A multicenter study of alternate-day prednisone therapy in patients with cystic fibrosis,☆☆,

Presented at the meeting of the American Pediatric Society, Washington, D.C., May 1993.
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Abstract

The purpose of this study was to evaluate the efficacy and safety of alternate-day prednisone therapy in treating patients with mild-to-moderate cystic fibrosis during a 4-year period. In 15 North American cystic fibrosis centers, we screened 320 patients and enrolled 285 patients from April 1986 to December 1987. Patients were randomly assigned to take prednisone, 1 mg/kg per dose or 2 mg/kg per dose, or a matching placebo given on alternate days. Lung function, clinical status, hospitalizations, growth, and steroid side effects were monitored. During the first 24 months the percentage of the predicted forced vital capacity was greater in the 1 mg/kg group (p <0.0001) and the 2 mg/kg group (p <0.01) when each was compared with placebo. Patients in the 1 mg/kg group had a higher percentage of predicted forced vital capacity than placebo patients during the entire 48 months (p <0.0025), but only in the group of patients who were colonized with Pseudomonas aeruginosa at baseline. For 48 months, the 1 mg/kg group had a higher percentage of predicted forced expiratory volume in 1 second than patients given placebo (p <0.02). The prednisone-treated groups had a reduction in serum IgG concentrations (1 mg/kg vs placebo, p <0.007; 2 mg/kg vs placebo, p <0.003). From 6 months onward, height z scores fell in the 2 mg/kg group compared with those given placebo (p <0.001). For the 1 mg/kg group, height z scores were lower from 24 months. An excess of abnormalities in glucose metabolism was seen in the 2 mg/kg group compared with the placebo group (p <0.005). Our findings suggest a role for alternate-day prednisone therapy at a dose of 1 mg/kg in patients with mild to moderate cystic fibrosis. The benefit of improved lung function appears to outweigh the potential for adverse effects when the treatment period is less than 24 months. (J PEDIATR 1995;126:515-23)

Section snippets

METHODS

We conducted a 4-year double-blind, placebo-controlled, parallel-group multicenter trial of two different doses of prednisone therapy in patients with mild-to-moderate CF. A total of 285 patients were enrolled at 15 participating CF centers in the United States and Canada. Cystic fibrosis was diagnosed on the basis of clinical signs and symptoms and two elevated sweat chloride (or sweat sodium) values according to published standards.11 Inclusion criteria were as follows: age 6 to 14 years;

RESULTS

There were no significant differences among the treatment groups at the beginning of the study with the exception that the Pseudomonas aeruginosa colonization rate was higher in the 2 mg/kg group than in the other two groups (Table I). The patients were characterized clinically as having mild-to-moderate disease on the basis of their modified Shwachman clinical score, Brasfield radiologic score, and lung function measurements. Pulmonary function values were similar in all three groups (Table I

DISCUSSION

Our findings suggest a role for alternate-day prednisone therapy, 1 mg/kg, in the treatment of patients with CF who were aged 6 to 14 years and had mild-to-moderate clinical status and P. aeruginosa colonization. In this group of patients, there was significant improvement in FVC, first evident at 6 months and persisting throughout the 4 years of the study. The administration of prednisone yielded no significant clinical benefit in patients who were not colonized with P. aeruginosa. The higher

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    From the Section of Pediatric Pulmonology and Critical Care, Indiana University School of Medicine, Indianapolis; the Eudowood Division of Pediatric Respiratory Sciences, Johns Hopkins University School of Medicine, Baltimore, Maryland; the Section of Pediatric Pulmonology, Department of Pediatrics, Temple University School of Medicine, St. Christopher's Hospital for Children, Philadelphia, Pennsylvania; and the Cystic Fibrosis Foundation, Bethesda, Maryland

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