Table 2 Secondary outcome measures of toddlers
From: Recommendations for the design of therapeutic trials for neonatal seizures
Criteria | Description |
|---|---|
Post-neonatal onset epilepsy | |
Health | 1. Hospitalizations and/or surgeries 2. Medical visits; therapies, other allied professional support, and medications 3. Growth percentiles for height, weight, and head circumference 4. Cardiorespiratory, e.g., limited exercise tolerance, need for respiratory support 5. Gastrointestinal, e.g., need for a special diet or parenteral nutrition, presence of a stoma or gastrostomy |
Neurosensory outcomes | 1. Visual impairment a. Visual acuity, e.g., total blindness (i.e., no light perception), severe visual impairment, use of glasses b. Cerebral visual impairment 2. Hearing impairment, including response to hearing aids or cochlear implant a. Profound > 90 dB b. Severe 70–90 dB c. Moderate 40–70 dB |
Neuromotor outcomes | 1. Clinical neurological examination a. Hammersmith Infant Neurological Examination (HINE)74,79: 2 to 24 months b. Amiel-Tison Neurological Development from Birth to Six Years80: Birth to 6 years 2. Detailed assessment of motor performance a. Gross Motor Function Measure (GMFM)81: 6 months to 18 years b. Peabody Developmental Motor Scale, Second Edition (PDMS-2)82: Birth to 60 months 3. Diagnosis of cerebral palsy (CP) based on clinical examination and motor function scores, and classified according to the Surveillance of Cerebral Palsy in Europe (SCPE) criteria83,84 4. In children with CP, the Gross Motor Function Classification System (GMFCS) grades severity of motor impairment into 5 levels.85,86 |
Neurocognitive and language outcomes | 1. Difficulties in assessing cognition in infants and young children a. Requires attention, some motor function (especially fine motor) to perform tasks, and receptive language to understand. b. There are no established tools for assessment of children who have major sensory or motor impairments. c. At 2 years, there are no established assessment tools for more sophisticated cognitive functions (e.g., executive functions, abstract reasoning) which are still developing. 2. Standardized measures of cognitive and language abilities have been used, but care is required in their interpretation. Translations into many languages not available. Where possible, performance may better be compared to that of typically developing children, although this may not be practicable in the context of a trial. Where direct assessment is not possible, all available evidence should be collected to categorize performance in standard deviation score bands. a. Bayley Scales of Infant Development, Third Edition (BSID-III)87: 1 month to 42 months; 5 scales (cognitive, language, motor, social-emotional, and adaptive) b. Griffiths Mental Development Scales, Third Edition (GMDS III)88: Birth to 72 months; 5 scales (foundations of learning, language and communication, eye and hand coordination, personal and social-emotional, gross motor) c. Mullen Scales of Early Learning89: Birth to 68 months; 5 scales (gross motor, fine motor, visual reception, receptive language, expressive language) |
Combined adverse categorical neurodevelopmental outcomes | 1. By severity, e.g., British Association of Perinatal Medicine90 a. Severe b. Moderate c. Mild 2. For a dichotomous variable, generally children with moderate and severe impairment are combined into a single group (children with neurodevelopmental impairment), with a 2nd group of children with no or mild impairment. |
Functional outcomes | 1. Mobility, e.g., Gross Motor Classification System 2. Communication: Difficult to assess with standardized assessment tools when there are multiple primary languages 3. Adaptive function: Ability to perform self-help skills (e.g., dressing/undressing, self-feeding) 4. Standardized measures of functional outcome a. Pediatric Evaluation of Disability Inventory (PEDI)91: 6 months to 7.5 years; measures self-care, mobility, and social function; targeted for children with disabilities b. Vineland Adaptive Behavioral Scales, Second Edition (VABS-II)92: Birth to 90 years; measures adaptive behavior |
