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

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.

Assessing Dysphagia in the Child.

Swallowing problems in children can occur for a variety of reasons, and assessment varies based on the age of the child, underlying medical problems, and results of the clinical swallow evaluation. The need for interdisciplinary management with speech language pathologists skilled in the management of children with dysphagia is imperative to identify the components of swallowing that are impaired and provide specific recommendations for safe and adequate nutrition supporting growth, development, and oral feeding if possible. This study focuses on the types of assessment tools available and how and when they are utilized for children of different ages and abilities.

Exploration of the Utility of the Generic ICHOM Standard Set Measures in Evaluating the Speech of Patients with Cleft Lip/Palate.

Background: The International Consortium of Health Outcome Measurements (ICHOM) standard set for cleft care appraisal recommends clinicians assess articulation with percentage consonants correct (PCC) and velopharyngeal function with velopharyngeal competency rating (VPC-R). This study explores the utility and limitations of these generic measures in detecting cleft speech sound disorders by comparing them with two cleft-specific speech-rating systems, cleft audit protocol of speech-augmented Americleft modification (CAPS-A-AM) and Pittsburgh weighted speech scale (PWSS). Methods: Consecutive children with repaired, nonsyndromic cleft lip/palate, aged 5 years or older (n = 27) underwent prospective speech evaluations conducted at a single academic institution. These evaluations were conducted, recorded, and evaluated by blinded speech-language pathologists experienced with all tools. Results: When comparing measures of articulation, PCC scores correlated better with scores for relevant subcomponents of CAPS-A-AM than PWSS. When comparing measures of velopharyngeal function, VPC-R scores correlated well with relevant components of both scales. Using a "screening test versus diagnostic test" analogy, VPC-R ratings were 87.5% sensitive and 73.7% specific for detecting velopharyngeal dysfunction according to subcomponents of CAPS-A-AM, and 70.6% sensitive and 100% specific according to subcomponents of PWSS. Conclusions: This exploratory study demonstrates that PCC and VPC-R perform moderately well in detecting articulatory and velopharyngeal dysfunction in patients with cleft lip/palate; however, these tools cannot describe nuances of cleft speech sound disorder. Thus, although PCC and VPC-R adequately track basic minimum outcomes, we encourage teams to consider extending the standard set by adopting a cleft-specific measurement system for further evaluation of the tools.