Furlow Palatoplasty with Tensor Tenopexy for Otitis Media

描述（由申请人提供）：我们建议进行一项为期5年，前瞻性，随机，盲法，一项对照研究，评估了两种腭裂成形术在改善咽鼓管功能（ETF）和减少腭裂（CP）患者中耳炎伴积液（OME）和听力损失的患病率方面的比较效果，而不会引起不良事件或对其他功能产生不利影响。评估的腭裂成形术是Furlow双反Z成形术，双侧处理腭腭张量肌（mTVP）肌腱（FP）和该手术的修改，包括双侧张量肌腱固定术（MFP），即在mTVP肌腱横断之前将mTVP肌腱附着在同侧肱骨上。在4年的时间里，我们将招募120名6个月大的非综合征性CP受试者，他们被分类为Veau I-IV。手术程序将包括3-6个月大的唇裂修复（如果存在），3-6个月大的双侧放置通气管（VT），大约9-11个月大的指定腭成形术，并在腭成形术后重复双侧VT插入，以根据中耳（ME）状态的算法来研究结束。术后评估ME状态（耳镜检查，鼓室测量）将定期进行，并在3年终点进行ETF和听力测试。主要结果包括在5年研究期结束时达到3岁的受试者的ETF和腭裂成形术后OME和听力损失的发生率和患病率。次要结局包括作为临床随访的一部分对CP儿童进行评估的措施；也就是腭咽能力，语言发育以及矫正腭成形术的需要。我们比较这两种腭成形术，因为它们的预期差异影响仅在mTVP功能上。我们的预期是，在MFP组中，ETF会更好，OME和听力损失的患病率会更低，但在3年终点，两组之间的次要结局指标不会有差异。在12名受试者/组中，在涉及操作这些肌肉的每个步骤之前和之后，将在电刺激左侧mTVP和左侧提上睑腭肌（mLVP）时进行术中ETF记录。我们期望不同程序步骤的测试结果将阐明这些肌肉在CP患者的ETF中所起的作用。在该申请的竞争性更新中，我们计划对所有7岁以下的入组受试者进行类似评估，并在4岁、5岁、6岁和7岁时进行额外的结果评估。我们选择120个样本量是为了允许到7岁时有25%的辍学率，从而保留检验各种假设的统计能力。如果在3岁及以后的主要结果方面，MFP被证明比FP更好，并且与不良事件或对其他功能的不良影响无关，这些结果将为改变当前腭成形术的方式提供基础，重新强调减少CP疾病的耳科并发症。

DESCRIPTION (provided by applicant): We propose a 5-year, prospective, randomized, blinded, controlled study that evaluates the comparative efficacies of 2 methods of palatoplasty with respect to improving Eustachian tube function (ETF) and reducing the prevalences of otitis media with effusion (OME) and hearing loss in cleft palate (CP) patients without causing adverse events or having detrimental effects on the other functions affected by the presence of a CP. The palatoplasties evaluated are the Furlow double opposing Z plasty with bilateral transaction of the tensor veli palatini muscle (mTVP) tendon (FP) and a modification of that procedure that includes bilateral tensor tenopexy (MFP), i.e. the attachment of mTVP tendons to the ipsilateral hamulus before mTVP tendon transection. Over a 4 year period, we will enroll 120 non-syndromic CP subjects by age 6 months who are classified as Veau I-IV. The surgical procedures will include cleft lip repair (if present) at 3-6 months of age, bilateral placement of a ventilation tube (VT) at 3-6 months of age, the designated palatoplasty at approximately 9-11 months of age and repeat bilateral VT insertions post-palatoplasty to study end as dictated by an algorithm based on middle ear (ME) status. Post-operative assessment of the ME status (otoscopy, tympanometry) will be done at regular intervals and at the 3-year endpoint with inclusion of ETF and hearing tests. Primary outcomes include ETF and the incidences and prevalences of OME and hearing loss after palatoplasty for those subjects who achieve the age of 3 years by the end of the 5 year study period. Secondary outcomes include measures that are assessed in CP children as part of their clinical follow-up; i.e. velopharyngeal competence, speech development and the need for revision palatoplasty. We compare these 2 palatoplasty procedures because of their expected differential effects only on mTVP function. Our expectations are that ETF will be better and the prevalences of OME and hearing loss less in the MFP group, but that the secondary outcome measures will not be different between the 2 groups at the 3 year endpoint. In 12 subjects/group, intra-operative recordings of ETF will be done during electrical stimulation of the left mTVP and left Levator Veli Palatini muscle (mLVP) before and then after each step in the procedures that involve manipulation of those muscles. We expect that the test results at different procedural steps will clarify the role played in ETF by these muscles in CP patients. In a competing renewal of this application, we plan to include similar assessments on all enrolled subjects to age 7 with additional outcome evaluations at age 4, 5, 6 and 7 years. Our sample size of 120 was chosen to allow for a drop-out of 25% by 7 years of age and, thus, to retain statistical power to test the various hypotheses. If the MFP proves to be better than the FP with respect to the primary outcomes at age 3 and later years and is not associated with adverse events or untoward effects on the other functions, these results will provide a foundation to argue for changing the way that current palatoplasties are done with a renewed emphasis on reducing the otologic complications of the CP condition. PUBLIC HEALTH RELEVANCE: The prevalence of OM and hearing loss in CP infants approaches 100%, does not decrease after standard methods of palatoplasty and is greater than that for than that for non-CP patients at all ages. This prospective, blinded, randomized study compares the efficacies of the more standard Furlow palatoplasty with a modification of that technique, Furlow palatoplasty with tensor tenopexy, which was designed to improve ETF and, consequently, promote the earlier resolution of OM and hearing loss in those patients. If the modified Furlow palatoplasty is shown to be superior to the standard Furlow palatoplasty with respect to improving ETF, ME status and hearing without adverse consequences on the other functions affected by the presence of a CP, application of this revised procedure is expected to improve the quality of life in CP patients and redirect the focus of CP surgeons to improve the otologic outcomes in these patients.