Long-term outcomes of letrozole treatment for precocious puberty in girls with McCune-Albright syndrome.
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ABSTRACT: OBJECTIVE:McCune-Albright syndrome (MAS) is a rare disorder with a broad spectrum including precocious puberty (PP) due to recurrent estrogen-secreting ovarian cysts. This study evaluates the long-term safety and efficacy of letrozole treatment in large cohort of girls with MAS-associated PP. DESIGN:Retrospective cohort analysis. METHODS:Clinical data, including history and physical examination, bone age, and pelvic ultrasounds, were reviewed on 28 letrozole-treated girls. Adult height was reviewed for 42 historical controls. Outcomes included rate of skeletal maturation, growth velocity, predicted adult height and adult height. RESULTS:Twenty-eight girls received letrozole treatment. Treatment duration was 4.1?±?2.6 years (mean?±?1 s.d.) (range: 0.5-10.9) and mean follow-up was 6.0?±?3.3 years (range: 0.5-15.0), for a total of 135.9 person-years of follow-up. Letrozole treatment was highly effective at decreasing the rate of skeletal maturation, with a decline in change in bone age over change in chronological age (?BA/?CA) from 1.7 (IQR: 2.3) to 0.5 (IQR: 0.4) (P?
PROVIDER: S-EPMC5066167 | BioStudies |
REPOSITORIES: biostudies
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