Healthcare Resource Utilization and Disease Progression in Patients With Primary Hyperoxaluria: A Retrospective Cohort Study
Author(s)
Anne Helene Olsen, MSc, PhD1, Katarzyna Mosiewicz, PhD2, Jack Garcia Uranga, MSc2, Kirstine Belling, PhD2.
1Novo Nordisk A/S, Bagsvaerd, Denmark, 2Novo Nordisk A/S, Bagsværd, Denmark.
1Novo Nordisk A/S, Bagsvaerd, Denmark, 2Novo Nordisk A/S, Bagsværd, Denmark.
OBJECTIVES: Primary hyperoxaluria (PH), a family of rare autosomal recessive genetic disorders, can cause recurrent kidney stones, chronic kidney disease (CKD), and kidney failure. This study assessed healthcare resource utilisation (HCRU) and disease progression of PH versus CKD and background populations.
METHODS: Data from the UK’s Clinical Practice Research Datalink Aurum registry, linked to Hospital Episode Statistics and Office of National Statistics were analysed. Patients with PH or CKD were included if they had ≥1 year of registry enrolment before first diagnosis (index date). The background population was matched (1:20) based on age and index year for patients with PH. Population comparisons included 10 years of follow-up post index date (data extraction: May 2022). Weighting adjusted for confounding factors (birth year, gender, and index of multiple deprivation). Number of unique visits were determined using negative binomial distribution with patient years as exposure. Comparisons of time-to-first-occurrence of disease progression were determined by Kaplan-Meier and estimated by Cox proportional hazards.
RESULTS: Of 123 patients with any PH type, 29 (23.6%) had ≥1 CKD-related diagnosis. Mean ± SD age at first diagnosis was 40 ± 22 years (PH) and 72 ± 14 years (CKD). Patients with PH had general practitioner (GP) visit histories for kidney stones (38.2%), urinary tract infections (11.3%), and ureteric stent cystoscopic insertions (8.1%); inpatient histories included kidney (31.7%), ureter (21.9%), and bladder (5.7%) stones. Patients with PH generally had greater rates of GP, in-, and outpatient visits versus CKD and background populations. In the follow-up period, patients with PH had increased risk of dialysis (3-fold; P=0.007), liver transplant (158-fold; P<0.001), and kidney transplant (18.7-fold; P<0.001) versus patients with CKD, but slightly better survival rates (0.31-fold; P=0.042).
CONCLUSIONS: Patients with PH had increased disease burden and HCRU versus the CKD and background populations, representing a need to drive earlier PH diagnosis and treatment.
METHODS: Data from the UK’s Clinical Practice Research Datalink Aurum registry, linked to Hospital Episode Statistics and Office of National Statistics were analysed. Patients with PH or CKD were included if they had ≥1 year of registry enrolment before first diagnosis (index date). The background population was matched (1:20) based on age and index year for patients with PH. Population comparisons included 10 years of follow-up post index date (data extraction: May 2022). Weighting adjusted for confounding factors (birth year, gender, and index of multiple deprivation). Number of unique visits were determined using negative binomial distribution with patient years as exposure. Comparisons of time-to-first-occurrence of disease progression were determined by Kaplan-Meier and estimated by Cox proportional hazards.
RESULTS: Of 123 patients with any PH type, 29 (23.6%) had ≥1 CKD-related diagnosis. Mean ± SD age at first diagnosis was 40 ± 22 years (PH) and 72 ± 14 years (CKD). Patients with PH had general practitioner (GP) visit histories for kidney stones (38.2%), urinary tract infections (11.3%), and ureteric stent cystoscopic insertions (8.1%); inpatient histories included kidney (31.7%), ureter (21.9%), and bladder (5.7%) stones. Patients with PH generally had greater rates of GP, in-, and outpatient visits versus CKD and background populations. In the follow-up period, patients with PH had increased risk of dialysis (3-fold; P=0.007), liver transplant (158-fold; P<0.001), and kidney transplant (18.7-fold; P<0.001) versus patients with CKD, but slightly better survival rates (0.31-fold; P=0.042).
CONCLUSIONS: Patients with PH had increased disease burden and HCRU versus the CKD and background populations, representing a need to drive earlier PH diagnosis and treatment.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO129
Topic
Clinical Outcomes
Topic Subcategory
Clinician Reported Outcomes, Performance-based Outcomes
Disease
Rare & Orphan Diseases, Urinary/Kidney Disorders