| Intervention | Indications/timing | Comments |
| CFTR modulator | - Most patients, depending on genotype and age.
| - Refer to UpToDate content and table on CFTR modulator therapy. The type of CFTR modulator and strength of recommendation depend on the genotype.
|
| Airway clearance therapies |
| Inhaled DNase | - DNase and/or hypertonic saline are recommended for:
- Patients with moderate to severe lung disease, including those on HEMT.
- Patients who are not on HEMT.
- DNase is generally given once daily and hypertonic saline twice daily.
- Administer a short-acting bronchodilator before each treatment.
| - Expert opinion varies regarding thresholds for beginning these inhaled therapies. For those not on HEMT or with moderate to severe lung disease:
- UpToDate suggests initiating DNase for patients with chronic respiratory symptoms or declining pulmonary function, while the CFF recommends its use for all children 2 years and older regardless of symptoms[3,4].
- UpToDate suggests hypertonic saline for all age groups including infants, regardless of symptoms*[5], while the CFF recommends it for those 2 years and older[3,4].
- Regimen should be individualized, depending on patient preference and treatment burden (cost, time).
|
| Inhaled hypertonic saline |
| Airway clearance physiotherapy (chest physiotherapy) | - All patients who produce sputum, twice daily.
| - No modality has been proven better than others.
|
| Prevention of infection |
| Vaccinations | All patients: - Annual influenza vaccine.
- Pneumococcal vaccine, with PCV13 (or PCV15) and PPSV23, depending on age and prior immunization status[6].
- All other routine childhood vaccines.
- COVID-19 vaccines, following CDC guidelines.
| |
| Palivizumab | - We do not suggest prophylaxis with palivizumab for children with CF unless they have other indications.
| - Insufficient evidence for benefit[7,8].
|
| Infection control measures | - All patients, in all health care settings.
| - Contact precautions, physical separation of patients, hand hygiene, use of masks by patients[3].
|
| Bronchodilators |
| Inhaled beta-2 adrenergic receptor agonist | - All patients, before each dose of inhaled antibiotics, DNase, hypertonic saline, or airway clearance therapy session.
- As a rescue medication for acute symptoms, in patients with signs or symptoms of airway hyperresponsiveness.
| |
| Antiinflammatory therapy |
| Oral azithromycin | - All patients ≥6 months, at time of acquisition of P. aeruginosa*.
- Administered 3 times weekly.
- Do not give azithromycin to patients who are infected with nontuberculous mycobacteria.
| - Evidence is strongest for patients ≥6 years who are infected with P. aeruginosa[3,4] but is also effective for younger children when begun at the time that they first become infected with this organism[9]. Azithromycin is probably not effective for most patients who are not infected with P. aeruginosa[10].
- Benefits may be related to antiinflammatory and/or antibacterial properties.
|
| Ibuprofen (high-dose) | - Suggested for children 6 to 17 years with good lung function (FEV1 >60% predicted)[3,4].
- In clinical practice, this is not commonly used in the United States.
| - Not recommended for patients with more severe lung function abnormalities or those who are older than 18 years of age. Requires pharmacokinetic studies to ensure proper dosing and monitoring for adverse effects (gastrointestinal bleeding, kidney function impairment).
|
| Inhaled glucocorticoids | - Patients with definite signs and symptoms of asthma (including patients with asthmatic symptoms in the setting of ABPA)¶.
| - Not recommended for patients without these characteristics, due to little evidence for benefit and concerns about adverse effects on growth.
|
| Prevention of acute exacerbations |
| Inhaled tobramycin and/or aztreonam lysine | - All patients with persistent P. aeruginosa infection.
- Give twice daily for 28 days, alternating with 28 days off of treatment.
| - Refer to UpToDate content on antibiotic therapy for chronic pulmonary infection.
|
| Surveillance and early eradication of P. aeruginosa | - All patients without P. aeruginosa infection.
- Routine surveillance of sputum cultures or throat swabs, followed by eradication protocol if P. aeruginosa is detected.
| - Refer to UpToDate content on early eradication of P. aeruginosa.
|
| Neuraminidase inhibitor (eg, oseltamivir) | - Selected patients meeting criteria for post-exposure prophylaxis or treatment.
| - Refer to UpToDate content on seasonal influenza in children.
|
| Treatment of acute exacerbations |
| Systemic antibiotics | - All patients with an acute exacerbation.
| - Selection of antibiotics is guided by cultures of respiratory secretions. Refer to UpToDate content on treatment of acute pulmonary exacerbations in cystic fibrosis.
|
| Intensify airway clearance therapies | - All patients with an acute exacerbation.
| |
| Systemic glucocorticoids | - Short course for patients with characteristics of an acute asthma episode. A prolonged course may be indicated for patients with confirmed acute ABPA¶.
| - Although practice varies, UpToDate recommends against the use of systemic glucocorticoids except as noted.
|
| Respiratory support | - As needed, guided by prognosis and patient preferences.
| - Patient preferences should be established through discussions of advanced care directives, ideally occurring well before the patient requires the intervention.
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