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

腭裂修复术后腭咽功能不全：现有手术程序的比较有效性研究和指导个性化治疗方法的标准确定

• 批准号: 10030375
• 负责人: Thomas James Sitzman
• 金额: $ 84.22万
• 依托单位: PHOENIX CHILDREN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-07-01 至 2025-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10030375
• 关键词: Address; Affect; Air; Anatomy; Behavior; Child; Child Health; Childhood; Cleft Palate; Communication; Craniofacial Abnormalities; Deformity; Effectiveness; Enrollment; Evaluation; Exhibits; Goals; Growth; Interview; Knowledge; Language; Literature; Magnetic Resonance Imaging; Methods; Mission; Morphology; Muscle; National Institute of Dental and Craniofacial Research; Nose; Obstruction; Obstructive Sleep Apnea; Operative Surgical Procedures; Outcome; Palate; Participant; Pathologist; Patient Outcomes Assessments; Patient Selection; Patients; Performance; Physical shape; Procedures; Process; Provider; Quality of life; Research; Resolution; Risk; Selection Criteria; Self Perception; Speech; Speech Intelligibility; Subgroup; Surgeon; Symptoms; Testing; Velopharyngeal Insufficiency; base; comparative effectiveness study; compare effectiveness; cost; cost effective; early experience; effectiveness evaluation; experience; imaging modality; implementation science; improved; operation; palate repair; peer; personalized approach; personalized medicine; recruit; repaired; soft tissue

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.

腭咽闭合不全（VPI）发生在高达40%的腭裂修复术后患者中， 在讲话期间鼻音过重和鼻空气排放，这实质上限制了交流。VPI拥有 传统的治疗方法是咽成形术，有效率超过80%，但会导致阻塞性睡眠呼吸暂停 (OSA)15%-50%的患者。为了避免OSA的风险，一些外科医生已经开始进行腭部再修复来治疗VPI。早期经验表明，再次修复在解决VPI方面有效率为84%， 导致OSA，尽管结果根据患者选择的标准而变化很大。鉴于 再次修复以降低OSA风险的潜力，以及我们对其知识的实质性限制， 有效性，迫切需要比较腭修复术与咽成形术的有效性， 确定哪些患者最有可能从每次手术中受益。本建议满足了这一需要 通过三个具体目标：（1）比较腭部再修复与咽成形术的效果， 治疗VPI。(2)确定腭部的解剖异常，预测鼻音过度的解决 在腭修复和咽成形术之后。(3)确定实施的障碍和促进因素 个性化VPI程序选择。目标1和2将通过开展一项观察性活动来实现， 腭部再修复术与咽成形术在VPI患者术后的比较有效性研究 初步腭裂修复这项研究将在3年内从10个唇腭裂中心招募528名参与者， 与已知因素或疑似影响手术操作的因素进行对照的倾向评分匹配 手术后的分配和言语结果。目标1将比较两种手术的结果， VPI手术后12个月时的鼻音过重、鼻排气和阻塞性睡眠呼吸暂停综合症。总体比较 将进行程序，以及对具有特定检查和形态学检查的患者进行亚组分析。 的搜索.目标2将通过在VPI手术前获得受试者的咽部MRI来实现; MRI 能够直接评估导致VPI的软膜肌和软组织。目标2将测试 解剖异常的膜肌裂开，膜肌错位，和/或短膜，如所见 在MRI上，预测腭部再修复后鼻音过重的解决。目标3将使用实施科学 了解供应商当前VPI程序选择方法的方法，确定可能 延迟或鼓励实施基于目标中的调查结果的个性化VPI管理方法 1和2.这些目标的完成将首次提供腭部再修复与咽成形术的直接比较 对于VPI的治疗，建立预测成功治疗的形态和解剖特征 并确定实施个性化VPI程序的障碍和促进因素 选择.这项研究将对腭裂术后VPI患儿产生积极的影响 通过使外科医生能够为每个孩子选择最有效、危害最小的外科手术来进行腭修复。