The epidemiology and impact of growth hormone deficiency in adults: A population-based study in the UK

Study type
Protocol
Date of Approval
Study reference ID
22_002256
Lay Summary

Growth hormone deficiency (GHD) is a condition where not enough growth hormone is produced by the brain. It can be present at birth or develop later in life. GHD in adulthood can have significant negative effects on a person’s quality of life and may increase the risk of heart attacks and strokes as well as broken bones. The number of adults in the UK diagnosed and living with GHD, and the impact of GHD on their long-term health, have not been well studied. The purpose of this study is to look at the number of people affect by GHD broken down by age group, sex, ethnicity, social deprivation and geography. It will also investigate if there has been any change in the number of people diagnosed with GHD over the last 20 years and the proportion of people who were born with the condition compared to those acquiring it in adulthood. The study will additionally look at whether GHD increases the risk of people having heart attacks or strokes, of dying, or from suffering a broken hip or leg. This information is important to fill a gap in the current knowledge of GHD, to raise awareness of this condition and to support improved screening and treatment of GHD.

Technical Summary

There have not been any large population-based studies assessing the prevalence and incidence of growth hormone deficiency (GHD), especially in adults. There is also a limited understanding of the impacts of GHD on long term outcomes including cardiovascular disease and mortality. This proposed research will consist of a large population-based epidemiological study of GHD, including an assessment of current management trends in GHD, and the impact of GHD on patient outcomes. All adults (aged ≥ 18) registered in CPRD Aurum with valid linkage to hospital episode statistics, death registration data from the Office for National Statistics, and index of multiple deprivation data, will be eligible. People with GHD will be identified using clinical diagnosis codes recorded in primary care. GHD incidence rates and prevalence will be calculated both overall and in sociodemographic subgroups, using whole CPRD population denominators. Treatment patterns and treatment persistence after GHD diagnosis will be estimated using Kaplan-Meier method. To evaluate GHD outcomes, adults with GHD will be propensity score matched using a 1:4 ratio with unaffected adults. Primary outcomes will be compared: all-cause mortality, a composite of cardiovascular disease (non-fatal myocardial infarction, non-fatal stroke), and lower limb fractures, time and will be assessed prospectively up to 10-years using Cox proportional hazards models adjusted for sociodemographic and additional outcome specific risk factors. Secondary outcomes of time-off work and unemployment will be evaluated using the same approach. All outcomes will also be assessed in a subgroup of the cohort with evidence of GHD treatment. This study will provide robust date on the epidemiology and impact of GHD in adults that will support the clinical management of people with GHD, including screening and treatment, and raise awareness of the condition in clinical practice.

Health Outcomes to be Measured

Primary outcomes:
Incident GHD;
Clinical outcomes comprising: all-cause mortality; cardiovascular disease (non-fatal myocardial infarction, non-fatal stroke), lower limb fractures

Secondary GHD impact outcomes, comprising: growth hormone replacement treatment, time off work for illness, unemployment

Collaborators

Wing Sin Chiu - Chief Investigator - Pfizer Ltd - UK
Emma Jones - Corresponding Applicant - Momentum Data Ltd
Andrew McGovern - Collaborator - Momentum Data Ltd
Anita Lynam - Collaborator - Momentum Data Ltd
Mary Araghi ( Gerino ) - Collaborator - Pfizer Ltd - UK
Muhammad (Ashkan) Dashtban - Collaborator - Momentum Data Ltd
Serhan Bahit - Collaborator - Momentum Data Ltd

Linkages

HES Admitted Patient Care;ONS Death Registration Data;Patient Level Index of Multiple Deprivation