There are rare skin conditions called pemphigus and bullous pemphigoid that cause large and widespread blisters. These skin conditions can occur at any age but is most often seen in people when they are middle-aged or older. The conditions tend to be a long-lasting, and some types can be life-threatening without treatment. Medications are usually given to control the blisters, with the most common form of treatment being steroids. However, long-term treatment with steroids can come with serious side effects such as high blood pressure, glaucoma, as well as milder effects such as diarrhoea, vomiting, and stomach discomfort.
We will use anonymous data from GP and hospital records from England to describe the pattern of pemphigus and bullous pemphigoid symptoms and signs, as well as treatment and clinical management. We will describe the number of people diagnosed with the conditions each year and the healthcare interactions they have and treatments they receive. The results of our study will provide an overview of the treatment for the rare conditions of pemphigus and bullous pemphigoid, to guide any possible improvements and standardisation in patient care to improve the wellbeing of people with the conditions.
Previous UK studies, including those using data from the Clinical Practice Research Datalink (CPRD) and Hospital Episode Statistics (HES) data, show a rise in pemphigus and bullous pemphigoid incidence along with high mortality between the late 1990s and mid-2010s. Pemphigus vulgaris (PV) is the most common form of pemphigus, with annual incidence in the UK previously estimated to be 0.7 per 100,000 person-years. Similarly, studies investigating bullous pemphigoid have reported incidences between 0.2 and 4.3 per 100 000 person years, with higher incidence (7.6 and 8.4 per 100,000 person-years) being reported in recent years. Treatment of pemphigus and bullous pemphigoid entails management of clinical symptoms rather than cure with oral prednisolone the mainstay treatment.
While separate CPRD studies have looked at aspects of the burden and treatment of pemphigus and bullous pemphigoid, we aim to provide a complete summary of the disease epidemiology including incidence, prevalence, and mortality, along with detailed examination of treatment practices including a treatment pathway mapping and healthcare resource use for a consistent patient population using 12 years of CPRD-HES-ONS linked data. Our descriptive retrospective cohort study will include five groups: all bullous pemphigoid patients, all pemphigus patients, specifically PV patients, specifically pemphigus foliaceus (PF) patients, and broader definition of PV and PF patients. We will describe the baseline demographics, lifestyle, and clinical characteristics of patients in the five pre-defined groups. We will describe the annual hospital activity and number of patients who receive treatment, stratified by monotherapy and combination therapy for immunomodulatory, steroid, and non-immunosuppressant treatments. We will also describe the treatment pathway for active disease, including the number of patients who receive non-steroidal immunosuppressant therapy, and the time to remission for each therapy. To assess the treatment pathway further, we will describe the number and proportion of patients who receive regular high-dose steroids.
Incidence, prevalence and mortality; Treatment provision including immunosuppressive and immunomodulatory therapies; Treatment pathway including order of treatment given; Routine high-dose steroid; Hospital activity
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
Boglarka Kovacs - Collaborator - Health iQ Ltd ( UK ) t/a CorEvitas
Caoimhe Rice - Collaborator - Health iQ Ltd ( UK ) t/a CorEvitas
Gulsah Akin Unal - Collaborator - Health iQ Ltd ( UK ) t/a CorEvitas
Judith Ruzangi - 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
Seth Jarvis - Collaborator - Health iQ Ltd ( UK ) t/a CorEvitas
Gulsah Akin Unal - Collaborator - Health iQ Ltd ( UK ) t/a CorEvitas
Sara Carvalho - Collaborator - Health iQ Ltd ( UK ) t/a CorEvitas
HES Accident and Emergency;HES Admitted Patient Care;HES Outpatient;ONS Death Registration Data;Patient Level Index of Multiple Deprivation