Cystic Fibrosis: A Disease in Transformation, yet More Work to Be Done!

Bonnie W Ramsey^{1

2}, Scott C Bell^{3

4

5}

Affiliations

¹ Department of Pediatrics University of Washington School of Medicine Seattle, Washington.
² Seattle Children's Research Institute Seattle, Washington.
³ Adult Cystic Fibrosis Centre The Prince Charles Hospital Brisbane, Australia.
⁴ Children's Health Research Centre The University of Queensland Brisbane, Australia.
⁵ Translational Research Institute Brisbane, Australia.

Editorial

Bonnie W Ramsey et al. Am J Respir Crit Care Med. 2022.

. 2022 Mar 1;205(5):487-489.

doi: 10.1164/rccm.202112-2782ED.

Bonnie W Ramsey^{1

2}, Scott C Bell^{3

4

5}

¹ Department of Pediatrics University of Washington School of Medicine Seattle, Washington.
² Seattle Children's Research Institute Seattle, Washington.
³ Adult Cystic Fibrosis Centre The Prince Charles Hospital Brisbane, Australia.
⁴ Children's Health Research Centre The University of Queensland Brisbane, Australia.
⁵ Translational Research Institute Brisbane, Australia.

No abstract available

Comment on

Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial.
Nichols DP, Paynter AC, Heltshe SL, Donaldson SH, Frederick CA, Freedman SD, Gelfond D, Hoffman LR, Kelly A, Narkewicz MR, Pittman JE, Ratjen F, Rosenfeld M, Sagel SD, Schwarzenberg SJ, Singh PK, Solomon GM, Stalvey MS, Clancy JP, Kirby S, Van Dalfsen JM, Kloster MH, Rowe SM; PROMISE Study group. Nichols DP, et al. Am J Respir Crit Care Med. 2022 Mar 1;205(5):529-539. doi: 10.1164/rccm.202108-1986OC. Am J Respir Crit Care Med. 2022. PMID: 34784492 Free PMC article.
Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on CFTR Function in Patients with Cystic Fibrosis and One or Two F508del Alleles.
Graeber SY, Vitzthum C, Pallenberg ST, Naehrlich L, Stahl M, Rohrbach A, Drescher M, Minso R, Ringshausen FC, Rueckes-Nilges C, Klajda J, Berges J, Yu Y, Scheuermann H, Hirtz S, Sommerburg O, Dittrich AM, Tümmler B, Mall MA. Graeber SY, et al. Am J Respir Crit Care Med. 2022 Mar 1;205(5):540-549. doi: 10.1164/rccm.202110-2249OC. Am J Respir Crit Care Med. 2022. PMID: 34936849

1. Middleton PG, Mall MA, Dřevínek P, Lands LC, McKone EF, Polineni D, et al. VX17-445-102 Study Group Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med . 2019;381:1809–1819. - PMC - PubMed
1. Heijerman HGM, McKone EF, Downey DG, Van Braeckel E, Rowe SM, Tullis E, et al. VX17-445-103 Trial Group 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. - PMC - PubMed
1. Nichols DP, Paynter AC, Heltshe SL, Donaldson SH, Frederick CA, Freedman SD, et al. PROMISE Study Group Clinical effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: a clinical trial Am J Respir Crit Care Med 2022205529–539 - PMC - PubMed
1. Graeber SY, Vitzthum C, Pallenberg ST, Naehrlich L, Stahl M, Rohrbach A, et al. Effects of elexacaftor/tezacaftor/ivacaftor therapy on CFTR function in patients with cystic fibrosis and one or two F508del alleles Am J Respir Crit Care Med 2022205540–549 - PubMed
1. Nichols DP, Donaldson SH, Frederick CA, Freedman SD, Gelfond D, Hoffman LR, et al. PROMISE: working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy. J Cyst Fibros . 2021;20:205–212. - PMC - PubMed