在正常受试者和患有囊性纤维化症的患者中吸入复合人类DNase. 一个第一阶段的研究
M L Aitken1, W Burke, G McDonald
1Division of Pulmonary and Critical Care, University of Washington, Seattle 98195.
JAMA
|April 8, 1992
概括
复合人类DNase (rhDNase) 吸入对于囊性纤维化患者和健康人群来说是安全的. 短期的rhdnase疗法可以改善肺功能,减少囊性纤维化成年人的呼吸障碍.
科学领域:
- 肺部医学 肺部医学
- 药理学 药理学是指药理学的学科.
- 生物技术是生物技术.
背景情况:
- 囊性纤维化 (CF) 是一种影响肺功能的遗传性疾病.
- 重组人体DNase (rhDNase) 是一种用于CF治疗的酶.
- 在CF患者和健康个体中评估rhDNase的安全性至关重要.
研究的目的:
- 评估气溶液rhdnase的安全性和耐受性.
- 为了评估rhDNase对CF患者肺功能的影响.
- 为了确定rhdnase治疗的潜在不良反应.
主要方法:
- 一项非随机试验,涉及12名健康受试者和14名CF患者 (12人完成).
- rhDNase通过吸入,每天三次,周一至周五两周.
- 监测了肺功能测试,血清DNase水平和抗DNase抗体.
主要成果:
- rhDNase吸入被很好地容忍,没有严重或过敏反应.
- 没有参与者产生抗rhDNase抗体.
- 患有CF的患者显示肺功能 (FVC,FEV1) 和呼吸障碍得分有所改善.
结论:
- 气溶式rhdnase对健康人群和患有CF的成年人来说都是安全的.
- 短期的rhdnase治疗显示出改善CF患者肺功能的潜力.
更多相关视频
相关概念视频
Cystic Fibrosis: Pathogenesis
Cystic fibrosis (CF), an autosomal recessive disorder, significantly affects the function of exocrine glands. This genetically inherited disease is characterized by the production of thick and sticky mucus, which can severely affect various organs and systems in the body.
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation, but...
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation, but...
Cystic Fibrosis: Management
Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
Sinus disease and chronic sinusitis...
Sinus disease and chronic sinusitis...


