Pediatric Year in Review

PEDIATRIC YEAR IN REVIEW 4 UPDATE IN CYSTIC FIBROSIS MANAGEMENT Bessonova L, Volkova N, Higgins M, Bengtsson L, Tian S, Simard C, Konstan MW, Sawicki GS, Sewall A, Nyangoma S, Elbert A, Marshall BC, Bilton D. Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor. Thorax. 2018 Aug;73(8):731-740. PMID: 29748252 Fay JF, Aleksandrov LA, Jensen TJ, Cui LL, Kousouros JN, He L, Aleksandrov AA, Gingerich DS, Riordan JR, Chen JZ. Cryo-EM Visualization of an Active High Open Probability CFTR Anion Channel. Biochemistry. 2018 Oct 30;57(43):6234-6246 PMID: 30281975 Crawford KJ, Downey DG. Theratyping in cystic fibrosis. Curr Opin Pulm Med. 2018 Nov;24(6):612-617. PMID: 30124524 Hart NJ, Aramandla R, Poffenberger G, Fayolle C, Thames AH, Bautista A, Spigelman AF, Babon JAB, DeNicola ME, Dadi PK, Bush WS, Balamurugan AN, Brissova M, Dai C, Prasad N, Bottino R, Jacobson DA, Drumm ML, Kent SC, MacDonald PE, Powers AC. Cystic fibrosis-related diabetes is caused by islet loss and inflammation. JCI Insight. 2018 Apr 19;3(8). PMID: 29669939 Plasschaert LW, Žilionis R, Choo-Wing R, Savova V, Knehr J, Roma G, Klein AM, Jaffe AB. A single-cell atlas of the airway epithelium reveals the CFTR-rich pulmonary ionocyte. Nature. 2018 Aug;560(7718):377-381. PMID: 30069046 Zhang Z, Liu F, Chen J. Molecular structure of the ATP- bound, phosphorylated human CFTR. Proc Natl Acad Sci U S A. 2018 Dec 11;115(50):12757-12762. PMID: 30459277 Rho J, Ahn C, Gao A, Sawicki GS, Keller A, Jain R. Disparities in Mortality of Hispanic Patients with Cystic Fibrosis in the United States. A National and Regional Cohort Study. Am J Respir Crit Care Med. 2018 Oct 15;198(8):1055-1063. PMID:29742360 Moran A, Pillay K, Becker D, Granados A, Hameed S, Acerini CL. ISPAD Clinical Practice Consensus Guidelines 2018: Management of cystic fibrosis-related diabetes in children and adolescents. Pediatr Diabetes. 2018 Oct;19 Suppl 27:64-74. PMID: 30094886