Cystic Fibrosis Treatments

CYSTIC FIBROSIS TREATMENTS
The Cystic Fibrosis (CF) Foundation established the Pulmonary Clinical Practice Guidelines Committee to assist healthcare providers with the use of chronic medications for the maintenance of lung health in CF patients. Since the 2007 publication of the guidelines, several novel medications have been approved for use in the US and additional data have been published on therapies previously reviewed. The committee conducted an assessment of the current evidence to develop updated recommendations on the use of currently available chronic treatments for CF lung disease.
Generic	Brand	Strength	Form	Dose
INHALED ANTIBIOTICS¹
aztreonam	Cayston	75mg	pwd for inh via nebulization	Adults and Children: <7yrs: Not established. Use a short-acting bronchodilator 15mins–4hrs before each dose; or a long-acting bronchodilator 30mins–12hrs before starting therapy. ≥7yrs: 75mg by nebulization 3 times daily (≥4hrs apart) for 28 days (then 28 days off).
tobramycin	Tobi	300mg/5mL	soln for oral inh	Adults and Children: <6yrs: Not established. Give in alternate 28-day cycles (28 days on, 28 days off). ≥6yrs: 300mg via oral inh over 15mins twice daily, as close to every 12hrs as possible (must be ≥6hrs apart). Give last when using multiple inhalation therapies.
tobramycin	Tobi Podhaler	28mg/cap	dry pwd for oral inh	Adults and Children: <6yrs: Not established. Give in alternate 28-day cycles (28 days on, 28 days off). ≥6yrs: Inhale contents of 4 caps with Podhaler device twice daily, as close to every 12hrs as possible (must be ≥6hrs apart). Give last when using multiple inhalation therapies.
NSAIDS²
ibuprofen	Advil	200mg	tabs, caps, caplets, liqui-gels	Adults: ≥18yrs: Insufficient data for chronic use. Children: 6–17yrs (FEV1 >60% predicted): maintain peak plasma concentration of 50–100mcg/mL.
	Advil	100mg/5mL	susp
	Motrin	200mg	tabs, caps, caplets, gelcaps
	Motrin	100mg/5mL	susp
CFTR MODULATORS
ivacaftor	Kalydeco³	150mg	tabs	Adults and Children: <1month or <6mos (with hepatic impairment and/or on concomitant moderate or strong CYP3A inhibitors): Not recommended. 1–<6mos (born at a gestational age <37wks): not evaluated. 1–<2mos (≥3kg): 5.8mg packet every 12hrs; 2–<4mos (≥3kg): 13.4mg packet every 12hrs; 4–<6mos (≥5kg): 25mg packet every 12hrs. 6mos–<6yrs (5–<7kg): 25mg packet every 12hrs; (7–<14kg): 50mg packet every 12hrs; (≥14kg): 75mg packet every 12hrs. ≥6yrs: 150mg tab every 12hrs. Hepatic impairment, concomitant moderate or strong CYP3A inhibitors: reduce dosing frequency; see full labeling
ivacaftor	Kalydeco³	5.8mg, 13.4mg, 25mg, 50mg, 75mg; per packet	oral granules⁴
lumacaftor/ ivacaftor	Orkambi³	100mg/125mg, 200mg/125mg	tabs	Adults: ≥12yrs: 2 tabs (200mg/125mg) every 12hrs. Currently taking strong CYP3A inhibitors: initially 1 tab (200mg/125mg) once daily for 1st week then continue with recommended daily dose. Hepatic impairment (moderate): 2 tabs in the AM and 1 tab in the PM; (severe): max 1 tab in the AM and 1 tab in the PM, or less; use with caution. Children: <1yr: Not established. 1–2yrs (7–<9kg): 1pkt (75mg/94mg) every 12hrs; (9–<14kg): 1pkt (100mg/125mg) every 12hrs; (≥14kg): 1 pkt (150mg/188mg) every 12hrs. 2–5yrs (<14kg): 1 pkt (100mg/125mg) every 12hrs; (≥14kg): 1 pkt (150mg/188mg) every 12hrs. 6–11yrs: 2 tabs (100mg/125mg) every 12hrs. Currently taking strong CYP3A inhibitors: initially 1 pkt every other day (1–5yrs) or 1 tab once daily (≥6yrs) for 1st week then continue with recommended daily dose. Hepatic impairment: 1–5yrs (moderate): 1 pkt in the AM and 1 pkt every other day in the PM; (severe): max 1 pkt in the AM, or less; ≥6yrs (moderate): 2 tabs in the AM and 1 tab in the PM; (severe): max 1 tab in the AM and 1 tab in the PM, or less; all: use with caution.
lumacaftor/ ivacaftor	Orkambi³	75mg/94mg, 100mg/125mg, 150mg/188mg; per packet	oral granules⁴
tezacaftor/ ivacaftor + ivacaftor	Symdeko³	50mg/75mg + 75mg, 100mg/150mg + 150mg	tabs	Adults and Children: <6yrs: Not established. 6–<12yrs (<30kg): 1 tab (50mg/75mg) in the AM and 1 tab (75mg) in the PM, approx. 12hrs apart; 6–<12yrs (≥30kg) or ≥12yrs: 1 tab (100mg/150mg) in the AM and 1 tab (150mg) in the PM, approx. 12hrs apart. Concomitant moderate CYP3A inhibitors (6–<12yrs [<30kg]): alternate 1 tab (50mg/75mg) in the AM or 1 tab (75mg) in the AM every other day; (6–<12yrs [≥30kg]) or ≥12yrs: alternate 1 tab (100mg/150mg) in the AM or 1 tab (150mg) in the AM every other day. Concomitant strong CYP3A inhibitors (6–<12yrs [<30kg]): 1 tab (50mg/75mg) in the AM twice weekly, approx. 3–4 days apart; (6–<12yrs [≥30kg]) or ≥12yrs: 1 tab (100mg/150mg) in the AM twice weekly, approx. 3–4 days apart. Moderate hepatic impairment (6–<12yrs [<30kg]): 1 tab (50mg/75mg) once daily, or less frequently if severe; (6–<12yrs [≥30kg]) or ≥12yrs: 1 tab (100mg/150mg) once daily, or less frequently if severe.
elexacaftor/ tezacaftor/ ivacaftor + ivacaftor	Trikafta³	50mg/25mg/ 37.5mg + 75mg, 100mg/50mg/ 75mg + 150mg	tabs	Adults: ≥12yrs: 2 tabs (100mg/50mg/75mg) in the AM and 1 tab (ivacaftor 150mg) in the PM, approx. 12hrs apart. Moderate hepatic impairment (not recommended; if needed, use with caution at reduced dose), concomitant moderate or strong CYP3A inhibitors: see full labeling. Children: <2yrs: Not established. 2–<6yrs (<14kg): 1 packet (80mg/40mg/60mg) in the AM and 1 packet (ivacaftor 59.5mg) in the PM, approx. 12hrs apart; (≥14kg): 1 packet (100mg/50mg/75mg) in the AM and 1 packet (ivacaftor 75mg) in the PM, approx. 12hrs apart. 6–<12yrs (<30kg): 2 tabs (50mg/25mg/37.5mg) in the AM and 1 tab (ivacaftor 75mg) in the PM, approx. 12hrs apart; (≥30kg): 2 tabs (100mg/50mg/75mg) in the AM and 1 tab (ivacaftor 150mg) in the PM, approx. 12hrs apart. Moderate hepatic impairment (not recommended; if needed, use with caution at reduced dose), concomitant moderate or strong CYP3A inhibitors: see full labeling.
elexacaftor/ tezacaftor/ ivacaftor + ivacaftor	Trikafta³	80mg/40mg/ 60mg + 59.5mg, 100mg/50mg/ 75mg + 75mg; per packet	oral granules⁴
MUCOLYTICS
dornase alfa	Pulmozyme	1mg/mL	soln for oral inh	Adults and Children: <3mos: Not recommended. Use appropriate nebulizer (jet or vibrating mesh). Do not dilute. <5yrs: 2.5mg once daily via nebulization; may increase to 2.5mg twice daily (see full labeling).
hypertonic saline	—	3%, 7%	soln for oral inh	Adults and Children: <6yrs: Not established. ≥6yrs: 4mL/dose via nebulization twice daily.
mannitol	Bronchitol	40mg/cap	dry pwd for oral inh	Adults: Perform Bronchitol Tolerance Test (BTT) prior to administration. Use a short-acting bronchodilator 5–15mins before each dose. Add-on treatment: 400mg (10 caps) twice daily (AM & PM) via provided inhaler; PM dose should be ≥2–3hrs before bedtime. Children: Not established.
NOTES
Key: CFTR = cystic fibrosis transmembrane conductance regulator; inh = inhalation; MWF = monday, wednesday, friday; NSAIDS = Nonsteroidal anti-inflammatory drugs; pkt = packet; pwd = powder; RTI = respiratory tract infection ¹ Refer to the CF Foundation’s Pulmonary Clinical Practice Guidelines Committee for their recommendations on the use of oral antibiotics for maintenance therapy. ² The guidelines recommended the use of ibuprofen to prevent the loss of lung function in patients with FEV1 >60% predicted. The Committee updated the guidelines to limit the use of ibuprofen to only children aged 6-17yrs. ³ Take with fat-containing food (eg, eggs, butter, peanut butter, cheese pizza, whole-milk dairy products). ⁴ Mix oral granules with 1 tsp (5mL) of soft-food or liquid (eg, pureed fruits/vegetables, yogurt, applesauce, water, milk, juice, breast milk, or infant formula) and completely consume within 1hr. Not an inclusive list of medications and/or official indications. Please see drug monograph at www.eMPR.com and/or contact company for full drug labeling.
REFERENCE
Mogayzel PJ, Naureckas ET, Robinson KA, et al. Cystic fibrosis pulmonary guidelines: chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2013 Apr 1;187(7):680-9. doi: 10.1164/rccm.201207-1160oe. (Rev. 4/2024)

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