Journal
MITOCHONDRION
Volume 13, Issue 1, Pages 15-24Publisher
ELSEVIER SCI LTD
DOI: 10.1016/j.mito.2012.11.002
Keywords
Mitochondrial disease; Outcome measures; Clinical trial; Patient perspective
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Funding
- ZonMW (The Netherlands Organization for Health Research and Development)
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Since some drug intervention effects are only experienced by the patient, organizations such as the Food and Drug Administration prefer clinically meaningful outcome measures. Here, we evaluated which symptoms and limitations in daily life are most burdensome to pediatric patients with mitochondrial disorders and their parents, using two questionnaires. In a study of 78 patients, the most burdensome complaints included fatigue, behavior and speech disturbances, epilepsy and muscle weakness and a high degree of limitations in daily activities was found. Importantly, there was a discrepancy between what symptoms metabolic pediatricians estimated would be most burdensome compared to the patients'/caretakers' opinion. To include feasible and relevant outcome measures in intervention studies, the experience and opinions of patients and caretakers should therefore be heard. (c) 2012 Elsevier B.V. and Mitochondria Research Society. All rights reserved.
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