Exploring the efficacy of testosterone undecanoate in male children with 5α-reductase deficiency

Importance:Children with 5-alpha-reductase deficiency(5α-RD)and hypospadias present with micropenis,which makes it difficult to obtain sufficient tissue for urethral reconstruction.Objective:We investigated the therapeutic effects of oral testosterone undecanoate and established a standard androgen treatment protocol for patients with 5α-RD with micropenis.Methods:Patients with 5α-RD were treated with oral testosterone undecanoate for 3 months as a course.All patients were treated with no more than 3 courses.If the penile length(PL)reached 2.5 cm(the minimum criterion for surgery)or greater than or equal to−2.5 standard deviations(SDs)(lower limit of normal),testosterone undecanoate was considered to be effective.Results:The median age of 90 patients with 5α-RD was 1.7 years(0.9,3.1 years).The baseline PL was 1.9±0.6 cm before treatment.At the end of the first course,the PL of 63 patients(70%)reached 2.5 cm,and 49 patients(54%)reached greater than or equal to−2.5 SDs.After two treatment courses,the PL of 81 patients(90%)reached 2.5 cm,and 90 patients(100%)reached greater than or equal to−2.5 SDs.After three courses,the PL of all patients reached 2.5 cm,and all patients reached a PL greater than or equal to−2.5 SDs.No abnormal increase was observed in height-SD score,weight-SD score,or ratio of bone age to chronological age during the 1-3-year follow-up.Interpretation:After 3-9 months of treatment,PL increased to the target length.No severe adverse reactions were observed during follow-up.Testosterone undecanoate was safe and effective in children with 5α-RD with micropenis.