2024年囊性纤维化年度回顾
Cystic Fibrosis Year in Review 2024.
作者信息
Savant Adrienne P
机构信息
Department of Pediatrics, Manning Family Children's, New Orleans, Louisiana, USA.
Department of Pediatrics, Tulane University, New Orleans, Louisiana, USA.
出版信息
Pediatr Pulmonol. 2025 Aug;60(8):e71222. doi: 10.1002/ppul.71222.
In 2024, important advances for people with cystic fibrosis (CF) were published. Important guidelines for newborn screening and care of infants diagnosed with CF transmembrane conductance regulator (CFTR)-Related Metabolic Syndrome/Cystic Fibrosis Screen Positive Inconclusive Diagnosis (CRMS/CFSPID) were published alongside related key lessons from individual programs. Work continues to improve growth and nutrition and treat pulmonary exacerbations. New position papers on care delivery and the care team in the post-CFTR modulator era were developed next to continued information related to CFTR modulator use on treatment burden simplification and side effects, such as mental health and use during pregnancy. The aim of this review is to provide high-level information that may lead to changes in clinical care.
2024年，有关囊性纤维化（CF）患者的重要进展得以发表。针对新生儿筛查以及被诊断患有囊性纤维化跨膜传导调节因子（CFTR）相关代谢综合征/囊性纤维化筛查呈阳性但诊断不确定（CRMS/CFSPID）的婴儿护理的重要指南得以发布，同时还公布了各个项目的相关关键经验教训。在改善生长发育和营养状况以及治疗肺部急性加重方面的工作仍在继续。除了持续提供有关CFTR调节剂使用对治疗负担简化和副作用（如心理健康以及孕期使用）的信息外，还制定了关于CFTR调节剂时代护理提供和护理团队的新立场文件。本综述的目的是提供可能导致临床护理发生变化的高级信息。
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