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

Ivacaftor for the treatment of cystic fibrosis in children under six years of age. Expert Rev Respir Med. 2020 Mar 10;: Authors: Aoyama BC, Mogayzel PJ 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. PMID: 32154747 [PubMed - as supplied by publisher]
Source: Expert Review of Respiratory Medicine - Category: Respiratory Medicine Tags: Expert Rev Respir Med Source Type: research

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CONCLUSIONS: The presence of the ApoE4 allele does not influence the vitamin K status in CF patients who have never received vitamin K supplementation. PMID: 32633402 [PubMed - in process]
Source: European Review for Medical and Pharmacological Sciences - Category: Drugs & Pharmacology Tags: Eur Rev Med Pharmacol Sci Source Type: research
Conditions:   Cystic Fibrosis in Children;   Cystic Fibrosis Interventions:   Behavioral: Questionnaire;   Behavioral: Interview Sponsor:   Centre Hospitalier Intercommunal Creteil Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Publication date: 7 July 2020Source: Cell Reports, Volume 32, Issue 1Author(s): Mehdi Badaoui, Alice Zoso, Tahir Idris, Marc Bacchetta, Juliette Simonin, Sylvain Lemeille, Bernhard Wehrle-Haller, Marc Chanson
Source: Cell Reports - Category: Cytology Source Type: research
In this study, we sought to determine the role of AURKB in transcriptome changes in lung resident fibroblast during pulmonary fibrosis. Our results showed that AURKB regulates the transcriptional changes that are required for the fibroblast activation proce sses such fibroproliferation, myofibroblast survival and extracellular matrix deposition during pulmonary fibrosis. Finally, this study demonstrates that AURKB is a critcal regulator of fibroblast activation in IPF and hence serve as a target for therapeutic intervention.Grantee: Satish K MadalaGrantor: US Department of Defense fundsGrant ID: W81XWH-17-1-0666Grant Title...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Expression profiling by high throughput sequencing Mus musculus Source Type: research
Neutrophil elastase (NE) is a major protease in the airways of patients with cystic fibrosis (CF) that activates airway inflammation by several mechanisms. NE stimulates epithelial toll like receptors (TLR) resulting in cytokine upregulation and release, upregulates MUC5AC, a major airway mucin, degrades both phagocytic receptors and opsonins resulting in both neutrophil and macrophage phagocytic failure, generates oxidative stress via extracellular generation and uptake of heme free iron, and activates other proteases. Altogether, these mechanisms create a significant inflammatory challenge that impairs innate immune func...
Source: Frontiers in Pharmacology - Category: Drugs & Pharmacology Source Type: research
Pseudomonas aeruginosa is an opportunistic pathogen commonly infecting immunocompromised patients with diseases like cystic fibrosis (CF) and cancers and has high rates of recurrence and mortality. The treatment efficacy can be significantly worsened by the multidrug resistance (MDR) of P. aeruginosa, and there is increasing evidence showing that it is easy for this pathogen to develop MDR. Here, we identified a gene cluster, pltZ-pltIJKNOP, which was originally assumed to be involved in the biosynthesis of an antimicrobial pyoluteorin, significantly contributing to the antibiotic resistance of P. aeruginosa ATCC 27853. Mo...
Source: Frontiers in Microbiology - Category: Microbiology Source Type: research
Condition:   Cystic Fibrosis Interventions:   Drug: Omadacycline Injection [Nuzyra];   Drug: Omadacycline Oral Tablet [Nuzyra] Sponsors:   Paul Beringer;   Paratek Pharmaceuticals Inc Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Condition:   Cystic Fibrosis Intervention:   Drug: Treatment with polyethylene glycol (Macrogol 4000) Sponsor:   University Hospital, Bordeaux Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Condition:   Cystic Fibrosis Interventions:   Drug: Omadacycline Injection [Nuzyra];   Drug: Omadacycline Oral Tablet [Nuzyra] Sponsors:   Paul Beringer;   Paratek Pharmaceuticals Inc Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Abstract The opportunistic bacterial pathogen Pseudomonas aeruginosa causes acute and chronic infections that are notoriously difficult to treat. In people with cystic fibrosis, P. aeruginosa can cause lifelong lung infections, and isolation of mucoid P. aeruginosa, resulting from the overproduction of alginate, is associated with chronic infection. The histone-like protein AlgP has previously been implicated in the control of alginate gene expression in mucoid strains, but this regulation is unclear. To explore AlgP in further detail, we deleted algP in mucoid strains and demonstrated that the deletion of algP di...
Source: Microbiology - Category: Microbiology Authors: Tags: Microbiology Source Type: research
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