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成人生长激素缺乏症生长激素替代治疗 10 年生活质量变化:垂体功能减退控制和并发症研究分析。

Ten-year change in quality of life in adults on growth hormone replacement for growth hormone deficiency: an analysis of the hypopituitary control and complications study.

机构信息

Lilly Diabetes (D.M., R.Q.), Eli Lilly and Company, Indianapolis, Indiana 46285; Lilly Diabetes (W.F.B.), Eli Lilly and Company, Bad Homburg 61352, Germany; Lilly France (M.R.), 92521 Neuilly sur Seine, France; Department of Endocrinology/Medicine and CIBERER 747 (S.M.W.), Hospital S Pau, Universitat Autonoma de Barcelona, 08193 Cerdanyola del Vallès, Barcelona, Spain; Department of Endocrinology (C.J.S.), and Diabetes and Nutritional Medicine, Charité-Universitätsmedizin, Campus Mitte, 13353 Berlin, Germany.

出版信息

J Clin Endocrinol Metab. 2014 Dec;99(12):4581-8. doi: 10.1210/jc.2014-2892.

DOI:10.1210/jc.2014-2892
PMID:25233155
Abstract

CONTEXT

Previous studies showed improvement in impaired quality of life (QoL) in adult patients with growth hormone (GH) deficiency (GHD) who were treated with GH; improvement was sustained over a few years after GH therapy.

OBJECTIVE

To evaluate the QoL over 10 years.

DESIGN

This was a prospective observational study.

SETTING

The study was conducted in clinical practice.

PATIENTS

1436 adult patients with adult-onset (AO) GHD (mean age [standard deviation (SD)]: 49.0 [12.2] years; 49% female) and 96 with childhood-onset (CO) GHD (31.3 [10.0] years; 60% female) (total N = 1532).

INTERVENTION

GH therapy.

MAIN OUTCOME MEASURES

QoL was measured by Questions on Life Satisfaction-Hypopituitarism (QLS-H) in countries where validated questionnaires and normative data for calculation of Z-scores were available. Change in QoL was tested by Student's t test and predicted by mixed-model repeated measures (MMRM) analysis.

RESULTS

At study entry, patients had diminished QoL Z-scores (mean [SD] AO, -1.55 [1.69]; CO -0.98 [1.32]). The largest QoL improvements were in the first year: mean (SD) increase 0.77 (1.37) for AO (P < .001) and 0.50 (1.37) for CO (P < .001). The initial improvement from study entry remained statistically significant throughout 10 years for AO and in years 1 to 4, 6, and 7 for CO (P < .05). MMRM analysis predicted a greater QoL improvement in those who were not depressed, lived in Europe, had poorer Z-scores at entry, had lower body mass index at entry, and had no impaired vision.

CONCLUSION

These data suggest that GH replacement provides sustained improvement in QLS-H scores toward normality for up to 10 years.

摘要

背景

先前的研究表明,接受生长激素(GH)治疗的成人 GH 缺乏症(GHD)患者的生活质量(QoL)受损得到改善;GH 治疗后几年内持续改善。

目的

评估 10 年期间的 QoL。

设计

这是一项前瞻性观察研究。

设置

该研究在临床实践中进行。

患者

1436 名成年起病(AO)GHD 患者(平均年龄[标准差]:49.0[12.2]岁;49%为女性)和 96 名儿童起病(CO)GHD 患者(31.3[10.0]岁;60%为女性)(共 1532 名患者)。

干预措施

GH 治疗。

主要观察指标

在有经过验证的问卷和计算 Z 分数的正常数据的国家,通过生活满意度-垂体功能减退症问卷(QLS-H)评估 QoL。通过学生 t 检验测试 QoL 的变化,并通过混合模型重复测量(MMRM)分析预测。

结果

在研究开始时,患者的 QoL Z 分数降低(AO 的平均值[标准差]为-1.55[1.69];CO 为-0.98[1.32])。在第一年,QoL 改善最大:AO 的平均(标准差)增加 0.77(1.37)(P<.001),CO 的平均(标准差)增加 0.50(1.37)(P<.001)。从研究开始到 10 年,AO 的初始改善仍具有统计学意义,而在 CO 的第 1 年至第 4 年、第 6 年和第 7 年也具有统计学意义(P<.05)。MMRM 分析预测,无抑郁、居住在欧洲、入组时 Z 分数较低、入组时体重指数较低且无视力受损的患者 QoL 改善更大。

结论

这些数据表明,GH 替代治疗可使 QLS-H 评分持续改善,达到正常水平,最长可达 10 年。

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