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Cystic Fibrosis: A Disease for a Multidisciplinary Team. Kim Otsuka, MD Pediatric Pulmonary Fellow September 21, 2004 UoA PPC 2004 Workshop Lectures. Objectives. Brief overview of cystic fibrosis (CF) Review some of the “newer” pulmonary therapies in CF Highlight nutritional issues in CF

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cystic fibrosis a disease for a multidisciplinary team

Cystic Fibrosis: A Disease for aMultidisciplinary Team

Kim Otsuka, MD

Pediatric Pulmonary Fellow

September 21, 2004

UoA PPC 2004 Workshop Lectures

  • Brief overview of cystic fibrosis (CF)
  • Review some of the “newer” pulmonary therapies in CF
  • Highlight nutritional issues in CF
  • Highlight psychosocial issues in CF
  • Highlight respiratory care issues in CF
cf center team
CF Center Team
  • Center director
  • Associate center director
  • Clinic coordinator
  • Social worker
  • Respiratory therapist
  • Nurse
  • Registered dietitian
cystic fibrosis a recap
Cystic Fibrosis - A recap
  • Epidemiology
    • Most common life-threatening inherited disease
    • Autosomal recessive
    • Incidence 1:3200 live births (Caucasians)*
      • Hispanics 1:9200, Blacks 1:15,000, Asians 1:31,000
    • 1:28 Caucasians are carriers
      • Hispanics 1:48, Blacks 1:61; Asians 1:88

*Adapted from Hamosh A, et al. J Pediatr 1998; 132: 255-259

  • Single gene defect on chromosome 7
    • Dicovered in 1989
    • Cystic fibrosis transmembrane regulator (CFTR)
      • Most common F508
      • Over 1000 mutations identified

Zeitlin P, N Engl J Med 2004; 351: 606-8.

  • CFTR mutations affect epithelial cells, thus affecting:
    • Airways (sinuses, lungs)
    • Pancreas (endocrine and exocrine)
    • GI tract (liver/biliary system, intestines)
    • Reproductive organs
    • Skin
pulmonary pathophysiology
Pulmonary Pathophysiology
  • 2 theories
    • Composition hypothesis
      • Airway epithelia regulate the airway surface liquid (ASL) salt content that is important for lung defense
    • Volume hypothesis
      • Airway epithelia regulate the volume of liquid in the mucus and periciliary liquid layers of ASL for efficient ciliary mucus interactions and airway clearance.
pulmonary pathophysiology8
Pulmonary - Pathophysiology

Ratjen F, Coring G. Lancet 2003; 361: 681-89.

pulmonary pathophysiology9
Pulmonary Pathophysiology
  • CFTR mutation --> abnormal airway surface microenvironment --> thickened mucus, chronic infection, chronic inflammation --> damages airway ---> bronchiectasis, respiratory failure and often death
inflammation in cf
Inflammation in CF

Konstan MW, Davis PB. Advanced Drug Delivery Reviews 2002; 54: 1409-1423

treatment anti inflammatories

Konstan et al, NEJM 1995

85 patients

5 - 39 y/o

FEV1 > 60 % predicted

20-30 mg/kg of ibuprofen

peak plasma concentrations 50-100 mcg/mL



-2.17 + 0.57 vs. -3.60 + 0.55 (p=0.02)

weight better in ibuprofen group (p=0.02)

Adverse events

No difference between groups in study

Increased GI hemorrhage in f/u

Treatment: Anti-inflammatories
treatment anti inflammatories12

Multicenter US trial

double-blind, randomized, placebo-controlled trial

185 patients, >6 y/o

FEV1 >30% predicted

weight <40 kg: 250 mg azithromycin 3x/wk

weight >40 kg: 500 mg azithromycin 3x/wk


At 24 weeks into study

FEV1 gain 4.77% in azithromycin group

FEV1 loss -1.77% in placebo

Improved weight gain and decreased hospitalizations in azithromycin group

Adverse events: nausea, diarrhea, wheezing

Treatment: Anti-inflammatories

King M, Rubin BK. Advanced Drug Delivery Review 2002; 54: 1475-90

Dornase alfa

Fuchs, et al 1994

968 patients (youngest 5 y/o)

Randomized, double-blinded placebo controlled trial

placebo vs. once or twice daily DNAse


Improved QOL, health economics

Pulmonary exacerbations

once daily: RR 0.78 (CI 0.57-1.06; p=0.11)

twice daily: RR = 0.66

(CI 0.48-0.91; p=0.01)

FEV1 (improved p<0.01)

once daily 5.8 + 0.7

twice daily 5.7 + 0.7

Adverse events

Voice alteration, laryngitis, rash

  • Often unable to eradicate the organism
  • Determining optimal delivery mode for a drug is difficult
  • CF patients require higher doses
    • Altered volume of distribution
    • Rapid clearance of drugs
inhaled tobramycin tobi
Decreased density of pseudomonas

Decreased hospitalizations

Additive effect with DNase

Adverse events


Voice alteration

No hearing loss

No change in kidney function

Ramsey et al, 1999

520 patients, >6 y/o

Tobi 300 mg bid x 28 days q o month x 20 weeks

Inhaled Tobramycin (Tobi®)

Ramsey et al, NEJM 1999; 340: 23-30

nutritional issues18
Nutritional Issues
  • Nutrition is closely tied to overall health
  • Malnutrition in CF associated with
    • Stunted growth
    • Pubertal delay
    • Deterioration of lung function
    • Early death
escf study
ESCF Study
  • J. Pediatrics 2003
  • 931 patients
  • 3-6 years
  • Parameters evaluated:
    • Height, weight, s/sx of lung disease recorded at age 3 and 6
    • Respiratory tract culture < 3 y/o
    • 1 valid spirometry obtained when stable between 5.5 and 7.5 y/o
results age 3
Results - age 3

Konstan MW, J. Pediatr 2003; 142:624-30

results age 6
Results - age 6

Konstan MW, J. Pediatr 2003; 142:624-30

weight gain improves lung function by age 6
Weight gain improves lung function by age 6

Konstan MW, J. Pediatr 2003; 142:624-30

  • Indexes of growth and nutrition at age 3 associated with lower pulmonary function at age 6
  • S/sx of lung disease at age 3 are also associated with lower pulmonary function at age 6
  • Relative weight loss was associated with worse lung function
  • Relative weight gain was associated with improved lung function
care team responsibilities
Care team responsibilities
  • Monitor growth
  • Provide anticipatory counseling and plan interventional strategies for those who are undernourished or are at high risk
  • Dietitian
    • Provides discipline-specific expertise needed for optimal nutritional management
    • Provide expertise concerning developmental and behavioral aspects of eating
nutritional management
Nutritional management
  • Growth and nutritional status monitored every 3 months
  • Annual pubertal assessment
  • Especially important times for monitoring
    • First 12 months after diagnosis
    • Birth to 12 months for those diagnosed prenatally or by GDS
    • Peripubertal growth period
pancreatic insufficiency
85-90% affected

Malabsorption fats, protein, and other nutrients

Diagnostic tests

72 hour fecal fat

fecal elastase-1

PS patient re-evaluated annually


Pancreatic enzyme supplementation

Improved release with H2 blocker or PPI

Proprietary enzymes better than generic

Complication: fibrosing colonopathy

Taurine supplementation


Pancreatic Insufficiency
calorie intake
Calorie intake
  • Greater than RDA for those without CF
    • Weight goal is 100-110% IBW
  • Fat intake 35-40%
  • Protein 15-20%
  • Supplements
    • Oral: controversial
    • Enteral
the vitamins
Vitamin A

15-40% deficient

18% adults deficits in dark field adaptation

Negative acute phase reactant (do not draw with acute illness)

Vitamin D

10-40% deficient

Vitamin E

5-10% deficient

Vitamin K


Essential Fatty Acids

Minerals and Electrolytes





The Vitamins
bone monitoring
Children > 8 y/o with risk factors

Post-organ transplant

End-stage lung disease

Bone fracture with low-impact activity

Chronic corticosteroid use

Delayed pubertal development

Nutritional failure


DEXA lumbar spine

Annual serum calcium, phosphorous, intact PTH, 25-hydroxy vit D

Dietary assessment of calcium and vit D


Optimize nutrition and intake of vit D, K, calcium

Weight bearing physical activity

Bone monitoring
cystic fibrosis related diabetes
Cystic Fibrosis Related Diabetes
  • Leading comorbidity associated with CF
    • Prevalence increased by 173% from 1992-2002
    • Prevalence increases with age
  • 3-12% are reported to have diabetes
    • 14% of CF patients >14 years old
    • 25% of CF patients 35-44 years old
  • Average age of onset 18-21 y/o
  • Females > Males
glucose intolerance increases with age
Glucose Intolerance Increases with Age

Moran, et al. J. Pediatrics. 133(1998)


Analysis of survival at University of Minnesota has demonstrated that the rapid decline in survival can be attributed to females with CFRD since males with CFRD has ~equivalent suvival rates to males without CFRD

Finnkelstein et al. . J Pediatr 1988; 112: 373-7


Rate of Decline in Pulmonary Function is Related to Oral Glucose Tolerance Status

Milla CE et al: Am J Resp Crit Care Med 162:891-5, 2000

comparison of cfrd with type 1 and type 2 dm
Comparison of CFRD with type 1 and type 2 DM

Moran T: Mountain West Consortium June 24, 2004

therapy cfrd
Therapy - CFRD
  • Treatment: insulin
  • Control glucose to avoid acute/chronic complications
    • FPG 80-120 mg/dl
    • Post-prandial FSBS with + 30 mg/dl from pre-meal value
    • HgA1c < 7%
  • Maintain optimal nutritional status and growth
    • Continue high energy intake
pulmonary function tests
Pulmonary Function Tests
  • Spirometry
  • Respiratory rate
  • Pulse oximetry
  • Lung volume
  • Exercise studies
aerosol delivery systems
Aerosol Delivery Systems
  • They are not all equal
  • 2 important considerations
    • Volume
    • Concentration of therapeutic agent
airway clearance
Airway Clearance
  • Standard daily therapy for patients with cystic fibrosis
  • CF --> viscous airway secretions + poor clearance --> airway obstruction and infection --> airway injury, fibrosis --> bronchiectasis
  • Goal: enhance clearance of airway secretions
airway clearance40
Airway Clearance

Wagener JS, Headley AA. Respir Care 2003; 48: 234-45

airway clearance41
Chest physiotherapy

Accepted standard

Goal move peripheral mucus into central airways

Active cycle breathing

Autogenic drainage

PEP and flutter

low, high pressure


Intrapulmonary percussive ventilation

High-Frequency Chest Compression

Shears mucus away from airways

Modifies viscoelastic properties of mucus


Airway Clearance
typical day airway clearance aerosols
Typical Day Airway Clearance/Aerosols
  • Albuterol 2x/day (15-20 minutes each)
  • Dnase 1x/day (15-20 minutes)
  • Inhaled tobramycin 2x/day (15-20 minutes)
  • Chest clearance 2x/day (20-30 minutes)
  • Step up to 3-4x/day will illnesses
social issues
Social Issues
  • Address social concerns every 3 months
  • Complete assessment by an MSW or psychologist should be performed annually including
    • Changes in family structure and coping
    • School and work performance
    • Participation in age appropriate activities
    • Developmental and emotional issues
    • Vocational training and preparation for adult life
social issues45
Social Issues
  • Other
    • Concerns about CF patient care and understanding of disease manifestations and treatment
    • Compliance with treatment regimens
    • Financial issues related to the illness
    • Family planning when reaching reproductive age
psychosocial aspects
Psychosocial Aspects
  • WHO definition of health

“A State of complete physical, mental, and social well being and not merely the absence of disease or infirmity”

  • Health related quality of life (HRQOL)
    • Physical health
    • Mental health
    • Social function
    • Role function
    • General health perceptions
psychosocial aspects47
Overall psychosocial function is good

Problem areas

Exacerbations - neg impact on general and physical health

Increased vulnerability to stress of life

More dysfunction seen in families with older patients

Lower PFT’s had a greater impact on the parents than the patients

Patients >12 years increased distress with hospitalization

Compliance of treatment regimens improved in mother with less frequent social contacts and less satisfactory marital relationships

Psychosocial Aspects
psychosocial aspects48
Psychosocial Aspects
  • Intellectual function distributed normally
      • Inversely related to age
    • Academic function distributed normally
      • No changes with age
    • Lower levels of academic and intellectual functioning in lower SES classes
    • Age and SES account for 21% and 43% of variance in level of functioning
risk behaviors
Risk Behaviors
  • Cystic fibrosis patients vs. age-matched adolescent peers
    • Alcohol use 45.5% vs. 63.1%
    • Cigarettes 21.1% vs 53.3%
    • Marijuana use 9.7% vs 29.4%
    • Cocaine and other injectable drugs 2.6% vs. 5.7%
risk behaviors50
Risk Behaviors
  • H/O sexually activity 28.3% vs. 46.4%
    • No difference in risky sexual behaviors: condom use, no contraception, and pregnancy
  • Tendency for improved health promoting behaviors
    • Seat belt use, bicycle helmets
take home points
Take Home Points
  • CF management requires the coordinated efforts of a CF care team
  • Growth and nutrition is critical for pulmonary health
    • “Breakfast is as important as morning CPT”
  • All adolescents need risk behavior assessments