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

The aim of this study was to evaluate the ability of high-resolution comput erized tomography (HRCT) of the chest and chest x-rays (CXR) to determine e fficacy 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, respective ly (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 correl ated with the total CXR score (r = -0.7, P < 0.01). There was improvement i n the parents' assessments of the patients' well-being, with improvement in physical activity, decreased cough, sleep quality, and appetite in those s ubjects receiving rhDNase. We conclude that the administration of rhDNase was associated with improvem ent in the HRCT scan in CF patients younger than 5 years of age. Findings i ndicate that HRCT of the chest is useful and sensitive in studying response s to therapy in patients with CF lung disease. To our knowledge, this is th e first report of the use of HRCT to assess the effectiveness of a therapeu tic modality in so young a CF patient population. <(c)> 2001 Wiley-Liss. In c.