SynthesisJournal of Huntington's disease2014
Understanding the Outcomes Measures used in Huntington Disease Pharmacological Trials: A Systematic Review.
Synthesis in Journal of Huntington's disease, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 2 of them syntheses that pooled 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.
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.
Who cites it
20 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- A systematic review of performance-based functional capacity measures for use in Huntington's disease and evaluation of their suitability for clinical trials.Journal of Huntington's disease · 2025Pooled it
- The use of wearable/portable digital sensors in Huntington's disease: A systematic review.Parkinsonism & related disorders · 2021Pooled it
- Assessing the efficacy of dextromethorphan/quinidine in treating irritability in Huntington's disease.Journal of Huntington's disease · 2026Trial
- Sample enrichment for clinical trials to show delay of onset in huntington disease.Movement disorders : official journal of the Movement Disorder Society · 2019Trial
- Do cognitive tests capture symptoms that matter to people with Huntington's disease?Scientific reports · 2025Article
- Huntington's Disease Research Over Six Decades: Global Insights, Gaps, and Future Directions.Cureus · 2025Review
- Small RNAs in plasma extracellular vesicles define biomarkers of premanifest changes in Huntington's disease.Journal of extracellular vesicles · 2024Article
- Objectively characterizing Huntington's disease using a novel upper limb dexterity test.Journal of neurology · 2021Article
- Plasma neurofilament light in Huntington's disease: A marker for disease onset, but not symptom progression.Parkinsonism & related disorders · 2021Article
- Psychometric Properties and Validation of the Polish Version of the 12-Item World Health Organization Disability Assessment Schedule 2.0 in Patients with Huntington's Disease.Journal of clinical medicine · 2021Article
- Understanding speech and swallowing difficulties in individuals with Huntington disease: Validation of the HDQLIFE Speech Difficulties and Swallowing Difficulties Item Banks.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2021Article
- Responsiveness to change over time and test-retest reliability of the PROMIS and Neuro-QoL mental health measures in persons with Huntington disease (HD).Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2020Article
- Therapeutic Update on Huntington's Disease: Symptomatic Treatments and Emerging Disease-Modifying Therapies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2020Review
- Perceptions of the impact of chorea on health-related quality of life in Huntington disease (HD): A qualitative analysis of individuals across the HD spectrum, family members, and clinicians.Neuropsychological rehabilitation · 2020Article
- Psychometric properties and responsiveness of Neuro-QoL Cognitive Function in persons with Huntington disease (HD).Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2020Article
- Rapid and robust patterns of spontaneous locomotor deficits in mouse models of Huntington's disease.PloS one · 2020Article
- Rethinking Functional Outcome Measures: The Development of a Novel Upper Limb Token Transfer Test to Assess Basal Ganglia Dysfunction.Frontiers in neuroscience · 2018Article
- HDQLIFE: development and assessment of health-related quality of life in Huntington disease (HD).Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2016Article
- Validity of the 12-item World Health Organization Disability Assessment Schedule 2.0 (WHODAS 2.0) in individuals with Huntington disease (HD).Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2015Article
- Irritability in Huntington's Disease: Factor Analysis of Snaith's Irritability Scale.Movement disorders clinical practiceArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
Abstract
backgroundThe identification of the gene mutation causing Huntington disease has raised hopes for new treatments to ease symptoms and slow functional decline. As such, there has been a push towards designing efficient pharmacological trials (i.e., drug trials), especially with regard to selecting outcomes measures that are both brief and sensitive to changes across the course of the disease, from subtle prodromal changes, to more severe end-stage changes.
objectivesRecently, to aid in efficient development of new HD research studies, the National Institute of Neurological Disorders and Stroke (NINDS) published recommendations for measurement selection in HD. While these recommendations are helpful, many of the recommended measures have little published data in HD. As such, we conducted a systematic review of the literature to identify the most common outcomes measures used in HD clinical trials.
methodsMajor medical databases, including PubMed, Embase, CINAHL, and the Cochrane Central Register of Controlled Trials, were used to identify peer-reviewed journal articles in English from 2001 through April 2013; 151 pharmacological trials were identified.
resultsThe majority of HD clinical trials employed clinician-reported outcomes measures (93%); patient reported outcome measures (11%) and observer reported outcome measures (3%) were used with much less frequency.
conclusionsWe provide a review of the most commonly used measures across these trials, compare these measures to the clinical recommendations made by the NINDS working groups, and provide recommendations for selecting measures for future clinical trials that meet the Food and Drug Administration standards.
Indexed as
Identifiers
What OpenQuestion holds
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.