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Background <p>Primary hyperoxaluria (PH) is a family of three rare, autosomal recessive genetic disorders that can result in recurrent kidney stones, progressive chronic kidney disease (CKD), and kidney failure. PH prevalence is underestimated due to its varying presentation and lack of awareness; delays in diagnosis can lead to substantial burdens on the healthcare system.</p> Methods <p>This retrospective, observational claims analysis evaluated disease burden and cost of care in patients who had PH, PH with CKD, or CKD alone. Data from the Merative MarketScan Commercial Claims and Encounters databases and the Centers for Medicare and Medicaid Services Medicare Fee-for-Service Limited Data Set were assessed during the study period of January 1, 2017, to December 31, 2021. PH prevalence was calculated based on the sample population within each data source.</p> Results <p>The study sample included 326 patients who had PH; applying projection factors to the US population, an estimated 4500 patients had a diagnosis of PH in 2021. Among these patients, 37% were estimated to have PH with CKD (65% of whom had early CKD, 33% had advanced CKD, and 2% had stage reported as unknown). Patients who had CKD alone (<i>n</i> = 845) were matched with patients who had PH with CKD (<i>n</i> = 169). Patients who had PH with CKD were significantly more burdened with kidney stones (<i>p</i> &lt; 0.01) than patients who had CKD alone. Higher rates of pharmacotherapy and medical treatments were observed in patients who had PH with CKD versus patients who had CKD alone. Median semi-annual total all-cause healthcare costs were greater in patients who had PH with CKD than in patients with CKD alone, regardless of CKD stage ($54,154 in patients who had PH with advanced CKD vs. $35,016 in patients with advanced CKD alone; $9,784 in patients who had PH with early CKD vs. $5,572 in patients with early CKD alone).</p> Conclusions <p>CKD stage progression among patients who had PH is associated with increasing all-cause costs, suggesting that early diagnosis and treatment of PH to limit the progression to advanced CKD could represent an opportunity to alleviate not only PH symptoms, but also the healthcare cost burden.</p>

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Real-world burden of primary hyperoxaluria with chronic kidney disease in the United States: a retrospective administrative claims analysis

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年10月16日

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  • David S. Goldfarb,
  • Frank Modersitzki,
  • Jeffrey R. Skaar,
  • Olivia Li,
  • Sandra Salem,
  • Jing Voon Chen

摘要

Background

Primary hyperoxaluria (PH) is a family of three rare, autosomal recessive genetic disorders that can result in recurrent kidney stones, progressive chronic kidney disease (CKD), and kidney failure. PH prevalence is underestimated due to its varying presentation and lack of awareness; delays in diagnosis can lead to substantial burdens on the healthcare system.

Methods

This retrospective, observational claims analysis evaluated disease burden and cost of care in patients who had PH, PH with CKD, or CKD alone. Data from the Merative MarketScan Commercial Claims and Encounters databases and the Centers for Medicare and Medicaid Services Medicare Fee-for-Service Limited Data Set were assessed during the study period of January 1, 2017, to December 31, 2021. PH prevalence was calculated based on the sample population within each data source.

Results

The study sample included 326 patients who had PH; applying projection factors to the US population, an estimated 4500 patients had a diagnosis of PH in 2021. Among these patients, 37% were estimated to have PH with CKD (65% of whom had early CKD, 33% had advanced CKD, and 2% had stage reported as unknown). Patients who had CKD alone (n = 845) were matched with patients who had PH with CKD (n = 169). Patients who had PH with CKD were significantly more burdened with kidney stones (p < 0.01) than patients who had CKD alone. Higher rates of pharmacotherapy and medical treatments were observed in patients who had PH with CKD versus patients who had CKD alone. Median semi-annual total all-cause healthcare costs were greater in patients who had PH with CKD than in patients with CKD alone, regardless of CKD stage ($54,154 in patients who had PH with advanced CKD vs. $35,016 in patients with advanced CKD alone; $9,784 in patients who had PH with early CKD vs. $5,572 in patients with early CKD alone).

Conclusions

CKD stage progression among patients who had PH is associated with increasing all-cause costs, suggesting that early diagnosis and treatment of PH to limit the progression to advanced CKD could represent an opportunity to alleviate not only PH symptoms, but also the healthcare cost burden.