Primary hyperoxaluria (PH) is a family of rare genetic liver disorders which can lead to complications such as kidney stones and chronic kidney disease.
This study will investigate the disease burden for people with PH. Patient characteristics will be described (age, sex, what illnesses they had and what medicines they were being prescribed), as well as the characteristics of the area of their UKGP practice e.g. how deprived or wealthy the area was. The study will also describe visits to the health care system, and disease progression over time.
The study will add knowledge about how this disease affects the patients, and how the disease develops over time.
Primary hyperoxaluria (PH) is a family of rare, life-threatening genetic liver disorders which can lead to complications such as kidney stones and chronic kidney disease. The disease burden associated with PH has not been well characterized and thus further evidence is warranted. The aim of this study is to describe the disease burden for patients with a diagnosis of PH.
The study design is a retrospective cohort study. The study population will include patients with a diagnosis of PH from CPRD Aurum. The index date is defined as the first date of diagnosis. Patients must be research standard (registered as “acceptable” in the database) with at least one year of registration prior to their index date. Patients will be followed up until the minimum of (a) their transfer out date, (b) their CPRD derived death date (if applicable), and (c) the last collection date of their GP practice.
The study population will be compared to two comparator populations consisting of (a) patients with chronic kidney disease (CKD), and (b) a sample of patients without a PH diagnosis. The two comparator populations will be matched with the study population on age, gender, and multiple deprivation index.
Baseline characteristics at index date (age, sex, multiple deprivation index, disease history and prescribed drugs), and rates of health care resource utilisation (visits registered in HES and Aurum) will be presented for the study and the comparator populations. Rates for initiation of dialysis, liver and kidney transplants (HES), and mortality (ONS mortality data), as well as progression of eGFR (Aurum) will be presented for the study and CKD comparator populations.
Intended public health benefit:
The study will contribute with increased knowledge about the disease burden and disease progression for this patient group.
Number of inpatient hospitalisations; number of outpatient visits; number of GP visits; first occurrence of dialysis; liver transplant; kidney transplant; all-cause mortality; eGFR lab values
Anne Helene Olsen - Chief Investigator - Novo Nordisk A/S
Anne Helene Olsen - Corresponding Applicant - Novo Nordisk A/S
Azzeddine Zemam - Collaborator - Novo Nordisk A/S
Kirstine Belling - Collaborator - Novo Nordisk A/S
HES Admitted Patient Care;HES Outpatient;ONS Death Registration Data;Practice Level Index of Multiple Deprivation