Using “Real-World Data” to Study Cleft Lip/Palate Care: An Exploration of Speech Outcomes from a Multi-Center US Learning Health Network

To assess the ability of a cleft-specific multi-site learning health network registry to describe variations in cleft outcomes by cleft phenotypes, ages, and treatment centers. Observed variations were assessed for coherence with prior study findings.Cross-sectional analysis of prospectively collected data from 2019-2022.Six cleft treatment centers collected data systematically during routine clinic appointments according to a standardized protocol.714 English-speaking children and adolescents with non-syndromic cleft lip/palate.Routine multidisciplinary care and systematic outcomes measurement by cleft teams.Speech outcomes included articulatory accuracy measured by Percent Consonants Correct (PCC), velopharyngeal function measured by Velopharyngeal Competence (VPC) Rating Scale (VPC-R), intelligibility measured by caregiver-reported Intelligibility in Context Scale (ICS), and two CLEFT-Q™ surveys, in which patients rate their own speech function and level of speech distress.12year-olds exhibited high median PCC scores (91-100%), high frequency of velopharyngeal competency (62.50-100%), and high median Speech Function (80-91) relative to younger peers parsed by phenotype. Patients with bilateral cleft lip, alveolus, and palate reported low PCC scores (51-91%) relative to peers at some ages and low frequency of velopharyngeal competency (26.67%) at 5 years. ICS scores ranged from 3.93-5.0 for all ages and phenotypes. Speech Function and Speech Distress were similar across phenotypes.This exploration of speech outcomes demonstrates the current ability of the cleft-specific registry to support cleft research efforts as a source of "real-world" data. Further work is focused on developing robust methodology for hypothesis-driven research and causal inference.