Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data
Abstract Clinical trials demonstrate the short-term efficacy of dual CFTR modulators, but long-term real-world data is limited. We aimed to investigate the effects of 24-month lumacaftor/ivacaftor (LUM/IVA) therapy in pediatric CF patients (pwCF). This observational study included pwCF homozygous fo...
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Nature Portfolio
2025-01-01
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| Online Access: | https://doi.org/10.1038/s41598-025-86010-1 |
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| author | Marcell Imrei Adrienn F. Kéri Éva Gács Ildikó Gönczi Melinda Meláth Éva Kosaras Botond Demeter Csaba Péterfia Klára Vass Gyöngyi Székely Klementina Ocskay Andrea Párniczky |
| author_facet | Marcell Imrei Adrienn F. Kéri Éva Gács Ildikó Gönczi Melinda Meláth Éva Kosaras Botond Demeter Csaba Péterfia Klára Vass Gyöngyi Székely Klementina Ocskay Andrea Párniczky |
| author_sort | Marcell Imrei |
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| description | Abstract Clinical trials demonstrate the short-term efficacy of dual CFTR modulators, but long-term real-world data is limited. We aimed to investigate the effects of 24-month lumacaftor/ivacaftor (LUM/IVA) therapy in pediatric CF patients (pwCF). This observational study included pwCF homozygous for F508del mutation treated between 2021 and 2023. We report data for the first 24 months from therapy initiation. Variables were analyzed separately for ages 2–5, 6–11, and over 12. Data from 49 pwCF (median age: 9.3 years (5.5–14.2)) showed that ppFEV1 values after a transient increase at 12 months, decreased from 102% (82–114) at baseline to 87% (74–96) at 24 months. The decrease was more pronounced with higher initial ppFEV1. Median sweat chloride concentration decreased from 75 mmol/L (69–82) to 57 mmol/L (43–70) without any association with respiratory function change. Median BMI z-score increased from − 0.81 (− 1.37–0.49) to − 0.39 (− 0.88 to − 0.04) (p = 0.288), and the proportion of underweight and overweight children decreased. Skeletal muscle mass remained stable, while fat mass significantly increased (p = 0.011). Fecal elastase levels improved, especially among younger patients. These findings underscore the potential benefits of early initiation of CFTR modulator therapy in pediatric CF patients, highlighting improvements in nutritional status and pancreatic function. |
| format | Article |
| id | doaj-art-467540729e2e4076b7b2a4e4b9ef8141 |
| institution | Kabale University |
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| language | English |
| publishDate | 2025-01-01 |
| publisher | Nature Portfolio |
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| series | Scientific Reports |
| spelling | doaj-art-467540729e2e4076b7b2a4e4b9ef81412025-01-19T12:20:51ZengNature PortfolioScientific Reports2045-23222025-01-011511910.1038/s41598-025-86010-1Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world dataMarcell Imrei0Adrienn F. Kéri1Éva Gács2Ildikó Gönczi3Melinda Meláth4Éva Kosaras5Botond Demeter6Csaba Péterfia7Klára Vass8Gyöngyi Székely9Klementina Ocskay10Andrea Párniczky11Heim Pál National Pediatric InstituteHeim Pál National Pediatric InstituteHeim Pál National Pediatric InstituteHeim Pál National Pediatric InstituteHeim Pál National Pediatric InstituteVelkey László Child’s Health Center, Borsod-Abaúj-Zemplén County Central Hospital and University Teaching HospitalVelkey László Child’s Health Center, Borsod-Abaúj-Zemplén County Central Hospital and University Teaching HospitalDepartment of Pediatrics, Medical School, University of PécsJósa András Hospital, Szabolcs-Szatmár-Bereg County Hospitals and University Teaching HospitalPediatric Rehabilitation Unit, Kaposi Mór Teaching HospitalHeim Pál National Pediatric InstituteHeim Pál National Pediatric InstituteAbstract Clinical trials demonstrate the short-term efficacy of dual CFTR modulators, but long-term real-world data is limited. We aimed to investigate the effects of 24-month lumacaftor/ivacaftor (LUM/IVA) therapy in pediatric CF patients (pwCF). This observational study included pwCF homozygous for F508del mutation treated between 2021 and 2023. We report data for the first 24 months from therapy initiation. Variables were analyzed separately for ages 2–5, 6–11, and over 12. Data from 49 pwCF (median age: 9.3 years (5.5–14.2)) showed that ppFEV1 values after a transient increase at 12 months, decreased from 102% (82–114) at baseline to 87% (74–96) at 24 months. The decrease was more pronounced with higher initial ppFEV1. Median sweat chloride concentration decreased from 75 mmol/L (69–82) to 57 mmol/L (43–70) without any association with respiratory function change. Median BMI z-score increased from − 0.81 (− 1.37–0.49) to − 0.39 (− 0.88 to − 0.04) (p = 0.288), and the proportion of underweight and overweight children decreased. Skeletal muscle mass remained stable, while fat mass significantly increased (p = 0.011). Fecal elastase levels improved, especially among younger patients. These findings underscore the potential benefits of early initiation of CFTR modulator therapy in pediatric CF patients, highlighting improvements in nutritional status and pancreatic function.https://doi.org/10.1038/s41598-025-86010-1Normal-weight obesityFecal elastasePancreatic functionBody compositionCFTR modulator therapyLUM/IVA |
| spellingShingle | Marcell Imrei Adrienn F. Kéri Éva Gács Ildikó Gönczi Melinda Meláth Éva Kosaras Botond Demeter Csaba Péterfia Klára Vass Gyöngyi Székely Klementina Ocskay Andrea Párniczky Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data Scientific Reports Normal-weight obesity Fecal elastase Pancreatic function Body composition CFTR modulator therapy LUM/IVA |
| title | Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data |
| title_full | Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data |
| title_fullStr | Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data |
| title_full_unstemmed | Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data |
| title_short | Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data |
| title_sort | body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real world data |
| topic | Normal-weight obesity Fecal elastase Pancreatic function Body composition CFTR modulator therapy LUM/IVA |
| url | https://doi.org/10.1038/s41598-025-86010-1 |
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