. Genetics · 2021Article
- Tracking Huntington's Disease Progression Using Motor, Functional, Cognitive, and Imaging Markers.Movement disorders : official journal of the Movement Disorder Society · 2021Article
- hiPSCs for predictive modelling of neurodegenerative diseases: dreaming the possible.Nature reviews. Neurology · 2021Review
- Neuroimaging, Urinary, and Plasma Biomarkers of Treatment Response in Huntington's Disease: Preclinical Evidence with the p75Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2021Article
- A Multi-Study Model-Based Evaluation of the Sequence of Imaging and Clinical Biomarker Changes in Huntington's Disease.Frontiers in big data · 2021Article
- What is the Pathogenic CAG Expansion Length in Huntington's Disease?Journal of Huntington's disease · 2021Review
- Drugging DNA Damage Repair Pathways for Trinucleotide Repeat Expansion Diseases.Journal of Huntington's disease · 2021Review
- Rate of torque development and striatal shape in individuals with prodromal Huntington's disease.Scientific reports · 2020Article
Corrections and comments
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Authors and funding
15 authors at 7 institutions in 3 countries.
Funding
Abstract
objectiveThe identification of sensitive biomarkers is essential to validate therapeutics for Huntington disease (HD). We directly compare structural imaging markers across the largest collective imaging HD dataset to identify a set of imaging markers robust to multicenter variation and to derive upper estimates on sample sizes for clinical trials in HD.
methodsWe used 1 postprocessing pipeline to retrospectively analyze T1-weighted magnetic resonance imaging (MRI) scans from 624 participants at 3 time points, from the PREDICT-HD, TRACK-HD, and IMAGE-HD studies. We used mixed effects models to adjust regional brain volumes for covariates, calculate effect sizes, and simulate possible treatment effects in disease-affected anatomical regions. We used our model to estimate the statistical power of possible treatment effects for anatomical regions and clinical markers.
resultsWe identified a set of common anatomical regions that have similarly large standardized effect sizes (>0.5) between healthy control and premanifest HD (PreHD) groups. These included subcortical, white matter, and cortical regions and nonventricular cerebrospinal fluid (CSF). We also observed a consistent spatial distribution of effect size by region across the whole brain. We found that multicenter studies were necessary to capture treatment effect variance; for a 20% treatment effect, power of >80% was achieved for the caudate (n = 661), pallidum (n = 687), and nonventricular CSF (n = 939), and, crucially, these imaging markers provided greater power than standard clinical markers.
interpretationOur findings provide the first cross-study validation of structural imaging markers in HD, supporting the use of these measurements as endpoints for both observational studies and clinical trials. ANN NEUROL 2020;87:751-762.
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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.