Table 3.
Proposed outcome measures for the explorative study in children with mitochondrial encephalo(myo)pathy
| Test | ≥ 4 (%) | Mean | Priority |
|---|---|---|---|
| Gross Motor Function Measure (GMFM) | 100% | 4.7 | 100% |
| Pediatric Evaluation of Disabilities Inventory (PEDI-CAT) | 100% | 4.4 | 69% |
| Tardieu test for spasticity | 100% | 4.2 | 77% |
| Caregiver burden scale | 100% | 4.5 | 100% |
| Barry–Albright Dystonia Scale | 92% | 4.1 | 54% |
| 6-min walk test | 85% | 3.9 | 54% |
| 9-hole peg test | 85% | 3.8 | 23% |
| 10-m walk or run test | 85% | 3.8 | 38% |
| Scale for the Assessment and Rating of Ataxia (SARA) | 85% | 4.1 | 54% |
| Growth and weight gain | 85% | 4.2 | 54% |
| Alberta Infant Motor Skills (AIMS; for young children) | 85% | 3.9 | 62% |
| Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND; for young children) | 85% | 4.0 | 62% |
| Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) | 85% | 4.0 | 77% |
| 30 s sit to stand test | 77% | 3.8 | 38% |
| Pediatric Outcomes Data Collection Instrument (PODCI) | 77% | 3.7 | 54% |
| International Pediatric Mitochondrial Disease Scale (IPMDS) | 78% | 4.0 | 62% |
The items on which only good consensus was obtained are shown in italics