Essential tremor is a common nervous system disorder affecting movement and the usual function of parts of the body, as well as the awareness and thinking capabilities. It is estimated that around 1,000,000 people in the UK have it. It is understood the number of patients affected may be even higher than previously reported, largely because of issues with the diagnosis of patients, and challenges relating to treatment. Therefore, there is a need for a more thorough understanding of the effect and management of patients outside of clinical trials and in the real world.
This study will look back at anonymous GP or hospitals records of patients diagnosed with essential tremors between 2010 and 2021 in the UK. In doing this, an up-to-date overview of the disease will be provided by describing the characteristics of patients diagnosed with the disease and also provide a summary of the treatments they received after their diagnosis. Furthermore, the resources that were used as part of patient care, as well as the costs of patient care will be investigated. The findings from our study will inform on the extent of the burden of essential tremors in the UK, which will be useful in bringing public health awareness as well as guide the strategies in improving care and management of patients suffering from the disease.
There is an increasing need for a comprehensive understanding of the effect and management of essential tremor (ET) in the real-world. This study aims to characterise ET patient profiles by describing i) epidemiology, ii) patient characteristics and treatment patterns iii) healthcare resource utilization (HCRU) and costs, and iv) specialties of diagnosing/treating physicians. Exploratorily, we will investigate (i) mortality (ii) comorbidity and cardiovascular risk profile and (iii) incremental HCRU and cost burden compared to a matched healthy cohort.
This retrospective cohort study will use primary care data from CPRD Aurum, independently (epidemiology) or linked to secondary care (HES Outpatient and Admitted Patient Care) from 01/01/2010-31/12/2021 (CPRD only; 31/03/2021 linked to HES; or latest available). Patients with a confirmed ET diagnosis will be analysed, conditional on fulfilment of selection criteria.
Yearly ET epidemiology (point prevalence [2010 to 2021] and cumulative incidence [2011 to 2021]) will be determined and estimates standardized by age/sex will be extrapolated to the overall UK population. Patient characteristics will be summarized using appropriate descriptive statistics in the 12-month baseline period or follow-up post-diagnosis as applicable. Treatment use will be evaluated from 2010 to 2021 among the prevalent population and post-diagnosis for an overall newly diagnosed cohort, among which treatment sequences will be visualized using Sankey diagrams. Kaplan Meier analyses will describe time to treatment discontinuation, switch, non-persistence, and overall survival. HCRU and costs (extrapolated) post-ET diagnosis will be described longitudinally and cross-sectionally in the latest available calendar year. Following propensity score matching of the newly diagnosed ET cohort to a healthy cohort without ET, the incremental burden of ET will be assessed.
Completion of the proposed study will result in various public health benefits, including an up-to-date real-world epidemiologic profile and characterisation of patients with ET in the UK, describing the burden of disease and elucidating unmet needs.
Primary outcomes: Point prevalence at the end of each calendar year from 2010 to 2021; Cumulative incidence in each calendar year from 2011 to 2021;
Key secondary outcomes: Patient characteristics of the prevalent and newly diagnosed ET populations; Pharmacological and non-pharmacologic treatment use among the prevalent and newly diagnosed ET populations; Treatment sequences among the newly diagnosed ET population; Time-to-event outcomes including time to treatment initiation, discontinuation, switch, and non-persistence; GP/nurse consultations and extrapolated costs; All-cause and ET-related outpatient hospital specialist visits and extrapolated costs; All-cause and ET-related hospitalizations and extrapolated costs; ET-related prescriptions and extrapolated costs; Top inpatient/outpatient procedures; outpatient specialties of ET treating/diagnosing physicians
Marco Ghiani - Chief Investigator - Ingress-health HWM GmbH
Evi Zhuleku - Corresponding Applicant - Ingress-health HWM GmbH
Karen Appiah - Collaborator - Cytel Statistical Services and Software UK Limited
Keltie McDonald - Collaborator - Cytel Statistical Services and Software UK Limited
Sabrina Müller - Collaborator - Ingress-health HWM GmbH
Thomas Wilke - Collaborator - Ingress-health HWM GmbH
HES Admitted Patient Care;HES Outpatient