背景:Mounier-Kuhn综合征或先天性气管支气管肥大是一种罕见的疾病,其特征是胸腔内气管和主支气管扩张。该疾病的主要体征和症状包括咳嗽,脓性和大量咳痰,呼吸困难,打鼾,喘息,反复呼吸道感染.一些患者的疾病症状被认为是由于常驻气管支气管软化症引起的病理表现。虽然用于治疗这种疾病的治疗选择包括吸入支气管扩张剂,皮质类固醇,和高渗溶液,在治疗上没有达成共识。据报道,使用持续气道正压通气(CPAP)是治疗气管支气管软化症的潜在选择。但没有前瞻性研究证明其在这种情况下的疗效。
目的:目的是确定Mounier-Kuhn综合征患者是否存在气管支气管软化和最佳CPAP压力,以减少气管支气管塌陷,并分析肺通气的影响。并行,我们旨在评估阻塞性睡眠呼吸暂停/低通气综合征的患病率.
方法:这种介入,开放标签,单臂临床试验将纳入诊断为Mounier-Kuhn综合征的患者.患者评估将在门诊进行,涉及3次就诊。访问1将涉及社会人口统计的收集和登记,临床,和功能数据。访问2将需要多导睡眠图,支气管镜检查用于评估气管支气管软化,滴定降低气道塌陷程度的最佳压力,和电阻抗层析成像。在第3次访问中,将要求表现出塌陷区域减少的患者在吸气和强制呼气期间接受胸部计算机断层扫描,无论有无正压(滴定以确定最佳CPAP压力)。
结果:该协议是一个博士学位项目。该项目于2017年1月24日提交给机构审查委员会,并于2017年2月2日获得批准(巴西研究数据库编号CAAE64001317.4.000.0068)。患者评估于2018年4月开始。计划招募基于志愿者的可用性和临床稳定性,每月至少进行一次干预,以便在2020年底完成该项目。每次干预后将对每个案例进行初步分析,但详细的结果预计将在2021年第一季度报告。
结论:对于治疗Mounier-Kuhn综合征的最佳治疗方案尚无共识。使用正压可以保持塌陷的气道通畅,起到“气动支架”的作用,以减少气流阻塞的程度。这个,反过来,能促进胸腔分泌物的动员,改善肺通气。
背景:ClinicalTrails.govNCT03101059;https://clinicaltrials.gov/ct2/show/NCT03101059。
■DERR1-10.2196/14786。
BACKGROUND: Mounier-Kuhn syndrome or congenital tracheobronchomegaly is a rare disease characterized by dilation of the trachea and the main bronchi within the thoracic cavity. The predominant signs and symptoms of the disease include coughing, purulent and abundant expectoration, dyspnea, snoring, wheezing, and recurrent respiratory infection. Symptoms of the disease in some patients are believed to be pathological manifestations arising due to resident
tracheobronchomalacia. Although treatment options used for the management of this disease include inhaled bronchodilators, corticosteroids, and hypertonic solution, there is no consensus on the treatment. The use of continuous positive airway pressure (CPAP) has been reported as a potential therapeutic option for
tracheobronchomalacia, but no prospective studies have demonstrated its efficacy in this condition.
OBJECTIVE: The purpose of this is to identify the presence of
tracheobronchomalacia and an optimal CPAP pressure that reduces the tracheobronchial collapse in patients with Mounier-Kuhn syndrome and to analyze the repercussion in pulmonary ventilation. In parallel, we aim to evaluate the prevalence of obstructive sleep apnea/hypopnea syndrome.
METHODS: This interventional, open-label, single-arm clinical
trial will enroll patients who are diagnosed Mounier-Kuhn syndrome. Patient evaluation will be conducted in an outpatient clinic and involve 3 visits. Visit 1 will involve the collection and registration of social demographic, clinical, and functional data. Visit 2 will entail polysomnography, bronchoscopy for the evaluation of
tracheobronchomalacia, titration of the optimal pressure that reduces the degree of collapse of the airway, and electrical impedance tomography. In visit 3, patients exhibiting a reduction in collapse areas will be requested to undergo chest computed tomography during inspiration and forced expiration with and without positive pressure (titrated to determine optimal CPAP pressure).
RESULTS: This protocol is a doctorate project. The project was submitted to the institutional review board on January 24, 2017, and approval was granted on February 2, 2017 (Brazilian Research database number CAAE 64001317.4.000.0068). Patient evaluations started in April 2018. Planned recruitment is based on volunteers\' availability and clinical stability, and interventions will be conducted at least once a month to finish the project at the end of 2020. A preliminary analysis of each case will be performed after each intervention, but detailed results are expected to be reported in the first quarter of 2021.
CONCLUSIONS: There is no consensus on the best treatment options for managing Mounier-Kuhn syndrome. The use of positive pressure could maintain patency of the collapsed airways, functioning as a \"pneumatic stent\" to reduce the degree of airflow obstruction. This, in turn, could promote mobilization of thoracic secretion and improve pulmonary ventilation.
BACKGROUND: ClinicalTrails.gov NCT03101059; https://clinicaltrials.gov/ct2/show/NCT03101059.
UNASSIGNED: DERR1-10.2196/14786.