Table 1 Examinations performed in the Extended LAST STRONG study
Outcome domain | Assessments | Age |
|---|---|---|
Medical history | ||
Past | Perinatal period, motor milestones | All |
Current | Functional abilities, comorbidities, devices, treatments | All |
Neurological examination and functional measurements | ||
Muscle function | Muscle strength assessment (Medical Research Council, MRC) | ≥ 5 years |
Hand-Held dynamometry (HHD) | ≥ 5 years | |
The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) | < 2 years | |
Hammersmith Infant Neurological Examinations (HINE) | < 2 years | |
Motor Function Measurement (MFM)-20/32 | ≥ 2 years | |
Graded and timed function tests; ambulant patients only 1. 30 s sit to stand, climb 4 stairs, rise from floor 2. 6-Minute Walk test (6MWT) | ≥ 2 years ≥ 5 years | |
Functional Ambulation Classification (FAC) | ≥ 5 years | |
Vignos and Brooke scale | ≥ 2 years | |
Contractures | Goniometry | ≥ 2 years |
Other | Coordination, gait, reflexes, cranial and facial muscles, dysmorphic features | ≥ 2 years |
Imaging | ||
Muscles | Quantitative muscle ultrasound | All |
Quantitative muscle magnetic resonance imaging (MRI) of the lower extremities | ≥ 10 years | |
Bone density | Dual-energy X-ray absorptiometry (DEXA) | ≥ 2 years |
X-ray of the hand | 2–17 years | |
Respiratory assessment | ||
Respiratory system | Spirometry (forced expiratory volume in the first second (FEV1), forced vital capacity (FVC), vital capacity (VC), peak cough flow (PCF)) | ≥ 5 years |
Maximum Expiratory Pressure (MEP), Maximum Inspiratory Pressure (MIP), Sniff Nasal Inspiratory Pressure (SNIP) | ≥ 5 years | |
Questionnaires | ||
Activities and participation | ACTIVLIM | ≥ 6 years |
Impact on Participation and Autonomy (IPA) | ≥ 18 years | |
Egen Klassifikation version 2 (EK2) | ≥ 12 years | |
Borg Rating Scale of Perceived Exertion; prior to and after 6MWT (Borg RPE) | ≥ 5 years | |
Fatigue | Checklist Individual Strength (CIS) | ≥ 18 years |
Pediatric Quality of Life Inventory (PedsQL) (Multidimensional Fatigue Scale; Child Self-report and/or parent proxy) | 2 − 17 years | |
Pain | McGill pain questionnaire | ≥ 12 years |
Wong-Baker Faces Pain rating scale | ≥ 2 years | |
Quality of life | Pediatric Quality of Life Inventory (PedsQL) (Generic Core Scale, Neuromuscular Module; Child Self-report and/or parent proxy) | 2–17 years |
Research and Development-36 (RAND-36) | ≥ 18 years | |
Individualized Neuromuscular Quality of Life (INQoL) | ≥ 18 years | |
Accelerometry | ||
Accelerometry | Accelerometry using GENEActiv for eight consecutive days | ≥ 2 years |