The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation <18 years of age and to describe the long-term safety and effectiveness of Kalydeco in CF patients with the R117H-CFTR mutation overall and in patients ≥18 years. The long-term safety and effectiveness of Kalydeco will be examined in totality through the evaluation of the primary outcome measures.
Description: Spirometry will be performed according to the standard procedure at each site, and FEV1 values as recorded in the registry will be evaluated. All descriptive and summary data collected for FEV1 will be repeated for FVC
Measure: Lung function measurements (percent predicted forced expiratory volume in 1 second [FEV1] and forced vital capacity [FVC]) Time: 36 MonthsDescription: Pulmonary exacerbation data will be collected as recorded in the registry.
Measure: Pulmonary exacerbations, use of IV antibiotics Time: 36 MonthsDescription: Height and weight measurements as recorded in the registry will be evaluated. BMI, BMI-for-age z-score, and weight-for-age z-score will be derived
Measure: Nutritional parameters (body mass index [BMI], BMI-for-age z-score, weight, and weight-for-age z-score) Time: 36 MonthsDescription: Death will be collected from the registry database.
Measure: Death or transplantation Time: 36 monthsDescription: Hospitalizations will be collected from the registry database.
Measure: Hospitalizations Time: 36 MonthsDescription: Information for the above shown CF-related complications as recorded in the registry will be evaluated
Measure: Selected Complications (Symptomatic sinus disease, Pulmonary complications, CF-related diabetes (CFRD) and distal intestinal obstruction syndrome (DIOS), Hepatobiliary complications, Pancreatitis) Time: 36 MonthsDescription: Data on microorganisms as recorded in the registry will be evaluated
Measure: Select pulmonary microorganisms (e.g., P. aeruginosa, S. aureus) Time: 36 MonthsCohort
There is one SNP
A Study in US Cystic Fibrosis Patients With the R117H-CFTR Mutation to Confirm the Long-term Safety and Effectiveness of Kalydeco, Including Patients <18 Years of Age, Combining Data Captured in the Cystic Fibrosis Foundation Registry From an Interventional Cohort and a Non-Interventional Cohort. --- R117H ---
A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric Patients The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation <18 years of age and to describe the long-term safety and effectiveness of Kalydeco in CF patients with the R117H-CFTR mutation overall and in patients ≥18 years. --- R117H ---
A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric Patients The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation <18 years of age and to describe the long-term safety and effectiveness of Kalydeco in CF patients with the R117H-CFTR mutation overall and in patients ≥18 years. --- R117H --- --- R117H ---
A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric Patients The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation <18 years of age and to describe the long-term safety and effectiveness of Kalydeco in CF patients with the R117H-CFTR mutation overall and in patients ≥18 years. --- R117H --- --- R117H --- --- R117H ---
Inclusion Criteria: Non Interventional Cohort - Male or female with confirmed diagnosis of CF - Must have at least 1 allele of the R117H-CFTR mutation - Enrolled in the US CFF Patient Registry - With a record of Kalydeco treatment initiation from 01 January 2015 through 31 December 2016 Historical Cohort - Patients with CF in the CFF Patient Registry as of 01 January 2009 - Must have at least 1 allele of the R117H-CFTR mutation - Patients with no evidence of any prior Kalydeco exposure Inclusion Criteria: Non Interventional Cohort - Male or female with confirmed diagnosis of CF - Must have at least 1 allele of the R117H-CFTR mutation - Enrolled in the US CFF Patient Registry - With a record of Kalydeco treatment initiation from 01 January 2015 through 31 December 2016 Historical Cohort - Patients with CF in the CFF Patient Registry as of 01 January 2009 - Must have at least 1 allele of the R117H-CFTR mutation - Patients with no evidence of any prior Kalydeco exposure Cystic Fibrosis Fibrosis Cystic Fibrosis Patient follow-up (i.e., collection of outcomes data after treatment initiation) in the Non-Interventional Cohort will be at least 36 months. --- R117H ---
Inclusion Criteria: Non Interventional Cohort - Male or female with confirmed diagnosis of CF - Must have at least 1 allele of the R117H-CFTR mutation - Enrolled in the US CFF Patient Registry - With a record of Kalydeco treatment initiation from 01 January 2015 through 31 December 2016 Historical Cohort - Patients with CF in the CFF Patient Registry as of 01 January 2009 - Must have at least 1 allele of the R117H-CFTR mutation - Patients with no evidence of any prior Kalydeco exposure Inclusion Criteria: Non Interventional Cohort - Male or female with confirmed diagnosis of CF - Must have at least 1 allele of the R117H-CFTR mutation - Enrolled in the US CFF Patient Registry - With a record of Kalydeco treatment initiation from 01 January 2015 through 31 December 2016 Historical Cohort - Patients with CF in the CFF Patient Registry as of 01 January 2009 - Must have at least 1 allele of the R117H-CFTR mutation - Patients with no evidence of any prior Kalydeco exposure Cystic Fibrosis Fibrosis Cystic Fibrosis Patient follow-up (i.e., collection of outcomes data after treatment initiation) in the Non-Interventional Cohort will be at least 36 months. --- R117H --- --- R117H ---
Inclusion Criteria: Non Interventional Cohort - Male or female with confirmed diagnosis of CF - Must have at least 1 allele of the R117H-CFTR mutation - Enrolled in the US CFF Patient Registry - With a record of Kalydeco treatment initiation from 01 January 2015 through 31 December 2016 Historical Cohort - Patients with CF in the CFF Patient Registry as of 01 January 2009 - Must have at least 1 allele of the R117H-CFTR mutation - Patients with no evidence of any prior Kalydeco exposure Inclusion Criteria: Non Interventional Cohort - Male or female with confirmed diagnosis of CF - Must have at least 1 allele of the R117H-CFTR mutation - Enrolled in the US CFF Patient Registry - With a record of Kalydeco treatment initiation from 01 January 2015 through 31 December 2016 Historical Cohort - Patients with CF in the CFF Patient Registry as of 01 January 2009 - Must have at least 1 allele of the R117H-CFTR mutation - Patients with no evidence of any prior Kalydeco exposure Cystic Fibrosis Fibrosis Cystic Fibrosis Patient follow-up (i.e., collection of outcomes data after treatment initiation) in the Non-Interventional Cohort will be at least 36 months. --- R117H --- --- R117H --- --- R117H ---
Inclusion Criteria: Non Interventional Cohort - Male or female with confirmed diagnosis of CF - Must have at least 1 allele of the R117H-CFTR mutation - Enrolled in the US CFF Patient Registry - With a record of Kalydeco treatment initiation from 01 January 2015 through 31 December 2016 Historical Cohort - Patients with CF in the CFF Patient Registry as of 01 January 2009 - Must have at least 1 allele of the R117H-CFTR mutation - Patients with no evidence of any prior Kalydeco exposure Inclusion Criteria: Non Interventional Cohort - Male or female with confirmed diagnosis of CF - Must have at least 1 allele of the R117H-CFTR mutation - Enrolled in the US CFF Patient Registry - With a record of Kalydeco treatment initiation from 01 January 2015 through 31 December 2016 Historical Cohort - Patients with CF in the CFF Patient Registry as of 01 January 2009 - Must have at least 1 allele of the R117H-CFTR mutation - Patients with no evidence of any prior Kalydeco exposure Cystic Fibrosis Fibrosis Cystic Fibrosis Patient follow-up (i.e., collection of outcomes data after treatment initiation) in the Non-Interventional Cohort will be at least 36 months. --- R117H --- --- R117H --- --- R117H --- --- R117H ---