Evidence map›Paper›PMID 42004889›Full record

ReviewAlzheimer's & dementia (New York, N. Y.)

Neuroprotection trial design in progressive supranuclear palsy: challenges and solutions.

Lawrence I Golbe, Ronald G Thomas

Abstract readReview
In one paragraph

Review in Alzheimer's & dementia (New York, N. Y.). The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
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

2 citing papers in PubMed.

  1. Utility of Progression Rate Since Symptom Onset in Predicting Subsequent Survival in Progressive Supranuclear Palsy.Movement disorders : official journal of the Movement Disorder Society · 2026
    Article
  2. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

2 authors.

Lawrence I GolbeDepartment of Neurology Division of Movement Disorders Rutgers Robert Wood Johnson Medical School New Brunswick New Jersey USA.ORCID https://orcid.org/0000-0002-4373-3365
Ronald G ThomasDivision of Biostatistics and Bioinformatics Herbert Wertheim School of Public Health & Human Longevity Science, University of California San Diego San Diego California USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionWe review the specific challenges posed to neuroprotective trial design by progressive supranuclear palsy (PSP), a rare disorder difficult to diagnose and to assess quantitatively.

methodsFocusing on reducing the size and cost of trials, we review elements of PSP neuroprotection trial methodology and formulate a new minimum clinically important difference (MCID) for PSP to be used as a potential outcome milestone.

resultsWhile the original, 28-item version of the PSP Rating Scale (PSPRS) remains the best-validated and most widely published clinical outcome measure, promising improvements include the PSPRS-15 and -10; magnetic resonance imaging (MRI) volumetry; wearable movement-sensitive devices; platform, futility, basket, factorial, adaptive, and multistage designs; customized endpoints; baseline predictor stratification; and the replacement of a continuous disability scale with attainment of milestones, possibly including our newly calculated MCID. DISCUSSION: The specific assessment tools we deem most valuable could be used individually or in combination to reduce the required N and cost of PSP neuroprotection trials.

Indexed as

adaptive trialsbaseline predictor stratificationbasket trialscustomized endpointsfactorial trialsfutility trialsminimum clinically important differencemovement‐sensing wearable devicesMRI volumetrymult‐stage trialsneuroprotection trialsplatform trialsprogressive supranuclear palsy (PSP)PSP Rating Scalerating scales

Identifiers

PMID42004889
PMCPMC13084695

What OpenQuestion holds

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Registered trials

None linked

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.