Use of computerized tomography and chest x-rays in evaluating efficacy of aerosolized recombinant human DNase in cystic fibrosis patients younger than age 5 years: A preliminary study
Use of computerized tomography and chest x-rays in evaluating efficacy of aerosolized recombinant human DNase in cystic fibrosis patients younger than age 5 years: A preliminary study
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DOI：
10.1002/ppul.1061
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发表时间：
2001-05-01
影响因子：
3.1
通讯作者：
Strouse, PJ
中科院分区：
文献类型：
--
作者：
Nasr, SZ;Kuhns, LR;Strouse, PJ
本研究的目的是评价胸部高分辨率计算机断层扫描(HRCT)和胸部X线片(CXR)判断吸入重组人DNA酶(RhDNase)治疗5岁以下囊性纤维化(CF)患者疗效的能力。这是一项随机、双盲、安慰剂对照的初步研究，对象为密歇根大学安娜堡分校囊性纤维化中心(Ann Arbor，MI)的12名年龄在5岁以下的CF患者。用先前验证的评分系统评估从基线到治疗第100天的HRCT和CXR评分的变化。结果显示，rhDNase组和安慰剂组之间HRCT评分的平均变化在95%水平上显著，rhDNase组和安慰剂组的平均变化+/-SE平均值分别为-1.00+/-0.53和0.58+/-0.24(P=0.02)。两组CXR评分差异无统计学意义。将HRCT评分与CXR评分进行相关性分析；只有小叶间隔增厚与CXR总评分显著相关(r=-0.7，P<；0.01)。在接受重组DNA酶治疗的受试者中，父母对患者幸福感的评估有所改善，体力活动有所改善，咳嗽、睡眠质量和食欲下降。我们得出结论，在5岁以下的CF患者中，应用重组人脱氧核糖核酸酶与HRCT扫描的改善有关。结果表明，胸部HRCT在研究慢性肺病患者的治疗反应方面是有用的和敏感的。据我们所知，这是第一份使用HRCT来评估如此年轻的CF患者群体中治疗方式的有效性的报告。2001年威利-里斯。Inc.
The aim of this study was to evaluate the ability of high-resolution computerized tomography (HRCT) of the chest and chest x-rays (CXR) to determine efficacy of inhaled recombinant human DNase (rhDNase) in cystic fibrosis (CF) patients younger than 5 years of age. A randomized, double-blind, placebo-controlled pilot study of 12 patients with CF younger than 5 years of age, attending the University of Michigan Cystic Fibrosis Center (Ann Arbor, MI) was conducted. The changes in the HRCT and CXR score from baseline to day 100 of therapy were assessed using a previously validated scoring system.The mean changes of HRCT scores between the rhDNase and placebo groups were found to be significant at the 95% level, with mean change +/- SE mean of -1.00 +/- 0.53 and 0.58 +/- 0.24 for rhDNase and placebo groups, respectively (P = 0.02). The difference in CXR score was not significant between the two groups. An analysis was performed to relate HRCT subscores to CXR score; only thickening of the intra-interlobular septae was significantly correlated with the total CXR score (r = -0.7, P < 0.01). There was improvement in the parents' assessments of the patients' well-being, with improvement in physical activity, decreased cough, sleep quality, and appetite in those subjects receiving rhDNase.We conclude that the administration of rhDNase was associated with improvement in the HRCT scan in CF patients younger than 5 years of age. Findings indicate that HRCT of the chest is useful and sensitive in studying responses to therapy in patients with CF lung disease. To our knowledge, this is the first report of the use of HRCT to assess the effectiveness of a therapeutic modality in so young a CF patient population. 2001 Wiley-Liss. Inc.