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Balancing precision and affordability in assessing infant development in large-scale mortality trials: secondary
Kristy P Robledo1, Ingrid Rieger2, Sarah Finlayson3
1NHMRC Clinical Trials Centre, The University of Sydney, Camperdown, New South Wales, Australia kristy.robledo@sydney.edu.au.
Insights
The Ages and Stages Questionnaire (ASQ-3) can screen for developmental delay in infants, but requires larger sample sizes compared to the Bayley Scales of Infant Development (Bayley-III). This approach may offer cost savings in large trials.
Area of Science:
- Neonatal research
- Developmental pediatrics
- Clinical trial methodology
Background:
- Large-scale mortality trials need reliable secondary assessments for infant impairment.
- The Ages and Stages Questionnaire (ASQ-3) is a parent-completed screening tool.
- The Bayley Scales of Infant Development (Bayley-III) is a clinician-administered diagnostic tool.
Purpose of the Study:
- To compare the effectiveness of ASQ-3 versus Bayley-III in classifying developmental impairment in infants.
- To evaluate the cost-effectiveness of using screening versus diagnostic tools in large clinical trials.
Main Methods:
- Secondary analysis of 405 children born <30 weeks gestation from the Australian Placental Transfusion Study.
- Children assessed around 2 years corrected age.
- Comparison of ASQ-3 (screening) and Bayley-III (diagnostic) for impairment classification.
Main Results:
- ASQ-3 demonstrated good predictive ability for developmental delay (AUC 0.75-0.99), with sensitivity 57%-100% and specificity 88%-90%.
- Using ASQ-3 could double the sample size but save $13M in assessment costs compared to Bayley-III.
- Potential cost savings may be offset by the need for larger sample sizes and upscaling.
Conclusions:
- In large trials, the high cost of precise diagnostic tools like Bayley-III may be prohibitive.
- Employing a less precise, calibrated screening tool (ASQ-3) with a larger sample size can be a more affordable strategy.
- Careful consideration of assessment precision, sample size, and overall cost is crucial for large-scale clinical trials.
Objective:
Large-scale mortality trials require reliable secondary assessments of impairment. We compared the Ages and Stages Questionnaire (ASQ-3), a screening tool self-administered by parents, in classifying impairment using the 'gold standard' Bayley Scales of Infant Development (Bayley-III), a diagnostic tool administered by trained assessors.
Design:
Analysis of 405 children around 2 years corrected age from the Australian Placental Transfusion Study, a trial conducted over 8 years.
Setting:
Secondary analysis of international, open-label, multicentre randomised trial.
Patients:
Children born <30 weeks gestation.
Interventions:
Immediate (<10 s) versus delayed (60 s+) cord clamping.
Main Outcomes:
ASQ-3 and Bayley-III assessments around 2 years corrected age. Impairment (or developmental delay) was defined as <2 SD below the mean (<70) for Bayley-III domains.
Results:
The area under the receiver operating curve for ASQ-3 domains predicting delay was 0.75-0.99. Sensitivity for predicting delay was 57%-100%, while specificity was 88%-90%.We modelled the cost and sample size using a less expensive, though less precise, screening assessment for impairment compared with a more costly diagnostic assessment. For detecting a 25% reduction in the relative risk of delay, using ASQ-3 rather than Bayley-III could require double the sample size (15 000 to 30 000), but outcome assessment cost savings would be US$13M (EUR$12M). However, assessment cost savings may be outweighed by upscaling.
Conclusions:
When measuring developmental outcomes in a large-scale clinical trial, using a more precise diagnostic tool may be financially prohibitive, so increasing the sample size and using a less precise but appropriately calibrated tool may be more affordable.
Trial Registration Number:
ACTRN12610000633088.
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