Evidence map›Paper›PMID 41665609›Full record

ArticleAnnals of clinical and translational neurology2026

Value of MRI Outcomes for Preventive and Early-Stage Trials in Spinocerebellar Ataxias 1 and 3.

Thiago J R Rezende, Emilien Petit, Young Woo Park, Sophie Tezenas du Montcel, James M Joers, Jonathan M DuBois, H Moore Arnold, Michal Povazan, Ipek Özdemir, Guita Banan and 17 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Annals of clinical and translational neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Review
  2. Early and Progressive Spinal Cord Atrophy in Spinocerebellar Ataxia Type 1.Movement disorders : official journal of the Movement Disorder Society · 2026
    Article
  3. Article
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

27 authors.

Thiago J R RezendeDepartment of Neurology, School of Medical Sciences, University of Campinas, Campinas, Brazil.ORCID https://orcid.org/0000-0001-8453-0313
Emilien PetitInserm, INRIA, CNRS, APHP, Paris Brain Institute, Sorbonne Université, Paris, France.
Young Woo ParkDepartment of Radiology, Center for Magnetic Resonance Research, University of Minnesota, Minneapolis, Minnesota, USA.
Sophie Tezenas du MontcelInserm, INRIA, CNRS, APHP, Paris Brain Institute, Sorbonne Université, Paris, France.ORCID https://orcid.org/0000-0002-2866-4330
James M JoersDepartment of Radiology, Center for Magnetic Resonance Research, University of Minnesota, Minneapolis, Minnesota, USA.ORCID https://orcid.org/0000-0002-8444-2185
Jonathan M DuBoisBiogen, Cambridge, Massachusetts, USA.
H Moore ArnoldBiogen, Cambridge, Massachusetts, USA.
Michal PovazanDepartment of Radiology and Radiological Sciences, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Ipek ÖzdemirDepartment of Radiology and Radiological Sciences, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Guita BananNorman Fixel Institute for Neurological Disorders, College of Medicine, University of Florida, Gainesville, Florida, USA.
Romain ValabregueInserm, INRIA, CNRS, APHP, Paris Brain Institute, Sorbonne Université, Paris, France.
Philipp EhsesGerman Center for Neurodegenerative Diseases (DZNE), Bonn, Germany.
Jennifer FaberGerman Center for Neurodegenerative Diseases (DZNE), Bonn, Germany.
Chiadi U OnyikeDepartment of Psychiatry and Behavioral Sciences, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Peter B BarkerDepartment of Radiology and Radiological Sciences, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Jeremy D SchmahmannAtaxia Center, Laboratory for Neuroanatomy and Cerebellar Neurobiology, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, Massachusetts, USA.
Eva-Maria RataiDepartment of Radiology, A. A. Martinos Center for Biomedical Imaging, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Sub H SubramonyNorman Fixel Institute for Neurological Disorders, College of Medicine, University of Florida, Gainesville, Florida, USA.ORCID https://orcid.org/0000-0002-8052-9240
Thomas H MareciNorman Fixel Institute for Neurological Disorders, College of Medicine, University of Florida, Gainesville, Florida, USA.
Khalaf O BusharaDepartment of Radiology, Center for Magnetic Resonance Research, University of Minnesota, Minneapolis, Minnesota, USA.
Henry PaulsonDepartment of Neurology, University of Michigan, Ann Arbor, Michigan, USA.
Thomas KlockgetherGerman Center for Neurodegenerative Diseases (DZNE), Bonn, Germany.
Alexandra DurrInserm, INRIA, CNRS, APHP, Paris Brain Institute, Sorbonne Université, Paris, France.
Tetsuo AshizawaStanley H. Appel Department of Neurology, Houston Methodist Research Institute and Weill Cornell Medicine, Houston, Texas, USA.
Christophe LengletDepartment of Radiology, Center for Magnetic Resonance Research, University of Minnesota, Minneapolis, Minnesota, USA.
Gülin ÖzDepartment of Radiology, Center for Magnetic Resonance Research, University of Minnesota, Minneapolis, Minnesota, USA.ORCID https://orcid.org/0000-0002-5769-183X
READISCA Investigators

Funding

University of Minnesota Clinical and Translational Science Institute (UMN CTSI)UL1TR000114 · NCATS · UNIVERSITY OF MINNESOTA · PI BLAZAR, BRUCE R · 2012 to 2015
$35.0M
TRD4 - Ultrahigh Field Engineering and SafetyP41EB027061 · NIBIB · UNIVERSITY OF MINNESOTA · PI Mehmet Akcakaya · 2019 to 2026
$11.7M
Supplementary funding for U01NS104326 Clinical Trial Readiness for SCA1 and SCA3 (“READISCA”)U01NS104326 · NINDS · METHODIST HOSPITAL RESEARCH INSTITUTE · PI ASHIZAWA, TETSUO, DURR, ALEXANDRA · 2018 to 2022
$6.1M
Institutional Center Cores for Advanced NeuroimagingP30NS076408 · NINDS · UNIVERSITY OF MINNESOTA · PI UGURBIL, KAMIL · 2012 to 2020
$4.2M
Advanced 3T for Structural and Functional Imaging at University of FloridaS10OD021726 · OD · UNIVERSITY OF FLORIDA · PI LONG, JOANNA R · 2017 to 2017
$2.0M
High Performance Connectome Upgrade for Human 3T MR ScannerS10OD017974 · OD · UNIVERSITY OF MINNESOTA · PI LIM, KELVIN O. · 2014 to 2014
$600k
BiogenNational Science Foundation DMR-1644779NCATS NIH HHS UL1 TR000114NIBIB NIH HHS P41 EB027061NIH HHS S10 OD017974NIH HHS S10 OD021726NIH HHS UL1TR000114NINDS NIH HHS P30 NS076408NINDS NIH HHS U01 NS104326ODCDC CDC HHS S10 OD017974University of Minnesota
6 · The paper itself

Abstract

objectiveTo examine the value of MRI outcomes as endpoints for preventive and early-stage trials of two polyglutamine spinocerebellar ataxias (SCAs).

methodsA cohort of 100 participants (23 SCA1, 63 SCA3, median Scale for the Assessment and Rating of Ataxia (SARA) score = 5, 42% preataxic, and 14 gene-negative controls) was scanned at 3T up to 6 times (median follow-up 3 years) in the READISCA study. Structural, microstructural, and neurochemical outcomes were assessed for sensitivity to change. Associations between change in MR and clinical and patient-reported outcomes were evaluated. Sample sizes were estimated for preventive trials (at preataxic stage) and early-stage trials using the most sensitive outcomes.

resultsInfratentorial volumes, middle cerebellar peduncle (MCP) diffusivities and select neurochemical outcomes were more sensitive to change than SARA in both SCAs with moderate-to-high effect sizes (Cohen's d ≥ 0.5). The pons volume was the most sensitive outcome at both preataxic (d = 1.09) and ataxic (d = 1.48) stages. The most sensitive MR measures overlapped between genotypes, except that SCA1 showed faster progression in the cerebellum and SCA3 in the pons. Changes in MCP diffusivities and pontine neurochemical measures were associated with changes in SARA and FARS-ADL (|r| = 0.28-0.47).

interpretationThe estimated sample sizes to detect a 50% reduction in progression with 80% power using the pons volume as primary outcome measure indicate the feasibility of preventive trials (n = 73 per arm in a 1-year trial) in common SCAs and predict a 6-fold reduction in required sample sizes relative to SARA in early interventional trials. These findings support the use of MRI endpoints in early-stage SCA trials.

Indexed as

Machado-Joseph DiseaseMagnetic Resonance ImagingMiddle Cerebellar PeduncleOutcome Assessment, Health CarePonsSpinocerebellar AtaxiasAdultDisease ProgressionFemaleHumansMaleMiddle AgedlongitudinalMRIspinocerebellar ataxia

Identifiers

PMID41665609
PMCPMC13358550

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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.