title: Efficacy and adverse effects of drugs used to treat adult cystic fibrosis.
abstract: BACKGROUND
  Cystic fibrosis (CF) is an autosomal recessive disease and is the most commonly seen monogenetic disease in Caucasians. The disease has various manifestations resulting from the abnormal thick secretions, most common being chronic lung infection and airway obstruction. Many new promising drugs have appeared on the horizon over the years. This review here is an attempt to bring together the various treatments being used to prolong and enhance the quality of life of CF patients.
  
  
  METHODS
  A literature review of published as well as ongoing clinical trials, meta-analysis and systematic reviews regarding the drugs used in CF management was carried out using PubMed and Ovid databases.
  
  
  CONCLUSIONS
  New concepts have been formed and some positive results in this direction have already led to the approval of cystic fibrosis transmembrane conductance regulator potentiator drug. Gene therapy and stem cell therapy are under development. The current therapies such as dornase alfa and pancreatic enzymes targeting the symptoms continue to evolve as they play an important complementary role. Development of new simple and cost-effective markers, which help assess the efficacy and safety of these constantly emerging new drugs, is also being investigated.
authors.affiliation: New York Medical College/Westchester Medical Center, Division of Pulmonary, Critical Care and Sleep Medicine , 100 Woods Road, Valhalla, NY 10595 , USA +1 914 493 7518 ; +1 914 493 8130 ; ravichopra77@gmail.com.
authors.forename: Ravi
authors.initials: R
authors.lastname: Chopra
authors.forename: Lisa
authors.initials: L
authors.lastname: Paul
authors.forename: Rajapriya
authors.initials: R
authors.lastname: Manickam
authors.forename: Wilbert S
authors.initials: WS
authors.lastname: Aronow
authors.forename: George P
authors.initials: GP
authors.lastname: Maguire
mesh_terms: D000328:Adult; D015415:Biomarkers / Q000378:metabolism; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000503:physiopathology / Q000628:therapy; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000187:drug effects / Q000378:metabolism; D015195:Drug Design; D064420:Drug-Related Side Effects and Adverse Reactions / Q000209:etiology; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D011788:Quality of Life; D033581:Stem Cell Transplantation / Q000379:methods
chemical_list: D015415:Biomarkers; D019005:Cystic Fibrosis Transmembrane Conductance Regulator
keywords: antibiotics; bronchodilators; cystic fibrosis; cystic fibrosis transmembrane conductance regulator modulators; new treatments