目的:介绍积极进行的翻修腭成形术与咽成形术治疗咽喉功能不全(VPI)的有效性比较研究的设计和方法。
方法:前瞻性观察多中心研究。
方法:美国和加拿大的12家医院。
方法:3-23岁有腭裂修复史并诊断为VPI的患者,总注册目标为528名参与者。
方法:翻修腭成形术和咽成形术(咽瓣或括约肌咽成形术),由他们的治疗团队为每个参与者选择。
方法:主要结果是鼻塞的消退,定义为通过对手术后12个月记录的标准语音样本进行盲性感知评估而确定的不存在一致的鼻塞。次要结果是新发阻塞性睡眠呼吸暂停的发生率。统计分析将使用倾向得分匹配来控制人口统计数据,病史,术前鼻塞严重程度,和术前影像学检查结果。
结果:研究招募开始于2021年2月。截至2022年9月,148名参与者注册,78人接受了VPI手术。预计入学人数将持续到2025年。术后评估的收集应在2026年底前完成,并在此后不久传播结果。
结论:正在美国和加拿大各地积极招募腭裂修复术后VPI患者,以评估手术结果的前瞻性观察性研究。这项研究将是迄今为止最大,最全面的VPI手术结果研究。
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).
METHODS: Prospective observational multicenter
study.
METHODS: Twelve hospitals across the United States and Canada.
METHODS: 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.
METHODS: Revision palatoplasty and pharyngoplasty (either pharyngeal flap or sphincter pharyngoplasty), as selected for each participant by their treatment team.
METHODS: 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.