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Long-term treatment of familial male-limited precocious puberty (testotoxicosis) with cyproterone acetate or ketoconazole

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Summary Background 

Familial male-limited precocious puberty (FMPP) or testotoxicosis is a rare gonadotrophin-independent form of sexual precocity caused by constitutively activating mutations of the LH receptor. Several clinical therapeutic approaches have been reported for this disorder, but with a paucity of long-term outcome data. Objective 

To evaluate the long-term treatment of testotoxicosis with cyproterone acetate or ketoconazole. Design 

A multicentric retrospective clinical study. Patients 

Ten boys from eight unrelated Brazilian families who carried known LH-receptor activating mutations were treated with 70 mg/m2 cyproterone acetate (n = 5) or 10 mg/kg ketoconazole (n = 5) for a mean period of 5 and 8 years, respectively. Measurements 

Chronological and bone ages, bone age/chronological age ratio, target height (TH) range, adult height, basal and GnRH-stimulated gonadotrophin levels and basal testosterone levels were assessed. Results 

Growth velocity decreased significantly during treatment with cyproterone acetate or ketoconazole when compared to pretreatment value in each group (P < 0·05). Bone age/chronological age ratio decreased significantly after cyproterone acetate or ketoconazole therapy. Basal testosterone levels were significantly lower in patients undergoing ketoconazole compared to cyproterone acetate treatment [0·6 ± 0·3 nmol/l (42 ± 21 ng/dl) vs. 5·6 ± 4·0 nmol/l (392 ± 280 ng/dl); P < 0·05], as expected. Secondary gonadotrophin-dependent precocious puberty occurred at a similar frequency (40%) in both groups. Five patients have attained adult height and two patients have already reached 90% of their adult height. Two of them achieved their TH range and one patient, for whom TH was not available, had an adult height of 0·3 SDS. Four boys (two in each group) did not attain their TH range. Conclusion 

Long-term treatment with cyproterone acetate or ketoconazole resulted in similar outcomes without important side-effects in boys with testotoxicosis. However, both therapies showed limited efficacy in attaining normal adult height.
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Document Type: Research Article

Affiliations: 1: Serviço de Endocrinologia Pediátrica, Instituto Materno Infantil, Recife, Brazil, 2: Serviço de Endocrinologia Pediátrica, Universidade de Campinas, Campinas, Brazil and 3: Serviço de Endocrinologia, Faculdade de Medicina da Universidade de São Paulo, Ribeirão Preto, Brazil

Publication date: July 1, 2008

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