Lead Clinical Pharmacist for adult cystic fibrosis, Leeds Teaching Hospitals NHS Trust, Leeds, UK.
Specialist Dietitian, Royal Brompton Hospital, London, UK.
Br J Hosp Med (Lond). 2021 Nov 2;82(11):1-6. doi: 10.12968/hmed.2021.0530. Epub 2021 Nov 22.
Cystic fibrosis is a life-limiting, inherited, multi-organ disease which affects many systems of the body. Until recently, treatments were only able to ameliorate symptoms, but the introduction of precision medications which modulate the underlying defect in the cystic fibrosis transmembrane conductance regulator () gene has changed this. Notably improvements in nutrition and lung function, reduced use of antibiotics and reduced occupation rates for hospital beds have been seen. This article summarises the discussion of a group of healthcare professionals from different specialties and an expert patient, representing their personal views and experience of treating patients who are using CFTR modulators. The discussion was sponsored by an unrestricted grant from Chiesi Limited (Manchester, UK).
囊性纤维化是一种危及生命的遗传性多器官疾病，会影响身体的多个系统。直到最近，治疗方法只能改善症状，但调节囊性纤维化跨膜电导调节因子（CFTR）基因中潜在缺陷的精准药物的出现改变了这一局面。营养和肺功能的改善、抗生素使用减少以及住院床位占用率降低等情况都有明显改善。本文总结了一组来自不同专业的医疗保健专业人员和一名专家患者的讨论，代表了他们在使用 CFTR 调节剂治疗患者方面的个人观点和经验。该讨论由 Chiesi Limited（英国曼彻斯特）的一项非限制拨款赞助。
