| Title: |
Effectiveness of lumacaftor/ivacaftor initiation in children with cystic fibrosis aged 2 through 5 years on disease progression: Interim results from an ongoing registry-based study |
| Authors: |
C. Kim; M. Higgins; L. Liu; N. Volkova; A. Zolin; L. Naehrlich; A. Pfleger; E. Lammertijn; D. Tješić-Drinković; P. Dřevínek; M. Jr. Macek; H. Vebert Olesen; N. Senecal; P. -R. Burgel; G. Fletcher; R. Padoan; A. Orenti; F. Ambrogi; S. Gambazza; E. Aleksejeva; A. -M. Charatsi; D. Zomer; E. Bakkeheim; L. Woźniacki; U. Krivec; C. Krantz; A. Lindblad; A. Jung; S. Clarke; S. B. Carr |
| Contributors: |
C. Kim; M. Higgin; L. Liu; N. Volkova; A. Zolin; L. Naehrlich; A. Pfleger; E. Lammertijn; D. Tješić-Drinković; P. Dřevínek; M.J. Macek; H. Vebert Olesen; N. Senecal; P.-. Burgel; G. Fletcher; R. Padoan; A. Orenti; F. Ambrogi; S. Gambazza; E. Aleksejeva; A.-. Charatsi; D. Zomer; E. Bakkeheim; L. Woźniacki; U. Krivec; C. Krantz; A. Lindblad; A. Jung; S. Clarke; S.B. Carr |
| Publisher Information: |
Elsevier |
| Publication Year: |
2024 |
| Collection: |
The University of Milan: Archivio Istituzionale della Ricerca (AIR) |
| Subject Terms: |
CFTR modulator; CFTR therapie; Children; Cystic fibrosi; Ivacaftor; Lumacaftor; Settore MED/01 - Statistica Medica |
| Description: |
Background: Lumacaftor/ivacaftor (LUM/IVA) has been shown to be safe and efficacious in people with cystic fibrosis (CF) ≥1 year of age. To assess the impact of early LUM/IVA initiation on CF disease progression, a 6-year observational study leveraging data from existing CF patient registries is being conducted in children with CF homozygous for F508del (F/F genotype) who were aged 2 through 5 years at treatment initiation. Here we present interim results from this study focusing on data from the European CF Society Patient Registry (ECFSPR). Methods: The LUM/IVA cohort included children in the ECFSPR who started LUM/IVA between 15 January 2019 and 31 December 2020. Longitudinal trends in growth parameters, pulmonary exacerbations, hospitalizations, safety outcomes, and other effectiveness outcomes in the LUM/IVA cohort were compared to those in two modulator-naïve cohorts: (i) matched concurrent cohort heterozygous for F508del and a minimal function mutation (F/MF concurrent comparator cohort) and (ii) matched concurrent cohort with the F/F genotype from countries without commercial access to LUM/IVA as of 2020 (F/F concurrent comparator cohort). Results: The LUM/IVA cohort matched to the F/MF concurrent comparator cohort had 681 children and the LUM/IVA cohort matched to the F/F concurrent comparator cohort had 183 children. LUM/IVA cohorts had increases in body mass index percentiles relative to the matched F/MF and F/F concurrent comparator cohorts (mean difference in change from baseline: 8.4 [95% CI: 5.5, 11.3] and 11.8 [95% CI: 5.9, 17.7], respectively). Increases in height and weight percentiles were also observed in the LUM/IVA cohort relative to the F/MF and F/F concurrent comparator cohorts. Reductions in pulmonary exacerbations and hospitalizations relative to baseline and the F/F concurrent comparator cohort were seen in 2021. Conclusions: This interim analysis showed favorable trends in clinical outcomes, including growth parameters, pulmonary exacerbations, and hospitalizations, suggesting an ... |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| Relation: |
info:eu-repo/semantics/altIdentifier/pmid/38402082; info:eu-repo/semantics/altIdentifier/wos/WOS:001252643500001; firstpage:1; lastpage:7; numberofpages:7; journal:JOURNAL OF CYSTIC FIBROSIS; https://hdl.handle.net/2434/1033168 |
| DOI: |
10.1016/j.jcf.2024.02.004 |
| Availability: |
https://hdl.handle.net/2434/1033168; https://doi.org/10.1016/j.jcf.2024.02.004 |
| Rights: |
info:eu-repo/semantics/openAccess |
| Accession Number: |
edsbas.75CCB9A |
| Database: |
BASE |