2015 Fiscal Year Final Research Report
Sex Hormone replacement therapy in patients with Prader-Willi syndrome
| Project/Area Number |
24791086
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| Research Category |
Grant-in-Aid for Young Scientists (B)
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| Allocation Type | Multi-year Fund |
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| Research Field |
Pediatrics
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| Research Institution | Dokkyo Medical University |
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Principal Investigator |
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| Project Period (FY) |
2012-04-01 – 2016-03-31
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| Keywords | Prader-Willi症候群 / 性腺機能低下症 / ホルモン補充療法 / 成人 |
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| Outline of Final Research Achievements |
Prader-Willi syndrome (PWS) has characteristics symptoms, short stature, intellectual disability, behavioral problems and hypogonadism, obesity, and reduced bone and muscle mass. Pubertal change, body composition and behavior were evaluated before and after 24 months of therapy. Serum testosterone, LH and FSH did not change. Increased pubic hair was observed in 16 of 22 patients (72.7%). Percent body fat decreased from 47.55 ± 2.06% to 39.75 ± 1.60% (n = 18) (p = 0.018). Bone mineral density increased from 0.8505 ± 0.0426 g/cm2 to 0.9035 ± 0.0465 g/cm2 (n = 18) (p = 0.036), and lean body mass increased from 18093.4 ± 863.0 g to 20312.1 ± 1027.2 g (n = 18) (p = 0.009). The Modified Overt Aggression Scale was unchanged, at 4.5 ± 2.0 and 3.0 ± 1.7 indicating no increase in aggression, and no behavioral problems were observed. Based on this pilot study, testosterone replacement with 125mg monthly is a potentially safe and useful intervention for adult males with PWS.
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| Free Research Field |
臨床遺伝学
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