Kann Peter H, Bergmann Simona, Bidlingmaier Martin, Dimopoulou Christina, Pedersen Birgitte T, Stalla Günter K, Weber Matthias M, Meckes-Ferber Stefanie
Division of Endocrinology & Diabetology, Philipp's University Marburg, D-35033, Marburg, Germany.
Endocrine Laboratory, Medizinische Klinik und Poliklinik IV, Ludwig-Maximilians University, 80336, Munich, Germany.
BMC Endocr Disord. 2018 Feb 13;18(1):10. doi: 10.1186/s12902-018-0237-3.
The adverse effects of growth hormone (GH) deficiency (GHD) in adults (AGHD) on metabolism and health-related quality of life (HRQoL) can be improved with GH substitution. This investigation aimed to design a score summarising the features of GHD and evaluate its ability to measure the effect of GH substitution in AGHD.
The Growth hormone deficiency and Efficacy of Treatment (GET) score (0-100 points) assessed (weighting): HRQoL (40%), disease-related days off work (10%), bone mineral density (20%), waist circumference (10%), low-density lipoprotein cholesterol (10%) and body fat mass (10%). A prospective, non-interventional, multicentre proof-of-concept study investigated whether the score could distinguish between untreated and GH-treated patients with AGHD. A 10-point difference in GET score during a 2-year study period was expected based on pre-existing knowledge of the effect of GH substitution in AGHD.
Of 106 patients eligible for analysis, 22 were untreated GHD controls (9 females, mean ± SD age 52 ± 17 years; 13 males, 57 ± 13 years) and 84 were GH-treated (31 females, age 45 ± 13 years, GH dose 0.30 ± 0.16 mg/day; 53 males, age 49 ± 15 years, GH dose 0.25 ± 0.10 mg/day). Follow-up was 706 ± 258 days in females and 653 ± 242 days in males. The GET score differed between the untreated control and treated groups with a least squares mean difference of + 10.01 ± 4.01 (p = 0.0145).
The GET score appeared to be a suitable integrative instrument to summarise the clinical features of GHD and measure the effects of GH substitution in adults. Exercise capacity and muscle strength/body muscle mass could be included in the GET score.
NCT number: NCT00934063 . Date of registration: 02 July 2009.
生长激素（GH）缺乏症（GHD）对成人（AGHD）的代谢及健康相关生活质量（HRQoL）的不良影响可通过GH替代治疗得到改善。本研究旨在设计一个评分系统，总结GHD的特征，并评估其测量AGHD中GH替代治疗效果的能力。
生长激素缺乏症及治疗效果（GET）评分（0 - 100分）评估（加权）：HRQoL（40%）、因病缺勤天数（10%）、骨密度（20%）、腰围（10%）、低密度脂蛋白胆固醇（10%）和体脂量（10%）。一项前瞻性、非干预性、多中心概念验证研究调查了该评分是否能区分未接受治疗和接受GH治疗的AGHD患者。基于此前关于AGHD中GH替代治疗效果的认识，预计在为期2年的研究期间GET评分会有10分的差异。
106例符合分析条件的患者中，22例为未接受治疗的GHD对照（9例女性，平均±标准差年龄52±17岁；13例男性，57±13岁），84例接受GH治疗（31例女性，年龄45±13岁，GH剂量0.30±0.16mg/天；53例男性，年龄49±15岁，GH剂量0.25±0.10mg/天）。女性随访时间为706±258天，男性为653±242天。未治疗对照组和治疗组的GET评分存在差异，最小二乘均值差异为 + 10.01±4.01（p = 0.0145）。
GET评分似乎是一个合适的综合工具，可用于总结GHD的临床特征并测量成人中GH替代治疗的效果。运动能力和肌肉力量/身体肌肉量可纳入GET评分。
NCT编号：NCT00934063。注册日期：2009年7月2日。
