Chris Landon, MD, FAAP, FCCP Ventura County Medical Center Ventura, California Audrius Plioplys, MD, CMD,FRCPC, FAAP Mercy Hospital Chicago, Illinois Reducing.

Chris Landon, MD, FAAP, FCCPChris Landon, MD, FAAP, FCCP

Ventura County Medical CenterVentura County Medical Center

Ventura, CaliforniaVentura, California

Audrius Plioplys, MD, CMD,FRCPC, FAAPAudrius Plioplys, MD, CMD,FRCPC, FAAP

Mercy HospitalMercy Hospital

Chicago, IllinoisChicago, Illinois

Reducing The Cost Of Reducing The Cost Of Pulmonary Impairment In Pulmonary Impairment In

Children With Neuromuscular Children With Neuromuscular DiseaseDisease

ObjectivesObjectives 1. Provide an overview of neuromuscular diseases 1. Provide an overview of neuromuscular diseases

and complications that predispose patients to and complications that predispose patients to frequent respiratory exacerbations.frequent respiratory exacerbations.

2. Identify treatment strategies/medically 2. Identify treatment strategies/medically appropriate care to meet needs of this difficult appropriate care to meet needs of this difficult patient population to promote improvements in patient population to promote improvements in quality of life, and positive clinical outcomes.quality of life, and positive clinical outcomes.

3. Discuss evidence supporting the effect of 3. Discuss evidence supporting the effect of airway clearance with The Vest system on airway clearance with The Vest system on reducing costs associated with pulmonary reducing costs associated with pulmonary complications/exacerbations. complications/exacerbations.

Neuromuscular Diseases: Neuromuscular Diseases: OverviewOverview

Children who experience varying degrees of Children who experience varying degrees of neurological/neuromuscular dysfunctionneurological/neuromuscular dysfunction

Diagnoses include: cerebral palsy, muscular Diagnoses include: cerebral palsy, muscular dystrophy, spinal muscular atrophy, brain dystrophy, spinal muscular atrophy, brain injury, consequences of infectious disease, injury, consequences of infectious disease, inherited metabolic disorders, etc.inherited metabolic disorders, etc.– One child in 1000 is institutionalized as a result One child in 1000 is institutionalized as a result

of profound disabilityof profound disability

Neuromuscular Diseases: Neuromuscular Diseases: OverviewOverview

NeuromuscularNeuromuscular GastroesophagealGastroesophageal Immune systemImmune system RespiratoryRespiratory PsychosocialPsychosocial

Multi-system assessment necessary to determine risk of pulmonary involvement:

Neuromuscular DiseasesNeuromuscular Diseases

Neuro assessmentNeuro assessment– Skeletal muscle controlSkeletal muscle control– Respiratory and ventilatory muscle abnormalitiesRespiratory and ventilatory muscle abnormalities– Weak/absent coughWeak/absent cough– Weak/absent gag reflexWeak/absent gag reflex– Upper airway control and coordinationUpper airway control and coordination– Seizure activitySeizure activity– SpasticitySpasticity

Assessment of complications that predispose to pulmonary involvement

Neuromuscular DiseasesNeuromuscular Diseases

Neuro assessment – DysphagiaNeuro assessment – Dysphagia– Oral motor dyskinesia/pseudobulbar palsyOral motor dyskinesia/pseudobulbar palsy– True bulbar palsyTrue bulbar palsy– Dysfunctional arousalDysfunctional arousal– Oral motor weaknessOral motor weakness– Increased secretionsIncreased secretions

Assessment of complications that predispose to pulmonary involvement

Neuromuscular DiseasesNeuromuscular Diseases

Neuro assessment – Oral Neuro assessment – Oral motor weaknessmotor weakness

– Muscular dystrophiesMuscular dystrophies

– MyopathiesMyopathies

– Neuromuscular junction Neuromuscular junction disordersdisorders

– Anterior horn cell Anterior horn cell disordersdisorders

Typical symptomsTypical symptoms

– Too weak to swallowToo weak to swallow

– Too weak to coughToo weak to cough

– Easily fatiguedEasily fatigued

– Head position Head position dependentdependent

Assessment of complications that predispose to pulmonary involvement

Neuromuscular DiseasesNeuromuscular Diseases

Neuro assessment – Neuro assessment – Increased secretionsIncreased secretions

– Autonomic Autonomic dysfunctiondysfunction

– Medication effectsMedication effects

– Frequent seizuresFrequent seizures

Typical symptomsTypical symptoms

– Constant droolingConstant drooling

– Worse with stress Worse with stress or infectionor infection

– Drowning in droolDrowning in drool

Assessment of complications that predispose to pulmonary involvement

Poor Swallow Control Poor Swallow Control (Dysphagia)(Dysphagia)

Oral motor dyskinesia / pseudobulbar palsyOral motor dyskinesia / pseudobulbar palsy True bulbar palsyTrue bulbar palsy Dysfunctional arousalDysfunctional arousal Oral motor weaknessOral motor weakness Increased secretionsIncreased secretions

Oral Motor Dyskinesia / Oral Motor Dyskinesia / Pseudobulbar PalsyPseudobulbar Palsy

Diffuse or bilateral cortical damageDiffuse or bilateral cortical damage Basal ganglia damageBasal ganglia damage Cerebellar – brainstem damageCerebellar – brainstem damage

Oral Motor Dyskinesia / Oral Motor Dyskinesia / Pseudobulbar PalsyPseudobulbar Palsy

Typical symptoms:Typical symptoms:– Increased gag, choking, vomitingIncreased gag, choking, vomiting– Nasal regurgitationNasal regurgitation– Tonguing – pushingTonguing – pushing– Poor tolerance of liquids and chunksPoor tolerance of liquids and chunks

True Bulbar PalsyTrue Bulbar Palsy

Cranial nerves 9, 10, 12Cranial nerves 9, 10, 12 Pontine-medullary damagePontine-medullary damage Arnold-Chiari malformationArnold-Chiari malformation Bulbar syrinxBulbar syrinx Moebius syndromeMoebius syndrome

True Bulbar PalsyTrue Bulbar Palsy

Typical symptomsTypical symptoms– Decreased gag, poor palate movementDecreased gag, poor palate movement– Pocketing of foodPocketing of food– RuminationRumination– DroolingDrooling– ““O” signO” sign

Dysfunctional ArousalDysfunctional Arousal

Disorder of excessive somnolenceDisorder of excessive somnolence Toxic encephalopathyToxic encephalopathy Drug induced stuporDrug induced stupor Autistic disorderAutistic disorder

Dysfunctional ArousalDysfunctional Arousal

Typical symptomsTypical symptoms– Must be reminded to swallowMust be reminded to swallow– Falls asleep while eatingFalls asleep while eating– Poor cough reflexPoor cough reflex– ““Q” sign, string bean signQ” sign, string bean sign

Oral Motor WeaknessOral Motor Weakness

MyopathiesMyopathies Muscular dystrophiesMuscular dystrophies Neuromuscular junction disordersNeuromuscular junction disorders Anterior horn cell disordersAnterior horn cell disorders

Oral Motor WeaknessOral Motor Weakness

Typical symptomsTypical symptoms– Too weak to swallowToo weak to swallow– Too weak to coughToo weak to cough– Easily fatiguedEasily fatigued– Head position dependentHead position dependent

Increased SecretionsIncreased Secretions

Autonomic dysfunctionAutonomic dysfunction Medication effectsMedication effects Frequent seizuresFrequent seizures

Increased SecretionsIncreased Secretions

Typical symptomsTypical symptoms– Constant droolingConstant drooling– Worse with stress or infectionWorse with stress or infection– Drowning in droolDrowning in drool

Poor Breathing ControlPoor Breathing Control

Central neurogenic hypoventilationCentral neurogenic hypoventilation Periodic breathing patternsPeriodic breathing patterns Ondine’s curseOndine’s curse Stupor and comaStupor and coma Thoracic weaknessThoracic weakness

Central Neurogenic Central Neurogenic HypoventilationHypoventilation

Diffuse cortical damageDiffuse cortical damage Poor hypoxic responsePoor hypoxic response Poor hypercarbic responsePoor hypercarbic response Worse with stress or infectionWorse with stress or infection

Periodic Breathing PatternsPeriodic Breathing Patterns

Cheyne-Stokes respirationCheyne-Stokes respiration Biot’s respirationBiot’s respiration Rett’s syndromeRett’s syndrome Apneustic breathingApneustic breathing

Ondine’s CurseOndine’s Curse

Waking versus sleeping centersWaking versus sleeping centers Defect in shift to automatic breathingDefect in shift to automatic breathing “… “… if I should die before I wake…”if I should die before I wake…”

Stupor and ComaStupor and Coma

Brainstem dysfunction from pressure Brainstem dysfunction from pressure Brainstem dysfunction from ischemiaBrainstem dysfunction from ischemia Brainstem suppression from drugsBrainstem suppression from drugs Brainstem degenerationBrainstem degeneration

Thoracic WeaknessThoracic Weakness

MyopathiesMyopathies Muscular dystrophiesMuscular dystrophies Neuromuscular junction disordersNeuromuscular junction disorders Anterior horn cell disordersAnterior horn cell disorders

Recognition of Neurogenic Recognition of Neurogenic Pulmonary Clearance ProblemsPulmonary Clearance Problems

Recognize the possible brain region Recognize the possible brain region involvedinvolved

Perform a careful historyPerform a careful history Perform a careful examinationPerform a careful examination Sleep study / Life ShirtSleep study / Life Shirt MRI – attention to brainstemMRI – attention to brainstem ? muscle studies ?? muscle studies ?

Gastroesophageal Function and Gastroesophageal Function and ComplicationsComplications

The Upper Airway-The Upper Airway-Swallowing and Swallowing and AspirationAspiration

Aspiration Associated Aspiration Associated PneumoniasPneumonias

Lower Esophageal Lower Esophageal Aspiration, Gastric Aspiration, Gastric Distention and Airway Distention and Airway RemodelingRemodeling

Gastroesophageal Reflux Gastroesophageal Reflux Disease (GERD)Disease (GERD)

Fundoplication Versus Fundoplication Versus Medication and Airway Medication and Airway ClearanceClearance

Nutrition and the Immune Nutrition and the Immune SystemSystem

The Faces of DysfunctionThe Faces of Dysfunction

Arching

Irritability

Regurgitation

Gagging and Choking Refusing Feedings

Failure To Thrive

Sandifer’s SyndromeSandifer’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 ReflexesCardiorespiratory Reflexes

Cardiorespiratory ReflexesCardiorespiratory Reflexes

RefluxateRefluxate

Refluxate ProtectionRefluxate Protection

The Immune SystemThe Immune System Genetic AbnormalitiesGenetic Abnormalities Nutritional Nutritional

Compromise of the Compromise of the Immune SystemImmune System

Stress and Immune Stress and Immune ResponseResponse

Recurrent Infection Recurrent Infection and Frequent Use of and Frequent Use of Antibiotics: The Antibiotics: The ImpactsImpacts

AllergiesAllergies Reactive Airway Reactive Airway

Disease (RAD)Disease (RAD) Airway Clearance Airway Clearance

TherapyTherapy

Respiratory Medical HistoryRespiratory Medical History

– Number of Pulmonary Infections AnnuallyNumber of Pulmonary Infections Annually

– Number of Hospital Admissions AnnuallyNumber of Hospital Admissions Annually

– Number of ER Admissions AnnuallyNumber of ER Admissions Annually

– Number of Courses of Antibiotics for Respiratory Number of Courses of Antibiotics for Respiratory Infections AnnuallyInfections Annually

– Immunization HistoryImmunization History

– History of Recurrent Infections with Respiratory History of Recurrent Infections with Respiratory Syncytial Virus (RSV)Syncytial Virus (RSV)

Quality Airway Clearance Therapy Quality Airway Clearance Therapy ShouldShould

•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 Neuromuscular Disease - Perioperative CareCare

High Risk For Post-Operative High Risk For Post-Operative ComplicationsComplications

AtelectasisAtelectasis PneumoniaPneumonia Respiratory FailureRespiratory Failure Need for prolonged ventilationNeed for prolonged ventilation TracheostomyTracheostomy DeathDeath

ProblemsProblems

Weak coughWeak cough Dyscoordinated swallowDyscoordinated swallow AspirationAspiration Difficulty clearing secretionsDifficulty clearing secretions Increased lower respiratory tract Increased lower respiratory tract

infectionsinfections

Respiratory WeaknessRespiratory Weakness

May not be apparent on physical examMay not be apparent on physical exam Respiratory failure when work of breathing Respiratory failure when work of breathing

is increasedis increased

Chronic Respiratory Muscle WeaknessChronic Respiratory Muscle Weakness

Reduced lung volumesReduced lung volumes MicroatelectasisMicroatelectasis V/Q mismatchV/Q mismatch ScoliosisScoliosis Decreased compliance of the chest wallDecreased compliance of the chest wall Decreased pulmonary complianceDecreased pulmonary compliance Hypoxemia only during sleepHypoxemia only during sleep Hypoventilation due to muscle weaknessHypoventilation due to muscle weakness Hypoventilation due to central hypoventilationHypoventilation due to central hypoventilation

Preoperative AssessmentPreoperative Assessment

Thorough historyThorough history Physical examinationPhysical examination Laboratory studiesLaboratory studies

Thorough HistoryThorough History

Frequency and severity of respiratory tract Frequency and severity of respiratory tract infectionsinfections

Pulmonary complications of previous surgeriesPulmonary complications of previous surgeries History suggestive of reactive airways diseaseHistory suggestive of reactive airways disease

– Even mildly increased airway obstruction may Even mildly increased airway obstruction may lead to respiratory failure in the postoperative lead to respiratory failure in the postoperative period in a patient with severe respiratory period in a patient with severe respiratory muscle weaknessmuscle weakness

Physical ExaminationPhysical Examination

Gag reflexGag reflex CoughCough Adequacy of aerationAdequacy of aeration Presence of adventitial lung Presence of adventitial lung

soundssounds

Ability To Cooperate With Post-Ability To Cooperate With Post-Operative Pulmonary TherapyOperative Pulmonary Therapy

General muscle strengthGeneral muscle strength Physical and intellectual capacityPhysical and intellectual capacity

Laboratory ExaminationsLaboratory Examinations

Chest x-rayChest x-ray Arterial blood gases or mixed Arterial blood gases or mixed

venous gas measurements and venous gas measurements and oximetryoximetry

Complete blood countComplete blood count

Pulmonary Function TestsPulmonary Function Tests

All children who are capable of performing themAll children who are capable of performing them– Lung volumesLung volumes– Pre and post bronchodilatorPre and post bronchodilator– Maximal inspiratory and expiratory mouth Maximal inspiratory and expiratory mouth

pressurespressures

» frequently decreased more than lung frequently decreased more than lung volumes and flowsvolumes and flows

»do not correlate with general muscle do not correlate with general muscle strengthstrength

Alternative/Competitive

Airway Clearance Modalities

Chest PhysiotherapyChest Physiotherapy

Mechanical techniques for the Mechanical techniques for the noninvasive clearance of excessive noninvasive clearance of excessive secretions and aspirated materials from secretions and aspirated materials from the airwaythe airway

Prevent, Treat, or At Least Delay Prevent, Treat, or At Least Delay The Effects of Mechanical,Infectious, The Effects of Mechanical,Infectious,

and Biochemical Sequelaeand Biochemical Sequelae prevent resistance to airflowprevent resistance to airflow

work of breathingwork of breathing

hyperinflationhyperinflation

atelectasisatelectasis

maldistribution of ventilationmaldistribution of ventilation

ventilation-perfusion ventilation-perfusion mismatchmismatch

Microorganisms and Host -Mediated Microorganisms and Host -Mediated Inflammatory MediatorsInflammatory Mediators

intrabronchial accumulation of intrabronchial accumulation of secretionssecretions

– vicious circle of infectionvicious circle of infection

– impaired clearanceimpaired clearance

– progressive airway damageprogressive airway damage

Improve Access To The Bronchial Improve Access To The Bronchial SurfaceSurface

Allow inhaled medications to penetrateAllow inhaled medications to penetrate

Effectiveness and SafetyEffectiveness and Safety

Easy to teach to patients and caregiversEasy to teach to patients and caregivers

Should not fatigue the patientShould not fatigue the patient

Time efficient and practicalTime efficient and practical

Whenever possible patient should be Whenever possible patient should be participantparticipant

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 15-30 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 ComplicationsRisk For Anesthetic Complications

Malignant hypothermiaMalignant hypothermia

Cardiac arrhythmiaCardiac arrhythmia

Excessive potassium releaseExcessive potassium release

MyoglobinuriaMyoglobinuria

Prolonged skeletal muscle contraction in Prolonged skeletal muscle contraction in muscular dystrophymuscular dystrophy

– succinylcholinesuccinylcholine

With Care We Can Minimize PostOp With Care We Can Minimize PostOp Risk Even In Severe Neuromuscular Risk Even In Severe Neuromuscular

DiseaseDisease

Ventilatory support planned for Ventilatory support planned for 24-48 hours post surgery24-48 hours post surgery

But avoiding more prolonged But avoiding more prolonged ventilationventilation

– disuse atrophydisuse atrophy

– respiratory muscle weaknessrespiratory muscle weakness

Chest Physiotherapy Is The Keystone To Chest Physiotherapy Is The Keystone To SuccessSuccess

Unable to perform incentive spirometryUnable to perform incentive spirometry

Clearance of secretions and prevention Clearance of secretions and prevention of atelectasis of atelectasis

Every 2 to 4 hours after surgeryEvery 2 to 4 hours after surgery

Frequency May Be Reduced If Tolerates Frequency May Be Reduced If Tolerates Extubation For 24 Hours Without Extubation For 24 Hours Without

ComplicationComplication

IPPB may be helpful in severe IPPB may be helpful in severe neurologic disease'neurologic disease'

Oxygen saturation should be >95%Oxygen saturation should be >95%

Hematocrit over 35%Hematocrit over 35%

Careful Attention To Fluid BalanceCareful Attention To Fluid Balance

Decreased pulmonary compliance and Decreased pulmonary compliance and hypoxemia if lung water is increasedhypoxemia if lung water is increased

Scoliosis repair even more critical Scoliosis repair even more critical settingsetting

Careful Attention To Even Minimal Careful Attention To Even Minimal BronchospasmBronchospasm

Bronchodilator aerosolsBronchodilator aerosols

Consider theophylline or steroidsConsider theophylline or steroids

Careful Attention To Atelectasis Careful Attention To Atelectasis PreventionPrevention

Bronchodilator aerosolsBronchodilator aerosols

Mucolytic aerosolsMucolytic aerosols

Vigorous CPT and suctioningVigorous CPT and suctioning

IPPBIPPB

Consider bronchoscopy Consider bronchoscopy

– removing secretionsremoving secretions

– obtaining cultureobtaining culture

Direct Antibiotic Therapy Against Direct Antibiotic Therapy Against Specific PathogensSpecific Pathogens

Normal mouth flora and anerobes Normal mouth flora and anerobes secondary to aspirationsecondary to aspiration

Consider surveillance cultures prior to Consider surveillance cultures prior to surgery surgery

Posterior pharyngeal culturesPosterior pharyngeal cultures

"Pulmonary evaluation of "Pulmonary evaluation of rehabilitation and therapy in rehabilitation and therapy in medically fragile children" medically fragile children" which is being submitted in which is being submitted in

response to the RFA HD#02-027response to the RFA HD#02-027

HYPOTHESIS: HYPOTHESIS:

Children and adolescents (6 to 18 years of Children and adolescents (6 to 18 years of age) with moderate to severe developmental age) with moderate to severe developmental disabilities with an adequate regimen of disabilities with an adequate regimen of pulmonary toilet can have an improved pulmonary toilet can have an improved quality of life. quality of life.

The development, validation and The development, validation and analysis of the three proposed tools will analysis of the three proposed tools will

facilitate the statistical evaluation of facilitate the statistical evaluation of specific indicators that correlate to risk specific indicators that correlate to risk of further deterioration in pulmonary of further deterioration in pulmonary

function.function.

This study will 1) further define the This study will 1) further define the scope and nature of respiratory failure in scope and nature of respiratory failure in

children, especially those with special children, especially those with special needs, 2) develop and evaluate acute and needs, 2) develop and evaluate acute and

chronic management strategies to chronic management strategies to prevent or minimize the development of prevent or minimize the development of chronic respiratory failure in children or chronic respiratory failure in children or

to ameliorate its effects.to ameliorate its effects.    

Recent scientific and engineering Recent scientific and engineering advances, including the availability of advances, including the availability of

innovative approaches and techniques for innovative approaches and techniques for measuring and treating pulmonary measuring and treating pulmonary dysfunction open new avenues of dysfunction open new avenues of

exploration to be utilized to enhance the exploration to be utilized to enhance the management and quality of life for management and quality of life for

children with respiratory failure.children with respiratory failure.

This study will focus on those children This study will focus on those children and adolescents with moderate to severe and adolescents with moderate to severe developmental disabilities, who reside in developmental disabilities, who reside in

residential care, skilled nursing, residential care, skilled nursing, domiciliary facilities and their group domiciliary facilities and their group

homes.homes.

Subjects will include ethnically diverse, male Subjects will include ethnically diverse, male and female subjects. and female subjects. Standard procedures Standard procedures will be utilized to gather comprehensive will be utilized to gather comprehensive baseline and follow-up data, score serial baseline and follow-up data, score serial

chest x-rays, evaluate quality of life chest x-rays, evaluate quality of life perceptions of caregivers, gather physiologic perceptions of caregivers, gather physiologic data related to cardiopulmonary evaluation data related to cardiopulmonary evaluation

at rest, exercise and sleep, structured at rest, exercise and sleep, structured protocols for airway clearance therapy and protocols for airway clearance therapy and routine follow-up at 14, 30, 90, 180 days and routine follow-up at 14, 30, 90, 180 days and

1 year and two years at the affiliated 1 year and two years at the affiliated institutional clinics routinely caring for these institutional clinics routinely caring for these

subjects. subjects.

Because of the negative impact and increased Because of the negative impact and increased cost of care related to increased risk of cost of care related to increased risk of

pulmonary compromise in this group we are pulmonary compromise in this group we are targeting development of broader definition of targeting development of broader definition of the medically complex child with a focus on the medically complex child with a focus on the structural and pathological changes in the the structural and pathological changes in the thoracopulmonary structures and proposing a thoracopulmonary structures and proposing a

chest radiograph scoring system to stage chest radiograph scoring system to stage disease progression and evaluate effectiveness disease progression and evaluate effectiveness of therapy in reducing pulmonary infections of therapy in reducing pulmonary infections

and their sequelaeand their sequelae. .

The chest radiograph scoring system is based The chest radiograph scoring system is based on currently recognized, landmark based on currently recognized, landmark based analysis of intrapulmonary evidence and analysis of intrapulmonary evidence and

generally recognized radiographic signs of long generally recognized radiographic signs of long term changes associated with chronic processes.term changes associated with chronic processes.

   Validation will be tested across a spectrum of radiology Validation will be tested across a spectrum of radiology

practitioners to determine if there is statistically significant practitioners to determine if there is statistically significant agreement in the interpretation of chest radiographs. agreement in the interpretation of chest radiographs. Additional data will be gathered to determine if there are Additional data will be gathered to determine if there are general hallmarks of disease progression across the group of general hallmarks of disease progression across the group of children studied and whether there is a correlation between children studied and whether there is a correlation between reduced incidence of pulmonary infection and any mode of reduced incidence of pulmonary infection and any mode of

airway clearance therapy.airway clearance therapy.

The definition proposed will recognize the The definition proposed will recognize the difference amongst the constellation of difference amongst the constellation of

diagnoses, but focus on the relevant diagnoses, but focus on the relevant similarities across this group of children as similarities across this group of children as relates to common structural abnormalities, relates to common structural abnormalities,

common sources of compromise that increase common sources of compromise that increase the risk of pulmonary complications, and risk the risk of pulmonary complications, and risk based analysis associating degree of disability based analysis associating degree of disability

with the prevalence of recurrent pulmonary with the prevalence of recurrent pulmonary infection.   infection.   

Baseline data will be collected utilizing a Baseline data will be collected utilizing a multifactor intake tool that will be multifactor intake tool that will be

completed utilizing the medical record completed utilizing the medical record and physical assessment at the time of and physical assessment at the time of

intake and  follow-up assessments at 30, intake and  follow-up assessments at 30,

60, 180 days, 1 year and 2 years.60, 180 days, 1 year and 2 years.      

A Caregiver Related Quality of Life tool A Caregiver Related Quality of Life tool will be administered at the same intervals will be administered at the same intervals

as stipulated above.  CRQOL will be as stipulated above.  CRQOL will be administered to all caregivers involved in administered to all caregivers involved in

a child’s direct care.   a child’s direct care.   

This comprehensive evaluation protocol will This comprehensive evaluation protocol will inform us as to the effectiveness of specific inform us as to the effectiveness of specific

modes of evaluation, measurement and modes of evaluation, measurement and outcome analysis as relates to the special needs outcome analysis as relates to the special needs child at risk for pulmonary complications and child at risk for pulmonary complications and

respiratory failure.  We will also be able to respiratory failure.  We will also be able to evaluate the effectiveness of specific evaluate the effectiveness of specific

therapeutic regimens and the impact of therapeutic regimens and the impact of consistently applied airway clearance therapyconsistently applied airway clearance therapy

National Airway Clearance National Airway Clearance RegistryRegistry

OverviewOverview RationaleRationale ObjectivesObjectives Design Design

NACR OverviewNACR Overview

Patients with pulmonary disorders often experience a vicious cycle of Patients with pulmonary disorders often experience a vicious cycle of obstruction, inflammation, infection, and destruction of lung tissue. In obstruction, inflammation, infection, and destruction of lung tissue. In many cases, this begins with airway clearance abnormalities. many cases, this begins with airway clearance abnormalities.

The National Airway Clearance Registry (NACR) is designed to The National Airway Clearance Registry (NACR) is designed to document the epidemiology and impact of airway clearance and document the epidemiology and impact of airway clearance and hypersecretion disorders, and the associated economic and human hypersecretion disorders, and the associated economic and human costs. costs.

Patients identified by participating physicians and healthcare teams Patients identified by participating physicians and healthcare teams will be able to use The Vest™ airway clearance system for a 60-day will be able to use The Vest™ airway clearance system for a 60-day screening trial. Following the screening trial, patients, with physician screening trial. Following the screening trial, patients, with physician input, will select standard medical management, or standard medical input, will select standard medical management, or standard medical management plus the Vest and be followed for up to three years. management plus the Vest and be followed for up to three years.

NACR RationaleNACR Rationale

Conducting separate clinical trials in numerous patient populations and Conducting separate clinical trials in numerous patient populations and disease states with airway clearance needs is impractical, time disease states with airway clearance needs is impractical, time consuming, and extremely expensive.consuming, and extremely expensive.

The similarities between the patient profiles and the pulmonary The similarities between the patient profiles and the pulmonary complications associated with impaired airway clearance will allow us complications associated with impaired airway clearance will allow us to take a different approach.to take a different approach.

The NACR will allow us to collect clinical data on numerous patient The NACR will allow us to collect clinical data on numerous patient populations and sub-groups while focusing on the key endpoints populations and sub-groups while focusing on the key endpoints associated with airway clearance-related problems.associated with airway clearance-related problems.

The Registry will use the power of large numbers compiled over time The Registry will use the power of large numbers compiled over time to answer key clinical questions.to answer key clinical questions.

Treatment RegistryTreatment Registry

ObservationalObservational research program for capturing research program for capturing actual experience in actual experience in clinical practiceclinical practice in order to gathering additional information on: in order to gathering additional information on:

» Post-market safety/tolerability of treatment(s)Post-market safety/tolerability of treatment(s)

» Health and economic outcomes of treatment(s)Health and economic outcomes of treatment(s)

» Cost-effectiveness of treatment(s)Cost-effectiveness of treatment(s)

» Epidemiology, e.g the clinical course of disease with and without Epidemiology, e.g the clinical course of disease with and without treatment(s) treatment(s)

*A registry typically involves more patients and a longer duration than a clinical trial*A registry typically involves more patients and a longer duration than a clinical trial

Considered the “Gold Considered the “Gold Standard”, more rigorous Standard”, more rigorous methodology, hypothesis-methodology, hypothesis-drivendriven

Considered observational, Considered observational, less rigor, hypothesis-less rigor, hypothesis-generatinggenerating

RandomizedRandomized Not randomizedNot randomized

Control groupControl group No concurrent control No concurrent control groupgroup

Restrictive Restrictive inclusion/exclusion criteriainclusion/exclusion criteria

Broad enrollment criteriaBroad enrollment criteria

Patient visits strictly Patient visits strictly defined by protocol defined by protocol

Recommended visits at Recommended visits at specific time intervalsspecific time intervals

Randomized Clinical Trial Data Registry

NACR Clinical ObjectivesNACR Clinical Objectives

Document the impact of airway clearance complications and airway clearance Document the impact of airway clearance complications and airway clearance therapy (ACT) on pulmonary health, healthcare utilization, and disease therapy (ACT) on pulmonary health, healthcare utilization, and disease progression among different patient populations. progression among different patient populations.

Document similarities and differences in clinical outcomes, healthcare Document similarities and differences in clinical outcomes, healthcare utilization, patient-reported treatment satisfaction and usage associated with utilization, patient-reported treatment satisfaction and usage associated with different forms of ACT. different forms of ACT.

Document and describe the ACT practices among different patient populations Document and describe the ACT practices among different patient populations in terms of ACTs used, prescribed protocols, and patient-reported treatment in terms of ACTs used, prescribed protocols, and patient-reported treatment satisfaction and usage.satisfaction and usage.

Determine predictors of successful outcomes for therapy with The Vest in Determine predictors of successful outcomes for therapy with The Vest in order to better define patient selection criteria for various patient populations.order to better define patient selection criteria for various patient populations.

Medical Advisory BoardMedical Advisory BoardMichael 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

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

NACR Site EligibilityNACR Site Eligibility

Clinical Centers within the United States that:Clinical Centers within the United States that: Regularly see patients that are prone to airway Regularly see patients that are prone to airway

clearance complicationsclearance complications Have a strong potential to enroll a minimum of 20 Have a strong potential to enroll a minimum of 20

registry patients within one year of participationregistry patients within one year of participation Have computer and internet accessHave computer and internet access Can designate an individual from their staff, in Can designate an individual from their staff, in

addition to the site physician(s) that can serve as addition to the site physician(s) that can serve as the key contact/coordinator for their sitethe key contact/coordinator for their site

NACR Patient Outcomes NACR Patient Outcomes Patient-reported outcomes: Patient-reported outcomes:

– Treatment satisfactionTreatment satisfaction

– Treatment adherenceTreatment adherence

– Symptom (cough and sputum) Symptom (cough and sputum) assessmentassessment

Clinical outcomes: Clinical outcomes: – Pulmonary functions (FEV1, Pulmonary functions (FEV1,

FVC, FEF25-75), tidal volume FVC, FEF25-75), tidal volume and vital capacityand vital capacity

– Respiratory rate, O2 saturation, Respiratory rate, O2 saturation, end-tidal CO2end-tidal CO2

Cost-related measures Cost-related measures (direct and indirect):(direct and indirect):– # non-routine physician # non-routine physician

office visitsoffice visits– # ER visits# ER visits– # hospitalizations, LOS# hospitalizations, LOS– # ICU visits, LOS# ICU visits, LOS– # Pulmonary exacerbations # Pulmonary exacerbations

requiring antibioticsrequiring antibiotics– Reduction in concomitant Reduction in concomitant

medications or therapiesmedications or therapies– Missed days from work or Missed days from work or

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