{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T21:19:52Z","timestamp":1790803192903,"version":"4.1.0"},"reference-count":28,"publisher":"Oxford University Press (OUP)","issue":"2","license":[{"start":{"date-parts":[[2020,1,1]],"date-time":"2020-01-01T00:00:00Z","timestamp":1577836800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/pages\/standard-publication-reuse-rights"}],"content-domain":{"domain":["www.atsjournals.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2020,1,15]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Rationale<\/jats:title>\n                    <jats:p>Lumacaftor\u2013ivacaftor is a CFTR (cystic fibrosis transmembrane conductance regulator) modulator combination recently approved for patients with cystic fibrosis (CF) homozygous for the Phe508del mutation.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objectives<\/jats:title>\n                    <jats:p>To evaluate the safety and effectiveness of lumacaftor\u2013ivacaftor in adolescents (\u226512 yr) and adults (\u226518 yr) in a real-life postapproval setting.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>The study was conducted in the 47 CF reference centers in France. All patients who initiated lumacaftor\u2013ivacaftor from January 1 to December 31, 2016, were eligible. Patients were evaluated for lumacaftor\u2013ivacaftor safety and effectiveness over the first year of treatment following the French CF Learning Society\u2019s recommendations.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Measurements and Main Results<\/jats:title>\n                    <jats:p>Among the 845 patients (292 adolescents and 553 adults) who initiated lumacaftor\u2013ivacaftor, 18.2% (154 patients) discontinued treatment, often owing to respiratory (48.1%, 74 patients) or nonrespiratory (27.9%, 43 patients) adverse events. In multivariable logistic regression, factors associated with increased rates of discontinuation included adult age group, percent predicted FEV1 (ppFEV1) less than 40%, and numbers of intravenous antibiotic courses during the year before lumacaftor\u2013ivacaftor initiation. Patients with continuous exposure to lumacaftor\u2013ivacaftor showed an absolute increase in ppFEV1 (+3.67%), an increase in body mass index (+0.73 kg\/m2), and a decrease in intravenous antibiotic courses by 35%. Patients who discontinued treatment had significant decrease in ppFEV1, without improvement in body mass index or decrease in intravenous antibiotic courses.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Lumacaftor\u2013ivacaftor was associated with improvement in lung disease and nutritional status in patients who tolerated treatment. Adults who discontinued lumacaftor\u2013ivacaftor, often owing to adverse events, were found at high risk of clinical deterioration.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1164\/rccm.201906-1227oc","type":"journal-article","created":{"date-parts":[[2019,10,11]],"date-time":"2019-10-11T03:01:50Z","timestamp":1570762910000},"page":"188-197","update-policy":"https:\/\/doi.org\/10.1164\/crossmarkpolicies","source":"Crossref","is-referenced-by-count":126,"title":["Real-Life Safety and Effectiveness of Lumacaftor\u2013Ivacaftor in Patients with Cystic Fibrosis"],"prefix":"10.1093","volume":"201","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0903-9828","authenticated-orcid":false,"given":"Pierre-R\u00e9gis","family":"Burgel","sequence":"first","affiliation":[{"name":"Universit\u00e9 de Paris, Institut Cochin, INSERM U1016, Paris, France"},{"name":"Respiratory Medicine and National Reference Cystic Fibrosis Reference Center, Cochin Hospital, Assistance 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