Exam 2 - Cystic Fibrosis

Question 1

Which class of CFTR mutation is most common?

Answer 1

Class II

Question 2

What is the name of most common CFTR mutation?

Answer 2

F508del

Question 3

What was the initial identified mutation for cystic fibrosis?

Answer 3

G551D

Question 4

What is the age indication for ivacaftor (Kalydeco)?

Answer 4

1 month or more

Question 5

What is the age indication for lumacaftor/ivacaftor (Orkambi)?

Answer 5

1 year or more

Question 6

What is the age indication for tezacaftor/ivacaftor (Symdeko)?

Answer 6

6 years or more

Question 7

What is the age indication for elexacaftor/tezacaftor/ivacaftor (Trikafta)?

Answer 7

2 years or more

Question 8

What genotype must patients have to qualify for ivacaftor (Kalydeco)?

Answer 8

heterozygous

Question 9

What genotype must patients have to qualify for lumacaftor/ivacaftor (Orkambi)?

Answer 9

homozygous

Question 10

What genotype must patients have to qualify for tezacaftor/ivacaftor (Symdeko)?

Answer 10

homo or heterozygous

Question 11

What genotype must patients have to qualify for elexacaftor/tezacaftor/ivacaftor (Trikafta)?

Answer 11

homo or heterozygous

Question 12

What is the MOA of the ivacaftor component?

Answer 12

facilitates the opening of the chloride channel (potentiator)

Question 13

What is the MOA of the lumacaftor, tezacaftor, and elexacaftor components?

Answer 13

chaperone that aids in trafficking of CFTR protein to cell membrane (corrector)

Question 14

What are the AEs of CFTRs? (3)

Answer 14

GI, rash, increased ALT/AST

Question 15

What are counseling points for CFTR modulators? (3)

Answer 15

take w/fat-containing meal, avoid grapefruit juice, dose reduction in hepatic dysfunction

Question 16

Which drugs cause CYP3A4 inhibition? (4)

Answer 16

erythromycin, clarithromycin, fluconzaole, itraconazole

Question 17

Which drugs cause CYP3A4 induction? (5)

Answer 17

rifampin, carbamazepine, phenobarbital, phenytoin, St. John’s wort

Question 18

What are the physiotherapy treatments for cystic fibrosis? (4)

Answer 18

high frequency chest wall oscillation (“vest” therapy), postural drainage, positive expiratory pressure, exercise

Question 19

What are the traditional pharmacotherapeutic treatments for cystic fibrosis? (4)

Answer 19

bronchodilators (SABAs), topical mucolyics (dornase alfa), hydrating agents (hypertonic saline), anti-inflammatory agents (azithromycin)

Question 20

What are the AEs of dornase alfa? (2)

Answer 20

voice hoarseness, rash

Question 21

What is the dosing frequency for azithromycin in cystic fibrosis?

Answer 21

tiw

Question 22

What are the treatments for pseudomonas infections in cystic fibrosis? (2)

Answer 22

tobramycin (TOBI as nebulized solution or dry powder device), aztreonam (inhaled)

Question 23

What is the dosing frequency for tobramycin in cystic fibrosis?

Answer 23

28 days on and 28 days off

Question 24

What are the AEs of tobramycin? (2)

Answer 24

voice alteration, tinnitus

Question 25

What are the AEs of aztreonam?

Answer 25

bronchospasm (pre-treat with SABA)

Question 26

What is the treatment for an acute exacerbation in cystic fibrosis with MSSA and no PA?

Answer 26

anti-staphylococcal penicillin or cephalosporin

Question 27

What is the treatment for an acute exacerbation in cystic fibrosis with MSSA and PA?

Answer 27

cefepime plus aminoglycoside

Question 28

What is the treatment for an acute exacerbation in cystic fibrosis with MRSA and no PA?

Answer 28

vancomycin or linezolid

Question 29

What is the treatment for an acute exacerbation in cystic fibrosis with MRSA and PA?

Answer 29

vancomycin or linezolid AND aminoglycoside plus beta-lactam (ceftazidime)

Question 30

What type of dosing is recommended for antibiotics in acute exacerbations in cystic fibrosis?

Answer 30

once-daily (extended-interval)

Question 31

What is the dosing duration for antibiotics in acute exacerbations in cystic fibrosis?

Answer 31

10-14 days

Question 32

What are monitoring parameters for antibiotics used in acute exacerbations of cystic fibrosis? (4)

Answer 32

symptom persistence/resolution, pulmonary function tests (FEV1, FVC), sputum culture and susceptibilities, BUN/SCr

Question 33

What are the goals for peak and trough in traditional dosing of aminoglycosides?

Answer 33

peak = 10-12, trough = <1.5 mcg/mL

Question 34

What are the goals for peak and trough in extended-interval dosing of aminoglycosides?

Answer 34

peak = 22.5-27.5, trough = <0.1 mcg/mL

Question 35

What are the goals for trough and AUC/MIC ratio for vancomycin?

Answer 35

trough = 10-20 mcg/mL, AUC/MIC = >/=400

Question 36

What is the age indication for azithromycin and or tobramycin treatment in cystic fibrosis?

Answer 36

6 plus