Objective | Endpoints | |
---|---|---|
Primary | To analyse somatropin drug utilization within routine clinical practice in a diverse population of children and adults, across all GH products To determine the place of long-acting GH in practice | Utilized GH preparations Dose and dosing schedule (daily vs. long-acting) Duration of treatment Persistence Switches between GH treatments Interruption/ discontinuation of GH treatment |
Secondary | Efficacy To describe the long-term efficacy of somatropin To assess the transition from adolescence to adulthood | Paediatric indications: Real final adult height (using stadiometer) Adult indication: Body composition (all methods) Delta IGF-I standard deviation score (SDS, change during treatment / normalization) Delta height standard deviation score (SDS) / delta lean body mass Body proportions (sitting height; standing height), Delta target height vs height SDS Lipid status, inflammation markers, IGF-I, blood pressure Analyses stratified according to different diagnosis groups |
Persistence | Persistence calculated as duration of time from initiation to discontinuation of therapy | |
Patient related outcomes (PRO) To describe patient PRO, including quality of life and patient experience over the initial 2 years of GH treatment, cross-sectional and long term (children and adults) | Satisfaction with treatment experience Quality of life, e.g., by SF-12 (in adults and adolescents) Additional validated self-reported or observed (e.g., parent- or partner-rated) QoL and PRO questionnaires can optionally be used | |
Safety To describe the long-term safety of somatropin | Number and rate per patient-years of Adverse events All causally related and/or temporally associated AEs | |
Exploratory | Exploratory: To assess / analyse additional parameters potentially modulated by GHT To assess subgroups of the population under observation, or to assess outcomes by participating centres | e.g., body composition by DXA |