Airway Collapse in Patients With Mounier-Kuhn Syndrome: Titration With Positive Pressure to Reduce Collapse

Evaluation of the Presence of Airway Collapse in Patients With Mounier-Kuhn Syndrome and Titration With Continuous Positive Pressure Through Non-invasive Mechanical Ventilation to Reduce Collapse

Mounier-Kuhn syndrome (MKS), or congenital tracheobronchiomegaly, is an entity characterized by dilation of the trachea and bronchi, associated with respiratory infections.The main signs and symptoms are cough, bulging and purulent expectoration, digital clubbing, dyspnoea, and wheezing.Some of these symptoms are believed to be due to excessive collapse of the intra-thoracic trachea and bronchi, resulting in airways obstruction of more than 50% . The purpose of this study is to identify and reduce tracheal collapse.

Study Overview

• Status: Unknown
• Conditions: Mounier-Kuhn Syndrome
• Intervention / Treatment: Procedure: Non invasive ventilation - Continuous Positive Airway Pressure

Detailed Description

Mounier-Kuhn syndrome (MKS), or congenital tracheobronchiomegaly, is an entity characterized by dilation of the trachea and bronchi, associated with respiratory infections. The prevalence among patients with respiratory symptoms is 0.4 to 1.6%. Its histological features include the atrophy or absence of longitudinal elastic fibers and smooth muscle cells of the airways, responsible for the structural alterations found, such as tracheobronchiomegaly, the presence of inter cartilaginous diverticula, bulging and dilation of the walls of the trachea and bronchi. The main signs and symptoms are cough, bulging and purulent expectoration, digital clubbing, dyspnoea, and wheezing and wheezing accompanied by recurrent respiratory infection. There may be association with other comorbidities such as gastroesophageal reflux disease, chronic obstructive pulmonary disease, bronchiectasis and obstructive sleep apnea / hypopnea syndrome (OSAS). Some of these symptoms are believed to be due to the tracheobronchial disease present in some patients, defined by excessive collapse of the intrathoracic trachea and bronchi resulting in airways obstruction of more than 50%. The main clinical impact is obstruction to expiratory airflow, with consequent air entrapment, reduction of cough and bronchial hygiene effectiveness, facilitating recurrent respiratory infections. Because it is a rare morbidity and little studied, the specific therapy is not consensual, and the main interventions are extrapolated from other pathologies. The use of non invasive mechanical ventilation (NIMV) with continuous positive airway pressure (CPAP) is reported as an option for treatment, however, there are no randomized studies proving its efficacy. The purpose of this study is to identify and reduce tracheal collapse and bronchi of SMK carriers with the use of positive pressure (CPAP-NIV) and to analyse their repercussion in the small airways.

• Study Type: Interventional
• Enrollment (Anticipated): 15
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Rafael Stelmach, MD-PHD; Phone Number: 551126615191; Email: rafael.stelmach@incor.usp.br
• Study Contact Backup: Name: Evelise Lima, MD; Phone Number: 551126615191; Email: eveliselima53@gmail.com

Study Locations

• Brazil
  • Sao Paulo, Brazil, 05403000
    • Recruiting
    • Divisao de Pneumologia, Instituto do Coracao, Hospital das Clinicas HCFMUSP, Faculdade de Medicina, Universidade de Sao Paulo
    • Contact: Rafael Stelmach, MD-PHD; Phone Number: 551126615191; Email: rafael.stelmach@incor.usp.br
    • Contact: Evelise Lima, MD; Phone Number: 551126615191; Email: eveliselima53@gmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Has Mounier-Khun Syndrome
• Accept and signed informed consent form

Exclusion Criteria:

• Other morbidity avoiding study procedures

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: MKS pax; Munier-Kuhn Syndrome patients

Procedure: Non invasive ventilation - Continuous Positive Airway Pressure; To identify, through bronchoscopy, the prevalence of collapse and whether it is possible to counteract an optimum pressure generated by NIV with CPAP that reduces tracheal and bronchial collapse in patients with SMK; To study the frequency of OSAS in patients with MKS ; Record reversal of collapse with CPAP using chest tomography; To identify the impact of CPAP on the distribution of pulmonary ventilation through the analysis of electrical impedance tomography.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of tracheal-bronchial collapse area before and after applied positive pressure through bronchoscopy	Identify the prevalence of collapse and whether it is possible to counteract an optimum pressure generated by NIV with CPAP that reduces tracheal and bronchial collapse in patients with SMK	18 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Apnea+hypopnea (AHI) index in patients with SMK	Study the frequency of OSAHS in patients with SMK;	24 months
Percentage of tracheal-bronchial collapse area before and after applied positive pressure measure using chest tomography	Record reversal of collapse with CPAP using chest tomography	24 months
Inspiratory and expiratory lung volumes before and after applied positive pressure measure using electrical impedance tomography.	To identify the impact of CPAP on the distribution of pulmonary ventilation through the analysis of electrical impedance tomography.	24 months

Collaborators and Investigators

• Sponsor: University of Sao Paulo General Hospital

Publications and helpful links

General Publications

• Lima E, Nakamura MAM, Genta PR, Rodrigues AJ, Athanazio RA, Rached S, Costa ELV, Stelmach R. Improving Airways Patency and Ventilation Through Optimal Positive Pressure Identified by Noninvasive Mechanical Ventilation Titration in Mounier-Kuhn Syndrome: Protocol for an Interventional, Open-Label, Single-Arm Clinical Trial. JMIR Res Protoc. 2020 Aug 14;9(8):e14786. doi: 10.2196/14786.

Study record dates

Study Major Dates

• Study Start (Actual): June 8, 2017
• Primary Completion (Anticipated): September 1, 2018
• Study Completion (Anticipated): April 1, 2019

Study Registration Dates

• First Submitted: March 23, 2017
• First Submitted That Met QC Criteria: April 3, 2017
• First Posted (Actual): April 4, 2017

Study Record Updates

• Last Update Posted (Actual): October 26, 2017
• Last Update Submitted That Met QC Criteria: October 24, 2017
• Last Verified: October 1, 2017

More Information

Terms related to this study

• Keywords: obstructive sleep apnea syndrome; Tracheal; Bronchial; Tracheobronchiomegaly; Collapse; Computer Tomography; Impedance Tomography
• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Disease; Congenital Abnormalities; Bronchial Diseases; Tracheal Diseases; Respiratory System Abnormalities; Syndrome; Shock; Tracheobronchomegaly
• Other Study ID Numbers: CAAE64001317400000068

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No