Table 2.
Proposed outcome measures for the natural history study in children with mitochondrial myopathy
| Test | ≥ 4 (%) | Mean | Priority adult-like | Priority infantile |
|---|---|---|---|---|
| 6-min walking test | 100% | 4.4 | 46% | |
| 30 s sit to stand test | 100% | 4.4 | 31% | |
| Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) | 100% | 4.7 | 92% | |
| Growth and weight gain | 100% | 4.6 | 92% | 92% |
| Caregiver burden scale | 100% | 4.7 | 92% | 100% |
| Grip strength | 92% | 4.2 | 46% | |
| New muscle endurance test (developed by RCMM) | 92% | 4 | 31% | |
| International Paediatric Mitochondrial Disease Scale (IPMDS) | 91% | 4.3 | 54% | 54% |
| PedsQL (multidimensional fatigue) | 91% | 4.2 | 46% | |
| Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) | 82% | 4 | 23% | 31% |
| Timed tests (stand up from floor) | 77% | 3.8 | 11% |
The item on which only good consensus was obtained is shown in italics