Cystic fibrosis year in review 2021

被引：4

作者：

Savant, Adrienne P. ^{[1
,2
]}

机构：

[1] Childrens Hosp New Orleans, Dept Pediat, New Orleans, LA USA

[2] Tulane Univ, Dept Pediat, New Orleans, LA 70118 USA

来源：

PEDIATRIC PULMONOLOGY | 2022年 / 57卷 / 07期

关键词：

advanced lung disease; CFTR modulators; cystic fibrosis; newborn screening; pulmonary exacerbations; LUNG-TRANSPLANT OUTCOMES; CLINICAL EFFECTIVENESS; PARALLEL-GROUP; DOUBLE-BLIND; ELEXACAFTOR/TEZACAFTOR/IVACAFTOR; IVACAFTOR; PEOPLE; CANADA; COMBINATION; TEZACAFTOR;

D O I：

10.1002/ppul.25948

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

People with cystic fibrosis (CF) have an amazing outlook with the treatment availability of highly effective modulators. Unfortunately, not all people with CF are eligible for modulators leading to continued pulmonary exacerbations and advanced lung disease. Additionally, optimizing diagnosis and evaluation for CF in the newborn period continues to be an area of focus for research. This review article will work to cover articles published in 2021 with high clinical relevance related to the above topics; however, due to the extensive body of research published, this review will not be comprehensive.

引用

页码：1590 / 1599

页数：10

共 84 条

[1] Females with cystic fibrosis have a larger decrease in sweat chloride in response to lumacaf tor/ivacaf tor compared to males [J].

Aalbers, B. L. ;

Hofland, R. W. ;

Bronsveld, I. ;

Groot, K. M. de Winter-de ;

Arets, H. G. M. ;

Kiviet, A. C. de ;

Ven, M. M. M. van Oirschot-van de ;

Kruijswijk, M. A. ;

Schotman, S. ;

Michel, S. ;

Ent, C. K. van der ;

Heijerman, H. G. M. .

JOURNAL OF CYSTIC FIBROSIS, 2021, 20 (01) :E7-E11

[2] Tobacco smoke exposure limits the therapeutic benefit of tezacaf tor/ivacaf tor in pediatric patients with cystic fibrosis [J].

Baker, Elizabeth ;

Harris, William T. ;

Rowe, Steven M. ;

Rutland, Sarah B. ;

Oates, Gabriela R. .

JOURNAL OF CYSTIC FIBROSIS, 2021, 20 (04) :612-617

[3] Updated guidance on the management of children with cystic fibrosis transmembrane conductance regulator-related metabolic syndrome/cystic fibrosis screen positive, inconclusive diagnosis (CRMS/CFSPID) [J].

Barben, Jurg ;

Castellani, Carlo ;

Munck, Anne ;

Davies, Jane C. ;

De Winter-de Groot, Karin M. ;

Gartner, Silvia ;

Kashirskaya, Nataliya ;

Linnane, Barry ;

Mayell, Sarah J. ;

McColley, Susanna ;

Ooi, Chee Y. ;

Proesmans, Marijke ;

Ren, Clement L. ;

Salinas, Danieli ;

Sands, Dorota ;

Sermet-Gaudelus, Isabelle ;

Sommerburg, Olaf ;

Southern, Kevin W. .

JOURNAL OF CYSTIC FIBROSIS, 2021, 20 (05) :810-819

[4] Newborn screening alone insufficient to improve pulmonary outcomes for cystic fibrosis [J].

Barreda, Christina B. ;

Farrell, Philip M. ;

Laxova, Anita ;

Eickhoff, Jens C. ;

Braun, Andrew T. ;

Coller, Ryan J. ;

Rock, Michael J. .

JOURNAL OF CYSTIC FIBROSIS, 2021, 20 (03) :492-498

[5] Triple Therapy for Cystic Fibrosis Phe508del-Gating and -Residual Function Genotypes [J].

Barry, Peter J. ;

Mall, Marcus A. ;

Alvarez, Antonio ;

Colombo, Carla ;

de Winter-de Groot, Karin M. ;

Fajac, Isabelle ;

McBennett, Kimberly A. ;

McKone, Edward F. ;

Ramsey, Bonnie W. ;

Sutharsan, Sivagurunathan ;

Taylor-Cousar, Jennifer L. ;

Tullis, Elizabeth ;

Ahluwalia, Neil ;

Jun, Lucy S. ;

Moskowitz, Samuel M. ;

Prieto-Centurion, Valentin ;

Tian, Simon ;

Waltz, David ;

Xuan, Fengjuan ;

Zhang, Yaohua ;

Rowe, Steven M. ;

Polineni, Deepika .

NEW ENGLAND JOURNAL OF MEDICINE, 2021, 385 (09) :815-825

[6] Outcomes of repeat sweat testing in cystic fibrosis newborn screen positive infants [J].

Bauer, Sarah E. ;

Wesson, Melissa ;

Oles, Sylwia K. ;

Ren, Clement L. .

PEDIATRIC PULMONOLOGY, 2021, 56 (06) :1521-1526

[7] CFTR modulator use in post lung transplant recipients [J].

Benninger, Lauryn A. ;

Trillo, Cesar ;

Lascano, Jorge .

JOURNAL OF HEART AND LUNG TRANSPLANTATION, 2021, 40 (12) :1498-1501

[8] Lumacaftor/ivacaftor in people with cystic fibrosis with an A455E-CFTR mutation [J].

Berkers, Gitte ;

van der Meer, Renske ;

Heijerman, Harry ;

Beekman, Jeffrey M. ;

Boj, Sylvia F. ;

Vries, Robert G. J. ;

van Mourik, Peter ;

Doyle, Jamie R. ;

Audhya, Paul ;

Yuan, Zheng ;

Kinnman, Nils ;

van der Ent, C. Kors .

JOURNAL OF CYSTIC FIBROSIS, 2021, 20 (05) :761-767

[9] Short-term effect of elexacaftor-tezacaftor-ivacaf tor on lung function and transplant planning in cystic fibrosis patients with advanced lung disease [J].

Bermingham, B. ;

Rueschhoff, A. ;

Ratti, G. ;

Nesmith, A. ;

Goodwin, D. ;

Gray, S. ;

Flume, P. ;

Solomon, G. M. ;

Cohen, L. ;

Garcia, B. .

JOURNAL OF CYSTIC FIBROSIS, 2021, 20 (05) :768-771

[10] Long-Term Outcomes in Real Life of Lumacaftor-Ivacaftor Treatment in Adolescents With Cystic Fibrosis [J].

Bui, Stephanie ;

Masson, Alexandra ;

Enaud, Raphael ;

Roditis, Lea ;

Dournes, Gael ;

Galode, Francois ;

Collet, Cyrielle ;

Mas, Emmanuel ;

Languepin, Jeanne ;

Fayon, Michael ;

Beaufils, Fabien ;

Mittaine, Marie .

FRONTIERS IN PEDIATRICS, 2021, 9

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