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:[1][2][3]
Medical Therapy
- Pharmacologic medical therapy is recommended among patients with [disease subclass 1], [disease subclass 2], and [disease subclass 3].
- Pharmacologic medical therapies for [disease name] include (either) [therapy 1], [therapy 2], and/or [therapy 3].
- Empiric therapy for [disease name] depends on [disease factor 1] and [disease factor 2].
- Patients with [disease subclass 1] are treated with [therapy 1], whereas patients with [disease subclass 2] are treated with [therapy 2].
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.
- 1.1 Recombinant human deoxyribonuclease I (rhDNase) enzyme
- 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.
- 1.1 Prophylaxis
- 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
- 1.1 Potentiators
References
- ↑ Ratjen FA (2009). "Cystic fibrosis: pathogenesis and future treatment strategies". Respir Care. 54 (5): 595–605. PMID 19393104.
- ↑ Edmondson C, Davies JC (2016). "Current and future treatment options for cystic fibrosis lung disease: latest evidence and clinical implications". Ther Adv Chronic Dis. 7 (3): 170–83. doi:10.1177/2040622316641352. PMC 4907071. PMID 27347364.
- ↑ Konstan MW, Ratjen F (2012). "Effect of dornase alfa on inflammation and lung function: potential role in the early treatment of cystic fibrosis". J. Cyst. Fibros. 11 (2): 78–83. doi:10.1016/j.jcf.2011.10.003. PMC 4090757. PMID 22093951.