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  1. Article ; Online: Cast no shadow: assessing the disease-modifying effects of elexacaftor/tezacaftor/ivacaftor.

    Barry, Peter J / Burgel, Pierre-Régis

    The European respiratory journal

    2023  Volume 62, Issue 6

    MeSH term(s) Humans ; Cystic Fibrosis/drug therapy ; Cystic Fibrosis/genetics ; Alleles
    Chemical Substances elexacaftor (RRN67GMB0V) ; ivacaftor (1Y740ILL1Z) ; tezacaftor
    Language English
    Publishing date 2023-12-07
    Publishing country England
    Document type Editorial ; Comment
    ZDB-ID 639359-7
    ISSN 1399-3003 ; 0903-1936
    ISSN (online) 1399-3003
    ISSN 0903-1936
    DOI 10.1183/13993003.02026-2023
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: Diagnosing Cystic Fibrosis in Adults.

    Barry, Peter J / Simmonds, Nicholas J

    Seminars in respiratory and critical care medicine

    2023  Volume 44, Issue 2, Page(s) 242–251

    Abstract: Diagnosing cystic fibrosis (CF) in adulthood is not a rare occurrence for CF centers despite the popular belief that the diagnosis is achieved almost universally in childhood by means of newborn screening or early clinical presentation. The purpose of ... ...

    Abstract Diagnosing cystic fibrosis (CF) in adulthood is not a rare occurrence for CF centers despite the popular belief that the diagnosis is achieved almost universally in childhood by means of newborn screening or early clinical presentation. The purpose of this review article is to highlight specific considerations of adult diagnosis of CF. Obtaining a diagnosis of CF at any age is exceptionally important to ensure optimal treatment, monitoring, and support. In the new era of more personalized treatment with the advent of transformative therapies targeting the underlying protein defect, accurate diagnosis is of increasing importance. This review highlights the diagnostic algorithm leading to a new diagnosis of CF in adults. The diagnosis is usually confirmed in the presence of a compatible clinical presentation, evidence of cystic fibrosis transmembrane conductance regulator (CFTR) protein dysfunction, and/or identification of variants in the
    MeSH term(s) Adult ; Humans ; Cystic Fibrosis/diagnosis ; Cystic Fibrosis/genetics ; Cystic Fibrosis/drug therapy ; Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Genetic Testing ; Mutation ; Neonatal Screening ; Prognosis
    Chemical Substances Cystic Fibrosis Transmembrane Conductance Regulator (126880-72-6)
    Language English
    Publishing date 2023-01-09
    Publishing country United States
    Document type Review ; Journal Article
    ZDB-ID 1183617-9
    ISSN 1098-9048 ; 1069-3424
    ISSN (online) 1098-9048
    ISSN 1069-3424
    DOI 10.1055/s-0042-1759881
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  3. Article: Diagnosing Cystic Fibrosis in Adults

    Barry, Peter J. / Simmonds, Nicholas J.

    Seminars in Respiratory and Critical Care Medicine

    (Cystic Fibrosis)

    2023  Volume 44, Issue 02, Page(s) 242–251

    Abstract: Diagnosing cystic fibrosis (CF) in adulthood is not a rare occurrence for CF centers despite the popular belief that the diagnosis is achieved almost universally in childhood by means of newborn screening or early clinical presentation. The purpose of ... ...

    Series title Cystic Fibrosis
    Abstract Diagnosing cystic fibrosis (CF) in adulthood is not a rare occurrence for CF centers despite the popular belief that the diagnosis is achieved almost universally in childhood by means of newborn screening or early clinical presentation. The purpose of this review article is to highlight specific considerations of adult diagnosis of CF. Obtaining a diagnosis of CF at any age is exceptionally important to ensure optimal treatment, monitoring, and support. In the new era of more personalized treatment with the advent of transformative therapies targeting the underlying protein defect, accurate diagnosis is of increasing importance. This review highlights the diagnostic algorithm leading to a new diagnosis of CF in adults. The diagnosis is usually confirmed in the presence of a compatible clinical presentation, evidence of cystic fibrosis transmembrane conductance regulator (CFTR) protein dysfunction, and/or identification of variants in the CFTR gene believed to alter protein function. Achieving the diagnosis, however, is not always straightforward as CFTR protein function exists on a continuum with different organs displaying varying sensitivity to diminution in function. We highlight the current knowledge regarding the epidemiology of CF diagnosed in adults and outline the various clinical presentations, including pulmonary and extrapulmonary, which are more common in this population. We expand on the stepwise testing procedures that lead to diagnosis, paying particular attention to additional levels of testing which may be required to achieve an accurate diagnosis. There continues to be an important need for both pulmonary and other specialists to be aware of the potential for later presentation of CF, as the improvements in treatment over decades have had large positive impacts on prognosis for people with this condition.
    Keywords cystic fibrosis ; diagnosis ; late presentation ; CFTR-related disorder ; nasal potential difference ; sweat test
    Language English
    Publishing date 2023-01-09
    Publisher Thieme Medical Publishers, Inc.
    Publishing place Stuttgart ; New York
    Document type Article
    ZDB-ID 1183617-9
    ISSN 1098-9048 ; 1069-3424
    ISSN (online) 1098-9048
    ISSN 1069-3424
    DOI 10.1055/s-0042-1759881
    Database Thieme publisher's database

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  4. Article ; Online: Editorial: A future focus on cystic fibrosis in an era of uncertainty despite opportunity.

    Barry, Peter J / Plant, Barry J

    Current opinion in pulmonary medicine

    2021  Volume 27, Issue 6, Page(s) 535–537

    MeSH term(s) Cystic Fibrosis/therapy ; Cystic Fibrosis Transmembrane Conductance Regulator ; Humans ; Uncertainty
    Chemical Substances Cystic Fibrosis Transmembrane Conductance Regulator (126880-72-6)
    Language English
    Publishing date 2021-09-01
    Publishing country United States
    Document type Editorial
    ZDB-ID 1285505-4
    ISSN 1531-6971 ; 1070-5287 ; 1078-1641
    ISSN (online) 1531-6971
    ISSN 1070-5287 ; 1078-1641
    DOI 10.1097/MCP.0000000000000826
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  5. Article: Progress in precision medicine in cystic fibrosis: a focus on CFTR modulator therapy.

    Tewkesbury, Daniel H / Robey, Rebecca C / Barry, Peter J

    Breathe (Sheffield, England)

    2022  Volume 17, Issue 4, Page(s) 210112

    Abstract: The genetic multisystem condition cystic fibrosis (CF) has seen a paradigm shift in therapeutic approaches within the past decade. Since the first clinical descriptions in the 1930s, treatment advances had focused on the downstream consequences of a ... ...

    Abstract The genetic multisystem condition cystic fibrosis (CF) has seen a paradigm shift in therapeutic approaches within the past decade. Since the first clinical descriptions in the 1930s, treatment advances had focused on the downstream consequences of a dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR) chloride ion channel. The discovery of the gene that codes for CFTR and an understanding of the way in which different genetic mutations lead to disruption of normal CFTR function have led to the creation and subsequent licensing of drugs that target this process. This marks an important move towards precision medicine in CF and results from clinical trials and real-world clinical practice have been impressive. In this review we outline how CFTR modulator drugs restore function to the CFTR protein and the progress that is being made in this field. We also describe the real-world impact of CFTR modulators on both pulmonary and multisystem complications of CF and what this will mean for the future of CF care.
    Language English
    Publishing date 2022-01-12
    Publishing country England
    Document type Journal Article ; Review
    ZDB-ID 2562899-9
    ISSN 2073-4735 ; 1810-6838
    ISSN (online) 2073-4735
    ISSN 1810-6838
    DOI 10.1183/20734735.0112-2021
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  6. Article ; Online: Editorial: The changing landscape of cystic fibrosis: new therapies, challenges and a global pandemic.

    Barry, Peter J / Plant, Barry J

    Current opinion in pulmonary medicine

    2020  Volume 26, Issue 6, Page(s) 668–670

    Keywords covid19
    Language English
    Publishing date 2020-11-13
    Publishing country United States
    Document type Editorial
    ZDB-ID 1285505-4
    ISSN 1531-6971 ; 1070-5287 ; 1078-1641
    ISSN (online) 1531-6971
    ISSN 1070-5287 ; 1078-1641
    DOI 10.1097/MCP.0000000000000734
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 4900: Show issues Location:
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  7. Article ; Online: Longitudinal effects of elexacaftor/tezacaftor/ivacaftor on liver tests at a large single adult cystic fibrosis centre.

    Tewkesbury, Daniel H / Athwal, Varinder / Bright-Thomas, Rowland J / Jones, Andrew M / Barry, Peter J

    Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society

    2023  Volume 22, Issue 2, Page(s) 256–262

    Abstract: Background: Elexacaftor/tezacaftor/ivacaftor (E/T/I) therapy has resulted in substantial improvements in health status for many with cystic fibrosis. Monitoring of liver tests is recommended due to observed rises in transaminases in trials and cases of ... ...

    Abstract Background: Elexacaftor/tezacaftor/ivacaftor (E/T/I) therapy has resulted in substantial improvements in health status for many with cystic fibrosis. Monitoring of liver tests is recommended due to observed rises in transaminases in trials and cases of hepatotoxicity. Comprehensive data in large populations of unselected individuals and those with established CF related liver disease (CFLD) is lacking.
    Methods: Patients prescribed E/T/I at a large, adult centre had liver tests monitored at least 3 monthly for 12 months. Changes in individual liver tests were analysed and abnormalities were compared in those with and without CFLD.
    Results: 255 of 267 eligible patients were included. Mild rises in median ALT, AST and bilirubin from baseline to 3 months (all p < 0.001) within normal limits were noted which were sustained. There were no differences in changes in liver tests between those with or without CFLD. There was a significant difference in alkaline phosphatase for those with raised levels at baseline versus those with normal baseline level (-18.5 vs +2.0 IU/L, p = 0.002). Clinically significant rises in ALT and AST occurred in 8 (3.1%) and 6 (2.4%) cases respectively, with derangements in 2 individuals attributed to therapy.
    Conclusions: E/T/I leads to a mild, likely clinically insignificant increase in ALT, AST and bilirubin after 3 months which is sustained but does not appear to increase further in the vast majority. Underlying CFLD should not be a barrier to treatment. Although there was a reduction in ALP when elevated at baseline, this was not unique to those with pre-existing CFLD.
    MeSH term(s) Humans ; Adult ; Cystic Fibrosis/diagnosis ; Cystic Fibrosis/drug therapy ; Cystic Fibrosis/genetics ; Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Aminophenols/adverse effects ; Benzodioxoles/adverse effects ; Bilirubin ; Liver ; Mutation
    Chemical Substances elexacaftor (RRN67GMB0V) ; ivacaftor (1Y740ILL1Z) ; tezacaftor ; Cystic Fibrosis Transmembrane Conductance Regulator (126880-72-6) ; Aminophenols ; Benzodioxoles ; Bilirubin (RFM9X3LJ49)
    Language English
    Publishing date 2023-01-19
    Publishing country Netherlands
    Document type Journal Article
    ZDB-ID 2084724-5
    ISSN 1873-5010 ; 1569-1993
    ISSN (online) 1873-5010
    ISSN 1569-1993
    DOI 10.1016/j.jcf.2023.01.007
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 6028: Show issues Location:
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    Zs.MO 459: Show issues

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  8. Article ; Online: After the Celebrations: Lessons from the New Era of Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy.

    Barry, Peter J / Jones, Andrew M

    Annals of the American Thoracic Society

    2019  Volume 16, Issue 2, Page(s) 189–190

    MeSH term(s) Aminophenols ; Cohort Studies ; Cystic Fibrosis ; Cystic Fibrosis Transmembrane Conductance Regulator ; Humans ; Ireland ; Quinolones ; Registries
    Chemical Substances Aminophenols ; Quinolones ; Cystic Fibrosis Transmembrane Conductance Regulator (126880-72-6) ; ivacaftor (1Y740ILL1Z)
    Language English
    Publishing date 2019-02-01
    Publishing country United States
    Document type Editorial ; Comment
    ZDB-ID 2717461-X
    ISSN 2325-6621 ; 1943-5665 ; 2325-6621
    ISSN (online) 2325-6621 ; 1943-5665
    ISSN 2325-6621
    DOI 10.1513/AnnalsATS.201810-664ED
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  9. Article ; Online: Triple combination cystic fibrosis transmembrane conductance regulator modulator therapy in the real world - opportunities and challenges.

    Barry, Peter J / Taylor-Cousar, Jennifer L

    Current opinion in pulmonary medicine

    2021  Volume 27, Issue 6, Page(s) 554–566

    Abstract: Purpose of review: The purpose of this review is to describe on-going and upcoming real-world studies that will aid the cystic fibrosis (CF) community in understanding the long-term efficacy, safety and challenges in utilizing this therapy and managing ... ...

    Abstract Purpose of review: The purpose of this review is to describe on-going and upcoming real-world studies that will aid the cystic fibrosis (CF) community in understanding the long-term efficacy, safety and challenges in utilizing this therapy and managing care.
    Recent findings: The triple combination of elexacaftor, tezacaftor and ivacaftor (ETI) has been demonstrated to improve lung function, weight and quality of life in children and adults with CF with at least one copy of Phe508del. Treatment with ETI will potentially be available for approximately 90% of the CF population and change the face of CF.
    Summary: In spite of early demonstrations of short-term efficacy and safety, for medications that may be given for much of someone's life, continued assessment of these outcomes is necessary. Furthermore, the CF community must evaluate and address the issues that arise with increased longevity including parenthood, preventive care management and the potential comorbidities of aging.
    MeSH term(s) Adult ; Aminophenols/therapeutic use ; Child ; Cystic Fibrosis/drug therapy ; Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Drug Combinations ; Humans ; Mutation ; Quality of Life
    Chemical Substances Aminophenols ; Drug Combinations ; Cystic Fibrosis Transmembrane Conductance Regulator (126880-72-6)
    Language English
    Publishing date 2021-09-03
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 1285505-4
    ISSN 1531-6971 ; 1070-5287 ; 1078-1641
    ISSN (online) 1531-6971
    ISSN 1070-5287 ; 1078-1641
    DOI 10.1097/MCP.0000000000000819
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  10. Article ; Online: Triple Therapy for Cystic Fibrosis Phe508del-Gating and -Residual Function Genotypes. Reply.

    Barry, Peter J / Mall, Marcus A / Polineni, Deepika

    The New England journal of medicine

    2021  Volume 385, Issue 23, Page(s) 2208

    MeSH term(s) Aminophenols/therapeutic use ; Chloride Channel Agonists ; Cystic Fibrosis/drug therapy ; Cystic Fibrosis/genetics ; Genotype ; Humans
    Chemical Substances Aminophenols ; Chloride Channel Agonists
    Language English
    Publishing date 2021-12-08
    Publishing country United States
    Document type Letter ; Comment
    ZDB-ID 207154-x
    ISSN 1533-4406 ; 0028-4793
    ISSN (online) 1533-4406
    ISSN 0028-4793
    DOI 10.1056/NEJMc2115966
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