{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,27]],"date-time":"2026-02-27T04:25:48Z","timestamp":1772166348997,"version":"3.50.1"},"reference-count":8,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2024,7,30]],"date-time":"2024-07-30T00:00:00Z","timestamp":1722297600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2024,7,30]],"date-time":"2024-07-30T00:00:00Z","timestamp":1722297600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100003196","name":"Ministero della Salute","doi-asserted-by":"publisher","award":["Ministero della Salute"],"award-info":[{"award-number":["Ministero della Salute"]}],"id":[{"id":"10.13039\/501100003196","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Ital J Pediatr"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Elexacaftor-tezacaftor-ivacaftor (ETI) is a transmembrane conductance regulator modulator that significantly improves lung function in patients affected by cystic fibrosis (CF). This triple drug is currently not indicated in liver transplant patients, as clinical trials including subjects with previous solid organ transplantation are lacking.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Case presentation<\/jats:title>\n                    <jats:p>We report on a liver transplant girl with CF-related advanced pulmonary disease meeting clinical criteria for lung transplant, who started the triple modulator because she could not get on the lung transplant waiting list due to psycho-social motivations. Since initiation of ETI therapy, she has experienced a significant improvement in respiratory function and quality of life, without adverse effects.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>This case shows that ETI therapy can represent a lifesaving drug for individuals without alternatives, even in liver transplant patients. The clinical benefits of the modulator overcome risks, which may be limited with a close drug monitoring of immunosuppressants serum levels and functional liver tests.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1186\/s13052-024-01713-x","type":"journal-article","created":{"date-parts":[[2024,7,30]],"date-time":"2024-07-30T16:02:43Z","timestamp":1722355363000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Lung transplant list withdrawal in a liver transplant patient thanks to elexacaftor-tezacaftor-ivacaftor: a case report"],"prefix":"10.1186","volume":"50","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8142-6729","authenticated-orcid":false,"given":"Arianna","family":"Traunero","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anna","family":"Galletti","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sergio","family":"Ghirardo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Egidio","family":"Barbi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Massimo","family":"Maschio","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2024,7,30]]},"reference":[{"key":"1713_CR1","unstructured":"Manufacturer\u2019s prescibing information for TRIKAFTA (elexacaftor-tezacaftor-ivacaftor). April 2023. https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2023\/217660s000lbl.pdf (Accessed on May 04, 2023)."},{"issue":"19","key":"1713_CR2","doi-asserted-by":"publisher","first-page":"1809","DOI":"10.1056\/NEJMoa1908639","volume":"381","author":"PG Middleton","year":"2019","unstructured":"Middleton PG, Mall MA, D\u0159ev\u00ednek P, et al. VX17-445-102 Study Group. Elexacaftor-Tezacaftor-Ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381(19):1809\u201319. https:\/\/doi.org\/10.1056\/NEJMoa1908639.","journal-title":"N Engl J Med"},{"issue":"5","key":"1713_CR3","doi-asserted-by":"publisher","first-page":"585","DOI":"10.1002\/lt.23389","volume":"18","author":"MR Miller","year":"2012","unstructured":"Miller MR, Sokol RJ, Narkewicz MR, Sontag MK. Pulmonary function in individuals who underwent liver transplantation: from the US cystic fibrosis foundation registry. Liver Transpl. 2012;18(5):585\u201393. https:\/\/doi.org\/10.1002\/lt.23389.","journal-title":"Liver Transpl"},{"issue":"2","key":"1713_CR4","doi-asserted-by":"publisher","first-page":"411","DOI":"10.1002\/ppul.25779","volume":"57","author":"H Ragan","year":"2022","unstructured":"Ragan H, Autry E, Bomersback T, et al. The use of elexacaftor\/tezacaftor\/ivacaftor in patients with cystic fibrosis postliver transplant: a case series. Pediatr Pulmonol. 2022;57(2):411\u20137. https:\/\/doi.org\/10.1002\/ppul.25779.","journal-title":"Pediatr Pulmonol"},{"issue":"2","key":"1713_CR5","doi-asserted-by":"publisher","first-page":"227","DOI":"10.1016\/j.jcf.2021.07.017","volume":"21","author":"CJ McKinzie","year":"2022","unstructured":"McKinzie CJ, Doligalski CT, Lobritto SJ, et al. Use of elexacaftor\/tezacaftor\/ivacaftor in liver transplant patients with cystic fibrosis. J Cyst Fibros. 2022;21(2):227\u20139. https:\/\/doi.org\/10.1016\/j.jcf.2021.07.017.","journal-title":"J Cyst Fibros"},{"key":"1713_CR6","doi-asserted-by":"publisher","unstructured":"Tachtatzis P, Spoletini G, Clifton I, Etherington C, Peckham D. Changes in liver biochemistry and tacrolimus levels following the introduction of elexacaftor\/tezacaftor\/ivacaftor in patients with cystic fibrosis and liver transplant. J Cyst Fibros. 2023 May 8:S1569-1993(23)00129-7. https:\/\/doi.org\/10.1016\/j.jcf.2023.04.023","DOI":"10.1016\/j.jcf.2023.04.023"},{"issue":"1","key":"1713_CR7","doi-asserted-by":"publisher","first-page":"64","DOI":"10.1164\/rccm.202011-4153OC","volume":"204","author":"PR Burgel","year":"2021","unstructured":"Burgel PR, Durieu I, Chiron R, French Cystic Fibrosis Reference Network Study Group, et al. Rapid Improvement after starting Elexacaftor-Tezacaftor-Ivacaftor in patients with cystic fibrosis and Advanced Pulmonary Disease. Am J Respir Crit Care Med. 2021;204(1):64\u201373. https:\/\/doi.org\/10.1164\/rccm.202011-4153OC.","journal-title":"Am J Respir Crit Care Med"},{"issue":"3","key":"1713_CR8","doi-asserted-by":"publisher","first-page":"489","DOI":"10.1016\/j.jcf.2022.01.012","volume":"21","author":"C Martin","year":"2022","unstructured":"Martin C, Reynaud-Gaubert M, Hamidfar R, et al. Sustained effectiveness of elexacaftor-tezacaftor-ivacaftor in lung transplant candidates with cystic fibrosis. J Cyst Fibros. 2022;21(3):489\u201396. https:\/\/doi.org\/10.1016\/j.jcf.2022.01.012.","journal-title":"J Cyst Fibros"}],"container-title":["Italian Journal of Pediatrics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s13052-024-01713-x.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1186\/s13052-024-01713-x\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s13052-024-01713-x.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,7,30]],"date-time":"2024-07-30T16:06:02Z","timestamp":1722355562000},"score":1,"resource":{"primary":{"URL":"https:\/\/ijponline.biomedcentral.com\/articles\/10.1186\/s13052-024-01713-x"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,7,30]]},"references-count":8,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2024,12]]}},"alternative-id":["1713"],"URL":"https:\/\/doi.org\/10.1186\/s13052-024-01713-x","relation":{"has-preprint":[{"id-type":"doi","id":"10.21203\/rs.3.rs-3363699\/v1","asserted-by":"object"}]},"ISSN":["1824-7288"],"issn-type":[{"value":"1824-7288","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,7,30]]},"assertion":[{"value":"20 September 2023","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"25 July 2024","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"30 July 2024","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and\/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. All experimental protocols were approved by the IRCCS Burlo Garofolo ethics committee. Written consent was obtained from the parents of patients under 16 years old.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Written consent was obtained from parents for participants under 18 years old. Written consent was obtained for the publication of potentially identifying images and clinical details.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"Arianna Traunero declares that she has no conflict of interest. Anna Galletti declares that she has no conflict of interest. Sergio Ghirardo declares that he has no conflict of interest. Egidio Barbi declares that he has no conflict of interest. Massimo Maschio declares that he has no conflict of interest.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"138"}}