Elexacaftor/tezacaftor/ivacaftor, a game-changer in cystic fibrosis: The Portuguese experience
Phase 3 trials of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) combination treatment in people with cystic fibrosis (CF) with ≥1 F508del-CFTR allele showed profound short-term effects on lung function, weight, and pulmonary exacerbations (PEx). The authors conducted a 12-month study to add evidenc...
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| Veröffentlicht in: | Pulmonary pharmacology & therapeutics 2024-12, Vol.87, p.102328, Article 102328 |
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| creator | Fragoso, E. Boaventura, R. Almeida, L. Amorim, A. Gamboa, F. Santos, A.S. Gonçalves, F. Cruz, C.M. Carreiro, A. Gonçalves, A.S. Teixeira, V. Azevedo, P. |
| description | Phase 3 trials of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) combination treatment in people with cystic fibrosis (CF) with ≥1 F508del-CFTR allele showed profound short-term effects on lung function, weight, and pulmonary exacerbations (PEx). The authors conducted a 12-month study to add evidence on the real-world long-term effectiveness and safety of CFTR modulator therapy with ELX/TEZ/IVA in Portuguese CF adult population.
Ambispective, multicentre, observational, real-life study involving all the Portuguese CF Reference Centres. Adult patients on treatment with ELX/TEZ/IVA combination outside clinical trials were included. Demographics, efficacy, and safety variables on the first 12 months of treatment were compared with the pre-treatment year.
132 adult people with CF were included, of which 119 completed 12 months treatment (mean duration of treatment 21.5 months). Mean age was 31.7 ± 11.0 years, 53 % patients were homozygous for the F508del variant, baseline sweat chloride was 86.7 ± 25.9 mmol/L and pre-treatment percent-predicted FEV1 was 77.9 ± 19.7 %. At 1 year, mean absolute change from baseline in FEV1 was +0.46L (95 % CI: 0.37, 0.55; p |
| doi_str_mv | 10.1016/j.pupt.2024.102328 |
| format | Article |
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Ambispective, multicentre, observational, real-life study involving all the Portuguese CF Reference Centres. Adult patients on treatment with ELX/TEZ/IVA combination outside clinical trials were included. Demographics, efficacy, and safety variables on the first 12 months of treatment were compared with the pre-treatment year.
132 adult people with CF were included, of which 119 completed 12 months treatment (mean duration of treatment 21.5 months). Mean age was 31.7 ± 11.0 years, 53 % patients were homozygous for the F508del variant, baseline sweat chloride was 86.7 ± 25.9 mmol/L and pre-treatment percent-predicted FEV1 was 77.9 ± 19.7 %. At 1 year, mean absolute change from baseline in FEV1 was +0.46L (95 % CI: 0.37, 0.55; p < 0.001) and +13.9 percentage points (95 % CI: 11.5, 16.2; p < 0.001). PEx episodes decreased by 78 % (p < 0.001) and hospitalizations for PEx decreased by 91.4 % (p < 0.001). Body mass index (BMI) increased 1.2 kg/m2 (95 % CI: 0.9, 1.5; p < 0.001). Mean sweat chloride variation was −44.5 mmol/L (95 % CI: −49.8, −39.2; p < 0.001). No correlation was found between sweat chloride and lung function (r = −0.116, p = 0.335). There were no major safety concerns. Of note, headache was reported in 7.6 % and neuropsychiatric manifestations occurred in 12.6 % treated patients, being anxiety and depressive disorders the most common.
ELX/TEZ/IVA treatment in Portuguese adults with CF was associated with significant improvement in lung function, a drop in PEx and PEx-related hospitalizations and increase in BMI at 12 months and was well tolerated. These results add knowledge to our understanding of clinical benefits and tolerability of ELX/TEZ/IVA. Careful evaluation of adverse effects of ELX/TEZ/IVA therapy and its determinants, mainly concerning mental health, are a research priority.]]></description><identifier>ISSN: 1094-5539</identifier><identifier>ISSN: 1522-9629</identifier><identifier>EISSN: 1522-9629</identifier><identifier>DOI: 10.1016/j.pupt.2024.102328</identifier><identifier>PMID: 39299648</identifier><language>eng</language><publisher>England: Elsevier Ltd</publisher><subject>Adolescent ; Adult ; Aminophenols - adverse effects ; Aminophenols - therapeutic use ; Benzodioxoles - adverse effects ; Benzodioxoles - therapeutic use ; Chloride Channel Agonists - adverse effects ; Chloride Channel Agonists - therapeutic use ; Cystic Fibrosis - drug therapy ; Cystic Fibrosis - genetics ; Cystic Fibrosis - physiopathology ; Cystic Fibrosis Transmembrane Conductance Regulator - genetics ; Drug Combinations ; Female ; Forced Expiratory Volume ; Humans ; Indoles - administration & dosage ; Indoles - adverse effects ; Indoles - pharmacology ; Indoles - therapeutic use ; Male ; Middle Aged ; Portugal ; Pyrazoles - adverse effects ; Pyrazoles - therapeutic use ; Pyridines - adverse effects ; Pyridines - therapeutic use ; Pyrrolidines - adverse effects ; Pyrrolidines - therapeutic use ; Quinolones - administration & dosage ; Quinolones - adverse effects ; Quinolones - therapeutic use ; Treatment Outcome ; Young Adult</subject><ispartof>Pulmonary pharmacology & therapeutics, 2024-12, Vol.87, p.102328, Article 102328</ispartof><rights>2024 Elsevier Ltd</rights><rights>Copyright © 2024 Elsevier Ltd. All rights reserved.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><cites>FETCH-LOGICAL-c237t-aac2d4a64fdc080c3db87f1803a3e20a36471169e35bc4074c397972a4fcbb393</cites><orcidid>0000-0001-7222-0094</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://dx.doi.org/10.1016/j.pupt.2024.102328$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>314,780,784,3550,27924,27925,45995</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/39299648$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Fragoso, E.</creatorcontrib><creatorcontrib>Boaventura, R.</creatorcontrib><creatorcontrib>Almeida, L.</creatorcontrib><creatorcontrib>Amorim, A.</creatorcontrib><creatorcontrib>Gamboa, F.</creatorcontrib><creatorcontrib>Santos, A.S.</creatorcontrib><creatorcontrib>Gonçalves, F.</creatorcontrib><creatorcontrib>Cruz, C.M.</creatorcontrib><creatorcontrib>Carreiro, A.</creatorcontrib><creatorcontrib>Gonçalves, A.S.</creatorcontrib><creatorcontrib>Teixeira, V.</creatorcontrib><creatorcontrib>Azevedo, P.</creatorcontrib><title>Elexacaftor/tezacaftor/ivacaftor, a game-changer in cystic fibrosis: The Portuguese experience</title><title>Pulmonary pharmacology & therapeutics</title><addtitle>Pulm Pharmacol Ther</addtitle><description><![CDATA[Phase 3 trials of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) combination treatment in people with cystic fibrosis (CF) with ≥1 F508del-CFTR allele showed profound short-term effects on lung function, weight, and pulmonary exacerbations (PEx). The authors conducted a 12-month study to add evidence on the real-world long-term effectiveness and safety of CFTR modulator therapy with ELX/TEZ/IVA in Portuguese CF adult population.
Ambispective, multicentre, observational, real-life study involving all the Portuguese CF Reference Centres. Adult patients on treatment with ELX/TEZ/IVA combination outside clinical trials were included. Demographics, efficacy, and safety variables on the first 12 months of treatment were compared with the pre-treatment year.
132 adult people with CF were included, of which 119 completed 12 months treatment (mean duration of treatment 21.5 months). Mean age was 31.7 ± 11.0 years, 53 % patients were homozygous for the F508del variant, baseline sweat chloride was 86.7 ± 25.9 mmol/L and pre-treatment percent-predicted FEV1 was 77.9 ± 19.7 %. At 1 year, mean absolute change from baseline in FEV1 was +0.46L (95 % CI: 0.37, 0.55; p < 0.001) and +13.9 percentage points (95 % CI: 11.5, 16.2; p < 0.001). PEx episodes decreased by 78 % (p < 0.001) and hospitalizations for PEx decreased by 91.4 % (p < 0.001). Body mass index (BMI) increased 1.2 kg/m2 (95 % CI: 0.9, 1.5; p < 0.001). Mean sweat chloride variation was −44.5 mmol/L (95 % CI: −49.8, −39.2; p < 0.001). No correlation was found between sweat chloride and lung function (r = −0.116, p = 0.335). There were no major safety concerns. Of note, headache was reported in 7.6 % and neuropsychiatric manifestations occurred in 12.6 % treated patients, being anxiety and depressive disorders the most common.
ELX/TEZ/IVA treatment in Portuguese adults with CF was associated with significant improvement in lung function, a drop in PEx and PEx-related hospitalizations and increase in BMI at 12 months and was well tolerated. These results add knowledge to our understanding of clinical benefits and tolerability of ELX/TEZ/IVA. Careful evaluation of adverse effects of ELX/TEZ/IVA therapy and its determinants, mainly concerning mental health, are a research priority.]]></description><subject>Adolescent</subject><subject>Adult</subject><subject>Aminophenols - adverse effects</subject><subject>Aminophenols - therapeutic use</subject><subject>Benzodioxoles - adverse effects</subject><subject>Benzodioxoles - therapeutic use</subject><subject>Chloride Channel Agonists - adverse effects</subject><subject>Chloride Channel Agonists - therapeutic use</subject><subject>Cystic Fibrosis - drug therapy</subject><subject>Cystic Fibrosis - genetics</subject><subject>Cystic Fibrosis - physiopathology</subject><subject>Cystic Fibrosis Transmembrane Conductance Regulator - genetics</subject><subject>Drug Combinations</subject><subject>Female</subject><subject>Forced Expiratory Volume</subject><subject>Humans</subject><subject>Indoles - administration & dosage</subject><subject>Indoles - adverse effects</subject><subject>Indoles - pharmacology</subject><subject>Indoles - therapeutic use</subject><subject>Male</subject><subject>Middle Aged</subject><subject>Portugal</subject><subject>Pyrazoles - adverse effects</subject><subject>Pyrazoles - therapeutic use</subject><subject>Pyridines - adverse effects</subject><subject>Pyridines - therapeutic use</subject><subject>Pyrrolidines - adverse effects</subject><subject>Pyrrolidines - therapeutic use</subject><subject>Quinolones - administration & dosage</subject><subject>Quinolones - adverse effects</subject><subject>Quinolones - therapeutic use</subject><subject>Treatment Outcome</subject><subject>Young Adult</subject><issn>1094-5539</issn><issn>1522-9629</issn><issn>1522-9629</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2024</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp9kEtPAjEUhRujEUT_gAszSxcO9DWPGjeG4CMh0QVubTqdO1DCPGxnCPjrLQIuXd2Tm3NP7vkQuiZ4SDCJR8th0zXtkGLK_YIymp6gPokoDUVMxanXWPAwipjooQvnlhjjhLPoHPWYoELEPO2jz8kKNkqroq3tqIXvozTrg7oLVDBXJYR6oao52MBUgd661uigMJmtnXH3wWwBwXtt227egYMANg1YA5WGS3RWqJWDq8McoI-nyWz8Ek7fnl_Hj9NQU5a0oVKa5lzFvMg1TrFmeZYmBUkxUwwoVizmCSGxABZlmvsWmolEJFTxQmcZE2yAbve5ja2__A-tLI3TsFqpCurOSUZwQqIk5dhb6d6q_fPOQiEba0plt5JgueMql3LHVe64yj1Xf3RzyO-yEvK_kyNIb3jYG8C3XBuw0ulfArmxoFuZ1-a__B_seopJ</recordid><startdate>20241201</startdate><enddate>20241201</enddate><creator>Fragoso, E.</creator><creator>Boaventura, R.</creator><creator>Almeida, L.</creator><creator>Amorim, A.</creator><creator>Gamboa, F.</creator><creator>Santos, A.S.</creator><creator>Gonçalves, F.</creator><creator>Cruz, C.M.</creator><creator>Carreiro, A.</creator><creator>Gonçalves, A.S.</creator><creator>Teixeira, V.</creator><creator>Azevedo, P.</creator><general>Elsevier Ltd</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><orcidid>https://orcid.org/0000-0001-7222-0094</orcidid></search><sort><creationdate>20241201</creationdate><title>Elexacaftor/tezacaftor/ivacaftor, a game-changer in cystic fibrosis: The Portuguese experience</title><author>Fragoso, E. ; Boaventura, R. ; Almeida, L. ; Amorim, A. ; Gamboa, F. ; Santos, A.S. ; Gonçalves, F. ; Cruz, C.M. ; Carreiro, A. ; Gonçalves, A.S. ; Teixeira, V. ; Azevedo, P.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c237t-aac2d4a64fdc080c3db87f1803a3e20a36471169e35bc4074c397972a4fcbb393</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2024</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Aminophenols - adverse effects</topic><topic>Aminophenols - therapeutic use</topic><topic>Benzodioxoles - adverse effects</topic><topic>Benzodioxoles - therapeutic use</topic><topic>Chloride Channel Agonists - adverse effects</topic><topic>Chloride Channel Agonists - therapeutic use</topic><topic>Cystic Fibrosis - drug therapy</topic><topic>Cystic Fibrosis - genetics</topic><topic>Cystic Fibrosis - physiopathology</topic><topic>Cystic Fibrosis Transmembrane Conductance Regulator - genetics</topic><topic>Drug Combinations</topic><topic>Female</topic><topic>Forced Expiratory Volume</topic><topic>Humans</topic><topic>Indoles - administration & dosage</topic><topic>Indoles - adverse effects</topic><topic>Indoles - pharmacology</topic><topic>Indoles - therapeutic use</topic><topic>Male</topic><topic>Middle Aged</topic><topic>Portugal</topic><topic>Pyrazoles - adverse effects</topic><topic>Pyrazoles - therapeutic use</topic><topic>Pyridines - adverse effects</topic><topic>Pyridines - therapeutic use</topic><topic>Pyrrolidines - adverse effects</topic><topic>Pyrrolidines - therapeutic use</topic><topic>Quinolones - administration & dosage</topic><topic>Quinolones - adverse effects</topic><topic>Quinolones - therapeutic use</topic><topic>Treatment Outcome</topic><topic>Young Adult</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Fragoso, E.</creatorcontrib><creatorcontrib>Boaventura, R.</creatorcontrib><creatorcontrib>Almeida, L.</creatorcontrib><creatorcontrib>Amorim, A.</creatorcontrib><creatorcontrib>Gamboa, F.</creatorcontrib><creatorcontrib>Santos, A.S.</creatorcontrib><creatorcontrib>Gonçalves, F.</creatorcontrib><creatorcontrib>Cruz, C.M.</creatorcontrib><creatorcontrib>Carreiro, A.</creatorcontrib><creatorcontrib>Gonçalves, A.S.</creatorcontrib><creatorcontrib>Teixeira, V.</creatorcontrib><creatorcontrib>Azevedo, P.</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><jtitle>Pulmonary pharmacology & therapeutics</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Fragoso, E.</au><au>Boaventura, R.</au><au>Almeida, L.</au><au>Amorim, A.</au><au>Gamboa, F.</au><au>Santos, A.S.</au><au>Gonçalves, F.</au><au>Cruz, C.M.</au><au>Carreiro, A.</au><au>Gonçalves, A.S.</au><au>Teixeira, V.</au><au>Azevedo, P.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Elexacaftor/tezacaftor/ivacaftor, a game-changer in cystic fibrosis: The Portuguese experience</atitle><jtitle>Pulmonary pharmacology & therapeutics</jtitle><addtitle>Pulm Pharmacol Ther</addtitle><date>2024-12-01</date><risdate>2024</risdate><volume>87</volume><spage>102328</spage><pages>102328-</pages><artnum>102328</artnum><issn>1094-5539</issn><issn>1522-9629</issn><eissn>1522-9629</eissn><abstract><![CDATA[Phase 3 trials of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) combination treatment in people with cystic fibrosis (CF) with ≥1 F508del-CFTR allele showed profound short-term effects on lung function, weight, and pulmonary exacerbations (PEx). The authors conducted a 12-month study to add evidence on the real-world long-term effectiveness and safety of CFTR modulator therapy with ELX/TEZ/IVA in Portuguese CF adult population.
Ambispective, multicentre, observational, real-life study involving all the Portuguese CF Reference Centres. Adult patients on treatment with ELX/TEZ/IVA combination outside clinical trials were included. Demographics, efficacy, and safety variables on the first 12 months of treatment were compared with the pre-treatment year.
132 adult people with CF were included, of which 119 completed 12 months treatment (mean duration of treatment 21.5 months). Mean age was 31.7 ± 11.0 years, 53 % patients were homozygous for the F508del variant, baseline sweat chloride was 86.7 ± 25.9 mmol/L and pre-treatment percent-predicted FEV1 was 77.9 ± 19.7 %. At 1 year, mean absolute change from baseline in FEV1 was +0.46L (95 % CI: 0.37, 0.55; p < 0.001) and +13.9 percentage points (95 % CI: 11.5, 16.2; p < 0.001). PEx episodes decreased by 78 % (p < 0.001) and hospitalizations for PEx decreased by 91.4 % (p < 0.001). Body mass index (BMI) increased 1.2 kg/m2 (95 % CI: 0.9, 1.5; p < 0.001). Mean sweat chloride variation was −44.5 mmol/L (95 % CI: −49.8, −39.2; p < 0.001). No correlation was found between sweat chloride and lung function (r = −0.116, p = 0.335). There were no major safety concerns. Of note, headache was reported in 7.6 % and neuropsychiatric manifestations occurred in 12.6 % treated patients, being anxiety and depressive disorders the most common.
ELX/TEZ/IVA treatment in Portuguese adults with CF was associated with significant improvement in lung function, a drop in PEx and PEx-related hospitalizations and increase in BMI at 12 months and was well tolerated. These results add knowledge to our understanding of clinical benefits and tolerability of ELX/TEZ/IVA. Careful evaluation of adverse effects of ELX/TEZ/IVA therapy and its determinants, mainly concerning mental health, are a research priority.]]></abstract><cop>England</cop><pub>Elsevier Ltd</pub><pmid>39299648</pmid><doi>10.1016/j.pupt.2024.102328</doi><orcidid>https://orcid.org/0000-0001-7222-0094</orcidid></addata></record> |
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| subjects | Adolescent Adult Aminophenols - adverse effects Aminophenols - therapeutic use Benzodioxoles - adverse effects Benzodioxoles - therapeutic use Chloride Channel Agonists - adverse effects Chloride Channel Agonists - therapeutic use Cystic Fibrosis - drug therapy Cystic Fibrosis - genetics Cystic Fibrosis - physiopathology Cystic Fibrosis Transmembrane Conductance Regulator - genetics Drug Combinations Female Forced Expiratory Volume Humans Indoles - administration & dosage Indoles - adverse effects Indoles - pharmacology Indoles - therapeutic use Male Middle Aged Portugal Pyrazoles - adverse effects Pyrazoles - therapeutic use Pyridines - adverse effects Pyridines - therapeutic use Pyrrolidines - adverse effects Pyrrolidines - therapeutic use Quinolones - administration & dosage Quinolones - adverse effects Quinolones - therapeutic use Treatment Outcome Young Adult |
| title | Elexacaftor/tezacaftor/ivacaftor, a game-changer in cystic fibrosis: The Portuguese experience |
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