The short-term aerosol administration of a high dose of tobramycin in patients with clinically stable cystic fibrosis is an efficacious and safe treatment for endobronchial infection with P. aeruginosa.

Chronic endobronchial bacterial infection evokes purulent airway secretions in patients with CF. The viscoelastic properties of these secretions is primarily due to the presence of polymerized DNA from degenerating leukocytes. Recombinant human DNase I (rhDNase) reduces the viscosity of CF sputum in vitro. To test the hypothesis that rhDNase would improve pulmonary function in children and adults with CF, we compared the efficacy and safety of 10-day administration of three doses of aerosolized rhDNase (0.6, 2.5, or 10.0 mg twice daily) in 181 outpatients using a randomized, placebo-controlled parallel design. Forced vital capacity (FVC) improved 10 to 12% (p < 0.05 to 0.001), and forced expiratory volume in one second (FEV1) improved 10 to 15% (p < 0.001) across all doses of rhDNase compared with placebo. The magnitude of effect was dose dependent for both FVC and FEV1 through study Day 21 (p < 0.001). rhDNase was associated with a decreased perception of dyspnea and an improved perception of well-being. No patients developed detectable anti-rhDNase antibodies or bronchial reactivity to rhDNase. Some patients experienced mild upper airway irritation, but no major adverse events were reported. Administration for 10 days of aerosolized rhDNase to pediatric and adult outpatients with CF improves lung function and is well tolerated. Although all three doses were efficacious, the greatest improvement in FEV1 and FEV1/FVC ratio was demonstrated in the 2.5 and 10.0 mg rhDNase treatment groups.

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Changes in calorie intake and weight gain were evaluated in five children with cystic fibrosis (CF) who received behavioral intervention and four children with CF who served as wait list controls. The behavioral intervention was a 6-week group treatment that provided nutritional education plus management strategies aimed at mealtime behaviors that parents find most problematic. The control group was identified prospectively and was evaluated on all dependent measures at the same points in time pre- and posttreatment as the intervention group. Difference scores on calorie intake and weight gain from pre- to posttreatment were compared between groups using t tests for independent samples. The behavioral intervention group increased their calorie intake by 1,032 calories per day, while the control group's intake increased only 244 calories per day from pre- to posttreatment [t(6) = 2.826, p = 0.03]. The intervention group also gained significantly more weight (1.7 kg) than the control group (0 kg) over the 6 weeks of treatment [t(7) = 2.588, p = 0.03] and demonstrated catchup growth for weight, as indicated by improved weight Z scores (-1.18 to -0.738). The control group showed a decline in weight Z scores over this same time period (-1.715 to -1.76). One month posttreatment, the intervention was replicated with two of the four children from the control group. Improved calorie intake and weight gain pre- to posttreatment were again found in these children. At 3- and 6-month follow-up study of children receiving intervention, maintenance of calorie intake and weight gain was confirmed. No changes were found on pulmonary functioning, resting energy expenditure, or activity level pre- to posttreatment. This form of early intervention appears to be promising in improving nutritional status and needs to be investigated over a longer period of time to evaluate the effects of treatment gains on the disease process.