Growth hormone treatment improves growth and clinical status in prepubertal children with cystic fibrosis: results of a multicenter randomized controlled trial.
BACKGROUND
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 (<or=25th percentile for height and weight) were randomized into two groups: daily rhGH treatment or no treatment groups for 1 yr. In yr 2, treatments were crossed over. Outcome measures included serial standardized height and weight, number of hospitalizations and antibiotic courses, random blood glucose levels, lean mass, bone mineral content, pulmonary function, nutritional intake, and CF quality of life questionnaires.


RESULTS
Groups were similar at baseline and prepubertal during the entire study. After 1 yr, GH showed significantly greater gain in height, weight, lean mass, and bone mineral content. Gain in height was similar regardless of baseline. There were fewer hospitalizations in the rhGH-treated group and improvement in CF quality of life questionnaires measures of weight and body image. There was no difference in pulmonary function between groups. Results were similar in those treated with rhGH the second year. After cessation of rhGH treatment, there was sustained effect for increased height and weight velocity, as well as accrual of bone mineral.


CONCLUSIONS
rhGH therapy improves height and weight, decreases the number of hospitalizations, and improves quality of life in prepubertal children with CF. These effects are sustained after rhGH is discontinued.
Hardin
Dana S
DS
Department of Pediatrics, University of Texas Southwestern Medical School, Texas 75390, USA. hardind@pediatrics.ohio-state.edu
Adams-Huet
Beverley
B
Brown
Daniel
D
Chatfield
Barbara
B
Dyson
Maynard
M
Ferkol
Thomas
T
Howenstine
Michelle
M
Prestidge
Claude
C
Royce
Frederick
F
Rice
Julie
J
Seilheimer
Dan K
DK
Steelman
Joel
J
Shepherds
Ross
R
D001827:Body Height / Q000187:drug effects; D001835:Body Weight / Q000187:drug effects; D015519:Bone Density / Q000187:drug effects; D002648:Child; D018592:Cross-Over Studies; D003550:Cystic Fibrosis / Q000188:drug therapy*; D005260:Female; D006128:Growth / Q000187:drug effects*; D006760:Hospitalization / Q000706:statistics & numerical data; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D011788:Quality of Life
D019382:Human Growth Hormone