The goal attainment scale in primary mitochondrial disease: Construct validity and lessons learned from a randomized controlled trial
GAS demonstrated some construct validity in relation to fatigue and depressive symptoms, showed limited overlap with conventional outcome measures and suffered from suboptimal implementation. Although exploratory, these findings suggest GAS may capture patient-relevant change in individuals with PMD. To realize its potential, standardized methodology and further validation are essential for its use as a robust outcome measure in future PMD trials.
Authors
- Edith H. C. Cup (ORCID: https://orcid.org/0000-0003-3452-9650)
- Karlien Mul (ORCID: https://orcid.org/0000-0001-8487-9478)
- Jan T. Groothuis (ORCID: https://orcid.org/0000-0003-3436-2475)
- Joanna IntHout (ORCID: https://orcid.org/0000-0002-6127-0747)
- Kristofoor E Leeuwenberg (ORCID: https://orcid.org/0000-0003-2204-308X)
- Jan Smeitink
- Mirian C.H. Janssen
Institutions
- Radboud University Nijmegen (NL)
- Radboud University Medical Center (NL)
- Radboud Institute for Molecular Life Sciences (NL)
- Khondrion (Netherlands) (NL)
Publication Details
- Journal
- Molecular Genetics and Metabolism
- Published
- 2026-03-03
- DOI
- https://doi.org/10.1016/j.ymgme.2026.109875
- Primary Topic
- Mitochondrial Function and Pathology
- Type
- article
- Field-Weighted Citation Impact
- 0.00