Comparing two methods of follow up in a multicentre randomised trial

Area of Science:

• Pediatric Medicine
• Clinical Trial Methodology
• Developmental Pediatrics

Background:

• Post-hemorrhagic ventricular dilatation (PHVD) is a serious complication in preterm infants.
• Accurate outcome measures are crucial for evaluating PHVD treatments in clinical trials.
• Parental input can offer valuable insights into a child's functional status.

Purpose of the Study:

• To assess the utility of a parental questionnaire for outcome measurement in a PHVD trial.
• To compare parental assessments with objective pediatric evaluations.
• To determine if parental responses can categorize child disability levels.

Main Methods:

• A questionnaire was administered to parents of 88 PHVD survivors before a 30-month pediatric assessment.
• Parental responses were compared with detailed pediatric findings, including Griffiths' mental development scales.
• A model using parental responses categorized children's disability (normal, impaired, disabled).

Main Results:

• High agreement between parents and pediatricians was observed for gross motor function (81-99%), feeding (91-99%), and language (85-93%).
• Parents identified 60% of disabled children, closely matching the pediatrician's assessment (66%).
• Parents accurately identified most severely disabled children, though less precise with subtle impairments.

Conclusions:

• Parental questionnaires demonstrate significant potential as outcome measures in PHVD trials.
• The methodology shows encouragement for further development and application in comparative group studies.
• This approach may enhance data collection efficiency and richness in pediatric clinical research.