Paediatric drugs
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Review
Managing the underlying cause of cystic fibrosis: a future role for potentiators and correctors.
Cystic fibrosis (CF), a severe genetic disease, is caused by mutations that alter the structure and function of CFTR, a plasma membrane channel permeable to chloride and bicarbonate. Defective anion transport in CF irreversibly damages the lungs, pancreas, liver, and other organs. CF mutations cause loss of CFTR function in multiple ways. ⋯ Because of multiple defects caused by the p. Phe508del mutation, it is probable that rescue of the mutant protein will require combined treatment with correctors having different mechanisms of action. This review evaluates the status of experimental and clinical research in pharmacotherapy for the CF basic defect.
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Fentanyl is a synthetic opioid commonly used as an anesthetic and also increasingly popular as a sedative agent in neonates. Initial dosage regimens in this population are often empirically derived from adults on a body weight basis. However, ontogenic maturation processes related to drug disposition are not necessarily always body weight correlates. We developed a predictive pharmacokinetic/pharmacodynamic model that includes growth and maturation physiologic changes for fentanyl in neonatal care. ⋯ Integrated pharmacokinetic/pharmacodynamic modeling showed that the usually prescribed dosage regimens of fentanyl in neonates may not always provide the optimum degree of sedation. The model could be used in optimal design of clinical trials for this vulnerable population. Prospective clinical testing is the reasonable next step.
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Gastroesophageal reflux (GER) is defined as the involuntary retrograde passage of gastric contents into the esophagus with or without regurgitation or vomiting. It is a frequently experienced physiologic condition occurring several times a day, mostly postprandial and causes no symptoms. These infants are also called 'happy spitters'. ⋯ Currently, North American Society for Pediatric Gasroenterology, Hepatology and Nutrition (NASPGHAN) and European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) practice guidelines concluded that there is insufficient evidence to justify the routine use of prokinetic agents. Esomeprazole (Nexium) is now approved in the US for short-term treatment of GERD with erosive esophagitis in infants aged from 1 to 12 months. Although Nissen fundoplication is now well established as a treatment option in selected cases of GERD in children, its role in neonates and young infants is unclear and is only reserved for selective infants who did not respond to medical therapy and have life-threatening complications of GERD.
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Asthma is the most common chronic disease in children in many low- and middle-income countries. In these settings, the burden of childhood asthma is increasing and is associated with severe disease. There are a number of challenges to providing optimal management of childhood asthma in such settings. ⋯ A low-cost spacer made from a plastic bottle is effective for use with a metered-dose inhaler, but use must be included in asthma educational initiatives. Educational programs for healthcare personnel and for the public that are culturally and language appropriate are needed for effective implementation of asthma guidelines. Socioeconomic and structural barriers to care within health services remain obstacles to achieving optimal treatment of asthma for many children.