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

Kristina Dunworth; Banafsheh Sharif-Askary; Lynn Grames; Carlee Jones; Jennifer Kern; Jillian Nyswonger-Sugg; Arthur Suárez; Karen Thompson; Jessica Ching; Brent Golden; Corinne Merrill; Phuong Nguyen; Kamlesh Patel; Carolyn R Rogers-Vizena; S Alex Rottgers; Gary B Skolnick; Alexander C Allori

doi:10.1177/10556656231207469

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

Cleft Palate Craniofac J. 2023 Oct 16:10556656231207469. doi: 10.1177/10556656231207469. Online ahead of print.

Authors

Kristina Dunworth¹, Banafsheh Sharif-Askary¹, Lynn Grames², Carlee Jones^{3

4}, Jennifer Kern^{3

5}, Jillian Nyswonger-Sugg^{3

5}, Arthur Suárez^{3

5}, Karen Thompson^{6

7}, Jessica Ching^{8

9}, Brent Golden¹⁰, Corinne Merrill¹¹, Phuong Nguyen¹², Kamlesh Patel^{2

11}, Carolyn R Rogers-Vizena^{6

13

14}, S Alex Rottgers^{15

16}, Gary B Skolnick¹¹, Alexander C Allori^{1

3

4}

Affiliations

¹ Department of Surgery, Division of Plastic, Maxillofacial, and Oral Surgery, Duke University School of Medicine, Durham, NC, USA.
² Cleft Palate and Craniofacial Institute, St. Louis Children's Hospital, St. Louis, USA.
³ Duke Cleft & Craniofacial Center, Duke Children's Hospital, Durham, NC, USA.
⁴ Division of Plastic, Maxillofacial, and Oral Surgery, Department of Surgery, Duke University Health System, Durham, NC, USA.
⁵ Department of Speech Pathology & Audiology, Duke University Hospital, Durham, NC, USA.
⁶ Cleft Lip and Palate Program/Craniofacial Program, Boston Children's Hospital, Boston, MA, USA.
⁷ Department of Otolaryngology and Communication Enhancement, Boston Children's Hospital, Boston, MA, USA.
⁸ University of Florida Craniofacial Center, University of Florida, Gainesville, FL, USA.
⁹ Division of Plastic and Reconstructive Surgery, Department of Surgery, University of Florida, Gainesville, FL, USA.
¹⁰ Pediatric Cleft Lip and Palate Surgery Program, Orlando Health Arnold Palmer Hospital for Children, Orlando, FL, USA.
¹¹ Division of Plastic and Reconstructive Surgery, Department of Surgery, Washington University School of Medicine in St. Louis, St. Louis, USA.
¹² Division of Plastic Surgery, Department of Surgery, University of Texas Health Science Center at Houston, Houston, TX, USA.
¹³ Department of Surgery, Harvard Medical School, Boston, MA, USA.
¹⁴ Department of Plastic and Oral Surgery, Boston Children's Hospital, Boston, MA, USA.
¹⁵ Cleft and Craniofacial Center, Johns Hopkins Children's Center, Baltimore, MD, USA.
¹⁶ Division of Plastic and Reconstructive Surgery, Department of Surgery, Johns Hopkins Medicine, Baltimore, MD, USA.

PMID: 37844605
DOI: 10.1177/10556656231207469

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.

Keywords: articulation; cleft lip; cleft lip and palate; cleft palate; nasality; nonsyndromic clefting; outcomes; pediatrics; registry; resonance; speech assessment; speech development; speech disorders; velopharyngeal dysfunction; velopharyngeal function.