{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,19]],"date-time":"2026-10-19T17:47:13Z","timestamp":1792432033330,"version":"4.5.0"},"reference-count":10,"publisher":"Georg Thieme Verlag KG","issue":"02","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Klin Padiatr"],"published-print":{"date-parts":[[2025,3]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Cystic fibrosis (CF) is a genetic disease that results from mutations in the CFTR\n                    gene. It primarily affects the lungs and digestive system. Recent advancements\n                    in the treatment of CF have been driven by highly effective therapies that\n                    modulate the function of the cystic fibrosis transmembrane conductance regulator\n                    (CFTR) protein, which target the underlying molecular defects in CFTR function.\n                    These modulators have been demonstrated to significantly improve lung function,\n                    weight gain, and quality of life for 90% of individuals with CF, particularly\n                    those with the F508del mutation. HEMT has also demonstrated potential benefits\n                    for pancreatic and liver function, and its early use in young children may delay\n                    or prevent disease progression. However, challenges remain in optimizing\n                    biomarkers and outcome measures for younger children, addressing side effects,\n                    and developing novel therapies for mutations not responsive to current\n                    treatments. This review focuses on the efficacy, safety, and future perspectives\n                    of HEMT in children under 12 years of age, emphasizing the importance of early\n                    intervention to improve long-term outcomes in CF patients.<\/jats:p>","DOI":"10.1055\/a-2449-3360","type":"journal-article","created":{"date-parts":[[2024,12,10]],"date-time":"2024-12-10T19:01:16Z","timestamp":1733857276000},"page":"66-72","source":"Crossref","is-referenced-by-count":1,"title":["CFTR Modulator Treatment in Children&lt;12 Years of Age \u2013 Status Quo\n                    and Challenges"],"prefix":"10.1055","volume":"237","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7232-7828","authenticated-orcid":false,"given":"Dominik","family":"Funken","sequence":"additional","affiliation":[{"name":"Department of Pediatric Pneumology, Allergology and Neonatology,\n                    Hannover Medical School Centre for Paediatrics and Adolescent Medicine,\n                    Hannover, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2680-3541","authenticated-orcid":false,"given":"Katharina","family":"Sch\u00fctz","sequence":"additional","affiliation":[{"name":"Department of Pediatric Pneumology, Allergology and Neonatology,\n                    Hannover Medical School Centre for Paediatrics and Adolescent Medicine,\n                    Hannover, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna-Maria","family":"Dittrich","sequence":"additional","affiliation":[{"name":"Department of Pediatric Pneumology, Allergology and Neonatology,\n                    Hannover Medical School Centre for Paediatrics and Adolescent Medicine,\n                    Hannover, Germany"},{"name":"German Center for Lung Research, BREATH location, Hannover Medical\n                    School, Hannover, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"194","published-online":{"date-parts":[[2024,12,10]]},"reference":[{"key":"ref1","doi-asserted-by":"crossref","first-page":"1252","DOI":"10.1164\/rccm.201507-1271LE","article-title":"Intestinal Current Measurements Detect Activation of Mutant CFTR in Patients\n                    with Cystic Fibrosis with the G551D Mutation Treated with Ivacaftor","volume":"192","author":"S Y Graeber","year":"2015","journal-title":"Am J Respir Crit Care Med"},{"key":"ref2","doi-asserted-by":"crossref","first-page":"540","DOI":"10.1164\/rccm.202110-2249OC","article-title":"Effects of Elexacaftor\/Tezacaftor\/Ivacaftor Therapy on CFTR Function in Patients\n                    with Cystic Fibrosis and One or Two F508del Alleles","volume":"205","author":"S Y Graeber","year":"2022","journal-title":"Am J Respir Crit Care Med"},{"key":"ref3","doi-asserted-by":"crossref","first-page":"1433","DOI":"10.1164\/rccm.201710-1983OC","article-title":"Effects of Lumacaftor-Ivacaftor Therapy on Cystic Fibrosis\n                    Transmembrane Conductance Regulator Function in Phe508del Homozygous Patients\n                    with Cystic Fibrosis","volume":"197","author":"S Y Graeber","year":"2018","journal-title":"Am J Respir Crit Care\n                    Med"},{"key":"ref5","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1016\/j.rmed.2017.08.026","article-title":"Ivacaftor for the p.Ser549Arg (S549R) gating mutation \u2013 The\n                    Israeli experience","volume":"131","author":"A Dagan","year":"2017","journal-title":"Respir Med"},{"key":"ref12","doi-asserted-by":"crossref","first-page":"193","DOI":"10.1016\/j.jcf.2022.08.008","article-title":"Cystic fibrosis transmembrane conductance regulator modulators and the exocrine\n                    pancreas: A scoping review","volume":"22","author":"M L Ramsey","year":"2023","journal-title":"Journal of Cystic Fibrosis"},{"key":"ref16","doi-asserted-by":"crossref","first-page":"e167957","DOI":"10.1172\/JCI167957","article-title":"Pharmacologic improvement of CFTR function rapidly decreases\n                    sputum pathogen density, but lung infections generally\n                    persist","volume":"133","author":"D P Nichols","year":"2023","journal-title":"J Clin Invest"},{"key":"ref35","doi-asserted-by":"crossref","first-page":"3381","DOI":"10.1002\/ppul.25065","article-title":"Persistent recovery of pancreatic function in patients with\n                    cystic fibrosis after ivacaftor","volume":"55","author":"D Munce","year":"2020","journal-title":"Pediatr\n                    Pulmonol"},{"key":"ref45","doi-asserted-by":"crossref","first-page":"721","DOI":"10.1016\/j.jcf.2022.04.005","article-title":"A case report of CFTR modulator administration via carrier mother to treat\n                    meconium ileus in a F508del homozygous fetus","volume":"21","author":"S Szentpetery","year":"2022","journal-title":"Journal of Cystic Fibrosis"},{"key":"ref49","doi-asserted-by":"crossref","first-page":"1265138","DOI":"10.3389\/fphar.2023.1265138","article-title":"Ocular development after highly effective modulator treatment\n                    early in life","volume":"14","author":"Y Zhu","year":"2023","journal-title":"Front Pharmacol"},{"key":"ref53","doi-asserted-by":"crossref","first-page":"1389586","DOI":"10.3389\/fphar.2024.1389586","article-title":"Readthrough-induced misincorporated amino acid ratios guide\n                    mutant-specific therapeutic approaches for two CFTR nonsense\n                    mutations","volume":"15","author":"A Premchandar","year":"2024","journal-title":"Front Pharmacol"}],"container-title":["Klinische P\u00e4diatrie"],"original-title":[],"language":"de","link":[{"URL":"http:\/\/www.thieme-connect.de\/products\/ejournals\/pdf\/10.1055\/a-2449-3360.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,11,27]],"date-time":"2025-11-27T22:30:03Z","timestamp":1764282603000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.thieme-connect.de\/DOI\/DOI?10.1055\/a-2449-3360"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,12,10]]},"references-count":10,"journal-issue":{"issue":"02","published-online":{"date-parts":[[2025,2,19]]},"published-print":{"date-parts":[[2025,3]]}},"URL":"https:\/\/doi.org\/10.1055\/a-2449-3360","archive":["Portico","CLOCKSS"],"relation":{},"ISSN":["0300-8630","1439-3824"],"issn-type":[{"value":"0300-8630","type":"print"},{"value":"1439-3824","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,12,10]]}}}