An investigation of the adverse outcomes and resource use associated with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) and their relationship with glucocorticoid (GC) treatment

Study type
Protocol
Date of Approval
Study reference ID
18_190
Lay Summary

Anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV) is a rare, serious and often life-threatening disease. AAV is associated with a number of adverse outcomes including the development of infections, heart and circulation problems and cancers. A number of effective treatments are available for AAV, however these treatments are themselves associated with adverse outcomes.

This study will utilise data collected in the course of routine clinical practice to extract data on a group of patients in England with AAV and will describe the frequency of occurrence of adverse outcomes (including infections, bone disease, heart and circulation disease, renal disease, eye conditions, metabolic conditions (e.g. diabetes) and mental health conditions) and the frequency of healthcare use (including general practice consultations, general practice prescriptions, inpatient hospital visits, outpatient visits) among such individuals. The study population will subsequently be characterised according to the extent of use of one of the main treatments for AAV, glucocorticoids, and the extent to which adverse outcomes and healthcare use in AAV patients differs according to the extent of glucocorticoid use will be explored.

The findings of the study will demonstrate the unmet needs of AAV patients.

Technical Summary

AAV is a rare, serious and often life-threatening disease. Morbidities associated with AAV include active vasculitis, infection, cardiovascular disease and malignancy. A number of available treatments are effective in reducing the morbidity and mortality associated with AAV, however these treatments are themselves associated with significant toxicity.

This study will utilise real world data to identify a population of patients in England with AAV and will describe the rate of infectious, bone, cardiovascular, renal, ocular, metabolic and mental health outcomes in the population. Healthcare use in the AAV population will also be described in terms of the rate of GP consultations, GP prescriptions, inpatient visits and outpatient visits.

The study population will subsequently be characterised according to the duration and dose of use of one of the main treatments for AAV, glucocorticoids (GCs), and the rate of the adverse outcomes and healthcare resource use measures described above will be estimated stratified by current and cumulative dose of GC prescribed.

The findings of the study will demonstrate the unmet needs of AAV patients and the potential of emerging 'steroid-sparing' treatments to reduce the treatment related morbidity associated with GC use in AAV.

Health Outcomes to be Measured

Infectious outcomes:
- Upper respiratory tract infection
- Lower respiratory tract infection
- Gastrointestinal infection
- Urinary tract infection
- Skin/wound infection
- Liver infection
- Vector Borne illnesses
- Tuberculosis
- Sexually transmitted disease/venereal disease

Bone outcomes:
- Osteoporosis
- Bone fractures

Ocular outcomes:
- Cataracts
Metabolic conditions:
- New-onset diabetes

Mental health outcomes:
- Depression
- Anxiety

Cardiovascular outcomes
- New-onset hypertension
- Vascular disease
- Stroke/TIA
- Peripheral vascular disease
- Venous thromboembolism

Renal outcomes:
- Chronic kidney disease
- End Stage Renal Disease
- Dialysis

Progressive multifocal leukoencephalopathy
Haemorrhagic cystitis/haematuria

Collaborators

Cormac Sammon - Chief Investigator - PHMR Associates Limited ( UK )
Cormac Sammon - Corresponding Applicant - PHMR Associates Limited ( UK )
Mihail Samnaliev - Collaborator - Maverex Ltd
Peter Rutherford - Collaborator - Vifor Pharma
Philip Spearpoint - Collaborator - Vifor Pharma

Linkages

HES Admitted Patient Care;HES Outpatient