{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,10]],"date-time":"2026-08-10T12:34:17Z","timestamp":1786365257900,"version":"build-2736575974"},"reference-count":12,"publisher":"The Pediatric Pharmacy Association","issue":"4","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,8]]},"abstract":"\n OBJECTIVE<\/jats:title>\n The management of cystic fibrosis (CF) requires intensive, chronic medication regimens that can have a high treatment burden and cost. De-escalation of chronic medications for patients with CF aged 12 years and older on elexacaftor-tezacaftor-ivacaftor (ETI) has been evaluated; however, data are lacking for patients aged 6 to 11 years.<\/jats:p>\n <\/jats:sec>\n \n METHODS<\/jats:title>\n \n This single-center, retrospective study evaluated the non-inferiority of chronic medication de-escalation following an institutional algorithm in children 6 to 11 years with CF by comparing the difference in percent predicted FEV\n 1<\/jats:sub>\n (ppFEV\n 1<\/jats:sub>\n ) at 3 to 12 months after ETI initiation. Children with CF who had at least 1 copy of F508del and were started on ETI between January 1, 2021, and December 31, 2022, were included. Secondary outcomes included changes in the number of chronic inhaled CF medications, presence of\n Pseudomonas<\/jats:italic>\n on a sputum culture, CF-related hospitalizations, and body mass index (BMI) z-score.\n <\/jats:p>\n <\/jats:sec>\n \n RESULTS<\/jats:title>\n \n Forty-one patients were included. The sample\u2019s baseline mean ppFEV\n 1<\/jats:sub>\n was 95%. The mean difference in ppFEV\n 1<\/jats:sub>\n between month 3 and month 12 on ETI was \u22121.37% (95% CI, \u22125.33 to 2.6). The lower bound of the 95% CI was less than the pre-defined non-inferiority margin (NIM) of \u22123.0%. Half of the patients had zero chronic inhaled medications by month 12 on ETI. Any\n Psuedomonas<\/jats:italic>\n decreased from 27.5% to 7.5% (p = 0.037) 1 year post-ETI initiation.\n <\/jats:p>\n <\/jats:sec>\n \n CONCLUSIONS<\/jats:title>\n Following the initiation of an institutional medication de-escalation algorithm, the number of chronic medications in children aged 6 to 11 years with CF on ETI was decreased. However, this study was unable to confirm the non-inferiority of chronic medication de-escalation in this younger population with CF.<\/jats:p>\n <\/jats:sec>","DOI":"10.5863\/jppt-25-00051","type":"journal-article","created":{"date-parts":[[2026,5,17]],"date-time":"2026-05-17T01:53:37Z","timestamp":1778982817000},"page":"501-508","source":"Crossref","is-referenced-by-count":1,"title":["De-escalation of Medications After Initiation of Elexacaftor-Tezacaftor-Ivacaftor in Children 6 to 11 Years of Age With Cystic Fibrosis"],"prefix":"10.5863","volume":"31","author":[{"given":"Eva M.","family":"Byerley","sequence":"first","affiliation":[{"name":"Department of Pharmacy (EMB, KJN, EMS) Nationwide Children\u2019s Hospital, Columbus, OH"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karen S.","family":"McCoy","sequence":"additional","affiliation":[{"name":"Section of Pulmonary Medicine (KSM, TJ), Nationwide Children\u2019s Hospital, Columbus, OH"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariah","family":"Eisner","sequence":"additional","affiliation":[{"name":"Biostatistics Resource at Nationwide Children\u2019s Hospital (ME), Nationwide Children\u2019s Hospital, Columbus, OH"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Terri","family":"Johnson","sequence":"additional","affiliation":[{"name":"Section of Pulmonary Medicine (KSM, TJ), Nationwide Children\u2019s Hospital, Columbus, OH"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kimberly J.","family":"Novak","sequence":"additional","affiliation":[{"name":"Department of Pharmacy (EMB, KJN, EMS) Nationwide Children\u2019s Hospital, Columbus, OH"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emily M.","family":"Stephan","sequence":"additional","affiliation":[{"name":"Department of Pharmacy (EMB, KJN, EMS) Nationwide Children\u2019s Hospital, Columbus, OH"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"3883","reference":[{"issue":"10059","key":"JPPT-25-00051-b01","series-title":"Lancet","first-page":"2519","article-title":"Cystic fibrosis","volume":"388","author":"Elborn","year":"2016","unstructured":"Elborn JS. Cystic fibrosis. Lancet. 2016;388(10059):2519\u20132531."},{"issue":"3","key":"JPPT-25-00051-b02","series-title":"J Cyst Fibros","first-page":"499","article-title":"Characterising burden of treatment in cystic fibrosis to identify priority areas for clinical trials","volume":"19","author":"Davies","year":"2020","unstructured":"Davies G, Rowbotham NJ, Smith S, et al. Characterising burden of treatment in cystic fibrosis to identify priority areas for clinical trials. J Cyst Fibros. 2020;19(3):499\u2013502."},{"issue":"12","key":"JPPT-25-00051-b03","series-title":"Pediatr Pulmonol","first-page":"1611","article-title":"Healthcare expenditures for privately insured US patients with cystic fibrosis, 2010-2016","volume":"53","author":"Grosse","year":"2018","unstructured":"Grosse SD, Do TQN, Vu M, et al. Healthcare expenditures for privately insured US patients with cystic fibrosis, 2010-2016. Pediatr Pulmonol. 2018;53(12):1611\u20131618."},{"issue":"12","key":"JPPT-25-00051-b04","series-title":"Am J Respir Crit Care Med","first-page":"1522","article-title":"A phase 3 open-label study of elexacaftor\/tezacaftor\/ivacaftor in children 6 through 11 years of age with cystic fibrosis and at least one F508del allele","volume":"203","author":"Zemanick","year":"2021","unstructured":"Zemanick ET, Taylor-Cousar JL, Davies J, et al. A phase 3 open-label study of elexacaftor\/tezacaftor\/ivacaftor in children 6 through 11 years of age with cystic fibrosis and at least one F508del allele. Am J Respir Crit Care Med. 2021;203(12):1522\u20131532."},{"key":"JPPT-25-00051-b05","article-title":"Vertex announces U.S. FDA approval for TRIKAFTA\u00ae (elexacaftor\/tezacaftor\/ivacaftor and ivacaftor) in children with cystic fibrosis ages 6 through 11 with certain mutations","year":"2021","unstructured":"Vertex Pharmaceutical Incorporated. 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Discontinuation versus continuation of hypertonic saline or dornase alfa in modulator treated people with cystic fibrosis (SIMPLIFY): results from two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials. Lancet Respir Med. 2023;11(4):329\u2013340."},{"issue":"1","key":"JPPT-25-00051-b07","series-title":"J Cyst Fibros","first-page":"32","article-title":"Impact of chronic medication de-escalation in patients with cystic fibrosis taking elexacaftor, tezacaftor, ivacaftor: a retrospective review","volume":"23","author":"Guenther","year":"2024","unstructured":"Guenther EL, McCoy KS, Eisner M, et al. Impact of chronic medication de-escalation in patients with cystic fibrosis taking elexacaftor, tezacaftor, ivacaftor: a retrospective review. J Cyst Fibros. 2024;23(1):32\u201337."},{"issue":"2","key":"JPPT-25-00051-b08","series-title":"J Cyst Fibros","first-page":"S98","article-title":"P113 Home reported outcomes (HERO-2) in people with cystic fibrosis taking elexacaftor\/tezacaftor\/ivacaftor: self-reported changes in use of chronic daily therapies at enrollment","volume":"22","author":"Ren","year":"2023","unstructured":"Ren C, Psoter K, Sabadosa K, et al. P113 Home reported outcomes (HERO-2) in people with cystic fibrosis taking elexacaftor\/tezacaftor\/ivacaftor: self-reported changes in use of chronic daily therapies at enrollment. J Cyst Fibros. 2023;22(2):S98."},{"issue":"1","key":"JPPT-25-00051-b09","series-title":"J Manag Care Spec Pharm","first-page":"26","article-title":"The cost of simplifying treatments for cystic fibrosis: Implications of the SIMPLIFY trial","volume":"30","author":"Gold","year":"2024","unstructured":"Gold LS, Hansen RN, Mayer-Hamblett N, et al. The cost of simplifying treatments for cystic fibrosis: Implications of the SIMPLIFY trial. J Manag Care Spec Pharm. 2024;30(1):26\u201333."},{"key":"JPPT-25-00051-b10","series-title":"2023 Cystic Fibrosis Foundation Patient Registry Highlights","year":"2024","unstructured":"Cystic Fibrosis Foundation. 2023 Cystic Fibrosis Foundation Patient Registry Highlights. Bethesda, Maryland; 2024."},{"issue":"14","key":"JPPT-25-00051-b11","series-title":"J Clin Med","article-title":"Pulmonary function tests in the evaluation of early lung disease in cystic fibrosis","volume":"12","author":"Walicka-Serzysko","year":"2023","unstructured":"Walicka-Serzysko K, Postek M, Borawska-Kowalczyk U, et al. Pulmonary function tests in the evaluation of early lung disease in cystic fibrosis. J Clin Med. 2023;12(14)."},{"key":"JPPT-25-00051-b12","series-title":"Lancet Reg Health Southeast Asia","first-page":"100434","article-title":"CFTR mutations and phenotypic correlations in people with cystic fibrosis: a retrospective study from a single centre in south India","volume":"27","author":"Varkki","year":"2024","unstructured":"Varkki SD, Aaron R, Chapla A, et al. CFTR mutations and phenotypic correlations in people with cystic fibrosis: a retrospective study from a single centre in south India. Lancet Reg Health Southeast Asia. 2024;27:100434."}],"container-title":["The Journal of Pediatric Pharmacology and Therapeutics"],"original-title":[],"link":[{"URL":"https:\/\/jppt.kglmeridian.com\/view\/journals\/jppt\/31\/4\/article-p501.xml","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/jppt.kglmeridian.com\/downloadpdf\/journals\/jppt\/31\/4\/article-p501.xml","content-type":"text\/html","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/jppt.kglmeridian.com\/downloadpdf\/journals\/jppt\/31\/4\/article-p501.xml","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,10]],"date-time":"2026-08-10T12:26:13Z","timestamp":1786364773000},"score":1,"resource":{"primary":{"URL":"https:\/\/jppt.kglmeridian.com\/view\/journals\/jppt\/31\/4\/article-p501.xml"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,8]]},"references-count":12,"journal-issue":{"issue":"4"},"URL":"https:\/\/doi.org\/10.5863\/jppt-25-00051","relation":{},"ISSN":["2331-348X","1551-6776"],"issn-type":[{"value":"2331-348X","type":"electronic"},{"value":"1551-6776","type":"print"}],"subject":[],"published":{"date-parts":[[2026,8]]}}}