Presentation (CTI)

OBJECTIVES: Atypical haemolytic uraemic syndrome (aHUS) is a rare life-threatening disease caused by complement dysregulation leading to the formation of blood clots throughout the body. Typically affecting the kidneys, if left untreated this condition can lead to kidney failure. Eculizumab is an effective treatment that prevents disease recurrence and progression, improving survival. The patient benefits of lifelong treatment are uncertain however, and therapy can have high costs for healthcare providers. This economic evaluation assessed the cost-effectiveness of stopping and replacing lifelong eculizumab therapy with a monitoring and retreatment strategy.
METHODS: A Markov model was used to estimate costs and quality adjusted life years (QALYs) for replacing lifelong eculizumab with a disease monitoring and retreatment strategy. Results from the Stopping Eculizumab Treatment Safely in aHUS (SETS aHUS) trial informed risks of disease recurrence and progression, utility values, and health care use. Time to relapse diagnosis was extrapolated using parametric survival functions.
RESULTS: Stopping treatment with a disease monitoring strategy increased average patient QALYs by 0.08 (95% CrI: -0.33 to 0.53) but reduced survival by 0.18 days over an 80-year time horizon (-0.0005 Life Years 95% CrI: -0.0029 to 0). It also reduced patient costs by £4,234,196 (95% CrI: -£684,495 to -£6,403,694) compared with the lifelong delivery of eculizumab. Results remained robust across multiple scenarios exploring uncertainties.
CONCLUSIONS: Stopping eculizumab therapy with a disease monitoring and retreatment protocol was cost-effective compared with the lifelong delivery of eculizumab. Its adoption is expected to reduce healthcare costs per patient and may improve quality of life.