1. Efficacy and safety of oxandrolone in growth hormone-treated girls with turner syndrome.
- Author
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Menke LA, Sas TC, de Muinck Keizer-Schrama SM, Zandwijken GR, de Ridder MA, Odink RJ, Jansen M, Delemarre-van de Waal HA, Stokvis-Brantsma WH, Waelkens JJ, Westerlaken C, Reeser HM, van Trotsenburg AS, Gevers EF, van Buuren S, Dejonckere PH, Hokken-Koelega AC, Otten BJ, and Wit JM
- Subjects
- Adolescent, Age Factors, Androgens administration & dosage, Androgens adverse effects, Blood Pressure drug effects, Chi-Square Distribution, Child, Child, Preschool, Dose-Response Relationship, Drug, Double-Blind Method, Drug Therapy, Combination adverse effects, Female, Humans, Netherlands, Puberty drug effects, Recombinant Proteins therapeutic use, Treatment Outcome, Virilism chemically induced, Body Height drug effects, Human Growth Hormone therapeutic use, Oxandrolone administration & dosage, Oxandrolone adverse effects, Turner Syndrome drug therapy
- Abstract
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 Ox in 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.
- Published
- 2010
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