AIMS: Identifying age-appropriate, comprehensive cognitive and behaviour COAs for infants and toddlers is challenging. While there are validated assessments, they are not singularly comprehensive. Using multiple scales results in conceptual overlap and may take hours to complete, thereby increasing burden on the patient, their family, clinical resources and potentially hindering validity, outcomes, and adherence. The aim of this study was to find a concise solution for infant and toddler cognitive and behavioural assessments in clinical trials.
METHODS: A literature review was conducted using Google Scholar and ePROVIDE, to identify infant and toddler cognitive and behaviour COAs. Four variables were used to determine unique and overlapping concepts and facilitate appropriate selection in a clinical trial setting: (1) domain coverage, (2) time to completion, (3) age of administration, and (4) availability of age equivalent (AE) scores.
RESULTS: Seven instruments were identified (Table 1). The BSDI was the most comprehensive instrument that provided AE scores but was limited to birth to 42 months. The PEDI allowed assessments through 21 years of age but missed the key milestone of language and motor function. While the VABS could be administered from birth continuously throughout development and adulthood, it does not assess language and cognition. Other scales were either more limited conceptually (GMSD and MSEL) or by age (NBAS and KABC). The majority were developed as diagnostic or baseline assessments, and only one (PEDI) could be administered as a computerized adaptive test (CAT). All identified COAs had a lengthy time to completion presenting a significant burden to patients, caregivers and clinical teams.
CONCLUSION: Our review did not yield a concise solution for patient-focused infant and toddler cognitive and behavioural assessments in clinical trials. The lack of conceptual coverage and congruency in age ranges for single COAs signals potential incomparability of results overtime, if multiple measures are needed. For children with severe developmental delays who fall below standardized scores, measures which yield AE scores offer comparability between instruments (i.e., concurrent validity) and enable measurement of ‘delay to progression’. A computer adaptive approach may be beneficial to minimize burden, reduce missing data, increase validity, and interpretability, if AE scores can be calculated.
