{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,4,3]],"date-time":"2025-04-03T15:24:30Z","timestamp":1743693870153,"version":"3.37.3"},"reference-count":30,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2023,9,20]],"date-time":"2023-09-20T00:00:00Z","timestamp":1695168000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Pulmonology"],"published-print":{"date-parts":[[2023,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:label\/><jats:p>Safety and efficacy data surrounding cystic fibrosis\u00a0transmembrane regulator\u00a0(CFTR) modulator administration for people with CF (pwCF) and severe lung disease elect has remained unclear as a result of exclusion from key trials.\u00a0A scoping review of English language articles from the period of 1 January\u00a02012, to 31 July\u00a02023 was conducted utilizing PubMed and EmBase databases with the following terms: \u201csevere lung disease\u201d OR \u201cadvanced lung disease\u201d AND \u201civacaftor OR lumacaftor OR tezacaftor OR elexacaftor\u201d; \u201ccystic fibrosis transmembrane conductance regulator\u201d AND \u201coff label drug use.\u201d Search results were reviewed by title and abstract for relevance.\u00a0Twenty articles specific to CFTR modulator use in the setting of severe lung disease were included for review, with few specific to pediatric\u2010aged pwCF. PwCF and severe lung disease experienced significant improvement in pulmonary function, body weight, number of IV antibiotic days, and quality of life. A few studies reported a transient decline in pulmonary function among pwCF shortly after LUM\/IVA initiation. However, preemptive reductions in the dose of LUM\/IVA may mitigate this reaction. ELE\/TEZ\/IVA utilization in pwCF and severe lung disease appears to be devoid of the transient decline in pulmonary function observed with LUM\/IVA while providing the same clinical benefit.\u00a0Current available data regarding use of CFTR modulators in pwCF and severe lung disease is reassuring; however, there remains a lack data regarding outcomes among the pediatric population including long\u2010term outcomes. Therefore, treatment decisions should be individualized and coproduced.<\/jats:p><\/jats:sec>","DOI":"10.1002\/ppul.26698","type":"journal-article","created":{"date-parts":[[2023,9,20]],"date-time":"2023-09-20T11:37:24Z","timestamp":1695209844000},"page":"3386-3392","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Use of CFTR modulators in special populations, part 2: Severe lung disease"],"prefix":"10.1002","volume":"58","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3933-473X","authenticated-orcid":false,"given":"Joseph","family":"Elijah","sequence":"first","affiliation":[{"name":"Department of Pharmacy Services Michigan Medicine Ann Arbor Michigan Medicine USA"},{"name":"Department of Clinical Pharmacy College of Pharmacy, University of Michigan Ann Arbor Michigan USA"},{"name":"School of Pharmacy and Pharmaceutical Sciences, Bouve College of Health Sciences Northeastern University Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Linda J.","family":"Fitzgerald","sequence":"additional","affiliation":[{"name":"Department of Pharmacy Services Michigan Medicine Ann Arbor Michigan Medicine USA"},{"name":"Department of Clinical Pharmacy College of Pharmacy, University of Michigan Ann Arbor Michigan USA"},{"name":"Sanofi Medical Affairs Bridgewater New Jersey USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4968-1460","authenticated-orcid":false,"given":"Hanna","family":"Phan","sequence":"additional","affiliation":[{"name":"Department of Pharmacy Services Michigan Medicine Ann Arbor Michigan Medicine USA"},{"name":"Department of Clinical Pharmacy College of Pharmacy, University of Michigan Ann Arbor Michigan USA"},{"name":"Susan B. Meister Child Health Evaluation and Research (CHEAR) Center University of Michigan Ann Arbor Michigan USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2023,9,20]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-0039-1694021"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1186\/1465-9921-4-8"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1105185"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMc1510466"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1709846"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1908639"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1183\/16000617.0112-2019"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2019.03.002"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.7326\/M18-0850"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2012.12.004"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1002\/rcr2.27"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1378\/chest.13-2397"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2015.01.008"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.24424"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.rmed.2020.106073"},{"key":"e_1_2_10_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2017.02.009"},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2017.03.003"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1155\/2018\/8394370"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2017.09.012"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12890-019-0866-y"},{"key":"e_1_2_10_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2019.12.006"},{"key":"e_1_2_10_23_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.25059"},{"key":"e_1_2_10_24_1","doi-asserted-by":"publisher","DOI":"10.1183\/23120541.00203-2020"},{"key":"e_1_2_10_25_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.resmer.2021.100829"},{"key":"e_1_2_10_26_1","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.03079-2020"},{"key":"e_1_2_10_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.rmed.2021.106646"},{"key":"e_1_2_10_28_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202011-4153OC"},{"key":"e_1_2_10_29_1","doi-asserted-by":"publisher","DOI":"10.1002\/prp2.1015"},{"key":"e_1_2_10_30_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.26318"},{"key":"e_1_2_10_31_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2016.11.001"}],"container-title":["Pediatric Pulmonology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ppul.26698","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,11,22]],"date-time":"2023-11-22T19:17:00Z","timestamp":1700680620000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ppul.26698"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,9,20]]},"references-count":30,"journal-issue":{"issue":"12","published-print":{"date-parts":[[2023,12]]}},"alternative-id":["10.1002\/ppul.26698"],"URL":"https:\/\/doi.org\/10.1002\/ppul.26698","archive":["Portico"],"relation":{},"ISSN":["8755-6863","1099-0496"],"issn-type":[{"type":"print","value":"8755-6863"},{"type":"electronic","value":"1099-0496"}],"subject":[],"published":{"date-parts":[[2023,9,20]]},"assertion":[{"value":"2023-08-18","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-09-11","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-09-20","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}