Growth hormone treatment improves growth and clinical status in prepubertal children with cystic fibrosis: results of a multicenter randomized controlled trial.

CONTEXT: This multicenter, randomized, controlled, crossover trial of prepubertal children with cystic fibrosis (CF) tests the hypotheses that recombinant human GH (rhGH) treatment 1) improves height, weight, lean mass, and bone content irrespective of baseline measures; 2) improves clinical status and quality of life; and 3) has continued effect after cessation after 1 yr of treatment. METHODS: Sixty-one prepubertal subjects (

Growth hormone improves bone mineral content in children with cystic fibrosis.

AIM: Osteoporosis and osteopenia have been reported as common complications of cystic fibrosis (CF); however, little is known about accrual of bone mineral in CF. The goal of our study was to measure bone mineral content (BMC) in non-acutely-ill, but poorly growing children with CF, and to determine the relationship between height, lean body mass and BMC. Our second aim was to evaluate the effect of one year of treatment with human recombinant growth hormone (GH) on total body BMC. METHODS: We measured total-body BMC using dual energy X-ray absorptiometry in 32 poorly growing (height < or =10th percentile for age) prepubertal Caucasian children (ages 7 years 6 months-12 years 9 months, 17 M and 15, F) with CF. BMC and lean tissue mass (LTM) were measured at baseline, at 6 months and one year. One half of the children were randomly assigned to receive treatment with GH (GHTX). Results were compared to reference data maintained for healthy children matched for age and ethnicity. Sex steroid and IGF-I levels were also measured. RESULTS: Children with CF exhibited lower total body BMC and LTM than age-, ethnicity- and gender-matched controls. This was still apparent when the data were matched for height and bone age. BMC correlated with height, LTM, and IGF-I levels. Although at baseline the groups were similar, the GHTX group demonstrated significantly greater increase in height, weight, LTM and BMC than the NonTX group. These differences remained despite correction for increase in height CONCLUSION: Our study is the first to evaluate BMC in children with CF and suggests that poor accumulation of bone mineral is a problem. We have further demonstrated that GH treatment improves accumulation of bone mineral.

Mucinous cystadenocarcinoma of the pancreas in an adolescent with cystic fibrosis.

Epidemiologic studies indicate that the overall risk of malignancy among patients with cystic fibrosis (CF) is similar to that of the general population. However, these studies and a number of case reports suggest that patients with CF may be at increased risk for the development of specific gastrointestinal cancers. Tumors of the esophagus, stomach, small and large bowels, liver and biliary tracts, and pancreas have been described. Previous reports of pancreatic cancers among patients with CF have included only adenocarcinoma in adults. This is the first description of a mucinous cystadenocarcinoma of the pancreas in an adolescent with CF. The tumor developed within a pancreatic cyst that had been initially identified 13 years before resection. Our report highlights the increased risk of pancreatic malignancy among patients who have CF and illustrates the premalignant potential of pancreatic cysts in this at-risk population. Further, it reinforces the need for careful surveillance and suggests a role for more aggressive diagnostic and therapeutic interventions for patients with atypical findings on pancreatic imaging studies.

A retrospective study of growth hormone use in adolescents with cystic fibrosis.

OBJECTIVES: Studies of growth hormone (GH) effectiveness in prepubertal children with cystic fibrosis (CF) have been published previously. We present a retrospective study of GH treatment in adolescents with CF. STUDY DESIGN: We performed a retrospective evaluation of data from 25 pubertal adolescents (ages 13 years 4 months to 16 years 11 months, Tanner stage III or IV). Thirteen (4 F) were followed for 1 year, then received 1 year of treatment with GH (GHTX). We compared the results with a 'control' group of 12 (4 F) whose growth was followed for 1 year (NonTX) but who were not treated with GH at the time of this review. Anthropometric data included: height, weight, lean tissue mass (LTM) and bone mineral content (BMC). Pulmonary function, number of hospitalizations, glycosylated haemoglobin (HbA1c), random blood glucose levels, IGF-I, oestradiol and testosterone levels are also reported. RESULTS: There was no difference between the groups at baseline. After 1 year, GHTX had significantly greater height and weight velocity, height and weight Z-scores, body mass index (BMI), LTM and BMC. Absolute pulmonary function was better and hospitalizations were fewer in the GHTX. No subject developed glucose intolerance or had any other side-effects. CONCLUSION: These results suggest that GH use in pubertal adolescents with CF safely improves height, body weight, bone mineralization and clinical status.

Growth hormone treatment enhances nutrition and growth in children with cystic fibrosis receiving enteral nutrition.

OBJECTIVES: Impaired longitudinal growth and poor weight gain are common and important problems in children with cystic fibrosis. This study evaluates the hypothesis that adjunctive growth hormone (GH) therapy augments the growth response to nutritional supplementation. STUDY DESIGN: We recruited 18 prepubertal children who received enteral nutritional supplementation for at least 2 years before enrollment. Nine were randomly assigned to receive no GH for 1 year, followed by 1 year of GH. Nine were randomly assigned to receive 1 year of GH followed by a second year of GH. Measurements included height, weight, pulmonary function, lean tissue mass, bone mineral content, hospitalizations, outpatient antibiotic use, and caloric intake. RESULTS: Growth hormone resulted in significant improvement in height, weight, bone mineral content, lean tissue mass, and number of hospitalizations. Pulmonary function was similar at baseline. Absolute forced vital capacity and forced expiratory volume in 1 minute significantly increased in GH treatment, but there was no significant change in percent predicted pulmonary function. Caloric intake was similar in both groups during both years. CONCLUSIONS: These results suggest that GH is a useful for enhancing growth in children with cystic fibrosis receiving enteral nutritional supplementation.