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

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.

Trial registration: NCT number: NCT00934063 . Date of registration: 02 July 2009.

Keywords: Clinical study; Growth hormone; Growth hormone deficiency; Quality of life.

Conflict of interest statement

Ethics approval and consent to participate

This study was performed in accordance with the Declaration of Helsinki. Ethical permissions were obtained from the Ethical Commission of the Chamber of Physicians of the German Federal State of Hessia (reference no FF 22/2009).

Written and informed consent for the registration and transfer of their personal data was obtained from all patients.

Consent for publication

Not applicable.

Competing interests

PHK has received research funding, honoraria for lectures and is a member of scientific boards for Novo Nordisk and Pfizer. MB has received research support, consultancy fees and/or speakers honoraria from Novo Nordisk, Pfizer, Sandoz, Novartis, IPSEN, OPKO and Genexine. BTP is an employee of Novo Nordisk A/S, Denmark. GKS has received speaker fees from Novo Nordisk and study investigator sponsored by Novo Nordisk. MMW is member of the NordiNet® ISC Board Novo Nordisk. SM-F is an employee and stockholder of Novo Nordisk Pharma GmbH, Germany. SB and CD have no conflicts of interest to declare.

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Figures

Fig. 1

Estimated difference in the GET score between control and GH-treated groups during follow-up visits (EAS) EAS, effectiveness analysis set; GET, Growth hormone deficiency and Efficacy of Treatment; GH, growth hormone.

Fig. 2

Mean (SD) change in IGF-I SDS from baseline to follow-up visits by gender IGF-I, insulin-like growth factor 1; SD, standard deviation; SDS, standard deviation score.