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PHAR359 > Cystic Fibrosis > Flashcards

Cystic Fibrosis Flashcards

1
Q

What is cystic fibrosis?

A

autosomal recessive disease
-two copies of the gene must be mutated for the person to be affected

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2
Q

What causes cystic fibrosis?

A

mutations in the CFTR gene
-the most common mutation is F508del

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3
Q

What is the role of CFTR?

A

regulates chloride ion across apical membranes
-airways, GIT, pancreas, kidney, sweat glands, and male reproductive glands

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4
Q

What does dysfunction of CFTR result in?

A

disruption of:
-chloride secretion
-sodium reabsorption
-water transport
mutations in CFTR impact composition of secretions and mucus
-results in thick mucous and secretions, which cause the manifestations of CF

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5
Q

What are the typical phenotypes of CF?

A

lung disease
pancreatic insufficiency
nutrient malabsorption
liver issues
male infertility

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6
Q

Describe the pathophysiology of CF.

A

lack of functional CFTR protein affects water balance in mucous
-decreased water component, which causes thick and sticky secretions
secretions obstruct ducts of various organs and small airways on the lungs
leads to chronic infections and/or inflammation

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7
Q

What is the impact of the thick secretions on the lungs?

A

thick lung secretions are hard to clear and leads to bacterial colonization

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8
Q

What are the lungs of a CF patient typically colonized with?

A

Pseudomonas aeruginosa
Staph aureus
H. influenzae
Stenotrophomonas maltophilia
Burkholderia cepacian

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9
Q

What is a key complication in CF?

A

pulmonary exacerbation
-increase in respiratory symptoms with systemic symptoms (malaise, anorexia, wt. loss)

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10
Q

What are the pulmonary symptoms seen in CF?

A

chronic and persistent, productive cough
-purulent and/or blood in sputum
dyspnea, wheezing, chest pain

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11
Q

What is done to monitor the lung function of a CF patient?

A

routine PFTs
-FEV1

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12
Q

What causes the gastrointestinal complications of CF?

A

result of thick secretions in GIT and causing obstructions

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13
Q

What are the many GI symptoms that can be seen in CF?

A

GERD
meconium ileus
intussusception
SIBO
constipation
rectal prolapse
pancreatic insufficiency
decreased absorption of fat-soluble vitamins (ADEK)

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14
Q

When does pancreatic insufficiency typically present in a CF patient?

A

present from birth in most patients

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15
Q

What does pancreatic insufficiency cause?

A

decreased volume of pancreatic secretions in ducts
-causes destruction of pancreas bc secretions are retained
leads to decreased absorption of lipids and fat-soluble vits
-steatorrhea, malnutrition, osteoporosis

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16
Q

What are the long-term complications of pancreatic insufficiency?

A

chronic pancreatitis
glucose intolerance
CF-related diabetes (pancreatic scarring)

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17
Q

What has become a more common complication of CF?

A

CFRD
-patients are living longer

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18
Q

How does CF cause infertility?

A

CFTR mutation causes a congenital absence of vas deferens
-greater than 95% male infertility due to absent or normal vas deferens

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19
Q

Can CF impact female fertility?

A

females have decrease fertility due to thick cervical mucus

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20
Q

What impact can CF have on the nasal cavity?

A

polyps in nose

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21
Q

What do nasal polyps predispose a CF patient to?

A

chronic sinusitis

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22
Q

What are the goals of therapy for CF?

A

maintain lung function as much as possible, ongoing
maintain adequate nutrition, ongoing
maintain QoL

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23
Q

What are the principles of CF pharmacotherapy?

A

CF pts should be followed and managed by a multidisciplinary team that specialize in CF care
medications are given lifelong
-monitor for AE and adherence
adherence is key - esp. inhaled therapies
-check adherence and inhaler technique q3-6mo or if worsening clinical status

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24
Q

What is the goal of the pulmonary therapies in CF?

A

increase airway clearance
-long term: slow progression of declining FEV1 and prevent lung transplant

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25
What is key with the administration of pulmonary therapies in CF?
the administration order
26
What is the correct administration order of the pulmonary therapies in CF?
bronchodilator hypertonic saline dornase alfa physical airway clearance inhaled antibiotics inhaled corticosteroids
27
Which CF patients are indicated for pulmonary therapies?
all CF patients 6 years and older -shown to improve FEV1, QoL, and decrease exacerbations
28
Which bronchodilators are used in CF?
short-acting medications -salbutamol, terbutaline, ipratropium
29
What is the use of bronchodilators in CF?
decrease the bronchospasm associated with the other inhaled therapies (hypertonic saline and antibiotics)
30
Which asthma drugs are not routinely recommended in CF?
long-acting bronchodilators inhaled corticosteroids montelukast
31
How is hypertonic saline administered?
nebules -requires a nebulizing machine for administration
32
What is the use of hypertonic saline in CF?
mucolytic: increase mucous clearance from airways by thinning out secretions -more water retained due to high concentration of saline -reduced viscosity and improved mucus rheology
33
What strength of hypertonic saline is used for CF?
usually start with 7% but can decrease to 3% if cannot tolerate
34
What are the adverse effects of hypertonic saline?
coughing sore throat wheeze bronchospasm
35
What is the use of dornase alfa in CF?
recombinant human deoxyribonuclease that cleaves DNA -decrease mucous viscosity and decrease inflammation
36
How is dornase alfa administered?
nebules
37
True or false: you can mix dornase alfa with other medications in the nebulizer
false
38
Which inhaled antibiotics are used in CF?
IV injectable liquids that are nebulized: -levofloxacin, aztreonam, colistimethate tobramycin (different formulations): -nebule, Tobi-podhaler, IV injection
39
What is the usual inhaled antibiotic regimen for CF?
28 days on, 28 days off cycle -help prevent resistance
40
Why do most CF patients require pancreatic enzyme replacement therapy?
pancreas is unable to secrete the necessary digestive enzymes to break down lipids -give exogenous lipase to break down fats and enhance vitamin and mineral uptake
41
What is the dose of pancreatic enzyme for a CF patient?
doses vary, depending on: -fat in each meal -number of meals/snacks per day -can also be weight-based
42
True or false: the pancreatic enzyme products are all interchangeable
false -delayed release, regular release, minimicrospheres for pediatric administration -many types of products with various lipase units
43
What are the adverse effects of pancreatic enzymes?
bloating, flatus, pain, loose stools steatorrhea (MCT oil may help) dyspepsia
44
Why are fat-soluble vitamins given to CF patients?
vitamin replacement to counter decreased absorption
45
Which vitamin product is used in CF?
separate OTC products can be used if nutritional requirements are being met -but very high doses may be required MVW Complete Formulation -lipid-based vitamin product, special formulation -Special Access Programme
46
True or false: CFTR modulators are not disease-modifying therapies
false they are disease-modifying therapies
47
Which CF patients are CFTR modulators used in?
patients with specific mutations -partially restore CFTR function in these patients -patients must have specific mutations and be managed by a CF-specialized physician
48
When should CFTR modulators be initiated in CF patients?
at the youngest age possible
49
What are examples of CFTR modulators?
ivacaftor (Kalydeco) lumacaftor/ivacaftor (Orkambi) tezacaftor/ivacaftor (Symdeko) elexcaftor/tezacaftor/ivacaftor (Trikafta)
50
Differentiate the two MOAs of Trikafta.
corrector: -helps to fix the flaws in the CFTR protein so that it can form the right shape and traffic to the cell surface potentiator: -binds to the defective protein at the cell surface and opens the chloride channel and holds it open so chloride ions can flow through
51
Which drugs in Trikafta are potentiators and which are correctors?
potentiator: ivacaftor corrector: elexacaftor, tezacaftor
52
How should Trikafta be taken?
must be taken with a fat containing food -might see pts with "fat chasers" as part of their nutrition regimen
53
Which CF patients is Trikafta approved for?
patients 2 years and older
54
Which dosage forms does Trikafta come as?
granules and tablets
55
What are the adverse effects of Trikafta?
elevated LFTs behavioral/psychiatric changes cataracts
56
What are the drug interactions of Trikafta?
many with 3A4 inhibitors and inducers
57
What are some monitoring parameters for Trikafta?
routine liver enzyme monitoring ophthalmological exam
58
Describe the efficacy of Trikafta.
significantly increases FEV1 after 8 wks vs placebo (adults) increased evidence of sustained FEV1 improvements when used for greater than 6 months evidence of improvement in lung function trajectory
59
What might occur for some patients on Trikafta with guidance from a CF team?
some patients may decrease their inhaled airway regimens
60
What are some other therapies that might be used in CF patients?
GERD and CFRD: treated similarly as non-CF populations -insulin for CFRD azithromycin: -chronic use considered for pts with bacterial colonization (S. aureus and H. influenzae) -not for prophylaxis but for potential anti-inflammatory high-dose ibuprofen: -slow the loss of lung function -plasma concentrations required increased nutritional needs: -high-calorie, high-protein diet -GT feeding when necessary
61
What is a CF exacerbation?
significant and sustained change from baseline in respiratory symptoms, pulmonary function or CXR
62
How are CF exacerbations managed?
minimum of 2 weeks IV antibiotic therapy -inhaled antibiotics are stopped
63
Which antibiotics are used for a CF exacerbation when the patient has no previous culture?
piperacillin/tazobactam or ceftazidime with IV tobramycin -coverage for most CF pathogens AND double-coverage of P.aeruginosa
64
What are the PK changes in CF patients?
extent and/or speed of oral absorption may be delayed (F unaffected) -due to gut and pancreas changes -decreased GI motility and absorption distribution changes -pts more likely to be leaner and shorter than avg -hypoalbuminemia enhanced clearance of medications -increased free fractions of drugs

PHAR359 (28 decks)

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