kalydeco治疗囊性纤维化的疗效？

The treatment effect of cystic fibrosis is obvious. After treatment with kalydeco, the patient's sweat chloride decreased and the body mass index increased significantly. It provided a substantial improvement in lung function, respiratory symptoms and body mass index, and could relieve uncomfortable symptoms such as cough, sputum, abdominal distension, abdominal pain, and abnormal changes in sweat.

cystic fibrosis

Cystic fibrosis (CF), an autosomal recessive disease, is the most common fatal genetic disorder affecting children and young adults. It is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene located on chromosome 7. This gene encodes a chloride channel that regulates the transport of salt and water across cell membranes. When CFTR becomes dysfunctional, the secretions become viscous, leading to pathology in multiple organ systems, most notably the lungs and gastrointestinal tract.

Although there is no cure for the underlying disease process, current therapies have increased overall survival rates for CF patients, with average life expectancy now at 48 years according to recent Canadian statistics. To date, the goals of CF treatment have been: preservation of lung function by minimizing lung infection and inflammation; recovery of baseline lung function, symptoms, and inflammation levels after acute respiratory exacerbation; and maintenance of adequate nutrition. Treatment strategies include a combination of physical therapy, pharmacological agents (ie, antibiotics, anti-inflammatory drugs, mucolytics), nutritional therapy (ie, high-calorie and high-fat diet), and pancreatic enzyme replacement therapy in patients with pancreatic insufficiency.

kalydeco drug introduction

Kalydeco is the first oral CFTR potentiator approved by Health Canada for the treatment of CF in patients 6 years and older with G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R or G970R mutations in the CFTR gene. The drug works by prolonging the time that activated CFTR channels remain open, thereby enhancing the regulation of chloride and water transport across cell membranes. It is available as a 150 mg oral tablet. Health Canada's recommended dose is 150 mg every 12 hours with fatty foods.

kalydeco for cystic fibrosis

One trial looked at kalydeco's effectiveness in treating cystic fibrosis. Background: Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at least one copy of the Phe508del CFTR mutation. In a phase 2 trial involving patients heterozygous for the Phe508del CFTR mutation and the minimal-function mutation (phe 508del-minimal-function genotype), the next-generation CFTR corrector elexacaftor, combined with tezacaftor and kalydeco, improved Phe508del CFTR function and clinical outcomes.

Methods: A phase 3, randomized, double-blind, placebo-controlled trial to confirm the efficacy and safety of elecafor-tizakafor-ivacarfor in patients 12 years of age or older with cystic fibrosis with the Phe508del-minimal function genotype. Patients were randomly assigned to receive 24 weeks of kalydeco or placebo. The primary endpoint was the absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) at week 4.

Results: A total of 403 patients were randomized and received at least one dose of active treatment or placebo. Relative to placebo, kalydeco- had 13.8 percentage points higher predicted FEV1 at 4 weeks and 14.3 percentage points higher at 24 weeks, 63% lower lung exacerbations, and 41.8 mmol lower sweat chloride concentrations per liter.

Kalydeco is effective in patients with cystic fibrosis, is generally safe, and has an acceptable side effect profile.

kalydeco

The recommended dose of kalydeco for adults and pediatric patients 6 years and older is 150 mg taken orally every 12 hours (total daily dose of 300 mg) with fatty foods, such as eggs, butter, peanut butter, cheese pizza, and whole-milk dairy products, such as whole milk, cheese, yogurt, breast milk, and infant formula.

If you miss a dose, take kalydeco with a fatty meal as soon as prescribed and within 6 hours of your usual dose. If you miss a dose of kalydeco and it is more than 6 hours after you usually take it, skip the dose. Do not take 2 doses of medicine at the same time to make up for a missed dose.

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