Aims: Familial hypophosphatemia (FH) commonly results in renal phosphate wasting, which leads to rickets, osteomalacia and other musculoskeletal consequences. X-linked hypophosphatemia, the most common form of FH, may be treated with burosumab. However, the healthcare utilization and economic burden of FH among burosumab-naïve patients has not been characterized in the USA. This study examined healthcare utilization and costs for burosumab-naïve patients with FH, compared with demographically matched controls without FH.
Methods: Using the Merative™ MarketScan® Commercial and Medicare US administrative claims databases, patients with >1 diagnosis code for FH (ICD10:E83.31) between 1/1/2018-12/31/2021, and continuous database enrollment for 12-months pre-and post-index were identified. The index date was the date of the first FH diagnosis. FH patients were demographically matched 1:3 to non-FH control patients based on age group, sex, geographic region, payer, and index year. Healthcare utilization and costs were assessed in the 12-month post-index period and adjusted to 2021 dollars using the medical care component of the Consumer Price Index. The Charlson Comorbidity index (CCI) score was reported in the 12-month pre-index period as a measure of baseline health status. Results were reported overall and stratified by age.
Results: Matched burosumab-naïve FH patients (n=570) and non-FH controls (n=1,710) were 57.0% female, 53.0% with an index year in 2018-2019, and with a mean (standard deviation [SD]) age of 47.2 (19.9) and 46.2 (18.3) years (10.4%, 76.2%, and 13.5% were <18, 18-64, and 65+ years respectively). Baseline CCI score was significantly greater among FH patients than controls (1.9 [2.6] vs. 0.2 [0.9], P<0.001). Annual all-cause healthcare utilization, mean number of healthcare visits, and mean healthcare costs were greater among FH patients vs controls (Table). Similar trends were observed among age-stratified FH patients and non-FH controls.
Conclusions: FH patients incur substantially higher healthcare utilization, costs, and comorbidity burden compared with non-FH controls.