Reducing The Cost Of
Pulmonary Impairment In
Children With Neuromuscular
Disease
Chris Landon, MD, FAAP, FCCP
Ventura County Medical Center
Ventura, California
Audrius Plioplys, MD, CMD,FRCPC, FAAP
Mercy Hospital
Chicago, Illinois
Objectives

1. Provide an overview of neuromuscular diseases
and complications that predispose patients to
frequent respiratory exacerbations.

2. Identify treatment strategies/medically
appropriate care to meet needs of this difficult
patient population to promote improvements in
quality of life, and positive clinical outcomes.

3. Discuss evidence supporting the effect of
airway clearance with The Vest system on
reducing costs associated with pulmonary
complications/exacerbations.
Neuromuscular Diseases:
Overview
Children who experience varying degrees of
neurological/neuromuscular dysfunction
 Diagnoses include: cerebral palsy, muscular
dystrophy, spinal muscular atrophy, brain
injury, consequences of infectious disease,
inherited metabolic disorders, etc.

– One child in 1000 is institutionalized as a result
of profound disability
Neuromuscular Diseases:
Overview
Multi-system assessment necessary to
determine risk of pulmonary involvement:
Neuromuscular
 Gastroesophageal
 Immune system
 Respiratory
 Psychosocial

Neuromuscular Diseases
Assessment of complications that
predispose to pulmonary involvement
 Neuro
–
–
–
–
–
–
–
assessment
Skeletal muscle control
Respiratory and ventilatory muscle abnormalities
Weak/absent cough
Weak/absent gag reflex
Upper airway control and coordination
Seizure activity
Spasticity
Neuromuscular Diseases
Assessment of complications that
predispose to pulmonary involvement
 Neuro
–
–
–
–
–
assessment – Dysphagia
Oral motor dyskinesia/pseudobulbar palsy
True bulbar palsy
Dysfunctional arousal
Oral motor weakness
Increased secretions
Neuromuscular Diseases
Assessment of complications that
predispose to pulmonary involvement

Neuro assessment – Oral
motor weakness

Typical symptoms
– Muscular dystrophies
– Too weak to swallow
– Myopathies
– Too weak to cough
– Neuromuscular junction
disorders
– Easily fatigued
– Anterior horn cell
disorders
– Head position
dependent
Neuromuscular Diseases
Assessment of complications that
predispose to pulmonary involvement

Neuro assessment –
Increased secretions

Typical symptoms
– Autonomic
dysfunction
– Constant drooling
– Medication effects
– Worse with stress
or infection
– Frequent seizures
– Drowning in drool
Poor Swallow Control
(Dysphagia)
Oral motor dyskinesia / pseudobulbar palsy
 True bulbar palsy
 Dysfunctional arousal
 Oral motor weakness
 Increased secretions

Oral Motor Dyskinesia /
Pseudobulbar Palsy
Diffuse or bilateral cortical damage
 Basal ganglia damage
 Cerebellar – brainstem damage

Oral Motor Dyskinesia /
Pseudobulbar Palsy

Typical symptoms:
–
–
–
–
Increased gag, choking, vomiting
Nasal regurgitation
Tonguing – pushing
Poor tolerance of liquids and chunks
True Bulbar Palsy
Cranial nerves 9, 10, 12
 Pontine-medullary damage
 Arnold-Chiari malformation
 Bulbar syrinx
 Moebius syndrome

True Bulbar Palsy

Typical symptoms
–
–
–
–
–
Decreased gag, poor palate movement
Pocketing of food
Rumination
Drooling
“O” sign
Dysfunctional Arousal
Disorder of excessive somnolence
 Toxic encephalopathy
 Drug induced stupor
 Autistic disorder

Dysfunctional Arousal

Typical symptoms
–
–
–
–
Must be reminded to swallow
Falls asleep while eating
Poor cough reflex
“Q” sign, string bean sign
Oral Motor Weakness
Myopathies
 Muscular dystrophies
 Neuromuscular junction disorders
 Anterior horn cell disorders

Oral Motor Weakness

Typical symptoms
–
–
–
–
Too weak to swallow
Too weak to cough
Easily fatigued
Head position dependent
Increased Secretions
Autonomic dysfunction
 Medication effects
 Frequent seizures

Increased Secretions

Typical symptoms
– Constant drooling
– Worse with stress or infection
– Drowning in drool
Poor Breathing Control
Central neurogenic hypoventilation
 Periodic breathing patterns
 Ondine’s curse
 Stupor and coma
 Thoracic weakness

Central Neurogenic
Hypoventilation
Diffuse cortical damage
 Poor hypoxic response
 Poor hypercarbic response
 Worse with stress or infection

Periodic Breathing Patterns
Cheyne-Stokes respiration
 Biot’s respiration
 Rett’s syndrome
 Apneustic breathing

Ondine’s Curse
Waking versus sleeping centers
 Defect in shift to automatic breathing
 “… if I should die before I wake…”

Stupor and Coma
Brainstem dysfunction from pressure
 Brainstem dysfunction from ischemia
 Brainstem suppression from drugs
 Brainstem degeneration

Thoracic Weakness
Myopathies
 Muscular dystrophies
 Neuromuscular junction disorders
 Anterior horn cell disorders

Recognition of Neurogenic
Pulmonary Clearance Problems
Recognize the possible brain region
involved
 Perform a careful history
 Perform a careful examination
 Sleep study / Life Shirt
 MRI – attention to brainstem
 ? muscle studies ?

Gastroesophageal Function and
Complications



The Upper AirwaySwallowing and
Aspiration
Aspiration Associated
Pneumonias
Lower Esophageal
Aspiration, Gastric
Distention and Airway
Remodeling



Gastroesophageal Reflux
Disease (GERD)
Fundoplication Versus
Medication and Airway
Clearance
Nutrition and the Immune
System
The Faces of Dysfunction
Arching
Failure To Thrive
Irritability
Gagging and Choking
Regurgitation
Refusing Feedings
Sandifer’s Syndrome
Characterized
by arching and
turning the head to the side
(opisthotonos) may give the
appearance of a seizure.
Functions to clear the esophagus
of acid reflux by increasing
intrathoracic pressure.
Cardiorespiratory Reflexes
Cardiorespiratory Reflexes
Refluxate
Refluxate Protection
The Immune System




Genetic Abnormalities
Nutritional

Compromise of the
Immune System
Stress and Immune 
Response
Recurrent Infection
and Frequent Use of
Antibiotics: The
Impacts
Allergies
Reactive Airway
Disease (RAD)
Airway Clearance
Therapy
Respiratory Medical History
–
Number of Pulmonary Infections Annually
–
Number of Hospital Admissions Annually
–
Number of ER Admissions Annually
–
Number of Courses of Antibiotics for Respiratory
Infections Annually
–
Immunization History
–
History of Recurrent Infections with Respiratory
Syncytial Virus (RSV)
Quality Airway Clearance Therapy
Should
•Clear secretions effectively and consistently
•Preserve lung function
•Reduce infectious exacerbations
•Reduce dependence on antibiotic therapy and other medications
•Reduce need for hospitalization and auxiliary medical services
•Delay disease progression
•Reduce the burden of care
•Enhance the quality of life
Neuromuscular Disease - Perioperative
Care
High Risk For Post-Operative
Complications






Atelectasis
Pneumonia
Respiratory Failure
Need for prolonged ventilation
Tracheostomy
Death
Problems





Weak cough
Dyscoordinated swallow
Aspiration
Difficulty clearing secretions
Increased lower respiratory tract
infections
Respiratory Weakness


May not be apparent on physical exam
Respiratory failure when work of breathing
is increased
Chronic Respiratory Muscle Weakness









Reduced lung volumes
Microatelectasis
V/Q mismatch
Scoliosis
Decreased compliance of the chest wall
Decreased pulmonary compliance
Hypoxemia only during sleep
Hypoventilation due to muscle weakness
Hypoventilation due to central hypoventilation
Preoperative Assessment



Thorough history
Physical examination
Laboratory studies
Thorough History



Frequency and severity of respiratory tract
infections
Pulmonary complications of previous surgeries
History suggestive of reactive airways disease
– Even mildly increased airway obstruction may
lead to respiratory failure in the postoperative
period in a patient with severe respiratory
muscle weakness
Physical Examination
Gag reflex
 Cough
 Adequacy of aeration
 Presence of adventitial lung
sounds

Ability To Cooperate With PostOperative Pulmonary Therapy


General muscle strength
Physical and intellectual capacity
Laboratory Examinations
Chest x-ray
 Arterial blood gases or mixed
venous gas measurements and
oximetry
 Complete blood count

Pulmonary Function Tests

All children who are capable of performing them
– Lung volumes
– Pre and post bronchodilator
– Maximal inspiratory and expiratory mouth
pressures
»frequently decreased more than lung
volumes and flows
»do not correlate with general muscle
strength
Alternative/Competitive
Airway Clearance Modalities
Chest Physiotherapy

Mechanical techniques for the
noninvasive clearance of excessive
secretions and aspirated materials from
the airway
Prevent, Treat, or At Least Delay
The Effects of Mechanical,Infectious,
and Biochemical Sequelae

prevent resistance to airflow

work of breathing

hyperinflation

atelectasis

maldistribution of ventilation

ventilation-perfusion
mismatch
Microorganisms and Host -Mediated
Inflammatory Mediators

intrabronchial accumulation of
secretions
– vicious circle of infection
– impaired clearance
– progressive airway damage
Improve Access To The Bronchial
Surface

Allow inhaled medications to penetrate
Effectiveness and Safety

Easy to teach to patients and caregivers

Should not fatigue the patient

Time efficient and practical

Whenever possible patient should be
participant
Mechanical Percussors
Mechanical Percussors
•Theory: Shock waves transmit through chest loosening secretions
in the airways—often dependant on positioning to drain mucus into
upper airways
•Technique: Patient is positioned for optimal drainage of lobe to be
percussed with each area being treated for 2-5 minutes
•Considerations: Patient’s mental and physical limitations may
impede, technique dependant, labor intensive, and positioning
tolerance
•Patient’s Cost: $80-150
Positive Expiratory Pressure
(PEP)
Manufacturers: DHD Healthcare & Pari Respiratory Equipment, Inc.
Positive Expiratory Pressure (PEP)
•Theory: Prolonged exhalation with positive pressure stabilizes
smaller airways open allowing inhaled air to pass and then on
exhalation move secretions into larger airways
•Technique: Patient exhales through flow restrictor via mask or
mouth piece and then performs “huff” cough—typical treatment
last about 15-20 minutes
•Considerations: Patient’s mental and physical limitations may
impede, technique dependant, for ages >4, unit is portable, and
maybe self administered
•Patient’s Cost: $30
Flutter Valve
Manufacturer: Scandipharm
Flutter Valve
•Theory: Intermittent positive pressure breaths with airway
oscillation vibrates the airway walls and loosens secretions—
accelerated expiratory flow rates move secretions to larger airways
•Technique: Patient exhales through the device causing the steel
ball to oscillate rapidly for 5-6 breaths than coughs and repeats for
a minimum of 15 minutes—the position/angle of the device
determines intensity of vibration
•Considerations: For ages > 5, technique dependant, requires
cognitive and physical function, portable, and may be self
administered
•Patient’s Cost: $90
Acapella
Manufacturer: DHD Healthcare
Acapella
•Theory: Intermittent positive pressure breaths with airway
oscillation vibrates the airway walls and loosens secretions—
accelerated expiratory flow rates move secretions to larger airways
•Technique: Patient actively exhales through the device causing
rapid oscillation for 10-20 breaths than coughs and repeats for
between 15-25 minutes—the dial on the end of the device
determines intensity of vibration
•Considerations: May not be appropriate for younger patients
(<5y/o), technique dependant, requires cognitive and physical
function, portable, is not position dependant, and may be self
administered
•Patient’s Cost: $88
Intrapulmonary Percussive Ventilation
(IPV)
Manufacturer: Percussionaire
Intrapulmonary Percussive Ventilation (IPV)
•Theory: High Frequency positive pressure air puffs are thought to
open collapsed areas with in the lung and help air get behind mucus
plugs aiding in clearance
•Technique: During inspiration the patient/caregiver depresses
button that triggers delivery of up to 300 bursts of air per minute—
the button is released during exhalation and the cycle repeated for
20 minutes, followed by coughing to clear secretions
•Considerations: May be used by vent patients, technique
dependant, requires adequate cognitive and physical abilities, for
ages >5, increased chances of air swallowing, position independent,
may be self administered, and may be used for nebulized meds.
•Patient’s Cost: $8,200--??warranty
PercussiveNeb
Manufacturer: Vortran Medical Technology1, Inc.
PercussiveNeb
•Theory: High Frequency positive pressure air puffs are thought to
open collapsed areas with in the lung and help air get behind mucus
plugs aiding in clearance
•Technique: During inhalation the patient receives nebulized
medication/saline, while on exhalation the unit delivers positive
pressure burst up to 800 times per minute—typical treatment lasts
between 10-20 minutes followed by coughing to clear secretions
• Considerations: Technique dependant, requires adequate cognitive
and physical abilities, for ages >5, increased chances of air
swallowing, position independent, may be self administered, and may
be used for nebulized meds.
•Patient’s Cost: $140
Cough Assist
(InExsufflator)
Manufacturer: J. H. Emerson Co.
Cough Assist (InExsufflator)
•Theory: Positive pressure to the airway, followed by a rapid
transition to negative pressure produces cough like expiratory flows
aiding in secretion clearance from larger airways
•Technique: Via facemask, mouth piece, or trach adaptor , positive
pressure is delivered to the airway and then shifted rapidly to a
negative pressure for 4-5 breaths, followed by a resting period—
typical treatment time is between 10-15 minutes followed by
coughing to clear secretions
•Considerations: Technique dependant, requires caregiver/patient
cooperation, geared towards patients with weak cough function, and
may be self administered by some users
•Patient’s Cost: $6,000—2 year warranty
MedPulse
Manufacturer: ElectroMed, Inc.
Med Pulse
•Theory: High frequency chest wall oscillation clears secretions by
both creating shear forces between air and secretions in the lungs,
moving mucus towards larger airways, and decreasing the viscosity,
allowing for easier clearance
•Technique: Patient places vest around chest setting prescribed
pressure and frequency then turns unit on pausing at 5-10 minute
intervals to cough and clear secretions—treatment typically lasts 1530 minutes
•Considerations: For ages >2, technique independent, provides
passive therapy, may be used on patients with cognitive/physical
impairments, and treats all lobes simultaneously
•Patient’s Cost: $11,999 with vests costing $500 each—2 year
warranty
Hayek Oscillator
Manufacturer: Breasy Medical Equipment, Ltd.
Hayek Oscillator
•Theory: High frequency chest wall oscillation clears secretions by
both creating shear forces between air and secretions in the lungs,
moving mucus towards larger airways, and decreasing the
viscosity, allowing for easier clearance—the Hayek is primarily
designed as a non-invasive ventilator with a airway clearance mode
and uses both positive and negative pressure during oscillation
•Technique: Patient places shell snuggly around chest setting
prescribed pressure and frequency then turns unit on—we are
unaware of a standard protocol for use in airway clearance
•Considerations: Shell fit may be difficult with abnormally shaped
chests, little offered in the way of product information/support, for
use on neonates to adults, and weighs 110 pounds
•Patient’s Cost: Last reported around $20,000
Airway Clearance
Percussion and Postural Drainage
-Mechanical percussors
Expiratory Pressure Modalities
-Positive Expiratory Pressure (PEP)
-Flutter Valve
-Acapella
High-Frequency Intrapulmonary Positive Pressure
-Intrapulmonary Percussive Ventilation (IPV)
-PercussiveNeb
Intrapulmonary Positive/Negative Pressure
-Cough Assist (InExsufflator)
High-Frequency Chest Wall Oscillation
-MedPulse
-Hayek Oscillator
The Aristotelian tradition, defined tragedy as a
drama which concerns better than average
people (heroes, kings, gods) who suffer a
transition from good fortune to bad fortune,
and who speak in an elevated language.
Tragedy, in the Aristotelean tradition, serves
the purpose of purging the soul of the "fear
and pity" which most of us carry around
(Aristotle called this catharsis ).
In some ancient Greek drama, an
apparently insoluble crisis was solved by
the intervention of a god, often brought on
stage by an elaborate piece of equipment.
This "god from the machine" was literally
a deus ex machina.
We have a group of 5 patients for whom chest physiotherapy did not
achieve effective airway clearance and who where to small to be
effectively fit with Advanced Respiratory (formerly American
Biosystems) child-medium vest for oscillatory therapy. We changed the
configuration of the vest to a "belt" without occluding any of the surface
features of the vest. The Model 103 generator from Advanced
Respiratory was used. We were able to achieve an effective fit for each
of these children without significant changes in pressure transmitted to
the chest (.30 psig(vest) versus .38 psig(belt)). Pressures for therapy
were adjusted downward and no child received therapy at a pressure
setting higher than 4 (range 0 to 10) on the 103 Model
Generator. Frequencies for therapy were kept in the range of 10-16 Hz
(range 0-25 Hz). Therapy with the "belt" was well tolerated by this
group of children and effective therapy could be delivered. There were
no adverse events related to this modification. This simple modification
may be a helpful adjunct to achieving effective airway clearance therapy
in this unique group when chest physiotherapy fails to achieve the
desired results.
Risk For Anesthetic Complications

Malignant hypothermia

Cardiac arrhythmia

Excessive potassium release

Myoglobinuria

Prolonged skeletal muscle contraction in
muscular dystrophy
– succinylcholine
With Care We Can Minimize PostOp
Risk Even In Severe Neuromuscular
Disease

Ventilatory support planned for
24-48 hours post surgery

But avoiding more prolonged
ventilation
– disuse atrophy
– respiratory muscle weakness
Chest Physiotherapy Is The Keystone To
Success

Unable to perform incentive spirometry

Clearance of secretions and prevention
of atelectasis

Every 2 to 4 hours after surgery
Frequency May Be Reduced If Tolerates
Extubation For 24 Hours Without
Complication

IPPB may be helpful in severe
neurologic disease'

Oxygen saturation should be >95%

Hematocrit over 35%
Careful Attention To Fluid Balance

Decreased pulmonary compliance and
hypoxemia if lung water is increased

Scoliosis repair even more critical
setting
Careful Attention To Even Minimal
Bronchospasm

Bronchodilator aerosols

Consider theophylline or steroids
Careful Attention To Atelectasis
Prevention

Bronchodilator aerosols

Mucolytic aerosols

Vigorous CPT and suctioning

IPPB

Consider bronchoscopy
– removing secretions
– obtaining culture
Direct Antibiotic Therapy Against
Specific Pathogens

Normal mouth flora and anerobes
secondary to aspiration

Consider surveillance cultures prior to
surgery

Posterior pharyngeal cultures
"Pulmonary evaluation of
rehabilitation and therapy in
medically fragile children" which
is being submitted in response to
the RFA HD#02-027
HYPOTHESIS:

Children and adolescents (6 to 18 years of
age) with moderate to severe developmental
disabilities with an adequate regimen of
pulmonary toilet can have an improved
quality of life.
The development, validation and
analysis of the three proposed tools will
facilitate the statistical evaluation of
specific indicators that correlate to risk
of further deterioration in pulmonary
function.
This study will 1) further define the
scope and nature of respiratory failure in
children, especially those with special
needs, 2) develop and evaluate acute and
chronic management strategies to
prevent or minimize the development of
chronic respiratory failure in children or
to ameliorate its effects.
Recent scientific and engineering
advances, including the availability of
innovative approaches and techniques for
measuring and treating pulmonary
dysfunction open new avenues of
exploration to be utilized to enhance the
management and quality of life for
children with respiratory failure.
This study will focus on those children
and adolescents with moderate to severe
developmental disabilities, who reside in
residential care, skilled nursing,
domiciliary facilities and their group
homes.
Subjects will include ethnically diverse, male
and female subjects. Standard procedures will
be utilized to gather comprehensive baseline
and follow-up data, score serial chest x-rays,
evaluate quality of life perceptions of
caregivers, gather physiologic data related to
cardiopulmonary evaluation at rest, exercise
and sleep, structured protocols for airway
clearance therapy and routine follow-up at
14, 30, 90, 180 days and 1 year and two years
at the affiliated institutional clinics routinely
caring for these subjects.
Because of the negative impact and increased
cost of care related to increased risk of
pulmonary compromise in this group we are
targeting development of broader definition of
the medically complex child with a focus on
the structural and pathological changes in the
thoracopulmonary structures and proposing a
chest radiograph scoring system to stage
disease progression and evaluate effectiveness
of therapy in reducing pulmonary infections
and their sequelae.
The chest radiograph scoring system is based on
currently recognized, landmark based analysis
of intrapulmonary evidence and generally
recognized radiographic signs of long term
changes associated with chronic processes.

Validation will be tested across a spectrum of radiology
practitioners to determine if there is statistically significant
agreement in the interpretation of chest radiographs.
Additional data will be gathered to determine if there are
general hallmarks of disease progression across the group of
children studied and whether there is a correlation between
reduced incidence of pulmonary infection and any mode of
airway clearance therapy.
The definition proposed will recognize the
difference amongst the constellation of
diagnoses, but focus on the relevant
similarities across this group of children as
relates to common structural abnormalities,
common sources of compromise that increase
the risk of pulmonary complications, and risk
based analysis associating degree of disability
with the prevalence of recurrent pulmonary
infection.
Baseline data will be collected utilizing a
multifactor intake tool that will be
completed utilizing the medical record
and physical assessment at the time of
intake and follow-up assessments at 30,
60, 180 days, 1 year and 2 years.
A Caregiver Related Quality of Life tool
will be administered at the same intervals
as stipulated above. CRQOL will be
administered to all caregivers involved in
a child’s direct care.
This comprehensive evaluation protocol will
inform us as to the effectiveness of specific
modes of evaluation, measurement and
outcome analysis as relates to the special needs
child at risk for pulmonary complications and
respiratory failure. We will also be able to
evaluate the effectiveness of specific
therapeutic regimens and the impact of
consistently applied airway clearance therapy
National Airway Clearance
Registry
Overview
 Rationale
 Objectives
 Design

NACR Overview

Patients with pulmonary disorders often experience a vicious cycle of
obstruction, inflammation, infection, and destruction of lung tissue. In
many cases, this begins with airway clearance abnormalities.

The National Airway Clearance Registry (NACR) is designed to
document the epidemiology and impact of airway clearance and
hypersecretion disorders, and the associated economic and human
costs.

Patients identified by participating physicians and healthcare teams
will be able to use The Vest™ airway clearance system for a 60-day
screening trial. Following the screening trial, patients, with physician
input, will select standard medical management, or standard medical
management plus the Vest and be followed for up to three years.
NACR Rationale

Conducting separate clinical trials in numerous patient populations and
disease states with airway clearance needs is impractical, time
consuming, and extremely expensive.

The similarities between the patient profiles and the pulmonary
complications associated with impaired airway clearance will allow us
to take a different approach.

The NACR will allow us to collect clinical data on numerous patient
populations and sub-groups while focusing on the key endpoints
associated with airway clearance-related problems.

The Registry will use the power of large numbers compiled over time
to answer key clinical questions.
Treatment Registry
Observational research program for capturing actual experience in
clinical practice in order to gathering additional information on:
» Post-market safety/tolerability of treatment(s)
» Health and economic outcomes of treatment(s)
» Cost-effectiveness of treatment(s)
» Epidemiology, e.g the clinical course of disease with and without
treatment(s)
*A registry typically involves more patients and a longer duration than a clinical trial
Randomized Clinical Trial
Data Registry
Considered the “Gold
Standard”, more rigorous
methodology, hypothesisdriven
Considered observational,
less rigor, hypothesisgenerating
Randomized
Not randomized
Control group
No concurrent control
group
Restrictive
inclusion/exclusion criteria
Broad enrollment criteria
Patient visits strictly
defined by protocol
Recommended visits at
specific time intervals
NACR Clinical Objectives

Document the impact of airway clearance complications and airway clearance
therapy (ACT) on pulmonary health, healthcare utilization, and disease
progression among different patient populations.

Document similarities and differences in clinical outcomes, healthcare
utilization, patient-reported treatment satisfaction and usage associated with
different forms of ACT.

Document and describe the ACT practices among different patient populations
in terms of ACTs used, prescribed protocols, and patient-reported treatment
satisfaction and usage.

Determine predictors of successful outcomes for therapy with The Vest in
order to better define patient selection criteria for various patient populations.
Medical Advisory Board
Frank Accurso, M.D.
Children’s Hospital
Denver, CO
Andrew Gelfand, M.D.
Pediatric Pulmonary Specialists, PA
Dallas, TX
Dean Hess, Ph.D., R.R.T.
Massachusetts General Hospital
Harvard Medical School
Boston, MA
Steve JuliusM.D.
Scottish Rite Children’s Hospital
Atlanta, GA
Michael Konstan, M.D.
Rainbow Babies and
Children’s Hospital
Cleveland, OH
Ron Morton, M.D.
Kosair Children’s Hospital
Louisville, KY
Virginia Simson Nelson, M.D.
University of Michigan
C.S. Mott Children’s Hospital
Ann Arbor, MI 48109-0242
Kerstin Sobus, M.D.
Altru Health Institute
Grand Forks, ND 58206
Jean Stansbury, R.N., C.N.P.
Gillette Children’s Hospital-Outpatient Clinic
St. Paul, MN 55101
NACR Site Eligibility
Clinical Centers within the United States that:
 Regularly see patients that are prone to airway
clearance complications
 Have a strong potential to enroll a minimum of 20
registry patients within one year of participation
 Have computer and internet access
 Can designate an individual from their staff, in
addition to the site physician(s) that can serve as
the key contact/coordinator for their site
NACR Patient Outcomes

Patient-reported outcomes:
– Treatment satisfaction
– Treatment adherence
– Symptom (cough and sputum)
assessment

Clinical outcomes:
– Pulmonary functions (FEV1,
FVC, FEF25-75), tidal volume
and vital capacity
– Respiratory rate, O2 saturation,
end-tidal CO2

Cost-related measures
(direct and indirect):
– # non-routine physician
office visits
– # ER visits
– # hospitalizations, LOS
– # ICU visits, LOS
– # Pulmonary exacerbations
requiring antibiotics
– Reduction in concomitant
medications or therapies
– Missed days from work or
school