A retrospective cohort study examining the epidemiology and clinical management of immune thrombocytopenic purpura in England

Study type
Protocol
Date of Approval
Study reference ID
22_001945
Lay Summary

Immune thrombocytopenic purpura (ITP) is the name given to a blood condition in which the immune system mistakenly attacks itself. ITP symptoms include easy bruising and bleeding. ITP in some patients may be short lived (i.e., acute) and resolve within a few months or become long lasting (i.e., chronic). Generally, treatment of ITP involves management of symptoms when needed rather than cure.

In this study, we will use anonymous GP and hospital records to estimate the number of people who have ITP in England and describe the characteristics of these individuals. We will also describe the types of treatment people with ITP receive and when treatment is given in relation to the timing of diagnosis and the progression of the condition. Finally, we will describe the healthcare resource use and costs, in terms of GP surgery appointments, hospital admissions, hospital outpatient appointments and accident and emergency attendances, among people with ITP. The results of our study will provide an overview of the healthcare burden associated of ITP and treatments used for this rare condition, to guide any possible improvements and standardisation in patient care to enhance the wellbeing of people living with ITP.

Technical Summary

Immune thrombocytopenic purpura (ITP) is an autoimmune disorder affecting both children and adults, characterised by a platelet count. Clinical manifestations include petechiae, purpura, bruising, and overt bleeding. ITP can be further segregated as being either primary (idiopathic) or secondary. In the UK, the estimated incidence of ITP is 4 per 100,000 person-years, with prevalence in northern Europe estimated at 2.7 per 100,000. Currently treatment options include steroids as first line treatment and a variety of medications that can reduce the destruction of platelets or increase their production as second line.

We aim to provide a descriptive epidemiological summary of primary ITP for a longitudinal patient population using 10 years of Clinical Practice Research Datalink (CPRD), Hospital Episode Statistics (HES), and Office for National Statistics (ONS) deaths registrations linked data. Our descriptive retrospective cohort study will include patients with a recorded diagnosis of primary ITP (using two definitions to explore the impact of sensitivity and specificity). We will subclassify patients with primary ITP as acute, persistent, chronic and chronic with two prescriptions of thrombopoietin receptor agonists. We will estimate incidence and point prevalence and describe the baseline characteristics of patients with primary ITP and its subclassifications. We will also report the number of patients who receive treatment, stratified by monotherapy and combination therapy for immunomodulatory, steroid, and non-immunosuppressants. To assess the treatment pathway further, the order and frequency of treatments will be described. Among a chronic cohort, treatment history will be described as counts and percentages. Similarly, we will report the annual counts and percentages of patients on each treatment to determine changes over time. We will report the primary and secondary care healthcare resource use and cost including inpatient admissions, outpatient appointments, accident and emergency attendances and primary care appointments using means and medians.

Health Outcomes to be Measured

Incidence and prevalence; Treatment provision including immunosuppressive and immunomodulatory therapies; Treatment pathway including order and frequency of treatment; Snapshot of prevalent patients treatment history; Primary care and secondary care HCRU and cost

Collaborators

Jennifer Davidson - Chief Investigator - Health iQ Ltd ( UK ) t/a CorEvitas
Caitlin Winton - Corresponding Applicant - Health iQ Ltd ( UK ) t/a CorEvitas
Archie Farrer - Collaborator - Health iQ Ltd ( UK ) t/a CorEvitas
Caoimhe Rice - Collaborator - Health iQ Ltd ( UK ) t/a CorEvitas
Mico Hamlyn - Collaborator - Health iQ Ltd ( UK ) t/a CorEvitas
Sara Carvalho - Collaborator - Health iQ Ltd ( UK ) t/a CorEvitas

Former Collaborators

Sara Carvalho - Collaborator - Health iQ Ltd ( UK ) t/a CorEvitas

Linkages

HES Accident and Emergency;HES Admitted Patient Care;HES Outpatient;ONS Death Registration Data;Patient Level Index of Multiple Deprivation