Design of the Growth hormone deficiency and Efficacy of Treatment (GET) score and non-interventional proof of concept study

Kann, Peter H.; Bergmann, Simona; Bidlingmaier, Martin; Dimopoulou, Christina; Pedersen, Birgitte T.; Stalla, Günter K.; Weber, Matthias M. und Meckes-Ferber, Stefanie (2018): Design of the Growth hormone deficiency and Efficacy of Treatment (GET) score and non-interventional proof of concept study. In: BMC Endocrine Disorders 18:10

Abstract

Background: 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. Methods: 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. Results: 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). Conclusions: 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.

• Dokumententyp: Zeitschriftenartikel
• Fakultät: Medizin
• Themengebiete: 600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin und Gesundheit
• ISSN: 1472-6823
• Sprache: Englisch