Long-term effects of previous oxandrolone treatment in adult women with Turner syndrome.
OBJECTIVE
Short stature is a prominent feature of Turner syndrome (TS), which is partially overcome by GH treatment. We have previously reported the results of a trial on the effect of oxandrolone (Ox) in girls with TS. Ox in a dose of 0.03 mg/kg per day (Ox 0.03) significantly increased adult height gain, whereas Ox mg/kg per day (0.06) did not, at the cost of deceleration of breast development and mild virilization. The aim of this follow-up study in adult participants of the pediatric trial was to investigate the long-term effects of previous Ox treatment.


METHODS
During the previous randomized controlled trial, 133 girls were treated with GH combined with placebo (Pl), Ox 0.03, or Ox 0.06 from 8 years of age and estrogen from 12 years. Sixty-eight women (Pl, n=23; Ox 0.03, n=27; and Ox 0.06, n=18) participated in the double-blind follow-up study (mean age, 24.0 years; mean time since stopping GH, 8.7 years; and mean time of Ox/Pl use, 4.9 years). We assessed height, body proportions, breast size, virilization, and body composition.


RESULTS
Height gain (final minus predicted adult height) was maintained at follow-up (Ox 0.03 10.2±4.9 cm, Ox 0.06 9.7±4.4 cm vs Pl 8.0±4.6 cm). Breast size, Tanner breast stage, and body composition were not different between groups. Ox-treated women reported more subjective virilization and had a lower voice frequency.


CONCLUSIONS
Ox 0.03 mg/kg per day has a beneficial effect on adult height gain in TS patients. Despite previously reported deceleration of breast development during Ox 0.03 treatment, adult breast size is not affected. Mild virilization persists in only a small minority of patients. The long-term evaluation indicates that Ox 0.03 treatment is effective and safe.
Department of Endocrinology, Radboud University Nijmegen Medical Centre, HB Nijmegen, The Netherlands. k.freriks@aig.umcn.nl
Kim
K
Freriks
Theo C J
TC
Sas
Maaike A F
MA
Traas
Romana T
RT
Netea-Maier
Martin
M
den Heijer
Ad R M M
AR
Hermus
Jan M
JM
Wit
Janiëlle A E M
JA
van Alfen-van der Velden
Barto J
BJ
Otten
Sabine M P F
SM
de Muinck Keizer-Schrama
Martin
M
Gotthardt
Philippe H
PH
Dejonckere
Gladys R J
GR
Zandwijken
Leonie A
LA
Menke
Henri J L M
HJ
Timmers
D000328:Adult; D001827:Body Height / Q000187:drug effects*; D001940:Breast / Q000187:drug effects / Q000254:growth & development; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D005260:Female; D005500:Follow-Up Studies; D019382:Human Growth Hormone / Q000008:administration & dosage; D006801:Humans; D010074:Oxandrolone / Q000008:administration & dosage*; D016032:Randomized Controlled Trials as Topic; D016896:Treatment Outcome; D014424:Turner Syndrome / Q000188:drug therapy*; D014770:Virilism / Q000139:chemically induced
D019382:Human Growth Hormone; D010074:Oxandrolone