Table 1.

Recent studies published (from 2008-present) for treating precocious puberty on girls with MAS

Author (ref #)	Year of Pub	Drug (Action)	N (completed therapy)	Study Design	Duration of Treatment	Efficacy	Safety Concerns
Mieszczak, J., et al. (18)	2008	Anastrozole (3rd Generation Aromatase inhibitor)	28 (27)	Prospective, International, Multi-institutional, Open-label	12 months	No change in menses or BA advancement. GV z-scores decreased but not statistically significant	No significant adverse outcomes
Sims, EK., et al. (23)	2012	Fulvestrant (Pure Estrogen Receptor Blocker)	30 (29)	Prospective International, Multi-institutional, Open-label trial	12 months	Decreased menses*, decrease in BA advancement*. No statistically significant change in mean GV, no change in PAH	Generally well tolerated, most common event reported was reaction at the site (n=7), also reported vomiting (n=1), abdominal pain (n=1). No serious treatment-related adverse events
de, G.B.P.C., et al. (22)	2015	Tamoxifen (Selective Estrogen Receptor Modulator)	8	Retrospective	3–8 years	Stabilization of bone maturation*, cessation of menses, PAH increased* (however the 4 patients who reached FH were less than predicted)	No adverse events
Estrada, A., et al. (20)	2016	Letrozole (3rd Generation Aromatase Inhibitor)	28	Retrospective, Cohort study, Single center	6 months to 10.9 years	Decrease in BA advancement*, decreased GV z-scores*, decreased menses*, PAH increased*	No adverse events. No additional cases of ovarian torsion, one noted in initial pilot study

^*p<0.05

BA: Bone Age

GV: Growth Velocity

PAH: Predicted Adult Height