The aromatase inhibitor anastrozole is ineffective in the treatment of precocious puberty in girls with McCune-Albright syndrome.
BACKGROUND
Precocious puberty (PP) in girls with McCune-Albright syndrome (MAS) is characterized by episodic development of large unilateral ovarian cysts followed by sudden onset of vaginal bleeding. Some patients experience frequent bleeding as well as accelerated linear growth and advanced skeletal maturation. The use of anastrozole for the treatment of PP in this condition has not been well studied.


OBJECTIVE
The objective of the study was to determine the safety and efficacy of the aromatase inhibitor anastrozole for the treatment of PP in girls with MAS.


METHODS
This was a prospective international multicenter study in which subjects received anastrozole 1 mg daily for 1 yr.


METHODS
Twenty-eight girls 10 years of age or younger with MAS and progressive PP were enrolled.


METHODS
Vaginal bleeding, rate of skeletal maturation (change in bone age over change in chronological age), growth velocity, and uterine/ovarian volumes were measured. These indices were compared with a 6-month pretreatment interval.


RESULTS
No difference in vaginal bleeding (mean number of days per year) was noted. Mean change in DeltaBA/DeltaCA, which was 1.25 +/- 0.77 at baseline, was -0.25 +/- 1.02 at study end (P = 0.22). Average growth velocity z score was 1.40 +/- 3.15 at study entry and 0.26 +/- 2.71 at 12 months (P = 0.10). Mean ovarian/uterine volumes were unaffected by anastrozole, and no significant adverse events occurred.


CONCLUSIONS
Although it appears safe, anastrozole for 1 yr was ineffective in halting vaginal bleeding, attenuating rates of skeletal maturation, and linear growth in girls with MAS. Pharmacological strategies other than anastrozole should be pursued for the treatment of PP in this population.
Mieszczak
Jakub
J
James Whitcomb Riley Hospital for Children, 702 Barnhill Drive, Indianapolis, IN 46202, USA. jmieszcz@iupui.edu
Lowe
Elizabeth S
ES
Plourde
Paul
P
Eugster
Erica A
EA
D000077384:Anastrozole; D047072:Aromatase Inhibitors / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005359:Fibrous Dysplasia, Polyostotic / Q000188:drug therapy*; D006801:Humans; D009570:Nitriles / Q000009:adverse effects / Q000627:therapeutic use*; D011446:Prospective Studies; D011629:Puberty, Precocious / Q000188:drug therapy*; D014230:Triazoles / Q000009:adverse effects / Q000627:therapeutic use*; D014592:Uterine Hemorrhage / Q000188:drug therapy
D047072:Aromatase Inhibitors; D009570:Nitriles; D014230:Triazoles; D000077384:Anastrozole