Safety and appropriateness of growth hormon trreatments in Europe
Study Description
Background: Since 1985 recombinant growth hormone (GH) has been available. Initially it was used to treat cases of primary severe GH deficiency (GHD) but its use has since multiplied. Nowadays, various indications for GH treatment are included where childhood short stature is not only primarily due to deficient endogenous GH secretion, such as Turner syndrome, children born short for gestational age, chronic renal failure, idiopathic short stature and childhood cancer.
Efficacy of GH in children with severe GHD is undisputed and short term safety during treatment is generally considered satisfactory. For other indications, data on quality of life and long-term safety are still limited due to low numbers of patients within individual countries. Several experimental studies have raised concerns with regards to cancer risk and long-term mortality.
To overcome the problem of low numbers, an international consortium consisting of investigators from eight different European countries, including Switzerland, was constituted to study “Safety and Appropriateness of Growth hormone treatments in Europe” (SAGhE).
Aims
We aim to establish a cohort of young adults treated with GH in childhood due to different diagnoses, to describe the frequency of GH use in Switzerland since 1985, to assess the long-term efficacy and quality of life and to investigate the long-term safety of GH-treatment with focus on the risk of cancer and mortality in this cohort.
Methods
To identify all eligible patients, we will collect information from:
a) Patient lists of all paediatric endocrinology departments and practicing paediatric endocrinologists in Switzerland;
b) Databases: KIGS – Pfizer International Growth Database; databases of the SCCR, the Swiss Childhood Cancer Survivor Study and the Swiss Paediatric Renal Registry.
Relevant data will be extracted from hospital records and registry databases. Assessment of quality of life will be done via questionnaire survey. The number of cancer diagnoses and mortality will be assessed by deterministic and probabilistic linkage of the databases with the cantonal cancer registries in Switzerland and with the mortality statistics of the Swiss Federal Statistical Office. For participants living in cantons not covered by a cancer registry, cancer outcomes will be assessed via questionnaire survey.
The risk of cancer and mortality in the cohort will be compared to the risk in the general population by calculating standardized incidence rates and standardized mortality rates. For diagnoses with increased baseline risks (childhood cancer survivors, chronic renal failure), the risk in GH-treated patients will be compared to the risk in untreated patients with similar diagnoses.
Significance
The project will describe the use of GH in Switzerland and analyse long-term safety in the frame of a high-quality international collaborative study. Results will be presented to the public, guidelines committees and health authorities and are likely to influence future recommendations for treatment with GH in children, particularly in children suffering from cancer. Furthermore, prospective data collection will be continued and will serve as a resource for future research.
Publications
Publications can be expected for 2012
Study Team
- Kuehni C. Institute of Social and Preventive Medicine, University of Bern
- Sommer G. Institute of Social and Preventive Medicine, University of Bern
- Kuonen R. Institute of Social and Preventive Medicine, University of Bern
Applicants
- Mullis P. University Children Hospital, Inselspital Bern, Department for Paediatric Endocrinology, Diabetology and Metabolism
- Kuehni C., Bohlius J. Institute of Social and Preventive Medicine, University of Bern
- Clough-Gorr K. NICER, Institute of Social and Preventive Medicine, University of Zurich
- Grotzer M. University Chlidren’s Hospital, Zurich
Partners
Schweizerische Gesellschaft für pädiatrische Endokrinologie und Diabetologie (SPGED)
Funding
EU-FP7 call HEALTH-2007-3.1-5: Better use of medicines
Swiss Cancer League (Oncosuisse)
Project Duration
12.2009 - 11.2012
State
ongoing