{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,6,5]],"date-time":"2025-06-05T08:52:45Z","timestamp":1749113565400,"version":"3.37.3"},"reference-count":35,"publisher":"Springer Science and Business Media LLC","issue":"4","license":[{"start":{"date-parts":[[2024,9,27]],"date-time":"2024-09-27T00:00:00Z","timestamp":1727395200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"},{"start":{"date-parts":[[2024,9,27]],"date-time":"2024-09-27T00:00:00Z","timestamp":1727395200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Pulm Ther"],"published-print":{"date-parts":[[2024,12]]},"DOI":"10.1007\/s41030-024-00274-y","type":"journal-article","created":{"date-parts":[[2024,9,27]],"date-time":"2024-09-27T19:01:53Z","timestamp":1727463713000},"page":"427-438","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Comparison of Reporting Quality in National Cystic Fibrosis Patient Registries: Implications for Identifying Use of Novel CFTR Modulators"],"prefix":"10.1007","volume":"10","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4063-7682","authenticated-orcid":false,"given":"Owen W.","family":"Tomlinson","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Philip","family":"Mitchelmore","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Craig A.","family":"Williams","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2024,9,27]]},"reference":[{"issue":"10059","key":"274_CR1","doi-asserted-by":"publisher","first-page":"2519","DOI":"10.1016\/S0140-6736(16)00576-6","volume":"388","author":"JS Elborn","year":"2016","unstructured":"Elborn JS. Cystic fibrosis. Lancet. 2016;388(10059):2519\u201331.","journal-title":"Lancet"},{"issue":"4","key":"274_CR2","doi-asserted-by":"publisher","first-page":"403","DOI":"10.1016\/j.jcf.2013.12.003","volume":"13","author":"K De Boeck","year":"2014","unstructured":"De Boeck K, Zolin A, Cuppens H, Olesen HV, Viviani L. The relative frequency of CFTR mutation classes in European patients with cystic fibrosis. J Cyst Fibros. 2014;13(4):403\u20139.","journal-title":"J Cyst Fibros"},{"issue":"10","key":"274_CR3","doi-asserted-by":"publisher","first-page":"1160","DOI":"10.1038\/ng.2745","volume":"45","author":"PR Sosnay","year":"2013","unstructured":"Sosnay PR, Siklosi KR, Van Goor F, et al. Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene. Nat Genet. 2013;45(10):1160\u20137.","journal-title":"Nat Genet"},{"key":"274_CR4","doi-asserted-by":"publisher","DOI":"10.1016\/j.rmed.2022.106878","volume":"199","author":"M Desai","year":"2022","unstructured":"Desai M, Hine C, Whitehouse JL, Brownlee K, Charman SC, Nagakumar P. Who are the 10%?\u2014non eligibility of cystic fibrosis (CF) patients for highly effective modulator therapies. Respir Med. 2022;199: 106878.","journal-title":"Respir Med"},{"key":"274_CR5","doi-asserted-by":"publisher","first-page":"1662","DOI":"10.3389\/fphar.2019.01662","volume":"10","author":"M Lopes-Pacheco","year":"2020","unstructured":"Lopes-Pacheco M. CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine. Front Pharmacol. 2020;10:1662.","journal-title":"Front Pharmacol"},{"issue":"1","key":"274_CR6","doi-asserted-by":"publisher","first-page":"7234","DOI":"10.1038\/s41598-019-43652-2","volume":"9","author":"AR Habib","year":"2019","unstructured":"Habib AR, Kajbafzadeh M, Desai S, Yang CL, Skolnik K, Quon BS. A systematic review of the clinical efficacy and safety of CFTR modulators in cystic fibrosis. Sci Rep. 2019;9(1):7234.","journal-title":"Sci Rep"},{"issue":"19","key":"274_CR7","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. Elexacaftor\u2013tezacaftor\u2013ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381(19):1809\u201319.","journal-title":"N Engl J Med"},{"issue":"10","key":"274_CR8","doi-asserted-by":"publisher","first-page":"941","DOI":"10.1136\/archdischild-2020-320680","volume":"106","author":"C Edmondson","year":"2021","unstructured":"Edmondson C, Course CW, Doull I. Cystic fibrosis transmembrane conductance regulator modulators for cystic fibrosis: a new dawn? Arch Dis Child. 2021;106(10):941\u20135.","journal-title":"Arch Dis Child"},{"issue":"8","key":"274_CR9","doi-asserted-by":"publisher","first-page":"840","DOI":"10.1136\/thorax-2023-220100","volume":"78","author":"NJ Rowbotham","year":"2023","unstructured":"Rowbotham NJ, Smith S, Elliott ZC, et al. A refresh of the top 10 research priorities in cystic fibrosis. Thorax. 2023;78(8):840\u20133.","journal-title":"Thorax"},{"issue":"3","key":"274_CR10","doi-asserted-by":"publisher","first-page":"456","DOI":"10.1016\/j.jcf.2022.01.009","volume":"21","author":"J Guo","year":"2022","unstructured":"Guo J, Garratt A, Hill A. Worldwide rates of diagnosis and effective treatment for cystic fibrosis. J Cyst Fibros. 2022;21(3):456\u201362.","journal-title":"J Cyst Fibros"},{"issue":"6","key":"274_CR11","doi-asserted-by":"publisher","first-page":"1622","DOI":"10.1002\/ppul.26954","volume":"59","author":"J Guo","year":"2024","unstructured":"Guo J, King I, Hill A. International disparities in diagnosis and treatment access for cystic fibrosis. Pediatr Pulmonol. 2024;59(6):1622\u201330.","journal-title":"Pediatr Pulmonol"},{"issue":"7","key":"274_CR12","doi-asserted-by":"publisher","first-page":"1173","DOI":"10.1513\/AnnalsATS.201511-781OC","volume":"13","author":"EA Knapp","year":"2016","unstructured":"Knapp EA, Fink AK, Goss CH, et al. The Cystic Fibrosis Foundation Patient Registry. Design and methods of a national observational disease registry. Ann Am Thorac Soc. 2016;13(7):1173\u20139.","journal-title":"Ann Am Thorac Soc"},{"issue":"1","key":"274_CR13","doi-asserted-by":"publisher","first-page":"9","DOI":"10.1093\/ije\/dyx196","volume":"47","author":"D Taylor-Robinson","year":"2018","unstructured":"Taylor-Robinson D, Archangelidi O, Carr SB, et al. Data resource profile: the UK Cystic Fibrosis Registry. Int J Epidemiol. 2018;47(1):9\u201310e.","journal-title":"Int J Epidemiol"},{"issue":"Suppl 1","key":"274_CR14","doi-asserted-by":"publisher","first-page":"i9","DOI":"10.1136\/bmjqs-2013-002378","volume":"23","author":"MS Schechter","year":"2014","unstructured":"Schechter MS, Fink AK, Homa K, Goss CH. The Cystic Fibrosis Foundation Patient Registry as a tool for use in quality improvement. BMJ Qual Saf. 2014;23(Suppl 1):i9-14.","journal-title":"BMJ Qual Saf"},{"key":"274_CR15","unstructured":"European Cystic Fibrosis Society. ECFS Patient Registry Annual Report 2020. 2022."},{"key":"274_CR16","unstructured":"Cystic Fibrosis Registries. https:\/\/www.cysticfibrosisdata.org\/CFRegistries.htm. Accessed 2023 Jun 4."},{"key":"274_CR17","unstructured":"Monash University Department of Epidemiology and Preventive Medicine. The ACFDR Registry Annual Report 2021. Melbourne VIC, Australia: Cystic Fibrosis Australia; 2022. https:\/\/www.cysticfibrosis.org.au\/cf-data-registry\/."},{"key":"274_CR18","unstructured":"Cystic Fibrosis Canada. The Canadian Cystic Fibrosis Registry 2021 Annual Data report. Toronto ON, Canada: Cystic Fibrosis Canada; 2023. https:\/\/www.cysticfibrosis.ca\/our-programs\/cf-registry\/reports-other-resources."},{"key":"274_CR19","unstructured":"\u010cesk\u00fd registr cystick\u00e9 fibr\u00f3zy [Czech cystic fibrosis register]. P\u0159ehledov\u00e1 zpr\u00e1va n\u00e1rodn\u00edho registru CF pro rok 2021 [CF National Registry Summary Report for the year 2021]. Prague, Czechia: Centrem cystick\u00e9 fibr\u00f3zy p\u0159i Pediatrick\u00e9 klinice Fakultn\u00ed nemocnice v Motole a 2. l\u00e9ka\u0159sk\u00e9 fakulty Univerzity Karlovy; 2021. https:\/\/cfregistr.cz\/data\/."},{"key":"274_CR20","unstructured":"Vaincre la Mucoviscidose [Defeat Cystic Fibrosis]. Registre Fran\u00e7ais de la mucoviscidose bilan de donn\u00e9es [French cystic fibrosis registry data report]. Paris, France: Vaincre la Mucoviscidose; 2022. https:\/\/www.vaincrelamuco.org\/registredelamuco."},{"key":"274_CR21","unstructured":"Mukoviszidose e.V. [Cystic Fibrosis], Mukoviszidose Institut gGmbH [Cystic Fibrosis Institute]. German Cystic Fibrosis Registry Annual Report 2021. Bonn, Germany: Mukoviszidose e.V.; 2022. https:\/\/www.muko.info\/englisch-version\/registry."},{"key":"274_CR22","unstructured":"CFRI The Cystic Fibrosis Registry of Ireland. 2021 Annual Report CF Registry of Ireland. Dublin, Ireland: Cystic Fibrosis Registry of Ireland; 2021. https:\/\/cfri.ie\/annual-reports\/."},{"key":"274_CR23","unstructured":"Nederlandse Cystic Fibrosis Stichting [Dutch Cystic Fibrosis Foundation]. Dutch CF Registry 2021 [Infographic]. Baarn, Netherlands: Nederlandse Cystic Fibrosis Stichting; 2021. https:\/\/ncfs.nl\/onderzoek-naar-taaislijmziekte\/dutch-cf-registry\/."},{"key":"274_CR24","unstructured":"\u00c7ocuk Solunum Yolu Hastal\u0131klar\u0131 ve Kistik Fibrozis Derne\u011fi [Pediatric Respiratory Diseases and Cystic Fibrosis Society]. Ulusal Kistik Fibrozis Kay\u0131t Sistemi 2021 Y\u0131l\u0131 Verileri [National Cystic fibrosis Recording system Data for 2021]. Ankara, Turkey: \u00c7ocuk Solunum Yolu Hastal\u0131klar\u0131 ve Kistik Fibrozis Derne\u011fi; 2021. https:\/\/www.kistikfibrozisturkiye.org\/hasta-kayit-sistemi\/#yillik-raporlar."},{"key":"274_CR25","unstructured":"Cystic Fibrosis Trust. UK Cystic Fibrosis Registry 2021 Annual Data Report. London, UK: Cystic Fibrosis Trust; 2022. https:\/\/www.cysticfibrosis.org.uk\/about-us\/uk-cf-registry\/reporting-and-resources."},{"key":"274_CR26","unstructured":"Cystic Fibrosis Foundation. Patient Registry Annual Data Report 2021. Bethesda MD, USA: Cystic Fibrosis Foundation; 2022. https:\/\/www.cff.org\/medical-professionals\/patient-registry."},{"key":"274_CR27","unstructured":"World Bank Open Data. World Bank Open Data. https:\/\/data.worldbank.org. Accessed 2023 Jul 27."},{"key":"274_CR28","doi-asserted-by":"publisher","first-page":"678374","DOI":"10.3389\/fgene.2021.678374","volume":"12","author":"N Petrova","year":"2021","unstructured":"Petrova N, Balinova N, Marakhonov A, et al. Ethnic differences in the frequency of CFTR gene mutations in populations of the European and North Caucasian part of the Russian federation. Front Genet. 2021;12:678374.","journal-title":"Front Genet"},{"key":"274_CR29","doi-asserted-by":"publisher","first-page":"1163391","DOI":"10.3389\/fphar.2023.1163391","volume":"14","author":"M Zampoli","year":"2023","unstructured":"Zampoli M, Morrow BM, Paul G. Real-world disparities and ethical considerations with access to CFTR modulator drugs: mind the gap! Front Pharmacol. 2023;14:1163391.","journal-title":"Front Pharmacol"},{"issue":"10","key":"274_CR30","doi-asserted-by":"publisher","first-page":"2793","DOI":"10.3390\/cells10102793","volume":"10","author":"I Fajac","year":"2021","unstructured":"Fajac I, Sermet I. Therapeutic approaches for patients with cystic fibrosis not eligible for current CFTR modulators. Cells. 2021;10(10):2793.","journal-title":"Cells"},{"key":"274_CR31","unstructured":"EMA. European Medicines Agency. 2020. Kaftrio. https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/kaftrio.  Accessed 2023 Jun 4."},{"key":"274_CR32","unstructured":"NHS England. Landmark NHS deal to open up access to life-changing cystic fibrosis drug. 2020. https:\/\/www.england.nhs.uk\/2020\/08\/landmark-nhs-deal-to-open-up-access-to-life-changing-cystic-fibrosis-drug\/.  Accessed 2020 Nov 12."},{"key":"274_CR33","unstructured":"European Cystic Fibrosis Society. ECFS Patient Registry 2022 Annual Data Report. Karup, Denmark: European Cystic Fibrosis Society; 2024. www.ecfs.eu\/ecfspr"},{"issue":"1","key":"274_CR34","doi-asserted-by":"publisher","first-page":"23","DOI":"10.3390\/jcm10010023","volume":"10","author":"RVE Dagenais","year":"2020","unstructured":"Dagenais RVE, Su VCH, Quon BS. Real-world safety of CFTR modulators in the treatment of cystic fibrosis: a systematic review. J Clin Med. 2020;10(1):23.","journal-title":"J Clin Med"},{"issue":"2","key":"274_CR35","doi-asserted-by":"publisher","first-page":"339","DOI":"10.1016\/j.jcf.2021.10.002","volume":"21","author":"S Heo","year":"2022","unstructured":"Heo S, Young DC, Safirstein J, et al. Mental status changes during elexacaftor\/tezacaftor\/ivacaftor therapy. J Cyst Fibros. 2022;21(2):339\u201343.","journal-title":"J Cyst Fibros"}],"container-title":["Pulmonary Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s41030-024-00274-y.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s41030-024-00274-y\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s41030-024-00274-y.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,11,19]],"date-time":"2024-11-19T16:37:56Z","timestamp":1732034276000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s41030-024-00274-y"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,9,27]]},"references-count":35,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2024,12]]}},"alternative-id":["274"],"URL":"https:\/\/doi.org\/10.1007\/s41030-024-00274-y","relation":{},"ISSN":["2364-1754","2364-1746"],"issn-type":[{"type":"print","value":"2364-1754"},{"type":"electronic","value":"2364-1746"}],"subject":[],"published":{"date-parts":[[2024,9,27]]},"assertion":[{"value":"29 June 2024","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"16 September 2024","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"27 September 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":"Owen W Tomlinson reports conference fees from Chiesi Ltd., and speaker fees from Vertex Pharmaceuticals. Philip Mitchelmore reports no conflicts of interest. Craig A Williams reports no conflicts of interest.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"No ethical approval was required for this study, as it is a secondary analysis of publicly available data. References to the included databases are included within the reference list.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}},{"value":"No medical writing or editorial assistance (including the use of generative AI) was received during the course of writing this article.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Medical Writing\/Editorial Assistance"}},{"value":"For the purpose of open access, the author has applied a Creative Commons Attribution (CC BY-NC) licence to any Author Accepted Manuscript version arising from this submission.","order":5,"name":"Ethics","group":{"name":"EthicsHeading","label":"Rights Retention"}}]}}