OBJECTIVE:

To determine the cost-effectiveness of treatment with caffeine compared with placebo for apnea of prematurity in infants with birth weights less than 1250 g, from birth through 18 to 21 months' corrected age.

METHODS:

We undertook a retrospective economic evaluation of the cost per survivor without neurodevelopmental impairment by using individual-patient data from the Caffeine for Apnea of Prematurity clinical trial (N = 1869). We included direct medical costs either to the insurance payer or the hospital but excluded costs to parents and society, such as lost productivity. We used a price of $0.21/mg of generic caffeine citrate for our base-case analysis. All costs were expressed in 2008 Canadian dollars and discounted at 3%. The time horizon for this analysis extended through 18 to 21 months' corrected age to match the clinical trial. RESULTS: The mean cost per infant was$124 466 in the caffeine group and $133 505 in the placebo group (difference:$9039 [−14 749 to −3375]; adjusted P = .014). Cost-effectiveness analysis showed caffeine to be a dominant or “win-win” therapy: in >99% of 1000 bootstrap replications of the analysis, caffeine-treated infants had simultaneously better outcomes and lower mean costs. These results were robust to a 1000% increase in the individual resource items, including the price of caffeine citrate.

CONCLUSIONS:

In comparison with placebo, caffeine therapy for apnea of prematurity in infants weighing less than 1250 g is economically appealing for infants up to 18 to 21 months' corrected age.