Abstract: SA-PO1219
Economic Evaluation of Lifelong Lumasiran Therapy in Primary Hyperoxaluria Type 1
Session Information
- Transplantation: Clinical - Complications, Pediatrics, and Multi-Organ Considerations
October 24, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Transplantation
- 2002 Transplantation: Clinical
Authors
- Ojha, Vishnu Shankar, Mayo Clinic in Florida, Jacksonville, Florida, United States
- Baker, Lyle Wesley, Mayo Clinic in Florida, Jacksonville, Florida, United States
- Wadei, Hani, Mayo Clinic in Florida, Jacksonville, Florida, United States
- Porter, Ivan E., Mayo Clinic in Florida, Jacksonville, Florida, United States
Background
Lumasiran has introduced the possibility of kidney transplantation without liver transplantation in patients with PH1 by suppressing hepatic oxalate production. However, lumasiran requires lifelong therapy with substantial cumulative costs, necessitating economic evaluation against curative transplantation strategies.
Methods
A lifetime Markov state-transition model from a US healthcare payer perspective compared: (1) lifelong Lumasiran with kidney transplantation alone (KT+lumasiran), (2) sequential liver followed by kidney transplantation (LT→KT), and (3) simultaneous liver-kidney transplantation (SLKT) in PH1. Age 6 years was used as the starting age. Base-case assumptions included blended lifetime annual lumasiran costs of $650,000/year, 10-year graft survival of 72% for KT+lumasiran, 75% for LT→KT, and 80% for SLKT, with 5-year oxalate recurrence of 8% after KT+lumasiran. Model inputs were derived from clinical trials, transplant registries, and expert recommendations. Outcomes included lifetime costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs). Costs and QALYs were discounted at 3% annually.
Results
In the base-case analysis, KT+lumasiran generated the highest projected QALYs (19.2) compared with LT→KT (17.4) and SLKT (18.1). However, KT+lumasiran was associated with substantially higher lifetime costs ($18.9 million) relative to LT→KT ($4.6 million) and SLKT ($3.9 million). The estimated ICER for KT+lumasiran was approximately $7.9 million/QALY versus LT→KT and $13.6 million/QALY versus SLKT, exceeding conventional willingness-to-pay thresholds. Deterministic sensitivity analysis identified annual lumasiran acquisition cost as the dominant model driver, followed by graft survival and duration of therapy. Probabilistic sensitivity analysis demonstrated a probability of cost-effectiveness of <1% at a willingness-to-pay threshold of $100,000/QALY and <5% at $150,000/QALY. Threshold analyses suggested effective long-term annual lumasiran pricing would likely need to decrease below approximately $100,000–150,000/year to approach conventional cost-effectiveness thresholds.
Conclusion
Lifelong KT+lumasiran improved projected quality-adjusted survival, the strategy remained economically unfavorable compared to LT→KT or SLKT. These findings suggest that current lumasiran pricing represents a major barrier to long-term economic sustainability in PH1 management.