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

Abstract

Objective 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. Design Cross-sectional analysis of prospectively collected data from 2019–2022. Setting Six cleft treatment centers collected data systematically during routine clinic appointments according to a standardized protocol. Participants 714 English-speaking children and adolescents with non-syndromic cleft lip/palate. Intervention Routine multidisciplinary care and systematic outcomes measurement by cleft teams. Outcome Measures 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. Results 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. Conclusions 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.