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长期未经治疗的生长激素缺乏症(GHD)以及9年生长激素替代治疗对生长激素缺乏症成年患者生活质量(QoL)的影响。

The effect of long-term untreated growth hormone deficiency (GHD) and 9 years of GH replacement on the quality of life (QoL) of GH-deficient adults.

作者信息

Gilchrist F J, Murray R D, Shalet S M

机构信息

Department of Endocrinology, Christie Hospital, Manchester, UK.

出版信息

Clin Endocrinol (Oxf). 2002 Sep;57(3):363-70. doi: 10.1046/j.1365-2265.2002.01608.x.

Abstract

BACKGROUND

Quality of life (QoL) is reduced in GH-deficient adults compared with the normal population. Further support for the role of GH in the maintenance of QoL is derived from short-term studies of GH replacement in severely GH-deficient adults; these have predominantly reported beneficial effects, although the degree of improvement has often been modest. To date, however, there are few data to demonstrate whether this beneficial effect on QoL is maintained in the long term.

PATIENTS AND METHODS

This study consisted of the follow-up of 85 GH-deficient adults who completed the Nottingham Health Profile (NHP) and the Psychological General Well-Being Schedule (PGWB) self-rating questionnaires in 1992, as part of a 12-month double-blind randomized study of GH replacement. In 2001 we attempted to contact all 85 patients and asked them to complete the two questionnaires again. Follow-up data were obtained in 61 patients. The findings were analysed according to whether the individual had received GH continuously since completion of the initial study, received no further GH replacement, or received GH replacement for only part of the intervening time. Both the NHP and the PGWB give a total score and subsection scores for six specific areas of QoL. A high score correlates with increased morbidity in the NHP, and with reduced morbidity in the PGWB.

RESULTS

Fifty-nine patients completed the NHP at both time points. The patients who continued GH (n = 17) had significantly greater morbidity at baseline than patients who opted to discontinue therapy (n = 27), as reflected by the higher scores overall (5.7 +/- 4.0 vs. 2.8 +/- 3.5; P = 0.01) and in the energy subsection (47.0 +/- 34.7 vs. 13.2 +/- 28.6; P < 0.001). Over the study period energy levels improved in the patients who remained on GH therapy (47.0 +/- 34.7 vs. 25.7 +/- 31.0; P = 0.04). By contrast, a deterioration in the physical mobility subsection (2.4 +/- 5.4 vs. 8.2 +/- 16.7; P = 0.04) occurred in the patients who did not continue GH therapy, and no change occurred in the energy subsection. In the 36 patients who completed the PGWB significant differences were observed at baseline between patients who received GH replacement continuously (n = 10) and those who discontinued therapy (n = 21) in the overall score (67.2 +/- 14.1 vs. 86.8 +/- 14.7; P = 0.001); and in the subsections for anxiety (P = 0.04), depression (P = 0.04), well-being (P = 0.001), self-control (P = 0.04) and vitality (P < 0.001); the greater morbidity at baseline being observed in the patients who continued GH replacement. In the patients receiving GH continuously for 9 years the vitality subsection score improved significantly (7.7 +/- 2.4 vs. 12.5 +/- 3.2; P = 0.003), whereas no change in vitality score occurred in patients who did not continue GH therapy. The change in the energy subsection of the NHP and vitality subsection of the PGWB over the 9 years of the study were significantly different between the patients who opted to continue GH replacement and those who discontinued therapy (P = 0.008 and P < 0.001, respectively).

CONCLUSION

During this 9-year study, small but significant declines in health were observed in GH-deficient adults who remained untreated. By contrast, the patients who received GH continuously experienced improvements in energy levels while all other areas of QoL were maintained. The beneficial effects of GH on QoL are therefore maintained with long-term GH replacement and obviate the reduction in QoL seen over time in untreated GH-deficient adults.

摘要

背景

与正常人群相比,生长激素缺乏的成年人生活质量(QoL)较低。生长激素在维持生活质量方面作用的进一步证据来自对严重生长激素缺乏成年人进行生长激素替代治疗的短期研究;这些研究主要报告了有益效果,尽管改善程度通常不大。然而,迄今为止,几乎没有数据表明这种对生活质量的有益影响能否长期维持。

患者与方法

本研究对85名生长激素缺乏的成年人进行了随访,这些患者在1992年完成了诺丁汉健康量表(NHP)和心理总体幸福感量表(PGWB)自评问卷,这是一项为期12个月的生长激素替代双盲随机研究的一部分。2001年,我们试图联系所有85名患者,并要求他们再次完成这两份问卷。61名患者获得了随访数据。根据个体在完成初始研究后是否持续接受生长激素治疗、未接受进一步的生长激素替代治疗或仅在部分干预时间接受生长激素替代治疗对结果进行分析。NHP和PGWB都给出了生活质量六个特定领域的总分和子项分数。NHP中高分与发病率增加相关,PGWB中高分与发病率降低相关。

结果

59名患者在两个时间点都完成了NHP。持续接受生长激素治疗的患者(n = 17)在基线时的发病率明显高于选择停止治疗的患者(n = 27),总体得分(5.7±4.0对2.8±3.5;P = 0.01)和精力子项得分(47.0±34.7对13.2±28.6;P < 0.001)均反映了这一点。在研究期间,继续接受生长激素治疗的患者精力水平有所改善(47.0±34.7对25.7±31.0;P = 0.04)。相比之下,未继续接受生长激素治疗的患者身体活动子项出现恶化(2.4±5.4对8.2±16.7;P = 0.04),精力子项没有变化。在完成PGWB的36名患者中,持续接受生长激素替代治疗的患者(n = 10)和停止治疗的患者(n = 21)在基线时总体得分(67.2±14.1对86.8±14.7;P = 0.001)存在显著差异;在焦虑(P = 0.04)、抑郁(P = 0.04)、幸福感(P = 0.001)、自我控制(P = 0.04)和活力(P < 0.001)子项也存在显著差异;继续接受生长激素替代治疗的患者在基线时发病率更高。在持续接受生长激素治疗9年的患者中,活力子项得分显著改善(7.7±2.4对12.5±3.2;P = 0.003),而未继续接受生长激素治疗的患者活力得分没有变化。在研究的9年中,选择继续接受生长激素替代治疗的患者与停止治疗的患者在NHP精力子项和PGWB活力子项的变化存在显著差异(分别为P = 0.008和P < 0.001)。

结论

在这项为期9年的研究中,未接受治疗的生长激素缺乏成年人健康状况出现了虽小但显著的下降。相比之下,持续接受生长激素治疗的患者精力水平得到改善,同时生活质量的所有其他方面得以维持。因此,生长激素替代治疗对生活质量的有益影响在长期治疗中得以维持,避免了未治疗的生长激素缺乏成年人随时间推移出现的生活质量下降。

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