Skip to main content

Table 1 Objectives and endpoints

From: Investigating significant health trends in growth hormone treatments registry: rationale, aims and design of a nationwide prospective registry (study protocol)

 

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