An observational study of the characteristics, epidemiology and healthcare resource utilisation of patients with type 1 diabetes pre, peri, and post diagnosis in England.

Study type
Protocol
Date of Approval
Study reference ID
23_003695
Lay Summary

People with type 1 diabetes cannot make enough of the hormone insulin and need to have insulin injections throughout the day to make sure the amount of sugar in the blood is not too high. Balancing blood sugar levels can be challenging, especially during periods of illness and for younger patients. High blood sugar levels can affect organs in the body and lead to both acute and long-term complications that might require hospital admission, increase mortality and impact quality of life.

A research trial (TN-10) has shown a drug, Teplizumab, can delay the onset of type 1 diabetes in people who are over 8 years old, and at high risk of having the disease. The trial only included patients from the United States, Canada, Australia, and Germany. The planned study will describe the characteristics of patients with type 1 diabetes before, at and after diagnosis using English electronic healthcare data. The planned study will look at how similar the trial patients were to English patients with type 1 diabetes, to see whether the results of the TN-10 trial are relevant in the UK context. There will be a particular focus on whether age at diagnosis affects diabetes outcomes. The planned study hopes to understand more about type 1 diabetes patients in England to find out whether a delay to the start of type 1 diabetes could help patients to better manage their condition, and experience fewer complications over time.

Technical Summary

This research aims to characterise type 1 diabetes mellitus (T1DM) patients in England throughout pre, peri and post diagnosis. The aim is to investigate how delaying age at diagnosis of T1DM might affect patient outcomes and health resource utilisation. To establish the potential value of the drug Teplizumab in England should it receive regulatory approval.

The study will describe the demographics, clinical characteristics, comorbidities, complications, resource utilisation, and mortality of T1DM patients. For these descriptive analyses, CPRD Aurum will be used. A matched cohort will be used to compare healthcare resource use and all-cause mortality. Linkage to Hospital Episode Statistics will capture complications and secondary healthcare resource utilisation. ONS Death Data will be used to capture mortality. Patient Rural/ Urban classification and practice-level Index of Multiple Deprivation (IMD) will also be used. The mother-baby link will help describe maternal history of T1DM, as well as, explore rates of mental health diagnoses and healthcare resource use amongst mothers of T1DM children.

The study will explore impact from age at disease onset. A multivariate logistic regression model or mixed model for repeated measures will be used to explore whether age at diagnosis has an impact on average HbA1c in the immediate 2 years following diagnosis. A generalised linear model will explore whether age at disease onset impacts healthcare resource use. Cox regression models will explore time to first diabetes complication and all-cause mortality.

For investigating the comparability of English patients to the TN-10 trial population, both the CPRD Aurum and Gold databases will be explored, to determine availability of data and feasibility.

The research will provide public health benefit by establishing whether a delay in onset of T1DM could have beneficial effects on patient outcomes, and thus inform the potential opportunity represented by a treatment that delays the disease (e.g. Teplizumab).

Health Outcomes to be Measured

The following outcomes will be explored across one or more of the pre, peri and post diagnosis periods; clinical characteristics (i.e. body mass index (BMI); smoking status; history of comorbidities at baseline; Charlson Comorbidity Index (CCI) score; history of specified autoimmune disease; presentation characteristics (i.e. abdominal symptoms); diabetes complications (i.e. diabetic ketoacidosis (DKA) and diabetic nephropathy); measures of healthcare resource (i.e. primary care consultations and referrals); longer term clinical trajectory including HbA1c and time-to diabetes complications; Family history of T1DM; maternal history of T1DM; Mental health diagnoses amongst mothers of T1DM patients; and primary care recording of diabetes-related autoantibodies.

Collaborators

Jennifer Campbell - Chief Investigator - CPRD
Zara Cuccu - Corresponding Applicant - CPRD
Charlie Nicholls - Collaborator - Sanofi Aventis UK Holdings Limited (UK)
LICHEN HAO - Collaborator - Sanofi US Services Inc (USA)
Nancy Cui - Collaborator - Sanofi Aventis Groupe (France)
Richard Hudson - Collaborator - Sanofi Aventis UK Holdings Limited (UK)
Sonia Coton - Collaborator - CPRD
Tao Haskins-Coulter - Collaborator - CPRD
XUAN Li - Collaborator - Sanofi UK
Zara Cuccu - Collaborator - CPRD

Linkages

HES Accident and Emergency;HES Admitted Patient Care;HES Outpatient;ONS Death Registration Data;Practice Level Index of Multiple Deprivation;CPRD Aurum Ethnicity Record;CPRD Aurum Mother-Baby Link;CPRD GOLD Ethnicity Record;CPRD GOLD Mother-Baby Link;Rural-Urban Classification