cystic fibrosis pharmacology

what is cystic fibrosis?

• obstructs pancreas and prevents it from properly breaking down and absorbing food

what health challenges are related to CF?

• diabetes

• osteoporosis

• reproductive problems

CF patients result from deletion of three base pairs in ____’s nucleotide sequence

• CFTR

what does the deletion of the three base pairs cause?

• loss of phenylalanine

what is the mutation in CF patients called?

• ΔF508

the most common mutation destabilizes ____

• NBD1

what is the major permanent anion in CF?

• Cl

(EXAM QUESTION) CFTR role

• HCO3 secretion

(EXAM QUESTION) CFTR correctors

• class I, II, V, VI

(EXAM QUESTION) CFTR potentiators

• class III/IV

what are the class potentiators?

• reaches cell surface

potentiator example

• ivacaftor

corrector examples

• lumacaftor

• tezacaftor

what is the purpose of cystic fibrosis treatments?

• to clear mucus from the lungs

CF treatment examples

• tobramycin (inhaled)

• pulmozyme

• azithromycin

Ivacaftor side effects

• abdominal pain

• diarrhea

• dizziness

• rash

• upper respiratory tract

• headache

• hepatotoxicity

Lumacaftor

• CYP3A4 inducer

• hepatotoxicity

dornase alpha (pulmozyme)

• hydrolyzes the dna found in mucus in CF patients and reduces viscosity

dornase alpha dose

• concentration: 1 mg/kg

tobramycin

• treats lung infections

pulmozyme

• mucus thinning drug

azithromycin

• for pseudomonas aeruginosa bacteria