Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) is a new CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator treatment, used over the last few years, which has shown an improvement in different clinical outcomes in patients with cystic fibrosis (CF). The objective of this study was a systematic research of the literature on the efficacy and safety of this CFTR modulator on patients with CF. A search of Pubmed was conducted for randomized clinical trials and observational studies published from 2012 to September 2022. The included full manuscripts comprised nine clinical trials and 16 observational studies, whose participants were aged ≥12 years or were children 6–11 years old with at least one Phe508del mutation and/or advanced lung disease (ALD). These studies reported that ELX/TEZ/IVA has a significant positive effect on the lung function of patients with CF, by ameliorating parameters such as FEV1, LCI, pulmonary exacerbations or sweat chloride concentration, increasing BMI and improving quality of their life. Its role in cystic fibrosis-related diabetes (CFRD) is not yet clear. It was found that this new CFTR modulator has an overall favorable safety profile, with mild to moderate adverse events. Further studies are needed for a deeper understanding of the impact of CFTR modulators on other CF manifestations, or the possibility of treating with ELX/TEZ/IVA CF patients with rare CFTR mutations.
31 March 2014 in Endocrine, Metabolic & Immune Disorders - Drug Targets
In this review, clinical and epidemiological aspects of milk allergy along with current data on the structure and function of the main cow's milk allergens, are presented. Milk allergy is the most frequent food allergy in childhood. One of the reasons why allergy to cow’s milk shows its highest prevalence in children is its early introduction into the diets of infants when breast feeding is not possible. The major allergens are caseins, a-lactalbumin and β-lactoglobulin, but allergies to other minor proteins (immunoglobulins, bovine serum albumin) have also been reported. Milk allergenicity can be reduced by various processing methods (mainly hydrolysis), and processed formulas based on cow’s milk can often be safely introduced to children allergic to milk proteins. Cross reactivity has been described between different mammalian milks and between milk and meat or animal dander.