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高效调节剂治疗时代囊性纤维化中的性别差异。

Sex disparities in cystic fibrosis in the era of highly effective modulator treatment.

作者信息

McNally Emma, Casey Michelle

机构信息

Department of Respiratory Medicine, Beaumont Hospital, Dublin, Ireland.

Cystic Fibrosis Unit, Beaumont Hospital, Dublin , Ireland.

出版信息

BMC Pulm Med. 2025 May 2;25(1):212. doi: 10.1186/s12890-025-03621-0.

Abstract

Cystic fibrosis (CF) is a genetic disorder characterized by progressive lung disease and extra-pulmonary manifestations with notable sex disparities in disease outcomes. In this review we summarize the underlying mechanisms driving this sex disparity, with a particular focus on the role of sex hormones on CF lung disease pathophysiology. We explore how the introduction of highly effective modulator therapies (HEMT) may impact sex differences in outcomes and assess whether they have the potential to close the sex gap. While treatment with HEMT has led to better outcomes in the CF population as a whole, females with CF continue to experience worse pulmonary morbidity than males. There is a need for continued research in this area, particularly into the influence and therapeutic potential of sex hormones.

摘要

囊性纤维化(CF)是一种遗传性疾病,其特征为进行性肺部疾病和肺外表现,在疾病结局方面存在显著的性别差异。在本综述中,我们总结了导致这种性别差异的潜在机制,特别关注性激素在CF肺部疾病病理生理学中的作用。我们探讨了高效调节剂疗法(HEMT)的引入如何可能影响结局中的性别差异,并评估它们是否有缩小性别差距的潜力。虽然HEMT治疗在整个CF人群中带来了更好的结局,但CF女性的肺部发病率仍继续高于男性。这一领域需要持续研究,特别是关于性激素的影响和治疗潜力。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a6d9/12046722/55e4f44dfbed/12890_2025_3621_Fig1_HTML.jpg

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