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重组人生长激素持续皮下输注治疗婴儿早期先天性小阴茎的疗效与安全性

Efficacy and Safety of Continuous Subcutaneous Infusion of Recombinant Human Gonadotropins for Congenital Micropenis during Early Infancy
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作者信息

Stoupa Athanasia, Samara-Boustani Dinane, Flechtner Isabelle, Pinto Graziella, Jourdon Isabelle, González-Briceño Laura, Bidet Maud, Laborde Kathleen, Chevenne Didier, Millischer Anne-Elodie, Lottmann Henri, Blanc Thomas, Aigrain Yves, Polak Michel, Beltrand Jacques

出版信息

Horm Res Paediatr. 2017;87(2):103-110. doi: 10.1159/000454861. Epub 2017 Jan 12.

Abstract

BACKGROUND

Early postnatal administration of gonadotropins to infants with congenital hypogonadotropic hypogonadism (CHH) can mimic minipuberty, thereby increasing penile growth. We assessed the effects of gonadotropin infusion on stretched penile length (SPL) and hormone levels in infants with congenital micropenis.

METHODS

Single-center study including 6 males with micropenis in case of isolated CHH (n = 4), panhypopituitarism (n = 1), and partial androgen insensitivity syndrome (PAIS; n = 1). Patients were evaluated at baseline, monthly and at the end of the study through a clinical examination (SPL, testicular position and size), serum hormone assays (testosterone, luteinizing hormone, follicle-stimulating hormone, inhibin B, anti-Müllerian hormone [AMH]), and ultrasound of penis/testes.

RESULTS

In CHH, significant increases occurred in serum testosterone (from undetectable level to 3.5 ± 4.06 ng/mL [12.15 ± 14.09 nmol/L]), SPL (from 13.8 ± 4.5 to 42.6 ± 5 mm; p < 0.0001), inhibin B (from 94.8 ± 74.9 to 469.4 ± 282.5 pg/mL, p = 0.04), and AMH (from 49.6 ± 30.6 to 142 ± 76.5 ng/mL, p = 0.03). Micropenis was corrected in all patients, except one. On treatment, in the patient with PAIS, SPL was increased from 13 to 38 mm.

CONCLUSIONS: Early gonadotropin infusion is a safe, well-tolerated and effective treatment. The effect in PAIS has not been reported previously. Long-term follow-up is needed to assess the impact, if any, on future fertility and reproduction.
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摘要

背景

对先天性低促性腺激素性性腺功能减退(CHH)婴儿在出生后早期给予促性腺激素可模拟小青春期,从而促进阴茎生长。我们评估了促性腺激素输注对先天性小阴茎婴儿的阴茎拉伸长度(SPL)和激素水平的影响。

方法

单中心研究,纳入6例小阴茎男性患者,其中孤立性CHH患者4例、全垂体功能减退患者1例、部分雄激素不敏感综合征(PAIS)患者1例。在基线、每月及研究结束时对患者进行评估,评估内容包括临床检查(SPL、睾丸位置和大小)、血清激素检测(睾酮、促黄体生成素、促卵泡生成素、抑制素B、抗苗勒管激素[AMH])以及阴茎/睾丸超声检查。

结果

在CHH患者中,血清睾酮水平显著升高(从不可检测水平升至3.5±4.06 ng/mL[12.15±14.09 nmol/L]),SPL显著增加(从13.8±4.5增至42.6±5 mm;p<0.0001),抑制素B水平升高(从94.8±74.9增至469.4±282.5 pg/mL,p = 0.04),AMH水平升高(从49.6±30.6增至142±76.5 ng/mL,p = 0.03)。除1例患者外,所有患者的小阴茎均得到矫正。在PAIS患者治疗过程中,SPL从13 mm增至38 mm。

结论

早期促性腺激素输注是一种安全、耐受性良好且有效的治疗方法。PAIS患者的治疗效果此前未见报道。需要进行长期随访以评估其对未来生育和生殖的影响(如有)。

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