Elexacaftor/tezacaftor/ivacaftor in children aged 6-11 years with cystic fibrosis, at least one F508DEL allele, and advanced lung disease: A 24-week observational study

Affiliations

¹ Cystic Fibrosis Centre, Hospital San Carlo, Potenza, Italy.
² Lazio Reference Centre for Cystic Fibrosis, Policlinico Umberto I University Hospital, Rome, Italy.
³ Pediatric Pulmonology Unit, Città della Salute e della Scienza, Ospedale Infantile Regina Margherita, Turin, Italy.
⁴ Pediatric Cystic Fibrosis Centre, Azienda Universitaria Ospedaliera Consorziale Policlinico, Bari, Italy.
⁵ Cystic Fibrosis Center, Azienda Ospedaliera Brotzu, Cagliari, Italy.
⁶ Department of Medicine, Surgery and Dentistry, Scuola Medica Salernitana, Postgraduate School of Pediatrics, University of Salerno, Baronissi, Italy.

Observational Study

Donatello Salvatore et al. Pediatr Pulmonol. 2022 Sep.

. 2022 Sep;57(9):2253-2256.

doi: 10.1002/ppul.25980. Epub 2022 May 25.

¹ Cystic Fibrosis Centre, Hospital San Carlo, Potenza, Italy.
² Lazio Reference Centre for Cystic Fibrosis, Policlinico Umberto I University Hospital, Rome, Italy.
³ Pediatric Pulmonology Unit, Città della Salute e della Scienza, Ospedale Infantile Regina Margherita, Turin, Italy.
⁴ Pediatric Cystic Fibrosis Centre, Azienda Universitaria Ospedaliera Consorziale Policlinico, Bari, Italy.
⁵ Cystic Fibrosis Center, Azienda Ospedaliera Brotzu, Cagliari, Italy.
⁶ Department of Medicine, Surgery and Dentistry, Scuola Medica Salernitana, Postgraduate School of Pediatrics, University of Salerno, Baronissi, Italy.

No abstract available

References

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1. Heijerman HGM, McKone EF, Downey DG, et al. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial. Lancet. 2019;394:1940-1948. Epub October 31, 2019. Erratum in: Lancet. 2020;395(10238):1694. doi:10.1016/S0140-6736(19)32597-8
1. Bresnick K, Arteaga-Solis E, Millar SJ, Laird G, LeCamus C. Burden of cystic fibrosis in children <12 years of age prior to the introduction of CFTR modulator therapies. BMJ Open Respir Res. 2021;8(1):e000998. doi:10.1136/bmjresp-2021-000998
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