Table 5c. [Treatment of Manifestations in Individuals with Cystic Fibrosis]. - GeneReviews®

Manifestation/ Concern	Treatment	Considerations/Other
Pulmonary	Airway clearance to aid in mucus clearance ¹
Chronic pulmonary medications to open airway, ↓ sputum viscosity, promote expectoration of secretions, & deliver anti-inflammatory & antimicrobial medications ²	Recommended sequence for inhaled medications: Bronchodilator Hypertonic saline Dornase alfa Airway clearance Inhaled corticosteroids &/or long-acting beta agonist (for select persons) Aerosolized antibiotic
Standard treatments for pneumothorax or hemoptysis ³
Consider double lung transplant for those w/advanced lung disease. ⁴
Infection	Routine childhood vaccinations incl annual influenza vaccine
Use contact precautions (gown & gloves) for every encounter by every clinician.	Assume all people w/CF have transmissible bacteria.
Antibiotics (oral, inhaled, or IV) for chronic suppression & to treat pulmonary exacerbations Treat identified bacteria in affected persons & bacteria common in CF. ⁵	Eradication attempts are recommended for Pseudomonas aeruginosa. ⁶
ENT	Standard treatments for rhinosinusitis Referral to ENT specialist w/CF experience as needed Sinus surgery as needed to widen ostia &/or clear impacted secretions Polypectomy as needed for obstructive nasal polyps	If needed, evaluate w/sinus CT; but often reserved for pre-surgical planning or unresolved symptoms despite maximal therapy.
Exocrine pancreatic insufficiency	Consult w/nutritionist w/experience in CF. Pancreatic enzyme replacement therapy Nutrient-dense food & supplements Fat-soluble vitamin supplements	Consider zinc supplementations. Consider gastrostomy tube. ⁷
Distal intestinal obstructive syndrome	If obstructed, consult w/surgeon for intervention. If not obstructed, use laxatives. ⁸
Gastroesophageal reflux disease	Standard treatments
Liver disease	Persistent elevations in liver function tests are treated w/ursodiol. ⁹ Refer to hepatologist w/CF experience.	May progress to need for liver transplant.
Diabetes (CFRD)	Refer to endocrinologist familiar w/CFRD. Treat CFRD w/glucose monitoring & insulin therapy. ¹⁰
Infertility	Counseling regarding partner testing & preimplantation genetic testing ART, sperm donation, surrogacy, & adoption	Invasive procedures & cost of aspiration/extraction followed by IVF may be prohibitive for many. ¹¹
Males: Ultrasound to assess for absence of vas deferens ART involves eval by urologist. 2 techniques are widely used: microsurgical epididymal sperm aspiration or testicular biopsy & sperm extraction. Once sperm is obtained, IVF w/intracytoplasmic sperm injection is necessary as sperm counts & concentration are often too low for intrauterine insemination. ¹¹	Female partners of men w/CF will also need to undergo medical procedures assoc w/IVF regardless of underlying fertility status.
Females: ↓ fertility or infertility can be treated by several options available through ART. ¹² There are no currently established guidelines as to which modality should be used. While intrauterine insemination combined w/gonadotropin ovulation induction can be used, IVF is more co mmonly used. ¹¹	IVF offers ability of preimplantation genetic testing.
Salt loss syndromes / Dehydration	Salt supplementation Extra salt & water for hydration & salt loss in situations w/salt loss (e.g., ↑ heat/humidity, exercise)
Mental health	Standard treatments using age-appropriate psychotherapy & pharmacotherapy ¹³

Manifestation/
Concern

Treatment

Considerations/Other

Pulmonary

Airway clearance to aid in mucus clearance ¹

Chronic pulmonary medications to open airway, ↓ sputum viscosity, promote expectoration of secretions, & deliver anti-inflammatory & antimicrobial medications ²

Recommended sequence for inhaled medications:

Bronchodilator
Hypertonic saline
Dornase alfa
Airway clearance
Inhaled corticosteroids &/or long-acting beta agonist (for select persons)
Aerosolized antibiotic

Standard treatments for pneumothorax or hemoptysis ³

Consider double lung transplant for those w/advanced lung disease. ⁴

Infection

Routine childhood vaccinations incl annual influenza vaccine

Use contact precautions (gown & gloves) for every encounter by every clinician.

Assume all people w/CF have transmissible bacteria.

Antibiotics (oral, inhaled, or IV) for chronic suppression & to treat pulmonary exacerbations
Treat identified bacteria in affected persons & bacteria common in CF. ⁵

Eradication attempts are recommended for Pseudomonas aeruginosa. ⁶

ENT

Standard treatments for rhinosinusitis
Referral to ENT specialist w/CF experience as needed
Sinus surgery as needed to widen ostia &/or clear impacted secretions
Polypectomy as needed for obstructive nasal polyps

If needed, evaluate w/sinus CT; but often reserved for pre-surgical planning or unresolved symptoms despite maximal therapy.

Exocrine
pancreatic
insufficiency

Consult w/nutritionist w/experience in CF.
Pancreatic enzyme replacement therapy
Nutrient-dense food & supplements
Fat-soluble vitamin supplements

Consider zinc supplementations.
Consider gastrostomy tube. ⁷

Distal intestinal
obstructive
syndrome

If obstructed, consult w/surgeon for intervention.
If not obstructed, use laxatives. ⁸

Gastroesophageal
reflux disease

Standard treatments

Liver disease

Persistent elevations in liver function tests are treated w/ursodiol. ⁹
Refer to hepatologist w/CF experience.

May progress to need for liver transplant.

Diabetes (CFRD)

Refer to endocrinologist familiar w/CFRD.
Treat CFRD w/glucose monitoring & insulin therapy. ¹⁰

Infertility

Counseling regarding partner testing & preimplantation genetic testing
ART, sperm donation, surrogacy, & adoption

Invasive procedures & cost of aspiration/extraction followed by IVF may be prohibitive for many. ¹¹

Males:

Ultrasound to assess for absence of vas deferens
ART involves eval by urologist. 2 techniques are widely used: microsurgical epididymal sperm aspiration or testicular biopsy & sperm extraction.
Once sperm is obtained, IVF w/intracytoplasmic sperm injection is necessary as sperm counts & concentration are often too low for intrauterine insemination. ¹¹

Female partners of men w/CF will also need to undergo medical procedures assoc w/IVF regardless of underlying fertility status.

Females:

↓ fertility or infertility can be treated by several options available through ART. ¹² There are no currently established guidelines as to which modality should be used.
While intrauterine insemination combined w/gonadotropin ovulation induction can be used, IVF is more co mmonly used. ¹¹

IVF offers ability of preimplantation genetic testing.

Salt loss
syndromes /
Dehydration

Salt supplementation
Extra salt & water for hydration & salt loss in situations w/salt loss (e.g., ↑ heat/humidity, exercise)

Mental health

Standard treatments using age-appropriate psychotherapy & pharmacotherapy ¹³

From: Cystic Fibrosis

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Adam MP, Feldman J, Mirzaa GM, et al., editors.

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