{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,6]],"date-time":"2026-07-06T16:18:41Z","timestamp":1783354721506,"version":"3.54.6"},"reference-count":29,"publisher":"European Respiratory Society (ERS)","issue":"3","license":[{"start":{"date-parts":[[2025,11,27]],"date-time":"2025-11-27T00:00:00Z","timestamp":1764201600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["publications.ersnet.org"],"crossmark-restriction":true},"short-container-title":["ERJ Open Res"],"accepted":{"date-parts":[[2025,10,31]]},"published-print":{"date-parts":[[2026,5]]},"abstract":"<jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>\n                      Elexacaftor\/tezacaftor\/ivacaftor (ETI) triple therapy has been introduced as causal therapy for people with cystic fibrosis (pwCF) carrying at least one\n                      <jats:italic>F508del<\/jats:italic>\n                      allele. Data on the real-world effect of ETI in pwCF without any\n                      <jats:italic>F508del<\/jats:italic>\n                      allele are limited.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>In this observational cohort study, data from the German CF Registry for pwCF who received off-label ETI for up to 365\u2005days were used to study effects on lung function, nutritional status and sweat chloride concentration (SCC).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      616 pwCF (mean\u00b1\n                      <jats:sc>sd<\/jats:sc>\n                      age 28.8\u00b113.9\u2005years) were registered with ETI treatment within compassionate use (n=509; at least one\n                      <jats:italic>F508del<\/jats:italic>\n                      allele present) or off-label use (carrying non-\n                      <jats:italic>F508del<\/jats:italic>\n                      mutations likely to respond to ETI (n=81) or not (n=26)). After 1\u2005year, mean percentage predicted forced expiratory volume in 1\u2005s increased by 10.3\u00b111.0 (p&lt;0.0001) and 9.0\u00b110.7 (p&lt;0.0001) percentage points compared to baseline in the at least one\n                      <jats:italic>F508del<\/jats:italic>\n                      and non-\n                      <jats:italic>F508del<\/jats:italic>\n                      predicted responsive groups, and remained stable in the non-\n                      <jats:italic>F508del<\/jats:italic>\n                      predicted non-responsive group (p=0.31). Body mass index percentile increased in the at least one\n                      <jats:italic>F508del<\/jats:italic>\n                      and non-\n                      <jats:italic>F508del<\/jats:italic>\n                      predicted responsive groups (11.4\u00b115.2; p&lt;0.0001 and 5.0\u00b112.3; p&lt;0.05, respectively), and remained stable in the non-\n                      <jats:italic>F508del<\/jats:italic>\n                      predicted non-responsive group (p=0.14). Mean SCC after 3\u2005months decreased by 43.9\u00b124.6\u2005mmol\u00b7L\n                      <jats:sup>\u22121<\/jats:sup>\n                      (p&lt;0.0001) and 34.4\u00b122.9\u2005mmol\u00b7L\n                      <jats:sup>\u22121<\/jats:sup>\n                      (p&lt;0.0001), respectively, in the at least one\n                      <jats:italic>F508del<\/jats:italic>\n                      and non-\n                      <jats:italic>F508del<\/jats:italic>\n                      predicted responsive groups, and increased in the non-\n                      <jats:italic>F508del<\/jats:italic>\n                      predicted non-responsive group (14.8\u00b19.5\u2005mmol\u00b7L\n                      <jats:sup>\u22121<\/jats:sup>\n                      ; p&lt;0.05).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>\n                      Real-world data align with the results of randomised clinical trials for pwCF carrying at least one\n                      <jats:italic>F508del<\/jats:italic>\n                      allele. Clinical improvements in pwCF with a range of CF transmembrane conductance regulator mutations treated off-label with ETI provide compelling support for the recent ETI label extension in Europe.\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1183\/23120541.00667-2025","type":"journal-article","created":{"date-parts":[[2025,11,27]],"date-time":"2025-11-27T16:55:16Z","timestamp":1764262516000},"page":"00667-2025","update-policy":"https:\/\/doi.org\/10.1183\/ers-crossmark-policy","source":"Crossref","is-referenced-by-count":0,"title":["Off-label treatment with elexacaftor\/tezacaftor\/ivacaftor in people with cystic fibrosis: real-world evidence from the German CF Registry"],"prefix":"10.1183","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6941-8315","authenticated-orcid":false,"given":"Mirjam","family":"Stahl","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Florian S.","family":"Gerts","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvia","family":"Hafkemeyer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julia","family":"Wosniok","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lutz","family":"Naehrlich","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"on behalf of the German CF Registry of the Mukoviszidose e.V. and participating sites","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"81","published-online":{"date-parts":[[2025,11,27]]},"reference":[{"key":"2026070608550782000_12.3.00667-2025.1","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1038\/s41572-024-00538-6","article-title":"Cystic fibrosis","volume":"10","author":"Mall","year":"2024","journal-title":"Nat Rev Dis Primers"},{"key":"2026070608550782000_12.3.00667-2025.2","unstructured":"Naehrlich L , Burkhart M , Wosniok J . German CF Registry \u2013 Annual Report 2023. 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