Imel, Erik A.Li, ZhiyiHeerssen, Heather M.Princic, NicoleSchwartz, HanaZhao, YangDahir, Kathryn M.2024-12-092024-12-092024-10-24Imel EA, Li Z, Heerssen HM, et al. Real-World Clinical and Healthcare Resource Burden Among Burosumab-Naïve Patients With Familial Hypophosphatemia. J Endocr Soc. 2024;8(12):bvae185. Published 2024 Oct 24. doi:10.1210/jendso/bvae185https://hdl.handle.net/1805/44846Objective: To examine the real-world clinical and healthcare resource burden of familial hypophosphatemia (FH). Methods: In a retrospective, observational cohort study using MarketScan claims data from 2017 to 2021, clinical characteristics and healthcare resource utilization (HCRU) and costs were compared between burosumab-naïve pediatric and adult patients with ≥ 1 FH diagnosis code and matched controls without FH. Patient characteristics were evaluated at baseline, and disease characteristics, HCRU, and costs were evaluated over a 12-month follow-up period. Outcomes were analyzed descriptively. Costs were additionally analyzed using multivariate regression models. Results: Overall, 570 patients with FH and 1710 non-FH matched controls were included. Approximately 10% of study participants were aged < 18 years. Patients with FH had 7.8-fold higher mean baseline comorbidity (Charlson Comorbidity Index). The prevalence of morbidities over the 12-month follow-up period was higher in patients with FH than controls, including renal disease (33% vs 3%), arthralgia (25% vs 10%), osteoarthritis (17% vs 6%), and delayed growth/walking difficulty (16% vs 2%; all P < .001). All-cause HCRU was significantly greater for patients with FH than controls over follow-up, including the proportion of patients with at least one inpatient admission (60% vs 4%), outpatient emergency room visit (52% vs 16%), and outpatient pharmacy prescription (96% vs 71%; all P < .001). The mean annual total healthcare cost per patient was also 22.6-fold higher for patients with FH than controls (adjusted cost difference = $129 643; P < .001). Differences were apparent across all age groups. Conclusion: Compared with non-FH matched controls, burosumab-naïve patients with FH experienced multiple morbidities and had substantially higher HCRU and costs.en-USAttribution 4.0 InternationalDisease burdenFamilial hypophosphatemiaHealthcare costsHealthcare resource utilizationX-linked hypophosphatemia (XLH)BurosumabArticle