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Deprecated: Implicit conversion from float 215.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Am+J+Respir+Crit+Care+Med 2017 ; 195 (7): 912-20 Nephropedia Template TP
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Lumacaftor/Ivacaftor in Patients Aged 6?11 Years with Cystic Fibrosis and Homozygous for F508del-CFTR #MMPMID27805836
Milla CE; Ratjen F; Marigowda G; Liu F; Waltz D; Rosenfeld M
Am J Respir Crit Care Med 2017[Apr]; 195 (7): 912-20 PMID27805836show ga
Rationale: 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.Objectives: 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.Methods: 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.Measurements and Main Results: 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 FEV1 were observed (change from baseline at Week 24, +2.5 percentage points; 95% confidence interval [CI], ?0.2 to 5.2; P?=?0.0671). At Week 24, significant improvements from baseline were observed in sweat chloride (?24.8 mmol/L; 95% CI, ?29.1 to ?20.5; P?0.0001), body mass index z score (+0.15; 95% CI, 0.08 to 0.22; P?0.0001), Cystic Fibrosis Questionnaire-Revised respiratory domain score (+5.4; 95% CI, 1.4 to 9.4; P?=?0.0085), and lung clearance index based on lung volume turnover required to reach 2.5% of starting N2 concentration (?0.88; 95% CI, ?1.40 to ?0.37; P?=?0.0018).Conclusions: Lumacaftor/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).