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长期生长激素治疗:流行病学与体格发育结果

Long-Term GH therapy: epidemiology and auxologic outcome.

作者信息

Audí L, Gilabert A, Lloveras G, Martí-Henneberg C, Rodríguez-Hierro F, Vilardell E, Webb S, Canela J, Sedano E, Vicens-Calvet E

机构信息

Growth Hormone and Related Substances Utilization Advisory Council, Catalan Health Service, Barcelona, Spain.

出版信息

Horm Res. 2002;57(3-4):113-9. doi: 10.1159/000057961.

Abstract

OBJECTIVES

Epidemiologic and auxologic characteristics of patients treated with GH during childhood and adolescence and entered in a national registry in Catalonia were studied between 1988 and 1997. At the end of 1997, prevalence was 53.2 treatments/100,000 inhabitants aged 0-14 years. Maximum annual incidence rates were observed in 1990 and 1991 (34.0-35.6 cases/100,000 inhabitants aged 0-14 years).

STUDY DESIGN

Analysis of treatments terminated in 1993 (n = 548) revealed, for the three principal reasons for cessation of treatment ('near-final height', 'adequate height but further growth potential', and 'poor growth response'), that males began and ended treatment at older ages with a better auxologic situation in SDS than girls at the beginning and end of therapy in the first two subgroups, with a similar duration of therapy. Severe GH deficiency (GHD) [both multiple pituitary hormone deficiency (MPHD) and the most severe isolated GHD (IGHD-A)] was more frequent in the group ending treatment at 'near-final height', whereas cessation of therapy because of 'poor growth response' was more frequent in the group with 'other causes of short stature' and no demonstrable GHD by routine tests. In the near-final height group, after excluding Turner's syndrome, MPHD and GHD cases secondary to brain tumors and GH deficiencies associated with malformative syndromes, positive linear correlations were observed between HSDS at the end of treatment and HSDS at the beginning, predicted adult height SDS (PAHSDS) and target height SDS (THSDS). Multiple regression analysis showed that in this group of patients, 41.4% of the variability in HSDS increment can be explained by the equation: HSDS increment = -0.33 + 0.29 THSDS - 0.68 HSDS at the beginning of treatment.

RESULTS

The outcome showed a reasonable use of GH, since good-response cases generally continued treatment until final height whereas therapy was suspended in doubtful cases.

摘要

目的

研究1988年至1997年间在加泰罗尼亚进入国家登记系统的儿童及青少年接受生长激素(GH)治疗的流行病学和体格学特征。1997年末,患病率为每100,000名0至14岁居民中有53.2例接受治疗。1990年和1991年观察到最高年发病率(每100,000名0至14岁居民中有34.0 - 35.6例)。

研究设计

对1993年结束治疗的病例(n = 548)进行分析发现,对于治疗终止的三个主要原因(“接近最终身高”、“身高足够但仍有生长潜力”以及“生长反应不佳”),在前两个亚组中,男性开始和结束治疗的年龄较大,治疗开始和结束时在标准差评分(SDS)方面的体格学状况比女性更好,且治疗持续时间相似。严重生长激素缺乏(GHD)[包括多重垂体激素缺乏(MPHD)和最严重的孤立性生长激素缺乏(IGHD - A)]在因“接近最终身高”而结束治疗的组中更为常见,而因“生长反应不佳”而停止治疗在患有“其他身材矮小原因”且常规检查未显示明显GHD的组中更为常见。在接近最终身高组中,排除特纳综合征、继发于脑肿瘤的MPHD和GHD病例以及与畸形综合征相关的生长激素缺乏后,观察到治疗结束时的身高标准差评分(HSDS)与开始时的HSDS、预测成人身高标准差评分(PAHSDS)和目标身高标准差评分(THSDS)之间存在正线性相关性。多元回归分析表明,在该组患者中,HSDS增量变异性的41.4%可由以下方程解释:HSDS增量 = -0.33 + 0.29 THSDS - 0.68治疗开始时的HSDS。

结果

结果显示生长激素的使用合理,因为反应良好的病例通常持续治疗直至达到最终身高,而可疑病例则暂停治疗。

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