Lumacaftor/Ivacaftor in Patients Aged 6–11 Years with Cystic Fibrosis and Homozygous for F508del-CFTR
| العنوان: | Lumacaftor/Ivacaftor in Patients Aged 6–11 Years with Cystic Fibrosis and Homozygous for F508del-CFTR |
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| المؤلفون: | David Waltz, Felix Ratjen, Gautham Marigowda, Margaret Rosenfeld, Carlos Milla, Fang Liu |
| المصدر: | American Journal of Respiratory and Critical Care Medicine. 195:912-920 |
| بيانات النشر: | American Thoracic Society, 2017. |
| سنة النشر: | 2017 |
| مصطلحات موضوعية: | Male, Pulmonary and Respiratory Medicine, Spirometry, medicine.medical_specialty, Cystic Fibrosis, Aminopyridines, Cystic Fibrosis Transmembrane Conductance Regulator, Quinolones, Aminophenols, Critical Care and Intensive Care Medicine, Cystic fibrosis, Ivacaftor, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Forced Expiratory Volume, Internal medicine, medicine, Humans, Benzodioxoles, 030212 general & internal medicine, Child, Intensive care medicine, Adverse effect, Sweat test, medicine.diagnostic_test, business.industry, Lumacaftor, medicine.disease, Rash, Treatment Outcome, 030228 respiratory system, chemistry, Tolerability, Drug Therapy, Combination, Female, medicine.symptom, business, medicine.drug |
| الوصف: | Combination lumacaftor/ivacaftor has been shown to improve lung function and other endpoints in patients aged 12 years and older with cystic fibrosis and homozygous for F508del-CFTR, but it has not been assessed in younger patients.In this open-label phase III trial, we evaluated the safety, tolerability, pharmacodynamics, and efficacy of lumacaftor/ivacaftor combination therapy in patients aged 6-11 years with cystic fibrosis who were homozygous for F508del-CFTR.Patients (N = 58) received 200 mg lumacaftor/250 mg ivacaftor orally every 12 hours for 24 weeks in addition to their existing cystic fibrosis medications.Lumacaftor/ivacaftor was well tolerated; the safety profile was generally similar to that observed in larger lumacaftor/ivacaftor trials with older patients. Four patients discontinued (two because of drug-related adverse events: elevated liver transaminases, n = 1; rash, n = 1). No safety concerns were associated with spirometry. No significant changes in percent predicted FEVLumacaftor/ivacaftor was well tolerated in this young population; no new safety concerns were identified. Improvements in lung clearance index, sweat chloride, nutritional status, and health-related quality of life were observed after 24 weeks of treatment. Clinical trial registered with www.clinicaltrials.gov (NCT01897233). |
| تدمد: | 1535-4970 1073-449X 0189-7233 |
| DOI: | 10.1164/rccm.201608-1754oc |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3b9edeb7fe911c5173cf49285f3b4699 https://doi.org/10.1164/rccm.201608-1754oc |
| Rights: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....3b9edeb7fe911c5173cf49285f3b4699 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 15354970 1073449X 01897233 |
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| DOI: | 10.1164/rccm.201608-1754oc |