Abstract

Central line-associated bloodstream infections (CLABSIs) lead to significant morbidity and mortality in children with intestinal failure (IF). Ethanol lock prophylaxis (ELP) greatly reduces CLABSI frequency with minimal side effects. However, in the United States, a recently approved orphan drug designation for dehydrated alcohol has greatly increased 70% ethanol cost from about $10/day to $1000/day. We examined the cost-effectiveness of ELP in relation to these changes. We simulated a previously developed IF Markov model over 1 year. Costs were measured in 2020 US dollars and effectiveness in quality-adjusted life-years (QALYs). CLABSI rate with and without ELP was estimated from the largest available comparative observational study. The primary outcome was incremental cost-effectiveness ratio (ICER) between treatments. Secondary outcomes included CLABSI frequency. Sensitivity analyses on all model parameters were performed. In the base model, children with IF not using ELP accumulated $131,815 in costs and 0.32 QALYs per patient compared with $437,884 and 0.33 QALYs per patient in those using ELP. The ICER was nearly $17 million/QALY gained. ELP resulted in a 40% reduction in CLABSI frequency. ELP became cost-effective at $68/day and cost-saving at $63/day. Sensitivity analysis identified no other plausible parameter variation to reach the benchmark of $100,000/QALY gained. At the current price, ELP is not cost-effective for CLABSI prevention in children with IF in the United States. This study highlights the critical need for the approval of an affordable lock therapy option to prevent CLABSIs in these children.

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