Methods to Improve Expiratory Cough Flow and Lung Volume in Children With NMDs

NCT02342132 | Completed

• 1 other identifier: H14-00473
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

Children with neuromuscular diseases (NMDs) and weak chest muscles suffer frequent chest infections, hospital admissions and reduced life expectancy. Physiotherapy is widely used but there is limited research data to support choice of therapy. The investigators will study the clinical value of the three most commonly used chest physiotherapy devices in children with NMD's by measuring lung function tests before and after each of the three treatments. This is a pilot study. The best device will be selected for a long term study of early physiotherapy intervention in children with NMDs. The treatments that have been developed to break the cycle of decreasing lung volume and poor secretion clearance, in children with NMDs, are all based on the same logical but unproved theory of treatment:

1. 1.The loss of lung volume is reversed by applying positive pressure to the lungs via a face mask - literally reinflating the lungs with pressure. The three devices the investigators will test all provide positive pressure assistance during the inspiratory phase.
2. 2.The temporarily increased lung volume then allows higher expiratory flow rates which helps to clear secretions. The expiratory phase is assisted by manual chest compression or, in one device, helped by application of negative pressure during exhalation.
3. 3.Treatments combining positive pressure inhalation with assisted coughing during exhalation, will produce measurable improvements in lung volume and expiratory cough flow rates when tested in children with NMDs.
4. 4.One of the three devices tested will be superior to the others. This will provide research-based justification for a long term study of the value of early intervention in children with NMDs.

Conditions

Neuromuscular Diseases

Outcome Measures

Primary Outcomes (3)

• Lung Volume

  Assessed by measuring vital capacity (FVC). This is a widely used test that measures effective lung volume. There are well established pediatric predictive values for FVC.

  Therapy effect assessed for 2hrs post treatment

• Cough Expiratory Flows

  Cough flows needed for adequate clearance of airway secretions. This study will measure peak expiatory flow (PEF) for which normal values are easily accessible. The two measures are comparable in clinical use (8).

  Therapy effect assessed for 2hrs post treatment

• Muscle Strength

  Assessed by measuring maximal inspiratory pressure (MIP) and maximal expiratory pressures (MEP). These are obtained by inhaling and exhaling maximally through a manometer.

  Therapy effect assessed for 2hrs post treatment

Study Arms (1)

Respiratoy Therapy Devices

EXPERIMENTAL

Non-Invasive Bipap Ventilator, Mechanical Insufflator-Exsufflator, and High Frequency Percussive Oscillator are the three respiratory therapy devices that all participants will be using in a random order, in three consecutive days, one device per day.

Device: Non-invasive bipap ventilator; Device: Mechanical insufflator-exsufflator; Device: High frequency percussive oscillator

Interventions

Non-invasive bipap ventilator DEVICE

The non-invasive bipap ventilator will be used in the following respiratory treatment: bipap assisted inspiratory breaths (BAIB). The patient will be encouraged to take deep breaths at least 1.5 times greater than resting tidal volume. The pressure assistance provided by the bipap starts at 10cm H2O. Patients already on bipap will use their home settings. Starting pressure is then increased as necessary to meet the maximal inspiratory goal volume in steps of 2 up to a maximum 20cm H2O. Once the ideal pressure of support levels are established, the child will perform 3 cycles of 5 deep breaths. After each cycle the patient will pause and perform huffing and assisted coughing. The patient will be suctioned as necessary.

Respiratoy Therapy Devices

Mechanical insufflator-exsufflator DEVICE

A commercial mechanical insufflator-exsufflator will be used in the following treatment: Mechanical insufflation-exsufflation (MI-E). The pressure will be administered by a face mask. The optimal pressure for the test will start at 10 cm H2O and will in crease in steps of 5 to a maximum of 25 cm H2O. The insufflation phase will take 3 seconds followed by a 2 second exsufflation phase. Three cycles of treatment will be used. Between each cycle the subject will pause and perform huffing and assisted coughing. The patient will be suctioned as necessary.

Respiratoy Therapy Devices

High frequency percussive oscillator DEVICE

A commercial high frequency percussive oscillator will be used in the following treatment: combined oscillation and nebulisation. Pressures will be delivered by a face mask. At higher oscillation frequencies it is usually necessary to support the cheeks. Pressure will be gradually be increased to accustom the child to a maximum of 25cm H2O. This standard maximal pressure will ideally be used for every child. A treatment cycle will consist of 3 minutes at a frequency of 2 Hz and pressure of 15cm H2O followed by 3 minutes of 4 Hz at 20cm H2O, then 3 minutes of 5 Hz at 25cm H2O. Throughout each cycle, the patient will receive nebulized saline through the machine. After each cycle the patient will pause and perform huffing and assisted coughing. The patient will be suctioned as necessary.

Respiratoy Therapy Devices

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Child must have neuromuscular disease (of any diagnostic type)
• Child and family must be enrolled in the NMD clinic at Children's hospital
• Child must be old enough and cognitively able to perform lung function tests(usually about 5 to 6 years old)

You may not qualify if:

• Child or guardian refuses consent for the study
• Child has a tracheostomy
• Child is too young or cognitively unable to perform lung function tests
• Child has a facial asymmetry that prevents a good seal with the mask

Sponsors & Collaborators

• University of British Columbia: lead

Study Sites (1)

BC's Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada

Location

Related Publications (12)

• Hull J, Aniapravan R, Chan E, Chatwin M, Forton J, Gallagher J, Gibson N, Gordon J, Hughes I, McCulloch R, Russell RR, Simonds A. British Thoracic Society guideline for respiratory management of children with neuromuscular weakness. Thorax. 2012 Jul;67 Suppl 1:i1-40. doi: 10.1136/thoraxjnl-2012-201964. No abstract available.

  PMID: 22730428 BACKGROUND

• Suarez AA, Pessolano FA, Monteiro SG, Ferreyra G, Capria ME, Mesa L, Dubrovsky A, De Vito EL. Peak flow and peak cough flow in the evaluation of expiratory muscle weakness and bulbar impairment in patients with neuromuscular disease. Am J Phys Med Rehabil. 2002 Jul;81(7):506-11. doi: 10.1097/00002060-200207000-00007.

  PMID: 12131177 BACKGROUND

• McCool FD, Rosen MJ. Nonpharmacologic airway clearance therapies: ACCP evidence-based clinical practice guidelines. Chest. 2006 Jan;129(1 Suppl):250S-259S. doi: 10.1378/chest.129.1_suppl.250S.

  PMID: 16428718 BACKGROUND

• Jenkins HM, Stocki A, Kriellaars D, Pasterkamp H. Breath stacking in children with neuromuscular disorders. Pediatr Pulmonol. 2014 Jun;49(6):544-53. doi: 10.1002/ppul.22865. Epub 2013 Aug 16.

  PMID: 23956183 BACKGROUND

• Toussaint M, Boitano LJ, Gathot V, Steens M, Soudon P. Limits of effective cough-augmentation techniques in patients with neuromuscular disease. Respir Care. 2009 Mar;54(3):359-66.

  PMID: 19245730 BACKGROUND

• Homnick DN. Mechanical insufflation-exsufflation for airway mucus clearance. Respir Care. 2007 Oct;52(10):1296-305; discussion 1306-7.

  PMID: 17894900 BACKGROUND

• Chatwin M, Ross E, Hart N, Nickol AH, Polkey MI, Simonds AK. Cough augmentation with mechanical insufflation/exsufflation in patients with neuromuscular weakness. Eur Respir J. 2003 Mar;21(3):502-8. doi: 10.1183/09031936.03.00048102.

  PMID: 12662009 BACKGROUND

• Miske LJ, Hickey EM, Kolb SM, Weiner DJ, Panitch HB. Use of the mechanical in-exsufflator in pediatric patients with neuromuscular disease and impaired cough. Chest. 2004 Apr;125(4):1406-12. doi: 10.1378/chest.125.4.1406.

  PMID: 15078753 BACKGROUND

• McIlwaine MP, Alarie N, Davidson GF, Lands LC, Ratjen F, Milner R, Owen B, Agnew JL. Long-term multicentre randomised controlled study of high frequency chest wall oscillation versus positive expiratory pressure mask in cystic fibrosis. Thorax. 2013 Aug;68(8):746-51. doi: 10.1136/thoraxjnl-2012-202915. Epub 2013 Feb 13.

  PMID: 23407019 BACKGROUND

• Toussaint M, De Win H, Steens M, Soudon P. Effect of intrapulmonary percussive ventilation on mucus clearance in duchenne muscular dystrophy patients: a preliminary report. Respir Care. 2003 Oct;48(10):940-7.

  PMID: 14525630 BACKGROUND

• Reardon CC, Christiansen D, Barnett ED, Cabral HJ. Intrapulmonary percussive ventilation vs incentive spirometry for children with neuromuscular disease. Arch Pediatr Adolesc Med. 2005 Jun;159(6):526-31. doi: 10.1001/archpedi.159.6.526.

  PMID: 15939850 BACKGROUND

• Finder JD, Birnkrant D, Carl J, Farber HJ, Gozal D, Iannaccone ST, Kovesi T, Kravitz RM, Panitch H, Schramm C, Schroth M, Sharma G, Sievers L, Silvestri JM, Sterni L; American Thoracic Society. Respiratory care of the patient with Duchenne muscular dystrophy: ATS consensus statement. Am J Respir Crit Care Med. 2004 Aug 15;170(4):456-65. doi: 10.1164/rccm.200307-885ST. No abstract available.

  PMID: 15302625 BACKGROUND

MeSH Terms

Conditions

Neuromuscular Diseases

Condition Hierarchy (Ancestors)

Nervous System Diseases

Study Officials

• Mike Seear, MD

  British Columbia's Children's Hospital

  PRINCIPAL INVESTIGATOR

• Mike Seear, MD

  BCCH

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principle Investigator

Study Record Dates

• First Submitted: July 25, 2014
• First Posted: January 19, 2015
• Study Start: August 1, 2014
• Primary Completion: November 1, 2014
• Study Completion: February 1, 2015
• Last Updated: September 21, 2017

Record last verified: 2017-09

Locations

CA (1)