Growth Hormone Improves Weight Velocity and Height Velocity in Prepubertal Children with Cystic Fibrosis

Abstract

We undertook this study to determine if growth hormone treatment of prepubertal children with cystic fibrosis could improve their height and weight. Nine prepubertal children with cystic fibrosis were treated with human recombinant growth hormone for one year. Results obtained during this year were compared to similar measurements made for each patient for the one year prior to the treatment year. Anthropometric data including: height, height velocity, weight, weight velocity and skin fold thickness were measured at three month intervals. Pulmonary function and skeletal muscle strength were measured at three month intervals. Glucose tolerance was evaluated by HbAlc and by fasting blood glucose and insulin levels every three months. Our results demonstrate that growth hormone treatment resulted in significant improvement in height velocity and height Z scores. Weight increased in all subjects, with a significant increase in weight velocity (year prior to treatment = 1.7+/-1.0 kg/yr, treatment year = 3.8+/-1.6 kg/yr; p=0.03). Measurements of skin fold thickness suggests that lean body mass improved with growth hormone treatment. Pulmonary function improved in all but two patients, whose pulmonary function remained the same and muscle strength improved in all subjects. These results suggest that growth hormone used in prepubertal children with cystic fibrosis can improve height and weight and may improve lean body mass.

Recombinant Human Growth Hormone in the Treatment of Patients With Cystic Fibrosis

“…35,37,38 The duration of treatment with rhGH ranged from 6 months to 3 years. Ages of the patients in the studies ranged from 6 months to 13 years, [31][32][33][34][35][36]38 with the exception of 1 case report of a patient aged 18 years. 37 Doses of rhGH in the studies ranged from 0.16 to 0.35 mg/kg per week, 32,33,35,36,38 with the exception of 1 case report in which the dose was 2.2 mg/day.…”

“…31,34 Baseline measures of height and weight were inconsistently reported among the observational studies, but all patients had deficient height and weight for their age. [31][32][33][34][35][36][37][38] Data Synthesis A summary of the results for all key questions of both qualitative and quantitative synthesis and strength of evidence is listed in Table 3. Forest plots of pooled analyses are available at www.…”

Late Consequences of Chronic Pediatric Illness

“…Growth hormone treatment enhances nutrition, linear growth and weight in children with CF [38], in prepubertal children may improve height, weight, bone mineral content and lean body mass [39], and may reduce the number of hospitalizations with or without significantly changing pulmonary function, even in children receiving enteral nutritional supplementation [39,40]. Children homozygous for the Delta F508 mutation, which is associated with the most severe disease, fail to normalize growth despite improved care [41].…”

Advancing Outcome Measures for the New Era of Drug Development in Cystic Fibrosis

“…Height and weight velocities were also used as primary efficacy measures for several recombinant human growth hormone trials, demonstrating improved growth in children with CF whose height and weight were less than or equal to the 25th percentile (40)(41)(42). In addition, the phase 3 efficacy trials of inhaled tobramycin demonstrated improved weight gain in the treatment group (43).…”