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March 6, 2026Molecular Genetics and MetabolismOpen Access

The goal attainment scale in primary mitochondrial disease: Construct validity and lessons learned from a randomized controlled trial

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Authors

KLKristofoor E LeeuwenbergRadboud University NijmegenJIJoanna IntHoutRadboud University NijmegenJGJan T. GroothuisRadboud University Nijmegen

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Implication

A randomized controlled trial evaluates the construct validity of the Goal Attainment Scale in mitochondrial disease, suggesting implications for future trials.

Key Points

  • This research aims to assess the construct validity of the Goal Attainment Scale (GAS) in patients with primary mitochondrial diseases (PMD).
  • Retrospective analysis of data from a double-blind, randomized, placebo-controlled trial.
  • Comparison of GAS with the Canadian Occupational Performance Measure (COPM).
  • Categorization of GAS goals using WHO's International Classification of Functioning, Disability and Health.
  • Evaluation of implementation quality with GAS appraisal criteria.
  • GAS goals predominantly focused on fatigue or lack of energy (85% of goals).
  • Weak to moderate negative correlations were found with the Checklist Individual Strength and Beck Depression Inventory-II scores.
  • Moderate positive correlations observed between GAS and COPM scores.
  • No significant correlations with conventional outcome measures like the 6-min walk test or health surveys.
  • Only 6 out of 17 GAS implementation criteria were fully met.

Cite This Study

Leeuwenberg et al. (2026) studied this question.

synapsesocial.com/papers/69aa6eb1531e4c4a9ff58ef6https://doi.org/10.1016/j.ymgme.2026.109875
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