Table 1.
Results of the Delphi-based process
| Consensus | ||
|---|---|---|
| Percentage scores ≥ 4 | Mean score | |
| Myopathy | ||
| 6-min walk test | 100% | 4.4 |
| 30 s sit to stand test | 100% | 4.4 |
| Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) | 100% | 4.7 |
| Growth and weight gain | 100% | 4.6 |
| Caregiver burden scale | 100% | 4.7 |
| Grip strength | 92% | 4.2 |
| New muscle endurance test (developed by RCMM) | 92% | 4.0 |
| International Paediatric Mitochondrial Disease Scale (IPMDS) | 91% | 4.3 |
| PedsQL (multidimensional fatigue) | 91% | 4.2 |
| Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) | 82% | 4.0 |
| Timed tests (stand up from floor) | 77% | 3.8 |
| Hammersmith Functional Motor Scale | 64% | 3.6 |
| Accelerometer | 62% | 3.5 |
| Alberta Infant Motor Scale (AIMS) | 60% | 3.6 |
| Gait measurement (GAITRite) | 55% | 3.4 |
| PedsQL (general) | 54% | 3.5 |
| Attain stand test (30 s) | 46% | 2.9 |
| Timed tests (10-m walk) | 46% | 3.3 |
| North Star Ambulatory Assessment | 44% | 3.2 |
| Test of Masticating and Swallowing Solids (TOMASS) | 40% | 3.2 |
| Checklist individual strength (CIS) | 38% | 3.1 |
| PedsQL (neuromuscular) | 36% | 3.2 |
| Lateral step-up test (30 s) | 31% | 2.6 |
| Childhood Myositis Assessment Scale (CMAS) | 31% | 2.8 |
| Gross Motor Function Measure (GMFM) | 27% | 2.7 |
| Pediatric Evaluation of Disability Inventory (PEDI) | 23% | 2.6 |
| Eyelid ptosis and ophthalmoparesis tests | 18% | 2.3 |
| 9-hole peg test | 15% | 1.8 |
| Quantitative muscle power using hand-held dynamometer | 15% | 2.3 |
| Timed tests (5 stairs) | 15% | 2.2 |
| Quantitative myasthenia gravis test | 8% | 2.0 |
| 6-min mastication test | 0% | 1.8 |
| Encephalo(myo)pathy | ||
| Gross Motor Function Measure (GMFM) | 100% | 4.7 |
| Pediatric Evaluation of Disability Inventory (PEDI) | 100% | 4.4 |
| Spasticity: Tardieu scale | 100% | 4.2 |
| Caregiver burden scale | 100% | 4.5 |
| Barry–Albright Dystonia Scale | 92% | 4.1 |
| Scale for the assessment and rating of ataxia (SARA) | 85% | 4.1 |
| Growth and weight gain | 85% | 4.2 |
| Alberta Infant Motor Scale (AIMS) | 85% | 3.9 |
| Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) | 85% | 4.0 |
| 6-min walk test | 85% | 3.9 |
| 9-hole peg test | 85% | 3.8 |
| Timed tests (10-m walk) | 85% | 3.8 |
| Pediatric Outcomes Data Collection Instrument (PODCI) | 77% | 3.7 |
| 30 s sit to stand test | 77% | 3.8 |
| International Pediatric Mitochondrial Disease Scale (IPMDS) | 73% | 4.1 |
| CP classifications (CFCF, GMFCS, MACS) | 69% | 3.8 |
| Global assessment of three most important symptoms | 67% | 3.8 |
| Accelerometer | 54% | 3.5 |
| Timed tests (stand up from floor) | 46% | 3.1 |
| Perceive Recall Plan Perform (PRPP) | 38% | 2.8 |
| Peabody vocabulary test | 38% | 3.2 |
| PedsQL (general) | 33% | 3.1 |
| Test of Masticating and Swallowing Solids (TOMASS) | 31% | 3.0 |
| Gait measurement (GAITRite) | 31% | 3.0 |
| Timed tests (up and go) | 31% | 3.0 |
| Hypertonia Assessment Scale | 23% | 2.8 |
| Child Health Assessment Questionnaire (CHAQ) | 23% | 2.8 |
| KIDSCREEN | 23% | 2.7 |
| Frontal assessment battery | 23% | 2.9 |
| PedsQL (CP) | 15% | 2.7 |
| Luria | 15% | 2.8 |
| Spasticity: Ashworth scale | 8% | 2.6 |
| Timed tests (5 stairs) | 8% | 2.5 |
| Eyberg Child Behavior Inventory | 8% | 1.9 |
| Gross Motor Function Measure (GMFM) | 0% | 2.2 |
The items on which no strong consensus was reached are shown in italics