欧洲的囊性纤维化:改善肺功能和寿命 - - 谨慎乐观的理由,但挑战仍然存在
Eitan Kerem1, Annalisa Orenti2, Arianna Adamoli2
1Department of Paediatrics and CF Centre, Hebrew University Medical School, Hadassah Medical Center, Jerusalem, Israel kerem@hadassah.org.il.
The European respiratory journal
|February 1, 2024
概括
在高收入欧洲国家,肺功能和囊性纤维化 (CF) 患者的存活率有所改善,但在低收入国家却没有. 针对性的干预措施对于全球公平的CF护理至关重要.
科学领域:
- 肺部医学 肺部医学
- 遗传学 是一个遗传学.
- 公共卫生 公共卫生
背景情况:
- 肺功能下降是囊性纤维化 (CF) 严重程度和预后的关键指标.
- 在不同经济地区的CF患者中,医疗保健获取和结果存在差异.
- 人均国民总收入 (GNI) 是影响医疗保健资源分配和患者结果的重要因素.
研究的目的:
- 分析CF成年人的肺功能趋势,在欧洲国家与不同的国内生产总值水平.
- 评估经济因素对CF患者患病率和结局的影响.
- 根据国家收入确定CF护理和结果的差异.
主要方法:
- 利用了2011-2021年欧洲囊性纤维化协会患者登记数据.
- 收集关于年龄,FEV1,人体测量,基因型,呼吸道培养和CF相关糖尿病 (CFRD) 的年度数据.
- 按人均国内生产总值分层划分欧洲国家,以比较患者的治疗结果.
主要成果:
- 在欧洲范围内观察到FEV1%预测和47621名CF患者的存活率的持续改善.
- 预测的平均FEV1%在成年人中从63.6%增加到74.7%,在儿童中从85%增加到94.2%.
- 从2011年到2021年,成人pwCF人数增加了60% (13,312至21,168),而高收入/中等收入国家的增长更大.
- 在低收入欧洲国家,预测的FEV1%或成年人数没有显著的年增长.
结论:
- 在过去十年中,预测FEV1%,成年人数和生存率的显著改善仅限于高收入和中等收入的欧洲国家.
- 需要在低收入国家采取紧急干预措施,以解决缺少观察到的CF患者结果改善的问题.
- 在F508del突变患者的实质性进展突出了为所有CF突变开发治疗方法的需要,以确保公平的护理.
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