Cystic fibrosis medical therapy: Difference between revisions

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* Treatment for cystic fibrosis has targeted following consequences of the defect such as [[Gastrointestinal tract|GI]] and pulmonary [[mucus]] plugging and [[infection]].
* 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>
* 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>
==Medical Therapy==
===Cystic fibrosis===
*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].
===Disease Name===


* '''1 Stage 1 - Name of stage'''
* '''1 [[Mucolytic agent|Mucolytics]]'''
** 1.1 '''Specific Organ system involved 1'''
** 1.1 '''Recombinant human deoxyribonuclease I (rhDNase) enzyme'''
*** 1.1.1 '''Adult'''
*** Preferred regimen (1): [[Dornase alfa]]  
**** Preferred regimen (1): [[drug name]] 100 mg PO q12h for 10-21 days '''(Contraindications/specific instructions)'''  
**: '''Note (1)''': Cleave the [[extracellular]] [[DNA]] and aid [[airway]] clearance.
**** Preferred regimen (2): [[drug name]] 500 mg PO q8h for 14-21 days
** 1.2 '''Clevage of [[Disulfide bond|disulfide bonds]] in the [[Mucoprotein|mucoproteins]]'''
**** Preferred regimen (3): [[drug name]] 500 mg q12h for 14-21 days
*** Preferred regimen (1): [[Acetylcysteine|N-acetyl-L-cysteine]]  
**** Alternative regimen (1): [[drug name]] 500 mg PO q6h for 7–10 days 
**: '''Note (1):''' Also increase levels of the [[intracellular]] [[antioxidant]] [[glutathione]] (GSH) that protect against the [[neutrophil]]-driven tissue damage.  
**** Alternative regimen (2): [[drug name]] 500 mg PO q12h for 14–21 days
* '''2 [[Airway]] surface [[rehydration]]'''  
**** Alternative regimen (3): [[drug name]] 500 mg PO q6h for 14–21 days
*:* Preferred regimen (1): [[Hypertonic]] [[Saline (medicine)|saline]]
*** 1.1.2 '''Pediatric'''
*:: '''Note (1):''' As it may cause [[bronchoconstriction]], it is commonly used with an [[bronchodilator]].
**** 1.1.2.1 (Specific population e.g. '''children < 8 years of age''')
*:* Preferred regimen (2): Osmotic agents
***** Preferred regimen (1): [[drug name]] 50 mg/kg PO per day q8h (maximum, 500 mg per dose) 
*:: '''Note (2):''' [[Mannitol]] is a nonabsorbable [[sugar alcohol]] which provides an osmotic gradient on the airway surface
***** Preferred regimen (2): [[drug name]] 30 mg/kg PO per day in 2 divided doses (maximum, 500 mg per dose)
*:* Preferred regimen (3): Correction of [[Ion transporter|ion transport]]
***** Alternative regimen (1): [[drug name]]10 mg/kg PO q6h (maximum, 500 mg per day)
***** Alternative regimen (2): [[drug name]] 7.5 mg/kg PO q12h (maximum, 500 mg per dose)
***** Alternative regimen (3): [[drug name]] 12.5 mg/kg PO q6h (maximum, 500 mg per dose)
****1.1.2.2 (Specific population e.g. ''''''children < 8 years of age'''''')
***** Preferred regimen (1): [[drug name]] 4 mg/kg/day PO q12h（maximum, 100 mg per dose）
***** Alternative regimen (1): [[drug name]] 10 mg/kg PO q6h (maximum, 500 mg per day)
***** Alternative regimen (2): [[drug name]] 7.5 mg/kg PO q12h (maximum, 500 mg per dose) 
***** Alternative regimen (3): [[drug name]] 12.5 mg/kg PO q6h (maximum, 500 mg per dose)
** 1.2 '''Specific Organ system involved 2'''
*** 1.2.1 '''Adult'''
**** Preferred regimen (1): [[drug name]] 500 mg PO q8h
*** 1.2.2  '''Pediatric'''
**** Preferred regimen (1): [[drug name]] 50 mg/kg/day PO q8h (maximum, 500 mg per dose)


* 2 '''Stage 2 - Name of stage'''
* '''3 [[Anti inflammatory medications|Anti-Inflammatory agents]]'''
** 2.1 '''Specific Organ system involved 1 '''
*:* Preferred regimen (1): [[Non-steroidal anti-inflammatory drug|Nonsteroidal anti-inflammatory agents (NSAIDs)]]
**: '''Note (1):'''
*:: '''Note (1):''' [[Ibuprofen]] showed some benefit in young patients with mild disease in high [[Dose|doses]].
**: '''Note (2)''':
*:* Preferred regimen (2): Inhaled [[Corticosteroid|corticosteroids]]
**: '''Note (3):'''
*:* Preferred regimen (3): [[Leukotriene B4 receptor|LTB4 receptor]] [[Receptor antagonist|antagonists]]
*** 2.1.1 '''Adult'''
*:: '''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.
**** Parenteral regimen
*:* Preferred regimen (4): [[Azithromycin]]
***** Preferred regimen (1): [[drug name]] 2 g IV q24h for 14 (14–21) days
***** Alternative regimen (1): [[drug name]] 2 g IV q8h for 14 (14–21) days
***** Alternative regimen (2): [[drug name]] 18–24 MU/day IV q4h for 14 (14–21) days
**** Oral regimen
***** Preferred regimen (1): [[drug name]] 500 mg PO q8h for 14 (14–21) days
***** Preferred regimen (2): [[drug name]] 100 mg PO q12h for 14 (14–21) days
***** Preferred regimen (3): [[drug name]] 500 mg PO q12h for 14 (14–21) days
***** Alternative regimen (1): [[drug name]] 500 mg PO q6h for 7–10 days 
***** Alternative regimen (2): [[drug name]] 500 mg PO q12h for 14–21 days
***** Alternative regimen (3):[[drug name]] 500 mg PO q6h for 14–21 days
*** 2.1.2 '''Pediatric'''
**** Parenteral regimen
***** Preferred regimen (1): [[drug name]] 50–75 mg/kg IV q24h for 14 (14–21) days (maximum, 2 g)
***** Alternative regimen (1): [[drug name]] 150–200 mg/kg/day IV q6–8h for 14 (14–21) days (maximum, 6 g per day)
***** Alternative regimen (2):  [[drug name]] 200,000–400,000 U/kg/day IV q4h for 14 (14–21) days (maximum, 18–24 million U per day) ''''''(Contraindications/specific instructions)''''''
**** Oral regimen
***** Preferred regimen (1): [[drug name]] 50 mg/kg/day PO q8h for 14 (14–21) days  (maximum, 500 mg per dose)
***** Preferred regimen (2): [[drug name]] '''(for children aged ≥ 8 years)''' 4 mg/kg/day PO q12h for 14 (14–21) days (maximum, 100 mg per dose)
***** Preferred regimen (3): [[drug name]] 30 mg/kg/day PO q12h for 14 (14–21) days  (maximum, 500 mg per dose)
***** Alternative regimen (1):  [[drug name]] 10 mg/kg PO q6h 7–10 days  (maximum, 500 mg per day)
***** Alternative regimen (2): [[drug name]] 7.5 mg/kg PO q12h for 14–21 days  (maximum, 500 mg per dose)
***** Alternative regimen (3): [[drug name]] 12.5 mg/kg PO q6h for 14–21 days  (maximum,500 mg per dose)
** 2.2  '<nowiki/>'''''Other Organ system involved 2''''''
**: '''Note (1):'''
**: '''Note (2)''':
**: '''Note (3):'''
*** 2.2.1 '''Adult'''
**** Parenteral regimen
***** Preferred regimen (1): [[drug name]] 2 g IV q24h for 14 (14–21) days
***** Alternative regimen (1): [[drug name]] 2 g IV q8h for 14 (14–21) days
***** Alternative regimen (2): [[drug name]] 18–24 MU/day IV q4h for 14 (14–21) days
**** Oral regimen
***** Preferred regimen (1): [[drug name]] 500 mg PO q8h for 14 (14–21) days
***** Preferred regimen (2): [[drug name]] 100 mg PO q12h for 14 (14–21) days
***** Preferred regimen (3): [[drug name]] 500 mg PO q12h for 14 (14–21) days
***** Alternative regimen (1): [[drug name]] 500 mg PO q6h for 7–10 days 
***** Alternative regimen (2): [[drug name]] 500 mg PO q12h for 14–21 days
***** Alternative regimen (3):[[drug name]] 500 mg PO q6h for 14–21 days
*** 2.2.2 '''Pediatric'''
**** Parenteral regimen
***** Preferred regimen (1): [[drug name]] 50–75 mg/kg IV q24h for 14 (14–21) days (maximum, 2 g)
***** Alternative regimen (1): [[drug name]] 150–200 mg/kg/day IV q6–8h for 14 (14–21) days (maximum, 6 g per day)
***** Alternative regimen (2):  [[drug name]] 200,000–400,000 U/kg/day IV q4h for 14 (14–21) days (maximum, 18–24 million U per day)
**** Oral regimen
***** Preferred regimen (1):  [[drug name]] 50 mg/kg/day PO q8h for 14 (14–21) days  (maximum, 500 mg per dose)
***** Preferred regimen (2): [[drug name]] 4 mg/kg/day PO q12h for 14 (14–21) days (maximum, 100 mg per dose)
***** Preferred regimen (3): [[drug name]] 30 mg/kg/day PO q12h for 14 (14–21) days  (maximum, 500 mg per dose)
***** Alternative regimen (1):  [[drug name]] 10 mg/kg PO q6h 7–10 days  (maximum, 500 mg per day)
***** Alternative regimen (2): [[drug name]] 7.5 mg/kg PO q12h for 14–21 days  (maximum, 500 mg per dose)
***** Alternative regimen (3): [[drug name]] 12.5 mg/kg PO q6h for 14–21 days  (maximum,500 mg per dose)


 
* '''4 Anti-infective agents'''
 
** 1.1 '''Prophylaxis'''
{| class="wikitable"
*** Preferred regimen (1):  [[Flucloxacillin]]
! colspan="3" |Medical treatment in patients with Cystic fibrosis
**: '''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)
|-
** 1.2 '''Eradication of early infection'''
!'''Category'''
*** Preferred regimen (1):  [[Tobramycin]]
!'''Approaches'''
**: '''Note (1):''' If [[Pseudomonas aeruginosa|P. aeruginosa]] not detected and treated aggressively, this [[gram-negative]], [[Opportunistic infection|opportunistic]] bacterium will become [[Chronic (medical)|chronic]].
!Explanation
** 1.3 '''Suppression of chronic infection'''
|-
*** Preferred regimen (1):  [[Tobramycin]]
| rowspan="2" |[[Mucolytic agent|Mucolytic agents]]
*** Preferred regimen (2):  [[Colistin]]
|[[Dornase alfa]]
*** Preferred regimen (3):  [[Aztreonam]]
|Cleave the [[extracellular]] [[DNA]] and aid [[airway]] clearance
** 1.4 '''Acute exacerbations'''
|-
**: '''Note (1):''' Pulmonary exacerbations are treated with oral or IV [[Antibiotic|antibiotics]] depending on severity.
|[[Acetylcysteine|N-acetyl-L-cysteine]]
* '''5 [[Cystic fibrosis transmembrane conductance regulator|CFTR protein]] defect'''
|Also increase levels of the [[intracellular]] [[antioxidant]] [[glutathione]] (GSH) that protect against the [[neutrophil]]-driven tissue damage
** 1.1 '''Potentiators'''
|-
*** Preferred regimen (1): [[Ivacaftor]]
| rowspan="3" |[[Airway]] surface [[rehydration]]
**: '''Note (1):''' Enhance the activity of the [[Cystic fibrosis transmembrane conductance regulator|CFTR channel]] if it is correctly located.
|[[Hypertonic]] [[Saline (medicine)|saline]]
**: '''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)
|As it may cause [[bronchoconstriction]], it is commonly used with an [[bronchodilator]]
** 1.2 '''Correctors and combination therapy'''
|-
*** Preferred regimen (1): lumicaftor/[[ivacaftor]]
|[[Osmosis|Osmotic]] agents
|[[Mannitol]] is a nonabsorbable [[sugar]] [[alcohol]] which provides an [[Osmosis|osmotic]] gradient on the [[airway]] surface 
|-
|Correction of [[ion]] transport
|
|-
| rowspan="4" |[[Antimicrobial|Anti-infective agents]]
|[[Prophylaxis]]
|Anti-[[Staphylococcus|staphylococcal]] [[Antibiotic|antibiotics]] (such as [[flucloxacillin]]) until ~3 years of age is recommended to reduce the incidence of [[Staphylococcus aureus|methicillin-susceptible ''S. aureus'']] ([[Staphylococcus aureus|MSSA]])
|-
|Eradication of early [[infection]]
|If [[Pseudomonas aeruginosa|P. aeruginosa]] not detected and treated aggressively, this [[gram-negative]], [[Opportunistic infection|opportunistic bacterium]] will become chronic.
* North America: inhaled [[tobramycin]]
* Europe: multicentre trial is currently assessing whether IV or oral [[Antibiotic|antibiotics]] are superior + nebulized [[colistin]]
|-
|Suppression of chronic [[infection]]
|The most commonly used nebulized [[Antibiotic|antibiotics]] against [[Pseudomonas aeruginosa|P. aeruginosa]] are [[tobramycin]], [[colistin]] and [[aztreonam]].
|-
|Acute exacerbations
|Pulmonary exacerbations are treated with oral or [[Intravenous therapy|IV]] [[Antibiotic|antibiotics]] depending on severity.
|-
| rowspan="4" |[[Anti inflammatory medications|Anti-Inflammatory agents]]
|[[Non-steroidal anti-inflammatory drug|Nonsteroidal anti-inflammatory agents (NSAIDs)]]
|[[Ibuprofen]] showed some benefit in young patients with mild disease in high doses.
|-
|Inhaled [[Corticosteroid|corticosteroids]]
|
|-
|[[Leukotriene B4 receptor|LTB4 receptor]] antagonists
|[[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.
|-
|[[Azithromycin]]
|
|-
| rowspan="2" |[[Cystic fibrosis transmembrane conductance regulator|CFTR protein]] defect
|Potentiators
|Enhance the activity of the [[Cystic fibrosis transmembrane conductance regulator|CFTR channel]] if it is correctly located.
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)
|-
|Correctors and combination therapy
|lumicaftor/[[ivacaftor]]
|}


==References==
==References==

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

References

  1. ↑ Ratjen FA (2009). "Cystic fibrosis: pathogenesis and future treatment strategies". Respir Care. 54 (5): 595–605. PMID 19393104.
  2. ↑ 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.
  3. ↑ 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.
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