Cost-utility analysis of sequential therapies for pediatric-onset eosinophilic esophagitis

Topical corticosteroids, proton-pump inhibitors (PPI) and elimination diets (ED) are equally recommended as first-line therapies for pediatric eosinophilic esophagitis (EoE). Dupilumab (DUP) is usually used for refractoriness. We aim to assess the cost-utility of all potential sequential treatments for EoE.

A Markov model under the payer's perspective was used to describe induction, maintenance, and reinductions for nonresponders over 10 years. Dietary options included step-up restriction starting from two foods, and from cow's milk (CM). Topical corticosteroids viscous solutions, or budesonide orodispersible tablets (BOT) were considered. Nonresponsiveness led to esophageal dilation. Simulation parameters were defined by systematic review. We deterministically selected the most cost-effective permutation as reference. Incremental cost-utility ratios (ICUR) of each alternative sequence were compared to a willingness-to-pay (WTP) threshold of €30,000. Sequences with the highest number of quality-adjusted life-years (QALY) and the highest QALY below WTP were probabilistically explored. Sensitivity analysis was performed.

PPI-ED(CM)-BOT-DUP was the most cost-effective sequence, resulting in €2373/QALY and 7.20 QALY at the time horizon. DUP-BOT-PPI-ED(CM) gave the uppermost QALY, 7.42, but any iteration was cost-effective, yielding a median ICUR of €323,774/QALY. PPI-BOT-ED(CM)-DUP, however, had a higher chance of being under the WTP (83%), showed a median ICUR of €25,156/QALY and provided 7.31 QALY. An 83% reduction of DUP price was needed to make DUP-BOT-PPI-ED(CM) and PPI-BOT-ED(CM)-DUP equally as efficient.

BOT and CM-starting step-up ED are present in both effective and cost-effective sequential treatments for EoE. The option PPI-BOT-ED(CM)-DUP succeeded in approaching the most effective alternative while balancing costs.