Using "Real-World Data" to Study Cleft Lip/Palate Care: An Exploration of Speech Outcomes from a Multi-Center US...

Area of Science:

• Craniofacial anomalies
• Speech pathology
• Health informatics

Background:

• Cleft lip and palate (CLP) treatment requires standardized outcome measurement.
• Learning health networks can aggregate real-world data for research.
• Understanding variations in CLP outcomes is crucial for improving care.

Purpose of the Study:

• To evaluate a cleft-specific registry's capacity to identify speech outcome variations.
• To compare outcomes across different cleft phenotypes, age groups, and treatment centers.
• To assess the registry data's coherence with existing research.

Main Methods:

• Cross-sectional analysis of prospectively collected data (2019-2022).
• Data from 714 children/adolescents with non-syndromic CLP across six treatment centers.
• Speech outcomes measured included Percent Consonants Correct (PCC), Velopharyngeal Competence (VPC-R), Intelligibility in Context Scale (ICS), and CLEFT-Q™ surveys.

Main Results:

• Older children (12 years) generally showed better speech outcomes (PCC, VPC, Speech Function) than younger peers.
• Patients with bilateral cleft lip, alveolus, and palate exhibited lower PCC and VPC at age 5.
• Intelligibility (ICS) and patient-reported speech function/distress showed consistency across phenotypes.

Conclusions:

• The cleft registry effectively provides real-world data for cleft research.
• The registry can describe variations in speech outcomes based on cleft type and age.
• Future research will focus on hypothesis-driven studies and causal inference using registry data.