Wilmott R W, Amin R S, Colin A A, DeVault A, Dozor A J, Eigen H, Johnson C, Lester L A, McCoy K, McKean L P, Moss R, Nash M L, Jue C P, Regelmann W, Stokes D C, Fuchs H J
Division of Pulmonary Medicine, Children's Hospital Medical Center, Cincinnati, Ohio 45229-3039, USA.
Am J Respir Crit Care Med. 1996 Jun;153(6 Pt 1):1914-7. doi: 10.1164/ajrccm.153.6.8665055.
The goal of this study was to evaluate the safety and efficacy of recombinant human DNase (rhDNase) in hospitalized patients with cystic fibrosis (CF) experiencing acute pulmonary exacerbations. Eighty patients with documented CF were enrolled at 11 CF centers when admitted for antibiotic therapy. Patients were at least 5 yr old with a forced vital capacity (FVC) > or = 35% of predicted and an oxygen saturation > or = 90% on a fraction of inspired oxygen (FIO2) < 0.5. Patients were randomized to receive rhDNase 2.5 mg in 2.5 ml excipient twice a day (n = 43) or 2.5 ml excipient alone twice daily (n = 37) along with conventional treatment for exacerbations. Administration of rhDNase was not associated with acute adverse events or deaths, and no patients experienced allergic or anaphylactic reactions. Although forced expiratory volume in one second (FEV1) and FVC improved in both treatment groups during the double-blind period, there were no statistically significant differences in the mean change from baseline in FEV1 or FVC between the two groups. rhDNase therapy is safe and well tolerated in CF patients with acute exacerbations requiring hospitalization, but the study did not demonstrate a statistically significant therapeutic effect of rhDNase when added to a regimen of antibiotics and chest physical therapy.
本研究的目的是评估重组人脱氧核糖核酸酶（rhDNase）在因急性肺部加重而住院的囊性纤维化（CF）患者中的安全性和有效性。11个CF中心共纳入80例确诊为CF且因抗生素治疗入院的患者。患者年龄至少5岁，用力肺活量（FVC）≥预测值的35%，在吸入氧分数（FIO2）<0.5时氧饱和度≥90%。患者被随机分为两组，一组每天两次接受2.5 mg rhDNase溶于2.5 ml辅料中（n = 43），另一组每天两次仅接受2.5 ml辅料（n = 37），同时接受针对病情加重的常规治疗。rhDNase的使用与急性不良事件或死亡无关，且无患者发生过敏或过敏反应。虽然在双盲期两个治疗组的一秒用力呼气容积（FEV1）和FVC均有所改善，但两组间FEV1或FVC相对于基线的平均变化无统计学显著差异。对于因急性加重而需住院的CF患者，rhDNase治疗安全且耐受性良好，但该研究未证明在抗生素和胸部物理治疗方案中添加rhDNase具有统计学显著的治疗效果。
