在中国,囊性纤维化患者的进展和死亡率
Wangji Zhou1, Yaqi Wang1, Yanli Yang1
1Department of Pulmonary and Critical Care Medicine, State Key Laboratory of Complex Severe and Rare Diseases, State Key Laboratory of Common Mechanism Research for Maior Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Orphanet journal of rare diseases
|January 8, 2025
概括
这项研究详细介绍了中国的囊性纤维化 (CF) 进展和死亡率. 基线肺功能和恶化影响CF患者的结果,预后得分有助于生存预测.
科学领域:
- 肺部病理学 肺部病理学
- 罕见疾病 罕见疾病
- 临床遗传学 临床遗传学
背景情况:
- 囊性纤维化 (CF) 在中国很少见,与高加索人群相比,其临床和遗传特征不同.
- 关于中国CF患者的进展和死亡率的数据有限.
研究的目的:
- 描述中国队列中囊性纤维化病的临床进展和死亡率模式.
- 为了确定与这种人群中疾病进展和生存相关的因素.
主要方法:
- 67名来自北京联合医学院医院CF队列的患者的回顾性分析.
- 利用混合效应和通用线性回归模型进行进展分析.
- 使用生存分析和日志等级测试来评估生存率和预后因素.
主要成果:
- 成年人诊断的CF患者的胰腺功能不良较少,BMI高于儿童诊断的患者.
- 对于诊断时≤30岁的患者,FEV1%的预测每年下降1.17%.
- 预测更高的FEV1%和肺部恶化与CF进展相关;5年和10年生存率分别为96.7%和80.6%.
结论:
- 预测的基线FEV1%是中国CF进展和死亡率的关键因素.
- 肺部恶化加快了中国CF患者的肺功能下降.
- CF-ABLE和3年预后得分有效地预测了这一群体的不良结果.
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