ESPE Abstracts (2016) 86 P-P2-49

Evaluation of the Combination of Anti-androgen and Anti-estrogen Treatment in Classical Congenital Adrenal Hyperplasia in Boys: Retrospective Study of 11 Cases

Christine Lefevre, Jacques Weill, Harmony Mazoyer & Maryse Cartigny

Pediatric Endocrinology Lille University Hospital, Lille, France

Background: Final height (FH) is reduced in congenital adrenal hyperplasia (CAH), due both to overtreatment by hydrocortisone therapy and to advanced epiphyseal closure linked to hyperandrogenism by Hydrocortisone therapeutic insufficiency.

Objective and hypotheses: To evaluate the efficacy and safety of the addition of an androgen receptor competitor and an aromatase inhibitor to reduced hydrocortisone doses.

Method: Retrospective study. Eleven boys (chronological age 9.4±1.6 years, bone age -BA- 12.3±1.6 years, initially predicted final height –IPFH- −1.5±1.3 SDS) were administered a treatment combining hydrocortisone (6 mg/m2/d instead of 15.6±9.9 mg/m2/d previously), Testolactone (40 mg/kg/d) and later letrozole (2.5 mg/kg/d) and flutamide (10 mg/kg/d). The additive treatment was stopped when statural age = bone age or when bone age was 14 years. The average chronological age at the end of the treatment was 13.2±2 years. FH or near FH (NFH) were observed.

Results: FH or NFH were 171.3±6.6 cm (−0.7±1.1 SDS). FH or NFH minus IPFH was 0.9±0.6 SDS. No hepatic nor renal side effect were observed. Hydrocortisone dose reduction did not result into any sign of adrenal insufficiency and bone mineral density remained normal in 4/11 studied patients. 6/10 patients developed testicular adrenal rest tumours. Among four patients investigated at the end of treatment, three suffered from severe semen abnormalities.

Conclusion: The addition of anti-androgen and aromatase inhibitor to Hydrocortisone treatment has improved the prognosis of FH in male CAH patients. The combined treatment can be considered as generally safe, excepted for a presumption of impaired fertility. However, it must be taken into consideration that included patients are the most affected ones. Notwithstanding ethical issues, subsequent randomized studies are theoretically needed to evaluate precisely the accountability of the additive anti-androgen and anti-estrogenic treatment in most severe CAH.

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