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对普拉德-威利综合征成人患者进行的五年生长激素(GH)治疗

Five-years growth hormone (GH) treatment in adults with Prader-Willi syndrome.

作者信息

Höybye Charlotte

机构信息

Department of Endocrinology, Metabolism and Diabetology, Karolinska University Hospital, Stockholm, Sweden.

出版信息

Acta Paediatr. 2007 Mar;96(3):410-3. doi: 10.1111/j.1651-2227.2006.00051.x.

DOI:10.1111/j.1651-2227.2006.00051.x
PMID:17407467
Abstract

AIM

We have previously shown that 1 year of growth hormone (GH) treatment to adults with Prader-Willi syndrome (PWS) has beneficial effects on body composition. The aim of the present observational study was to re-evaluate our cohort, with focus on long-term GH treatment.

METHODS

Seven men and seven women, median age 31 years, were available for follow-up for 6 years. Nine were on GH treatment for 5 years. Body composition was measured with Dual Energy X-ray absorptiometry (DXA).

RESULTS

In six GH treated patients with genetically verified PWS there was a substantial increase in lean body mass of 5 kg (p = 0.031) and a concomitant, however, non-significant, decrease in body fat of 5% (p = 0.156). The changes in the genetically verified patients without GH treatment were small and unsystematic. No compliance problems were reported. Only one non-GH-treated woman developed overt diabetes.

CONCLUSION

Despite inherent behavioural problems it was possible to continue GH injections for 5 years with sustained favourable effects on body composition without clinically, significant side effects.

摘要

目的

我们之前已表明,对普拉德-威利综合征(PWS)成人患者进行1年的生长激素(GH)治疗对身体成分有有益影响。本观察性研究的目的是重新评估我们的队列,重点关注长期GH治疗。

方法

7名男性和7名女性,中位年龄31岁,可进行6年的随访。其中9人接受了5年的GH治疗。采用双能X线吸收法(DXA)测量身体成分。

结果

在6例经基因验证的接受GH治疗的PWS患者中,去脂体重显著增加5千克(p = 0.031),然而,体脂同时下降5%,但差异无统计学意义(p = 0.156)。未经GH治疗的经基因验证患者的变化较小且无系统性规律。未报告依从性问题。只有1名未接受GH治疗的女性患明显糖尿病。

结论

尽管存在固有的行为问题,但仍有可能持续进行5年的GH注射,对身体成分持续产生有利影响,且无临床显著副作用。

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