EFFECTS OF RHDNASE ON LUNG-FUNCTION AND N UTRITIONAL-STATUS IN A CYSTIC-FIBROSIS PEDIATRIC POPULATION

Background. - In 1994 we started recombinant human deoxyribonuclease ( rhDNase) in every cystic fibrosis (CF) patient whatever hits (her) cli nical condition, provided they were aged more than 5 years and forced vital capacity (FVC) was greater than or equal to 40%. Population and methods. - We reviewed retrospectively the effects of rhDNase in 69 CF children and adolescents during a 2-year follow-up. Patients (35 boys , 34 girls) received 2.5 mg of rhDNase once daily from a mean age of 8 .5 years (range 5-16.4). Baseline spirometric values (% predicted) and nutritional status were as followed: FVC = 84.8 +/- 21.7; forced expi ratory volume in 1 second (FEV1) = 80.8 +/- 22.2 peak flow = 89.7 +/- 34.2, forced expiratory fraction 25-75% (FEF 25-75) = 71.8 +/- 32.8; Z score weight/height = -0.41 +/- 1.14; Z score weight/age = 0.48 +/- 1 .25 body mass index = 15.4 +/- 1.8; caloric intake = 107 +/- 25% of re commended dietary allowances (RDA). Patients had a Shwachman-Kulczycki 's score of 87 +/- 9. Spirometric and nutritional data were analysed a fter 1, 3, 6, 12, 18 and 24 months of treatment and compared to baseli ne values (changes evaluated as percent change from mean baseline for spirometric data). Shwachman-Kulczycki's score was calculated after 24 months of rhDNase.Results. - An improvement of FVC (+10.7%, P < 0.001 and FEV1 (+12%, P < 0.01) was noted after one month of treatment and was maintained throughout the following 2 years around 8.7% (6.4-11.4) for FVC and 8.2%(7.3-9.1) for FEV1, P less than or equal to 0.01. Thi s was particularly observed in children aged 5 to 10 years, in boys an d in patients with a baseline FVC under 70% predicted. There was no si gnificant change in FEF 25-75. We observed an improvement of daily cal oric intake from the third month (P < 0.05) and of body mass index fro m the sixth month (P = 0.02). This was particularly noted in girls. Z score weight/age was improved only during the first 3 months of treatm ent while Z score weight/height increased only after a 2 year follow-u p. There was no significant change in Shwachman-Kulczycki's score afte r 24 months of rhDNase. Conclusion. - rhDNase in CF children is effect ive on lung function as well as on nutritional status and the response to this treatment can be evaluated after the first 3 months. (C) 1998 , Elsevier, Paris.