Related Experiment Video

Updated: Jul 31, 2025

Whole-brain Segmentation and Change-point Analysis of Anatomical Brain MRI—Application in Premanifest Huntington's Disease
09:06

Whole-brain Segmentation and Change-point Analysis of Anatomical Brain MRI—Application in Premanifest Huntington's Disease

Published on: June 9, 2018

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Defining Clinical Meaningfulness in Huntington's Disease

Jamie L Hamilton1, James A Mills2, Glenn T Stebbins3

  • 1Clinical Department, CHDI Management/CHDI Foundation, Princeton, New Jersey, USA.

Movement Disorders : Official Journal of the Movement Disorder Society
|May 6, 2023
PubMed
Abstract

Insights

This study establishes minimal clinically important difference (MCID) thresholds for Huntington's disease (HD) progression. Findings aid in interpreting clinical outcomes and guiding treatment decisions for HD patients.

Area of Science:

  • Neurology
  • Clinical Trials
  • Patient-Reported Outcomes

Background:

  • Minimal Clinically Important Difference (MCID) quantifies the smallest meaningful change in patient outcomes.
  • Anchor-based MCID methods link outcome changes to patient-perceived clinical importance.

Purpose of the Study:

  • To estimate longitudinal MCID for key outcome measures in Huntington's disease (HD) Stages 2 and 3.
  • To establish thresholds for meaningful change in HD progression.

Main Methods:

  • Utilized data from 11,070 participants in the global Enroll-HD observational study.
  • Employed linear mixed-effects models to calculate MCID for motor, cognitive, and functional measures over 12-36 months.
  • Used the SF-12 physical component summary score as the anchor for clinical importance.

Main Results:

  • MCID estimates varied significantly by HD progression stage and time frame.
  • Higher disease stage and longer observation periods correlated with increased MCID values.
  • Provided specific MCID values, e.g., a 3.6-point increase on the UHDRS Total Motor Score over 24 months in Stage 2.

Conclusions:

  • This is the first study to define MCID thresholds for Huntington's disease.
  • Results will enhance clinical interpretation of HD studies and inform treatment recommendations.
  • Findings support improved clinical decision-making and clinical trial design in HD research.
Keywords:
Huntington's diseaseminimal clinically important differenceoutcome measurespatient experience dataquality of life

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