Protocol for a Prospective Observational Study of Revision Palatoplasty Versus Pharyngoplasty for Treatment of Velopharyngeal Insufficiency Following Cleft Palate Repair.

Objective: To present the design and methodology for an actively enrolling comparative effectiveness study of revision palatoplasty versus pharyngoplasty for the treatment of velopharyngeal insufficiency (VPI).

Design: Prospective observational multicenter study.

Setting: Twelve hospitals across the United States and Canada.

Participants: Individuals who are 3-23 years of age with a history of repaired cleft palate and a diagnosis of VPI, with a total enrollment target of 528 participants.

Interventions: Revision palatoplasty and pharyngoplasty (either pharyngeal flap or sphincter pharyngoplasty), as selected for each participant by their treatment team.

Main outcome measure(s): The primary outcome is resolution of hypernasality, defined as the absence of consistent hypernasality as determined by blinded perceptual assessment of a standard speech sample recorded twelve months after surgery. The secondary outcome is incidence of new onset obstructive sleep apnea. Statistical analyses will use propensity score matching to control for demographics, medical history, preoperative severity of hypernasality, and preoperative imaging findings.

Results: Study recruitment began February 2021. As of September 2022, 148 participants are enrolled, and 78 have undergone VPI surgery. Enrollment is projected to continue into 2025. Collection of postoperative evaluations should be completed by the end of 2026, with dissemination of results soon thereafter.

Conclusions: Patients with VPI following cleft palate repair are being actively enrolled at sites across the US and Canada into a prospective observational study evaluating surgical outcomes. This study will be the largest and most comprehensive study of VPI surgery outcomes to date.