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The Aromatase Inhibitor Anastrozole Is Ineffective in the Treatment of Precocious Puberty in Girls with McCune-Albright Syndrome

Department of Pediatrics (J.M., E.A.E.), Section of Pediatric Endocrinology, Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, Indiana 46202; AstraZeneca (E.S.L.), Wilmington, Delaware 19850; and EUSA Pharma (P.P.), Doylestown, Pennsylvania 18901

Address all correspondence and requests for reprints to: Jakub Mieszczak M.D., James Whitcomb Riley Hospital for Children, 702 Barnhill Drive, Room 5960, Indianapolis, Indiana 46202. E-mail: jmieszcz{at}iupui.edu.

Context: 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.

Design and Settings: This was a prospective international multicenter study in which subjects received anastrozole 1 mg daily for 1 yr.

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

Main Outcome Measures: 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 BA/CA, 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.