Home Sleep Testing in Neuromuscular Disease Patients (HSTNMD)

Lay Summary Patients with severe neuromuscular develop hypoventilation, which leads to elevated carbon dioxide levels. Measuring only oxygen saturation levels with pulse oximetry may be inadequate. End tidal carbon dioxide levels or arterial blood gases should be measured periodically, depending on the clinical condition of the patient. A thorough review of systems will help define any problems. Patients who are hypoventilating often have elevated carbon dioxide levels at night and complain of a morning headache, restlessness or nightmares, and poor quality sleep. This may cause daytime sleepiness. Insufficient respiration with hypoxia may occur later, especially if the lung is damaged by chronic aspiration.

We propose to evaluate the use of the Nonin LifeSense monitor in home evaluation of respiration, oxygen level, heart rate, and carbon dioxide level and to develop interpretation of the results that will lead to appropriate interventions for apnea, and insufficient respiration.

Relevance to MDA Fewer than one per cent of the Muscular Dystrophy Association have pulmonologists as co-directors.Late referral of progressive restrictive lung disease leads to invasive support of respiratory failure. Early initiation of non invasive ventilation techniques requires patience on the part of the caregiver and exploration of mask interfaces and ventilation techniques. In addition, the development of new therapies, currently manifested through enhanced diagnostic accuracy, will require new signal for initiation and in the assessment of success or failure.

Aims Aim 1. To assess the utility of a small portable device (LifeSense Monitor Nonin Medical Inc. Plymouth Minnesota) with extended recording capabilty to provide accurate diagnosis of hypoventilation.

Aim 2. To provide an easily interpretable report defining sleep hypoxemia, hypercapnea, and apnea.

Aim 3. To promote early evaluation and treatment of the respiratory problems in centers that do not have pumonologists as these are essential to prognosis, whether of survival or of quality of life, in neuromuscular diseases.

Study Overview

• Status: Completed
• Conditions: Neuromuscular Disease
• Intervention / Treatment: Other: Evaluation for Nocturnal non invasive ventilation

• Study Type: Observational
• Enrollment (Actual): 9

Contacts and Locations

Study Locations

• United States
  • California
    • Ventura, California, United States, 93003
      • Pediatric Diagnostic Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 years to 50 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Study Population Description Patients from the Neuromuscular Disease Clinic at Ventura County Medical Center

Non-Probability Sample: invitation to volunteer

Neuromuscular Disease

Description

Inclusion Criteria:

• Clinical diagnosis of neuromuscular disease
• Must have home caregivers

Exclusion Criteria:

• No home caregiver

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

Number of groups / cohorts

4

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
1SevereRLD,NMD; Patients with FEV1<40%	Other: Evaluation for Nocturnal non invasive ventilation; Patients will be referred for evaluation for NNIV is indicated in patients with diurnal Pa,CO2 >6.0 Pa; and 2) nocturnal Sa,O2 <88% for 5 consecutive min. End tidal CO2 representing equivalent values will be referred for NNIV
2VerySevereRLD,NMD; FEV1<30%	Other: Evaluation for Nocturnal non invasive ventilation; Patients will be referred for evaluation for NNIV is indicated in patients with diurnal Pa,CO2 >6.0 Pa; and 2) nocturnal Sa,O2 <88% for 5 consecutive min. End tidal CO2 representing equivalent values will be referred for NNIV
3NINV; FEV1<25%,on non invasive ventilation	Other: Evaluation for Nocturnal non invasive ventilation; Patients will be referred for evaluation for NNIV is indicated in patients with diurnal Pa,CO2 >6.0 Pa; and 2) nocturnal Sa,O2 <88% for 5 consecutive min. End tidal CO2 representing equivalent values will be referred for NNIV
ModerateRLD,NMD; Patients with FEV1 40-50% of predicted	Other: Evaluation for Nocturnal non invasive ventilation; Patients will be referred for evaluation for NNIV is indicated in patients with diurnal Pa,CO2 >6.0 Pa; and 2) nocturnal Sa,O2 <88% for 5 consecutive min. End tidal CO2 representing equivalent values will be referred for NNIV

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
1. End tidal CO2 catheter is tolerated for six hours or more	3 months

Secondary Outcome Measures

Outcome Measure	Time Frame
1. Interpretable data are captured for oximetry, heart rate, and end-tidal CO2	3 months
2. An interpretive report is generated that will guide the non-pulmonary physician in potential interventions	6 months
2. Patients and parents are able to apply the monitors at home and a six hour data set is collected	3 months

Collaborators and Investigators

• Sponsor: Landon Pediatric Foundation

Publications and helpful links

General Publications

• Shneerson JM, Simonds AK. Noninvasive ventilation for chest wall and neuromuscular disorders. Eur Respir J. 2002 Aug;20(2):480-7. doi: 10.1183/09031936.02.00404002.
• Kumar SP, Sword D, Petty RK, Banham SW, Patel KR. Assessment of sleep studies in myotonic dystrophy. Chron Respir Dis. 2007;4(1):15-8. doi: 10.1177/1479972306074480.
• Kirk VG, Flemons WW, Adams C, Rimmer KP, Montgomery MD. Sleep-disordered breathing in Duchenne muscular dystrophy: a preliminary study of the role of portable monitoring. Pediatr Pulmonol. 2000 Feb;29(2):135-40. doi: 10.1002/(sici)1099-0496(200002)29:23.0.co;2-#.
• Birnkrant DJ, Panitch HB, Benditt JO, Boitano LJ, Carter ER, Cwik VA, Finder JD, Iannaccone ST, Jacobson LE, Kohn GL, Motoyama EK, Moxley RT, Schroth MK, Sharma GD, Sussman MD. American College of Chest Physicians consensus statement on the respiratory and related management of patients with Duchenne muscular dystrophy undergoing anesthesia or sedation. Chest. 2007 Dec;132(6):1977-86. doi: 10.1378/chest.07-0458.

Study record dates

Study Major Dates

• Study Start: July 1, 2008
• Primary Completion (Actual): September 1, 2008
• Study Completion (Actual): July 1, 2009

Study Registration Dates

• First Submitted: June 3, 2008
• First Submitted That Met QC Criteria: June 11, 2008
• First Posted (Estimate): June 12, 2008

Study Record Updates

• Last Update Posted (Estimate): August 13, 2014
• Last Update Submitted That Met QC Criteria: August 12, 2014
• Last Verified: August 1, 2014

More Information

Terms related to this study

• Keywords: Home sleep testing neuromuscular disease; Neuromuscular disease patients with FEV1<50% of predicted
• Additional Relevant MeSH Terms: Nervous System Diseases; Neuromuscular Diseases
• Other Study ID Numbers: LS01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No