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
ANTIBIOTICS
azithromycin Zithromax 250mg, 500mg tabs Adults and Children: ≥6yrs (18–35kg): 250mg 3 times weekly (MWF); (≥36kg): 500mg 3 times weekly (MWF).
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.
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.
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.
ANTI-INFLAMMATORY
ibuprofen Advil 200mg tabs, caps, caplets, liqui-gels Adults: ≥18yrs: Insufficient data for chronic use.
Children: 6–17yrs (FEV1 >60% predicted): 20–30mg/kg twice daily; max 3200mg/day. Maintain peak plasma concentration of 50–100mcg/mL.
100mg/5mL susp
Motrin 200mg tabs, caps, caplets, gelcaps
100mg/5mL susp
CFTR MODULATORS
ivacaftor Kalydeco* 150mg tabs Adults and Children: <4mos, 4–<6mos (with hepatic impairment and/or on concomitant moderate or strong CYP3A inhibitors): Not recommended. 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. Concomitant CYP3A inhibitors (≥6mos): reduce to twice weekly (strong inhibitors) or once daily (moderate inhibitors). Hepatic impairment (≥6mos): reduce to once daily for moderate impairment, once daily or less frequently if severe.
25mg, 50mg, 75mg; per packet oral granules
lumacaftor/ ivacaftor Orkambi* 100mg/ 125mg, 200mg/ 125mg tabs Adults: 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: <2yrs: Not established. 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 tab once daily or 1 pkt every other day (2–5yrs) for 1st week then continue with recommended daily dose. Hepatic impairment: 2–5yrs (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.
100mg/125mg, 150mg/188mg; per packet oral granules
tezacaftor/ ivacaftor + ivacaftor Symdeko* 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 Trikafta* 100mg/50mg/ 75mg + 150mg tabs Adults: 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): 2 tabs (100mg/50mg/75mg) in the AM on Day 1; and then 1 tab (100mg/50mg/75mg) in the AM on Day 2; continue alternating Day 1 and Day 2 dosing thereafter. Concomitant moderate CYP3A inhibitors: 2 tabs (100mg/50mg/75mg) in the AM on Day 1; and then 1 tab (ivacaftor 150mg) in the AM on Day 2; continue alternating thereafter. Concomitant strong CYP3A inhibitors: 2 tabs (100mg/50mg/75mg) twice a week in the AM, approx. 3–4 days apart.
Children: <12yrs: Not established.
MUCOLYTICS
dornase alfa Pulmozyme 1mg/mL soln for oral inh Adults and Children: <3mos: Not recommended. <5yrs: use only if there is a potential for benefit in pulmonary function or in risk of RTI. >5yrs: 2.5mg once daily via nebulization; some patients may benefit from twice daily.
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 Tolerant 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: <18yrs: Not established.
NOTES

Key: CFTR = cystic fibrosis transmembrane conductance regulator; inh = inhalation; MWF = monday, wednesday, friday; pkt = packet; pwd = powder; RTI = respiratory tract infection
* Take with fat-containing food (eg, eggs, avocados, nuts, butter, peanut butter, cheese pizza, whole-milk dairy products).
Mix oral granules with 1 tsp (5mL) of soft-food or liquid (eg, yogurt, applesauce, milk, or juice) and completely consume.

REFERENCE

DeSimone E, Tilleman J, Giles ME, Moussa B. Cystic Fibrosis: Update on Treatment Guidelines and New Recommendations. US Pharm. 2018;43(5)16-21.
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.

Created 3/2021