Cystic fibrosis medical therapy: Difference between revisions
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==Overview== | ==Overview== | ||
Medical treatments for patients with cystic fibrosis 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 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 agent|mucolytic agents]] ([[dornase alfa]], [[Acetylcysteine|N-acetyl-L-cysteine]]), [[airway]] surface [[rehydration]] ([[hypertonic]] [[Saline (medicine)|saline]], [[Osmosis|osmotic]] agents), [[Antimicrobial|anti-infective agents]] (for [[prophylaxis]], eradication of early [[infection]] and suppression of chronic [[infection]]), [[Anti inflammatory medications|anti-inflammatory agents]] ([[Non-steroidal anti-inflammatory drug|NSAIDs]], inhaled [[Corticosteroid|corticosteroids]], [[Leukotriene B4 receptor|LTB4 receptor]] [[Receptor antagonist|antagonists]] and [[Azithromycin]]) and potentiators of [[Cystic fibrosis transmembrane conductance regulator|CFTR protein]] defect. | ||
==Medical Therapy== | ==Medical Therapy== | ||
Medical treatments for patients with cystic fibrosis are include:<ref name="pmid19393104">{{cite journal |vauthors=Ratjen FA |title=Cystic fibrosis: pathogenesis and future treatment strategies |journal=Respir Care |volume=54 |issue=5 |pages=595–605 |year=2009 |pmid=19393104 |doi= |url=}}</ref><ref name="pmid27347364">{{cite journal |vauthors=Edmondson C, Davies JC |title=Current and future treatment options for cystic fibrosis lung disease: latest evidence and clinical implications |journal=Ther Adv Chronic Dis |volume=7 |issue=3 |pages=170–83 |year=2016 |pmid=27347364 |pmc=4907071 |doi=10.1177/2040622316641352 |url=}}</ref><ref name="pmid22093951">{{cite journal |vauthors=Konstan MW, Ratjen F |title=Effect of dornase alfa on inflammation and lung function: potential role in the early treatment of cystic fibrosis |journal=J. Cyst. Fibros. |volume=11 |issue=2 |pages=78–83 |year=2012 |pmid=22093951 |pmc=4090757 |doi=10.1016/j.jcf.2011.10.003 |url=}}</ref> | * Treatment for cystic fibrosis has targeted following consequences of the defect such as [[Gastrointestinal tract|GI]] and pulmonary [[mucus]] plugging and [[infection]]. | ||
* Medical treatments for patients with cystic fibrosis are include:<ref name="pmid19393104">{{cite journal |vauthors=Ratjen FA |title=Cystic fibrosis: pathogenesis and future treatment strategies |journal=Respir Care |volume=54 |issue=5 |pages=595–605 |year=2009 |pmid=19393104 |doi= |url=}}</ref><ref name="pmid27347364">{{cite journal |vauthors=Edmondson C, Davies JC |title=Current and future treatment options for cystic fibrosis lung disease: latest evidence and clinical implications |journal=Ther Adv Chronic Dis |volume=7 |issue=3 |pages=170–83 |year=2016 |pmid=27347364 |pmc=4907071 |doi=10.1177/2040622316641352 |url=}}</ref><ref name="pmid22093951">{{cite journal |vauthors=Konstan MW, Ratjen F |title=Effect of dornase alfa on inflammation and lung function: potential role in the early treatment of cystic fibrosis |journal=J. Cyst. Fibros. |volume=11 |issue=2 |pages=78–83 |year=2012 |pmid=22093951 |pmc=4090757 |doi=10.1016/j.jcf.2011.10.003 |url=}}</ref> | |||
===Cystic fibrosis=== | |||
* '''1 [[Mucolytic agent|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 bond|disulfide bonds]] in the [[Mucoprotein|mucoproteins]]''' | |||
*** Preferred regimen (1): [[Acetylcysteine|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 (medicine)|saline]] | |||
*:: '''Note (1):''' As it may cause [[bronchoconstriction]], it is commonly used with an [[bronchodilator]]. | |||
| | *:* Preferred regimen (2): Osmotic agents | ||
|N-acetyl-L-cysteine | *:: '''Note (2):''' [[Mannitol]] is a nonabsorbable [[sugar alcohol]] which provides an osmotic gradient on the airway surface | ||
*:* Preferred regimen (3): Correction of [[Ion transporter|ion transport]] | |||
* '''3 [[Anti inflammatory medications|Anti-Inflammatory agents]]''' | |||
| | *:* Preferred regimen (1): [[Non-steroidal anti-inflammatory drug|Nonsteroidal anti-inflammatory agents (NSAIDs)]] | ||
*:: '''Note (1):''' [[Ibuprofen]] showed some benefit in young patients with mild disease in high [[Dose|doses]]. | |||
*:* Preferred regimen (2): Inhaled [[Corticosteroid|corticosteroids]] | |||
*:* Preferred regimen (3): [[Leukotriene B4 receptor|LTB4 receptor]] [[Receptor antagonist|antagonists]] | |||
*:: '''Note (2):''' [[Leukotriene B4|Leukotriene B4 (LTB4)]] is produced by [[Macrophage|macrophages]] and [[Neutrophil|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):''' [[Antistaphylococcal penicillins|Anti-staphylococcal antibiotics]] (such as [[flucloxacillin]]) until ~3 years of age is recommended to reduce the [[incidence]] of [[methicillin]]-susceptible [[Staphylococcus aureus|S. aureus]] (MSSA) | |||
|Anti-staphylococcal antibiotics (such as flucloxacillin) until ~3 years of age is recommended to reduce the incidence of methicillin-susceptible | ** 1.2 '''Eradication of early infection''' | ||
*** Preferred regimen (1): [[Tobramycin]] | |||
**: '''Note (1):''' If [[Pseudomonas aeruginosa|P. aeruginosa]] not detected and treated aggressively, this [[gram-negative]], [[Opportunistic infection|opportunistic]] bacterium will become [[Chronic (medical)|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 [[Antibiotic|antibiotics]] depending on severity. | |||
* '''5 [[Cystic fibrosis transmembrane conductance regulator|CFTR protein]] defect''' | |||
** 1.1 '''Potentiators''' | |||
*** Preferred regimen (1): [[Ivacaftor]] | |||
| | **: '''Note (1):''' Enhance the activity of the [[Cystic fibrosis transmembrane conductance regulator|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 [[Cystic fibrosis transmembrane conductance regulator|CFTR channel]] is open) | |||
** 1.2 '''Correctors and combination therapy''' | |||
*** Preferred regimen (1): lumicaftor/[[ivacaftor]] | |||
The most significant advance in the treatment of CF over the last few years has been the development of | |||
==References== | ==References== | ||
{{Reflist|2}} | {{Reflist|2}} | ||
[[Category:Medicine]] | |||
[[Category:Up-To-Date]] | |||
[[Category:Gastroenterology]] | [[Category:Gastroenterology]] | ||
[[Category:Pediatrics]] | [[Category:Pediatrics]] | ||
[[Category:Pulmonology]] | [[Category:Pulmonology]] | ||
Latest revision as of 21:05, 6 April 2018
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]
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)
- 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.