Abstract
Cystic fibrosis (CF) is an autosomal recessive illness caused by the defective cystic fibrosis transmembrane conductance regulator (CFTR) gene. These patients suffer from repeated chronic sinuses and lung infections, resulting in frequent hospital admissions and antibiotic (Abx) courses. These are the major contributing factors responsible for a low health-related quality of life (HRQoL) and increasing the disease burden. The introduction and approval of CFTR modulators-lumacaftor (LUM) and ivacaftor (IVA) in 2015 by the US Food and Drug Administration (FDA) reduced the mortality and morbidity rates associated with the disease. In 2018, the FDA approved these drugs from age two and five years with two copies of F5806 del. This literature review aims to present the studies centered on the clinical effects of LUM/IVA. We searched for the relevant articles, from 2016 to 2020, in PubMed Central (PMC), Google Scholars, and Journal of Cystic Fibrosis. LUM/IVA has a broader range of effects. They showed marked improvement in the reduction of pulmonary exacerbations (PEx), Hospitalization rates, Abx use, and modification in forced expiratory volume in one second (FEV1) status of pre-existing severe lung disease. Now, there is a need for an initiative to conduct more clinical trials and studies in the future to assess and evaluate the long-term clinical benefits and safety of LUM/IVA therapy in all age groups.
Highlights
BackgroundCystic fibrosis (CF) is a chronic, progressive, and genetic disease affecting more than 90,000 people worldwide
The introduction and approval of CFTR modulators-lumacaftor (LUM) and ivacaftor (IVA) in 2015 by the US Food and Drug Administration (FDA) reduced the mortality and morbidity rates associated with the disease
The primary defect is the mutant cystic fibrosis transmembrane conductance regulator (CFTR) gene, which codes for CFTR protein, either absent or defective [1,2]
Summary
Cystic fibrosis (CF) is a chronic, progressive, and genetic disease affecting more than 90,000 people worldwide. The primary defect is the mutant cystic fibrosis transmembrane conductance regulator (CFTR) gene, which codes for CFTR protein, either absent or defective [1,2]. Defective CFTR protein acts as abnormal channels present on the body's mucous membranes as in the respiratory, gastrointestinal, and reproductive systems. These protein channels are responsible for chloride and fluid movement from cells of specific regions. The regular activity of the channel is halted by mutations either caused by protein production or processing, gating or, conduction abnormalities [3,4]. The results are the thick and sticky secretions instead of thin, normal secretions in targeted regions that are pathognomonic of these patients' signs and symptoms
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