Velopharyngeal insufficiency following cleft palate repair: a comparative effectiveness study of existing surgical procedures and identification of criteria to guide a personalized treatment approach

腭咽闭合不全(VPI)在高达40%的腭裂修复患者中发生，并导致 说话时鼻音过大和鼻气排出，极大地限制了交流。VPI有 传统的治疗方法是咽部成形术，其有效率超过80%，但会导致阻塞性睡眠呼吸暂停 (OSA)在15%-50%的患者中。为了避免OSA的风险，一些外科医生已经开始进行腭部修复以治疗VPI。早期经验表明，再次修复在解决VPI方面有效率为84%，而不是 导致OSA，尽管结果因患者选择标准的不同而有很大不同。给定 重新修复以降低阻塞性睡眠呼吸暂停综合征风险的可能性，以及我们对其认识的实质性限制 有效性，迫切需要比较腭部再修复和咽成形术的有效性，以及 确定哪些患者最有可能从每次手术中受益。本提案满足了这一需求 通过三个具体的目标：(1)比较腭裂修复术和咽成形术的疗效。 治疗VPI。(2)确定腭部解剖异常可预测上颌下垂的消退。 在腭部修复和咽部成形术之后。(3)确定实施的障碍和促进者 个性化的VPI程序选择。目标1和目标2将通过进行观测来实现 腭裂修复与咽成形术治疗VPI的疗效比较 初步的腭裂修复。这项研究将在3年内从10个唇裂中心招募528名参与者，使用 与已知因素或怀疑影响手术过程的因素进行对照的倾向评分匹配 术后作业和言语结果。目标1将比较两种程序的结果 VPI术后12个月的鼻音过大、鼻气流出和OSA。总体比较： 将进行程序，以及对具有特定检查和形态的患者进行亚组分析 标准。目标2将通过在VPI手术前对参与者进行腭咽MRI检查来实现 可直接评估引起VPI的膜肌和软组织。目标2将测试是否 可见的膜肌裂开、膜肌错位和/或短膜的解剖异常。 在MRI上，预测腭裂修复后高鼻音的消退。目标3将使用实施科学 了解供应商当前选择VPI程序的方法，确定可能 推迟或鼓励实施基于AIMS调查结果的个性化VPI管理方法 1和2.完成这些目标将首次将腭部修复与咽成形术进行直接比较 对于VPI的治疗，建立预测治疗成功的形态和解剖特征 每个流程，并确定实施个性化VPI流程的障碍和促进者 选择。建议的研究将对唇裂术后的VPI儿童产生显著的积极影响 通过使外科医生能够为每个儿童选择最有效、危害最小的外科手术来修复腭部。

Velopharyngeal insufficiency (VPI) occurs in up to 40% of patients following cleft palate repair and results in hypernasality and nasal air emission during speech, which substantially limits communication. VPI has traditionally been treated with pharyngoplasty, which is over 80% effective but leads to obstructive sleep apnea (OSA) in 15%–50% of patients. To avoid the risk of OSA, some surgeons have begun performing palate re-repair to treat VPI. Early experience suggests re-repair is 84% effective in resolving VPI and that it does not lead to OSA, although results vary substantially depending upon the criteria for patient selection. Given the potential of re-repair to reduce the risk of OSA, and the substantial limitations in our knowledge about its effectiveness, there is a critical need to compare the effectiveness of palate re-repair to pharyngoplasty and to determine which patients are most likely to benefit from each operation. This proposal addresses this need through three specific aims: (1) Compare the effectiveness of palate re-repair with that of pharyngoplasty for treating VPI. (2) Determine the anatomic abnormalities of the palate that predict resolution of hypernasality following palate re-repair and following pharyngoplasty. (3) Identify barriers and facilitators to implementing personalized VPI procedure selection. Aims 1 and 2 will be achieved by conducting an observational comparative-effectiveness study of palate re-repair versus pharyngoplasty in patients with VPI following their initial cleft palate repair. The study will enroll 528 participants over a 3-year period from 10 cleft centers, using propensity score matching to control for known factors or those suspected of influencing surgical procedure assignment and speech outcome after surgery. Aim 1 will compare the two procedures for the outcomes of hypernasality, nasal air emission, and OSA at 12 months after VPI surgery. An overall comparison of procedures will be performed, as will subgroup analyses for patients with specific exam and morphologic criteria. Aim 2 will be achieved by obtaining velopharyngeal MRI on participants prior to VPI surgery; MRI enables direct evaluation of the velar muscles and soft tissues responsible for VPI. Aim 2 will test whether the anatomic anomalies of velar muscle dehiscence, velar muscle malposition, and/or a short velum, as visualized on MRI, predict resolution of hypernasality following palate re-repair. Aim 3 will use implementation science methods to understand providers’ current approach to VPI procedure selection, identifying factors that may delay or encourage implementation of a personalized approach to VPI management built upon findings in Aims 1 and 2. Completion of these aims will provide the first direct comparison of palate re-repair to pharyngoplasty for the treatment of VPI, establish the morphologic and anatomic features that predict successful treatment with each procedure, and identify barriers and facilitators to implementing personalized VPI procedure selection. The proposed research will produce a significant positive impact for children with VPI after cleft palate repair by enabling surgeons to select the most effective, least harmful surgical procedure for each child.