TY - JOUR
T1 - VOCAL
T2 - An observational study of ivacaftor for people with cystic fibrosis and selected non–G551D-CFTR gating mutations
AU - Simmonds, Nicholas J.
AU - van der Ent, C. Kors
AU - Colombo, Carla
AU - Kinnman, Nils
AU - DeSouza, Cynthia
AU - Thorat, Teja
AU - Chew, Marci L.
AU - Chandarana, Keval
AU - Castellani, Carlo
N1 - Funding Information:
The authors thank all the people with cystic fibrosis who took part in the study and their families, as well as the principal investigators and research coordinators at each site. The authors thank all the site investigators and clinical research associates who collected data at study sites. The authors acknowledge the study investigators who were not included as authors: Antonio Manca, Donatello Salvatore, Harm Tiddens, Giovanna Pizzamiglio, Joanna Whitehouse, Edward F. Nash, Renske van der Meer, Hester van der Vaart, Petrus Merkus, Josje Altenburg, Christof Majoor, Giulia Paiola, Elena Spinelli, Nicola Ferrara, and Simona Cristadoro. The authors acknowledge Marianna Passiu, Ilaria Meneghelli, and Arianna Bisogno for their contributions to the study. Editorial coordination and support were provided by Francesca Francois, PharmD, MPH, of Vertex Pharmaceuticals Incorporated; Francesca Francois may own stock or stock options in that company. Medical writing and editorial support were provided under the direction of the authors by Christopher Edwards, PhD, CMPP, an employee of ArticulateScience, LLC, which received funding from Vertex Pharmaceuticals Incorporated.
Publisher Copyright:
© 2022 The Authors
PY - 2023/1
Y1 - 2023/1
N2 - Background: VOCAL was an observational study of the effect of long-term ivacaftor on real-world clinical outcomes and healthcare resource utilization (HCRU) in people with cystic fibrosis (pwCF) in Italy, the Netherlands, and the UK. Methods: pwCF aged ≥6 years with non–G551D-CFTR gating mutations were eligible. Prospective data were collected up to 48 months after enrollment; retrospective data were collected to ensure that 12 months of pre-ivacaftor data were available. Endpoints included absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) and measures of nutritional status. Pulmonary exacerbation (PEx) rates, HCRU, and respiratory microbiology during ivacaftor treatment were compared with data from the 12-month period before initiation. Results: Seventy-three eligible pwCF were enrolled and received ivacaftor; 65 (89.0%) completed the study (48 [65.8%] completed ≥48 months of ivacaftor). During the first 6 months of ivacaftor, ppFEV1, body mass index (BMI), and BMI-for-age z-score showed least-squares mean absolute improvements of 10.8 percentage points, 0.79 kg/m2, and 0.54, respectively; improvements were maintained through 48 months. Rates of PEx, antibiotic use due to PEx, and hospitalization decreased by >50% during ivacaftor treatment compared with before ivacaftor. The number of respiratory cultures and sputum was lower post-ivacaftor, as was the percentage of pwCF with positive respiratory cultures for 3 of 9 pathogens evaluated (Pseudomonas aeruginosa, Aspergillus fumigatus, Stenotrophomonas maltophilia). Reported safety results were consistent with CF and ivacaftor's known safety profile. Conclusions: These results demonstrate the positive long-term effectiveness of ivacaftor on clinical outcomes and HCRU in pwCF with non–G551D-CFTR gating mutations in real-world settings.
AB - Background: VOCAL was an observational study of the effect of long-term ivacaftor on real-world clinical outcomes and healthcare resource utilization (HCRU) in people with cystic fibrosis (pwCF) in Italy, the Netherlands, and the UK. Methods: pwCF aged ≥6 years with non–G551D-CFTR gating mutations were eligible. Prospective data were collected up to 48 months after enrollment; retrospective data were collected to ensure that 12 months of pre-ivacaftor data were available. Endpoints included absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) and measures of nutritional status. Pulmonary exacerbation (PEx) rates, HCRU, and respiratory microbiology during ivacaftor treatment were compared with data from the 12-month period before initiation. Results: Seventy-three eligible pwCF were enrolled and received ivacaftor; 65 (89.0%) completed the study (48 [65.8%] completed ≥48 months of ivacaftor). During the first 6 months of ivacaftor, ppFEV1, body mass index (BMI), and BMI-for-age z-score showed least-squares mean absolute improvements of 10.8 percentage points, 0.79 kg/m2, and 0.54, respectively; improvements were maintained through 48 months. Rates of PEx, antibiotic use due to PEx, and hospitalization decreased by >50% during ivacaftor treatment compared with before ivacaftor. The number of respiratory cultures and sputum was lower post-ivacaftor, as was the percentage of pwCF with positive respiratory cultures for 3 of 9 pathogens evaluated (Pseudomonas aeruginosa, Aspergillus fumigatus, Stenotrophomonas maltophilia). Reported safety results were consistent with CF and ivacaftor's known safety profile. Conclusions: These results demonstrate the positive long-term effectiveness of ivacaftor on clinical outcomes and HCRU in pwCF with non–G551D-CFTR gating mutations in real-world settings.
KW - CFTR gating mutation
KW - Cystic fibrosis
KW - Healthcare resource utilization
KW - Ivacaftor
KW - Lung function
KW - Real-world experience
UR - http://www.scopus.com/inward/record.url?scp=85130430556&partnerID=8YFLogxK
U2 - 10.1016/j.jcf.2022.05.007
DO - 10.1016/j.jcf.2022.05.007
M3 - Article
C2 - 35613999
AN - SCOPUS:85130430556
SN - 1569-1993
VL - 22
SP - 124
EP - 131
JO - Journal of Cystic Fibrosis
JF - Journal of Cystic Fibrosis
IS - 1
ER -