CFTR modulators

CFTR 调制器
  • 文章类型: Journal Article
    背景:elexacaftor/tezacaftor/ivacaftor(ETI)的出现为符合条件的CF成人带来了前所未有的临床益处。因此,慢性治疗是否可以安全地停止或适应这种新情况的问题已经成为一个非常有趣的问题。我们的目标是在意大利专家之间就ETI对CF肺病当前临床管理的影响达成共识。
    方法:从2021年12月至2022年4月,由国家CF科学协会认可的意大利专家小组通过改良的Delphi方法得出并分级了一组关于囊性纤维化成人肺部管理的声明。
    结果:专家组发表了13项声明,探讨了在吸入抗生素和粘液活性剂领域的可能修改;气道清除和身体活动;慢性大环内酯类和支气管扩张剂;以及肺移植转诊。专家认为最迫切需要探索的领域是ETI对吸入抗生素和肺移植的作用的影响。
    结论:从这项研究中得出的优先事项列表可能有助于指导和告知关于ETI对CF肺病及其临床管理影响的最紧迫领域的临床研究。
    BACKGROUND: The advent of elexacaftor/tezacaftor/ivacaftor (ETI) resulted in unprecedented clinical benefits for eligible adults with CF. As a result, the question of whether chronic treatments can be safely stopped or adapted to this new situation has become a matter of great interest. Our objective was to derive a consensus among Italian experts on the impact of ETI on the current clinical management of CF lung disease.
    METHODS: From December 2021 to April 2022 a panel of Italian experts endorsed by the national CF scientific society derived and graded a set of statements on the pulmonary management of adults with cystic fibrosis through a modified Delphi methodology.
    RESULTS: The panel produced 13 statements exploring possible modifications in the fields of inhaled antibiotics and mucoactives; airway clearance and physical activity; chronic macrolides and bronchodilators; and lung transplant referral. The areas that the experts considered most urgent to explore were the impact of ETI on the role of inhaled antibiotics and lung transplant.
    CONCLUSIONS: The list of priorities that emerged from this study could be useful to guide and inform clinical research on the most urgent area of impact of ETI on CF lung disease and its clinical management.
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  • 文章类型: Journal Article
    CFTR modulators associated with substantial clinical benefit are expected to rapidly improve the baseline condition of people with cystic fibrosis (PWCF) as well as decrease the rate of lung function decline, the occurrence of pulmonary exacerbations and likely even other disease complications. These changes in clinical status of PWCF introduced by clinically effective modulator therapy will have major repercussions on modalities of future CF drug development. As part of its \'Strategic Plan to speed up Access to new Drugs\', the European Cystic Fibrosis Society (ECFS) convened a meeting in Brussels on November 27th 2019 with relevant stakeholders (CF researchers and clinicians, patient organization and pharmaceutical company representatives, regulators, health technology assessors; see Acknowledgments for list of attendees) to discuss the future of clinical trials in cystic fibrosis (CF) in the context of HEMT entering the clinical arena. The following is the conclusion of the presentations and discussions. It is hoped that these concepts will be considered in future regulatory guidelines and may provide rationale and support for alternative trial designs.
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