| Summary: | **Background:** Autosomal dominant polycystic kidney disease (ADPKD), a hereditary nephropathy, eventually leads to end-stage renal disease (ESRD), typically by mid-life.
**Objectives:** The objective of this study was to assess real-world healthcare resource utilization and cost among commercially insured (COM) and Medicare Advantage (MAPD) ADPKD patients in addition to the cost profile by chronic kidney disease (CKD) stage.
**Methods:** Patients diagnosed with ADPKD (two or more claims) with ≥30 days of continuous medical and pharmacy benefits and no evidence of autosomal recessive polycystic kidney disease were selected (Optum Research Database and Impact National Benchmarking Database: 1/1/06–8/31/12). Plan and patient paid healthcare costs and resource utilization per patient per month (PPPM) were described in total and by insurance type. CKD stage was established based on serum creatinine laboratory values or dialysis-related codes. Adjusted, CKD stage-specific costs were predicted for 4 years using regression models.
**Results:** Of the 36,253,096 patients in the databases (1/1/06-8/31/12), 5,051 had evidence of ADPKD. Following exclusion criteria, 4,356 COM and 468 MAPD ADPKD patients remained. Total healthcare resource utilization and costs were high, and costs increased substantially from CKD stage 1–5. PPPM healthcare costs were 37% for ADPKD management and 52% for dialysis services. Predicted 4-year healthcare costs by CKD stage were $40,164 (stage 1), $33,397 (stage 2), $42,686 (stage 3), $148,402 (stage 4), and $207,548 (stage 5).
**Conclusions:** Healthcare resource utilization and costs associated with ADPKD were substantial, irrespective of payer type, and primarily driven by CKD stage. Of the total healthcare costs, 88% were ADPKD- and dialysis-related. Most impactful was the spike in predicted cost when patients progressed from CKD stage 3 to stage 4 (by 348%) after multivariate adjustment. These stage 4–associated costs are primarily due to ultimate progression into stage 5 and ESRD within the 4-year time frame.
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