Ivacaftor for the treatment of cystic fibrosis in children under six years of age.

Expert Review of Respiratory Medicine
Brianna C Aoyama, Peter J Mogayzel

Abstract

Introduction: Cystic fibrosis (CF) results from aberrant ion transport due to abnormalities or absence of the cystic fibrosis transmembrane conductance regulator (CFTR), a chloride transporter that resides on the apical surface of epithelial cells. A novel class of medications, known as CFTR modulators, specifically target the abnormal protein.Areas covered: Ivacaftor increases the open probability of CFTR located on the cell surface, leading to enhanced chloride transport, and has been shown to improve lung function, weight, and quality of life. We reviewed the sentinel studies that lead to the approval of the use of ivacaftor in people with CF age six months and older with at least one CFTR gene mutation that is responsive to ivacaftor based on clinical trial and/or in vitro data. Children with CF have the greatest potential to benefit from CFTR modulator therapy when it is initiated prior to the development of permanent damage; however, challenges remain regarding use of ivacaftor in the youngest pediatric population.Expert opinion: Ivacaftor is safe and effective CFTR modulator that can be prescribed in children over six months of age with at least one CFTR gene mutation that is responsive to ivacaftor.

References

Oct 23, 2009·Proceedings of the National Academy of Sciences of the United States of America·Fredrick Van GoorPaul Negulescu
Nov 19, 2010·The New England Journal of Medicine·Frank J AccursoBonnie W Ramsey
Oct 7, 2011·Proceedings of the National Academy of Sciences of the United States of America·Fredrick Van GoorPaul A Negulescu
Nov 4, 2011·The New England Journal of Medicine·Bonnie W RamseyUNKNOWN VX08-770-102 Study Group
Apr 18, 2013·American Journal of Respiratory and Critical Care Medicine·Jane C DaviesUNKNOWN VX08-770-103 (ENVISION) Study Group
Jul 31, 2013·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Fredrick Van GoorBeth J Hoffman
Jan 17, 2014·The Lancet. Respiratory Medicine·Michael P Boyle, Kris De Boeck
Jun 14, 2014·American Journal of Respiratory and Critical Care Medicine·Steven M RoweUNKNOWN GOAL Investigators of the Cystic Fibrosis Foundation Therapeutics Development Network
Oct 1, 2014·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Kris De BoeckMark Higgins
Jun 14, 2015·The Lancet. Respiratory Medicine·Richard B MossUNKNOWN VX11-770-110 (KONDUCT) Study Group
Jun 16, 2015·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Christopher M SiracusaDennis Drotar
Jul 2, 2015·American Journal of Respiratory and Critical Care Medicine·Gregory S SawickiMichael W Konstan
Oct 29, 2015·The New England Journal of Medicine·Abdul RehmanIbrahim A Janahi
Mar 12, 2016·Expert Opinion on Drug Safety·Susanna A McColley
Apr 4, 2018·American Journal of Respiratory and Critical Care Medicine·Felix RatjenMargaret Rosenfeld
Aug 21, 2018·Current Opinion in Pulmonary Medicine·Kathryn J Crawford, Damian G Downey
Aug 25, 2018·Frontiers in Pharmacology·Harriet CorvolLoïc Guillot
Oct 20, 2018·The New England Journal of Medicine·Dominic KeatingUNKNOWN VX16-445-001 Study Group
Feb 14, 2019·Cell Reports·Gitte BerkersCornelis K van der Ent
Mar 29, 2019·Science Translational Medicine·Xingshen SunJohn F Engelhardt
Mar 29, 2019·Science Translational Medicine·Qing SangLei Wang
May 6, 2019·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Margaret RosenfeldUNKNOWN KLIMB study group
Oct 2, 2019·The Lancet. Respiratory Medicine·Scott C BellFelix Ratjen
Nov 8, 2019·The New England Journal of Medicine·Peter G MiddletonUNKNOWN VX17-445-102 Study Group
Nov 26, 2019·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Alex H GiffordDavid P Nichols

❮ Previous
Next ❯

Citations

Sep 10, 2021·Pediatric Pulmonology·Ashlee AndersonLisa Lubsch
Nov 6, 2021·Pediatric Pulmonology·Megan E GabelSuzanne H Michel

❮ Previous
Next ❯

Related Concepts

Trending Feeds

COVID-19

Coronaviruses encompass a large family of viruses that cause the common cold as well as more serious diseases, such as the ongoing outbreak of coronavirus disease 2019 (COVID-19; formally known as 2019-nCoV). Coronaviruses can spread from animals to humans; symptoms include fever, cough, shortness of breath, and breathing difficulties; in more severe cases, infection can lead to death. This feed covers recent research on COVID-19.

Blastomycosis

Blastomycosis fungal infections spread through inhaling Blastomyces dermatitidis spores. Discover the latest research on blastomycosis fungal infections here.

Nuclear Pore Complex in ALS/FTD

Alterations in nucleocytoplasmic transport, controlled by the nuclear pore complex, may be involved in the pathomechanism underlying multiple neurodegenerative diseases including Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Here is the latest research on the nuclear pore complex in ALS and FTD.

Applications of Molecular Barcoding

The concept of molecular barcoding is that each original DNA or RNA molecule is attached to a unique sequence barcode. Sequence reads having different barcodes represent different original molecules, while sequence reads having the same barcode are results of PCR duplication from one original molecule. Discover the latest research on molecular barcoding here.

Chronic Fatigue Syndrome

Chronic fatigue syndrome is a disease characterized by unexplained disabling fatigue; the pathology of which is incompletely understood. Discover the latest research on chronic fatigue syndrome here.

Evolution of Pluripotency

Pluripotency refers to the ability of a cell to develop into three primary germ cell layers of the embryo. This feed focuses on the mechanisms that underlie the evolution of pluripotency. Here is the latest research.

Position Effect Variegation

Position Effect Variagation occurs when a gene is inactivated due to its positioning near heterochromatic regions within a chromosome. Discover the latest research on Position Effect Variagation here.

STING Receptor Agonists

Stimulator of IFN genes (STING) are a group of transmembrane proteins that are involved in the induction of type I interferon that is important in the innate immune response. The stimulation of STING has been an active area of research in the treatment of cancer and infectious diseases. Here is the latest research on STING receptor agonists.

Microbicide

Microbicides are products that can be applied to vaginal or rectal mucosal surfaces with the goal of preventing, or at least significantly reducing, the transmission of sexually transmitted infections. Here is the latest research on microbicides.

Related Papers

Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
Fredrick Van GoorBeth J Hoffman
European Respiratory Review : an Official Journal of the European Respiratory Society
Isabelle Sermet-Gaudelus
Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
S CarterEdward F McKone
© 2021 Meta ULC. All rights reserved