ESPE Abstracts (2019) 92 P3-218

Clinical Observation of Oral Testosterone Undecanoate Treatmentforchildren with 5-Alpha-Reductase Deficiency

Ying Liu1, Chunxiu Gong2

1Department of Pharmacy, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China. 2Department of Endocrinology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China

Objective: Clinical Observation of fifty-two patients with 5-alpha-reductase deficiency (5 - alpha-reductasedeficiency, 5α-RD) with oral testosterone undecanoate. To evaluate the efficacy and drug safety, find the optimum age of medication.

Methods: Prospective self-controlled comparison methods were used to analyze the clinical data and follow-up results in sixty-nine patients who are diagnosed with 5-alpha-reductase deficiency by ** hospital with continuous oral testosterone undecanoate capsules (2~3 mg/Kg/day, maximum dose of 80 mg/day) from March 2009 to April 2018.

Results: Sixty-nine patients with 5-alpha-reductase deficiency were included in the study, ranging in age from 0.1 to 8 years old, with an average age of (2.2±1.7)years old. The clinical manifestations were small penis with different degrees of hypospadias and 4 cases of pure small penis. A total of 68 cases were treated with oral testosterone undecanoate capsules (PL>2.5SD).The effective rate of the first course of treatment was 47.92%, the effective rate of the second course was 91.67%, and the optimal length of penis was reached in the second course. The length of growth of the penis in the first course was (0.83 + 0.47) cm, the length in the second course was (0.60 + 0.35) cm, and the final length was about (3.09 + 0.53) cm. The efficacy of undecanted testosterone before puberty was significant in these patients, and the fitting curve analysis showed that there was no correlation between the efficacy of drug and age before puberty (R2 was far less than 1), and there was no significant difference in the efficacy among all age groups (P>0.05).The height, weight, BMI, bone age and sex hormone levels of the patients were regularly monitored without affecting the physical growth and development of the patients. Two patients were followed up to 18 years old, and there was no significant difference between the DSD of final height and expected target height.

Conclusion: Theseageof 5-alpha-reductase deficiency patients was stratified. Their clinical manifestations was almost small penis with different degrees of hypospadias. The effective rate of oral undecanted testosterone was 91.67%, and the effect of the first course was better than that of the second course before puberty. There was no correlation between drug efficacy and age in prepubertal. The drug's short-term safety was relatively high and had less effect on bone age and target height with these patients.

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