Cystic fibrosis medical therapy

Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Shaghayegh Habibi, M.D.[2]

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Overview

Medical treatments for patients with cystic fibrosis has targeted following consequences of the defect such as GI and pulmonary mucus plugging and infection. Treatment include mucolytic agents (dornase alfa, N-acetyl-L-cysteine), airway surface rehydration (hypertonic saline, osmotic agents), anti-infective agents (for prophylaxis, eradication of early infection and suppression of chronic infection), anti-inflammatory agents (NSAIDs, inhaled corticosteroids, LTB4 receptor antagonists and Azithromycin) and potentiators of CFTR protein defect.

Medical Therapy

• Treatment for cystic fibrosis has targeted following consequences of the defect such as GI and pulmonary mucus plugging and infection.
• Medical treatments for patients with cystic fibrosis are include:

Cystic fibrosis

• 1 Mucolytics
  • 1.1 Recombinant human deoxyribonuclease I (rhDNase) enzyme
    • Preferred regimen (1): Dornase alfa

    Note (1): Cleave the extracellular DNA and aid airway clearance.

  • 1.2 Clevage of disulfide bonds in the mucoproteins
    • Preferred regimen (1): N-acetyl-L-cysteine

    Note (1): Also increase levels of the intracellular antioxidant glutathione (GSH) that protect against the neutrophil-driven tissue damage.

• 2 Airway surface rehydration

  • Preferred regimen (1): Hypertonic saline

  Note (1): As it may cause bronchoconstriction, it is commonly used with an bronchodilator.

  • Preferred regimen (2): Osmotic agents

  Note (2): Mannitol is a nonabsorbable sugar alcohol which provides an osmotic gradient on the airway surface

  • Preferred regimen (3): Correction of ion transport

• 3 Anti-Inflammatory agents

  • Preferred regimen (1): Nonsteroidal anti-inflammatory agents (NSAIDs)

  Note (1): Ibuprofen showed some benefit in young patients with mild disease in high doses.

  • Preferred regimen (2): Inhaled corticosteroids
  • Preferred regimen (3): LTB4 receptor antagonists

  Note (2): Leukotriene B4 (LTB4) is produced by macrophages and PMNs in response to infection and plays a significant role in inflammatory response.

  • Preferred regimen (4): Azithromycin

• 4 Anti-infective agents
  • 1.1 Prophylaxis
    • Preferred regimen (1): Flucloxacillin

    Note (1): Anti-staphylococcal antibiotics (such as flucloxacillin) until ~3 years of age is recommended to reduce the incidence of methicillin-susceptible S. aureus (MSSA)

  • 1.2 Eradication of early infection
    • Preferred regimen (1): Tobramycin

    Note (1): If P. aeruginosa not detected and treated aggressively, this gram-negative, opportunistic bacterium will become chronic.

  • 1.3 Suppression of chronic infection
    • Preferred regimen (1): Tobramycin
    • Preferred regimen (2): Colistin
    • Preferred regimen (3): Aztreonam
  • 1.4 Acute exacerbations

    Note (1): Pulmonary exacerbations are treated with oral or IV antibiotics depending on severity.

• 5 CFTR protein defect
  • 1.1 Potentiators
    • Preferred regimen (1): Ivacaftor

    Note (1): Enhance the activity of the CFTR channel if it is correctly located.

    Note (2): The most significant advance in the treatment of CF over the last few years has been the development of Ivacaftor (Ivacaftor increases the time the CFTR channel is open)

  • 1.2 Correctors and combination therapy
    • Preferred regimen (1): lumicaftor/ivacaftor