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: 10832139
• 负责人: Thomas James Sitzman
• 金额: $ 3.95万
• 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/10832139
• 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; Length; 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; Risk Reduction; Selection Criteria; Self Perception; Speech; Speech Intelligibility; Subgroup; Surgeon; Symptoms; Testing; Velopharyngeal Insufficiency; Visualization; 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.

腭咽功能不全（VPI）发生在高达40%的腭裂修复后的患者，并导致

项目成果

Does Notching Along the Nasal Velar Surface During Nasopharyngoscopy Predict Discontinuity of the Underlying Levator Veli Palatini Muscle?

鼻咽镜检查过程中沿鼻软腭表面刻痕是否预示着提肌帆帕拉蒂尼肌肉的不连续性？

• DOI: 10.1177/10556656231161991
• 发表时间: 2023
• 期刊: The Cleft palate-craniofacial journal : official publication of the American Cleft Palate-Craniofacial Association
• 作者: Perry,JamieL;Kinter,Sara;Williams,JessicaL;Snodgrass,TaylorD;Sitzman,ThomasJ
• 通讯作者: Sitzman,ThomasJ

VPI Management in SATB2 Syndrome: Use of MRI to Evaluate Anatomy and Physiology in Non-Cleft VPI.

• DOI: 10.1177/10556656221106888
• 发表时间: 2023-11
• 期刊: CLEFT PALATE CRANIOFACIAL JOURNAL
• 影响因子: 1.1
• 作者: Perry, Jamie L.;Williams, Jessica L.;Snodgrass, Taylor D.;Sitzman, Thomas J.
• 通讯作者: Sitzman, Thomas J.

Establishing a Clinical Protocol for Velopharyngeal MRI and Interpreting Imaging Findings.

建立腭咽 MRI 临床方案并解释影像学结果。

• DOI: 10.1177/10556656221141188
• 发表时间: 2024
• 期刊: The Cleft palate-craniofacial journal : official publication of the American Cleft Palate-Craniofacial Association
• 作者: Perry,JamieL;Snodgrass,TaylorD;Gilbert,ImaniR;Sutton,BradleyP;Baylis,AdrianeL;Weidler,EricaM;Tse,RaymondW;Ishman,StaceyL;Sitzman,ThomasJ
• 通讯作者: Sitzman,ThomasJ

Standardization of Videorecorded Speech Samples for Children with Cleft Palate: Methods to Facilitate High-Quality Speech Outcomes Data Collection.

腭裂儿童录像语音样本的标准化：促进高质量语音结果数据收集的方法。

• DOI: 10.1177/10556656231217645
• 发表时间: 2023
• 期刊: The Cleft palate-craniofacial journal : official publication of the American Cleft Palate-Craniofacial Association
• 作者: Baylis,AdrianeL;Cummings,CaitlinE;Lien,KariM;Sitzman,ThomasJ;Kirschner,RichardE;Chapman,KathyL;CORNetandVPI-OPSConsortiumsandAmericleftSpeechGroup
• 通讯作者: CORNetandVPI-OPSConsortiumsandAmericleftSpeechGroup

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

修复腭成形术与咽成形术治疗腭裂修复术后腭咽功能不全的前瞻性观察研究方案。

• DOI: 10.1177/10556656221147159
• 发表时间: 2024
• 期刊: The Cleft palate-craniofacial journal : official publication of the American Cleft Palate-Craniofacial Association
• 作者: Sitzman,ThomasJ;Baylis,AdrianeL;Perry,JamieL;Weidler,EricaM;Temkit,M'hamed;Ishman,StaceyL;Tse,RaymondW;VPI-OPSStudyConsortium
• 通讯作者: VPI-OPSStudyConsortium