TY - JOUR
T1 - Efficacy and Safety of Oxandrolone in Growth Hormone-Treated Girls with Turner Syndrome
AU - Menke, Leonie A.
AU - Sas, Theo C. J.
AU - de Muinck Keizer-Schrama, Sabine M. P. F.
AU - Zandwijken, Gladys R. J.
AU - de Ridder, Maria A. J.
AU - Odink, Roelof J.
AU - Jansen, Maarten
AU - Delemarre-van de Waal, Henriëtte A.
AU - Stokvis-Brantsma, Wilhelmina H.
AU - Waelkens, Johan J.
AU - Westerlaken, Ciska
AU - Reeser, H. Maarten
AU - Paul van Trotsenburg, A. S.
AU - Gevers, Evelien F.
AU - van Buuren, Stef
AU - Dejonckere, Philippe H.
AU - Hokken-Koelega, Anita C. S.
AU - Otten, Barto J.
AU - Wit, Jan M.
PY - 2010
Y1 - 2010
N2 - Context and Objective: GH therapy increases growth and adult height in Turner syndrome (TS). The benefit to risk ratio of adding the weak androgen oxandrolone (Ox) to GH is unclear. Design and Participants: A randomized, placebo-controlled, double-blind, dose-response study was performed in 10 centers in The Netherlands. One hundred thirty-three patients with TS were included in age group 1 (2-7.99 yr), 2 (8-11.99 yr), or 3 (12-15.99 yr). Patients were treated with GH (1.33 mg/m(2) . d) from baseline, combined with placebo(Pl) or Oxin low (0.03 mg/kg . d) or conventional (0.06 mg/kg . d) dose from the age of 8 yr and estrogens from the age of 12 yr. Adult height gain (adult height minus predicted adult height) and safety parameters were systematically assessed. Results: Compared with GH + Pl, GH + Ox 0.03 increased adult height gain in the intention-to-treat analysis (mean +/- SD, 9.5 +/- 4.7 vs. 7.2 +/- 4.0 cm, P <0.02) and per-protocol analysis (9.8 +/- 4.9 vs. 6.8 +/- 4.4 cm, P = 0.02). Partly due to accelerated bone maturation (P <0.001), adult height gain on GH + Ox 0.06 was not significantly different from that on GH + Pl (8.3 +/- 4.7 vs. 7.2 +/- 4.0 cm, P = 0.3). Breast development was slower on GH + Ox (GH + Ox 0.03, P = 0.02; GH + Ox 0.06, P = 0.05), and more girls reported virilization on GH + Ox 0.06 than on GH + Pl (P <0.001). Conclusions: In GH-treated girls with TS, we discourage the use of the conventional Ox dosage (0.06 mg/kg . d) because of its low benefit to risk ratio. The addition of Ox 0.03 mg/kg . d modestly increases adult height gain and has a fairly good safety profile, except for some deceleration of breast development. (J Clin Endocrinol Metab 95: 1151-1160, 2010)
AB - Context and Objective: GH therapy increases growth and adult height in Turner syndrome (TS). The benefit to risk ratio of adding the weak androgen oxandrolone (Ox) to GH is unclear. Design and Participants: A randomized, placebo-controlled, double-blind, dose-response study was performed in 10 centers in The Netherlands. One hundred thirty-three patients with TS were included in age group 1 (2-7.99 yr), 2 (8-11.99 yr), or 3 (12-15.99 yr). Patients were treated with GH (1.33 mg/m(2) . d) from baseline, combined with placebo(Pl) or Oxin low (0.03 mg/kg . d) or conventional (0.06 mg/kg . d) dose from the age of 8 yr and estrogens from the age of 12 yr. Adult height gain (adult height minus predicted adult height) and safety parameters were systematically assessed. Results: Compared with GH + Pl, GH + Ox 0.03 increased adult height gain in the intention-to-treat analysis (mean +/- SD, 9.5 +/- 4.7 vs. 7.2 +/- 4.0 cm, P <0.02) and per-protocol analysis (9.8 +/- 4.9 vs. 6.8 +/- 4.4 cm, P = 0.02). Partly due to accelerated bone maturation (P <0.001), adult height gain on GH + Ox 0.06 was not significantly different from that on GH + Pl (8.3 +/- 4.7 vs. 7.2 +/- 4.0 cm, P = 0.3). Breast development was slower on GH + Ox (GH + Ox 0.03, P = 0.02; GH + Ox 0.06, P = 0.05), and more girls reported virilization on GH + Ox 0.06 than on GH + Pl (P <0.001). Conclusions: In GH-treated girls with TS, we discourage the use of the conventional Ox dosage (0.06 mg/kg . d) because of its low benefit to risk ratio. The addition of Ox 0.03 mg/kg . d modestly increases adult height gain and has a fairly good safety profile, except for some deceleration of breast development. (J Clin Endocrinol Metab 95: 1151-1160, 2010)
U2 - https://doi.org/10.1210/jc.2009-1821
DO - https://doi.org/10.1210/jc.2009-1821
M3 - Article
C2 - 20061421
SN - 0021-972X
VL - 95
SP - 1151
EP - 1160
JO - Journal of clinical endocrinology and metabolism
JF - Journal of clinical endocrinology and metabolism
IS - 3
ER -