TY - JOUR
T1 - Efficacy and Safety of Long-Term Continuous Growth Hormone Treatment in Children with Prader-Willi Syndrome
AU - de Lind van Wijngaarden, R.F.A.
AU - Siemensma, E.P.C.
AU - Festen, D.A.M.
AU - Otten, B.J.
AU - van Mil, E.G.A.H.
AU - Rotteveel, J.
AU - Odink, R.J.H.
AU - van Bindels-de Heus, G.C.B.
AU - Leeuwen, M.
AU - Haring, D.A.P.J.
AU - Bocca, G.
AU - Houdijk, E.C.A.M.
AU - Hoorweg-Nijman, J.J.G.
AU - Vreuls, R.C.F.M.
AU - Jira, P.E.
AU - Trotsenburg, A.S.P.
AU - Bakker, B.
AU - Schroor, E. J.
AU - Pilon, J.W.
AU - Wit, J.M.
AU - Drop, S.L.S.
AU - Hokken-Koelega, A.C.S.
AU - Bindels-de Heus, G. C. B. Karen
AU - Haring, Danny A. J. P.
PY - 2009
Y1 - 2009
N2 - Background: Children with Prader-Willi syndrome (PWS) have abnormal body composition and impaired growth. Short-term GH treatment has beneficial effects. Objectives: The aim of the study was to investigate effects of long-term continuous GH treatment on body composition, growth, bone maturation, and safety parameters. Setting: We conducted a multicenter prospective trial. Design: Fifty-five children with a mean +/- SD age of 5.9 +/- 3.2 yr were followed during 4 yr of continuous GH treatment (1mg/m(2).d). Data were annually obtained in one center: fat percentage (fat%) and lean body mass (LBM) by dual-energy x-ray absorptiometry, height, weight, head circumference, bone age, blood pressure, and fasting IGF-I, IGF binding protein-3, glucose, insulin, glycosylated hemoglobin, total cholesterol, high-density lipoprotein, and low-density lipoprotein. SD scores (SDS) were calculated according to Dutch and PWS reference values (SDS and SDSPWS). Results: Fat% SDS was significantly lower after 4 yr of GH treatment (P <0.0001). LBMSDS significantly increased during the first year (P = 0.02) but returned to baseline values the second year and remained unchanged thereafter. Mean +/- SD height normalized from -2.27 +/- 1.2 SDS to -0.24 +/- 1.2 SDS (P <0.0001). Head circumference SDS increased from -0.79 +/- 1.0 at start to 0.07 +/- 1.1 SDS after 4 yr. BMISDSPWS significantly decreased. Mean +/- SD IGF-I and the IGF-I/IGF binding protein-3 ratio significantly increased to 2.08 +/- 1.1 and 2.32 +/- 0.9 SDS, respectively. GH treatment had no adverse effects on bone maturation, blood pressure, glucose homeostasis, and serum lipids. Conclusions: Our study in children with PWS shows that 4 yr of continuous GH treatment (1 mg/m(2).d) improves body composition by decreasing fat% SDS and stabilizing LBMSDS and head circumference SDS and normalizes height SDS without adverse effects. Thus, long-term continuous GH treatment is an effective and safe therapy for children with PWS. (J Clin Endocrinol Metab 94: 4205-4215, 2009)
AB - Background: Children with Prader-Willi syndrome (PWS) have abnormal body composition and impaired growth. Short-term GH treatment has beneficial effects. Objectives: The aim of the study was to investigate effects of long-term continuous GH treatment on body composition, growth, bone maturation, and safety parameters. Setting: We conducted a multicenter prospective trial. Design: Fifty-five children with a mean +/- SD age of 5.9 +/- 3.2 yr were followed during 4 yr of continuous GH treatment (1mg/m(2).d). Data were annually obtained in one center: fat percentage (fat%) and lean body mass (LBM) by dual-energy x-ray absorptiometry, height, weight, head circumference, bone age, blood pressure, and fasting IGF-I, IGF binding protein-3, glucose, insulin, glycosylated hemoglobin, total cholesterol, high-density lipoprotein, and low-density lipoprotein. SD scores (SDS) were calculated according to Dutch and PWS reference values (SDS and SDSPWS). Results: Fat% SDS was significantly lower after 4 yr of GH treatment (P <0.0001). LBMSDS significantly increased during the first year (P = 0.02) but returned to baseline values the second year and remained unchanged thereafter. Mean +/- SD height normalized from -2.27 +/- 1.2 SDS to -0.24 +/- 1.2 SDS (P <0.0001). Head circumference SDS increased from -0.79 +/- 1.0 at start to 0.07 +/- 1.1 SDS after 4 yr. BMISDSPWS significantly decreased. Mean +/- SD IGF-I and the IGF-I/IGF binding protein-3 ratio significantly increased to 2.08 +/- 1.1 and 2.32 +/- 0.9 SDS, respectively. GH treatment had no adverse effects on bone maturation, blood pressure, glucose homeostasis, and serum lipids. Conclusions: Our study in children with PWS shows that 4 yr of continuous GH treatment (1 mg/m(2).d) improves body composition by decreasing fat% SDS and stabilizing LBMSDS and head circumference SDS and normalizes height SDS without adverse effects. Thus, long-term continuous GH treatment is an effective and safe therapy for children with PWS. (J Clin Endocrinol Metab 94: 4205-4215, 2009)
U2 - https://doi.org/10.1210/jc.2009-0454
DO - https://doi.org/10.1210/jc.2009-0454
M3 - Article
C2 - 19837938
SN - 0021-972X
VL - 94
SP - 4205
EP - 4215
JO - Journal of clinical endocrinology and metabolism
JF - Journal of clinical endocrinology and metabolism
IS - 11
ER -