Higher Rates of Complications and Health-Care Resource Utilisation in Achondroplasia Compared to the General Population: A Matched Cohort Study Using the Cprd-HES Database
Author(s)
Pimenta J1, Irving M2, Cheung MS2, Mazzeo L1, Landis S1, Mukherjee S1
1BioMarin, London, UK, 2Guy's and St Thomas' NHS Foundation Trust, London, UK
Presentation Documents
OBJECTIVES: Achondroplasia (ACH) is a rare skeletal dysplasia condition, resulting in disproportionate short stature and multiple complications. We aimed to estimate disease burden and health-care resource use (HCRU) in ACH patients compared to general population controls. METHODS: A matched retrospective cohort study was conducted using the Clinical Practice Research Datalink (CPRD-GOLD) linked to Hospital Episode Statistics (HES) in England. A cohort of ACH patients (study index date first record of ACH between 01/01/1987-31/12/2018) were index date matched (1:4) by age, sex, general practitioner (practice level) and linkage ability to HES to control patients, defined as those with no evidence of skeletal/growth disorders. Event rates per 100 person-year were calculated for a pre-defined set of complications, medical imaging (skeletal x-rays, DEXA scans etc), in-patient surgeries and HRCU by age-group; rate ratios (RR) with 95% CIs were used to compare between cohorts. RESULTS: We identified 275 ACH cases and 1,064 matched controls; 51% female, median age 28 years (IQR 7-40 years). Considering all pre-defined complications, complications were twice as likely to be reported among ACH patients than controls (RR = 1.98 [1.89-2.08], ranging from RR=1.13 [1.00-1.28] for cardiovascular conditions to RR=8.31 [4.72-14.61] for developmental issues. Furthermore, significantly higher rates of medical imaging (RR=2.48 [2.31-2.65]) and in-patient surgeries (RR 9.80 [7.84-12.26]) comprising mainly of ear/nose/throat and orthopaedic procedures, were reported among ACH patients. ACH patients reported significantly more HCRU than controls; GP visits RR=1.53 [1.52-1.55], hospital out-patient RR=2.44 [2.38-2.50] and in-patient visits RR=2.18 [2.06-2.30]. Complications and HCRU were highest among youngest (≤10y) and oldest (≥60y) age-groups. CONCLUSIONS: In a real-world clinical setting, ACH patients experience significantly higher rates of complications, medical imaging, surgeries and healthcare resource use compared to an appropriate matched general population cohort, consistently throughout life. These findings may have impact for proactive clinical management and patient care.
Conference/Value in Health Info
2021-11, ISPOR Europe 2021, Copenhagen, Denmark
Value in Health, Volume 24, Issue 12, S2 (December 2021)
Code
POSC186
Topic
Clinical Outcomes, Economic Evaluation, Epidemiology & Public Health, Real World Data & Information Systems
Topic Subcategory
Clinician Reported Outcomes, Health & Insurance Records Systems
Disease
Musculoskeletal Disorders, Rare and Orphan Diseases