What is the treatment for shortness of breath in cystic fibrosis (CF)?

Treatment of Shortness of Breath in Cystic Fibrosis

Airway clearance techniques (ACTs) combined with dornase alfa should be the first-line treatment for shortness of breath in cystic fibrosis patients, with positive expiratory pressure (PEP) devices being the preferred ACT due to their effectiveness, safety, and ability to be self-administered. 1, 2

Airway Clearance Techniques

Airway clearance is fundamental to managing dyspnea in CF patients, as it addresses the underlying pathophysiology of mucus obstruction. The following techniques are recommended based on evidence:

First-Line ACT Options:

1. Positive Expiratory Pressure (PEP) therapy

   • Recommended over conventional chest physiotherapy 3
   • Grade B recommendation (fair evidence, intermediate benefit) 3
   • Reduces exacerbation rates when used with a mask for at least one year 4
   • Can be self-administered without assistance 3

2. Conventional Chest Physiotherapy

   • Includes postural drainage, percussion, and vibration
   • Effective for increasing mucus clearance but effects are modest 3
   • Grade C recommendation (fair evidence, small benefit) 3
   • Requires assistance from a caregiver 5

3. Autogenic Drainage

   • Can be performed without assistance and in one position 3
   • Grade C recommendation (low evidence, small benefit) 3
   • Involves controlled breathing at different lung volumes 5

4. Oscillating PEP devices

   • Alternative to conventional chest physiotherapy 3
   • Grade I recommendation (low evidence, conflicting benefit) 3
   • Examples include Flutter device and high-frequency chest wall oscillation 3

5. Huffing technique

   • Recommended as an adjunct to other methods 3
   • Grade C recommendation (low evidence, small benefit) 3
   • Consists of forced expirations without glottic closure 3

Pharmacological Management

First-Line Pharmacological Therapy:

• Dornase alfa (Pulmozyme)
  • Indicated for management of CF to improve pulmonary function 2
  • Reduces risk of respiratory infections requiring parenteral antibiotics 2
  • Demonstrated improvement in FEV1 by 7.9-9.0% within 8 days of starting treatment 2
  • Can be continued or initiated during acute respiratory exacerbations 2
  • Dosing: 2.5 mg once daily via nebulizer (consider twice daily in patients >21 years or with FVC >85%) 2

Bronchodilator Therapy:

• Should be administered before airway clearance to mobilize secretions 1
• Regular reassessment of bronchodilator response is needed as it can vary over time 1

Comprehensive Management Approach

1. Assess severity of shortness of breath

   • Evaluate baseline pulmonary function
   • Determine if acute exacerbation is present

2. For daily management:

   • Start with bronchodilator therapy
   • Administer dornase alfa once daily (2.5 mg)
   • Perform PEP therapy or preferred ACT at least once daily
   • Include huffing technique as an adjunct

3. For acute exacerbations:

   • Intensify ACT frequency (2-4 times daily)
   • Consider adding antibiotics based on severity 1
   • Continue dornase alfa therapy 2
   • Aggressive treatment of Pseudomonas aeruginosa if present 1

4. For severe shortness of breath:

   • Consider nebulized antibiotics when oral antibiotics and postural drainage are unsuccessful 1
   • More frequent ACTs may be needed

Important Considerations

• No single ACT has been proven superior to others; however, PEP therapy offers advantages of self-administration and comparable efficacy to conventional chest physiotherapy 3, 6, 5

• Exercise should be incorporated as an adjunctive therapy for airway clearance and overall health 6, 7

• Regular microbiologic monitoring is essential to guide antimicrobial therapy 1

• Treatment burden is substantial; prioritize therapies based on individual response 1

• Long-term effects of ACTs on quality of life, exacerbation rates, hospitalizations, and mortality remain uncertain 3, 8

• The introduction of CFTR modulators may impact the need for and approach to airway clearance techniques, but further research is needed 7

Pitfalls to Avoid

• Manually assisted cough may be detrimental in patients with airflow obstruction and should not be used 3

• Failing to reassess bronchodilator response regularly may lead to suboptimal therapy 1

• Neglecting daily airway clearance, even when asymptomatic, can lead to mucus buildup and exacerbations 6

• Overlooking the importance of proper technique in performing ACTs can reduce their effectiveness