Introduction: Pulmonary hypertension affects a significant number of premature and low-birth weight infants, and it is associated with increased morbidity and mortality. The standard of care currently recommends cardiac catheterization prior to initiation of pulmonary vasodilator therapies, including enteral sildenafil. This analysis sought to evaluate the cost-utility of catheterization-obligate treatment in pulmonary hypertension (PH), as compared to empiric initiation of sildenafil based on echocardiographic findings alone in preterm infants with bronchopulmonary dysplasia (BPD).

Methods: A Markov-state transition model was constructed to simulate the clinical scenario of a preterm infant with echocardiographic evidence of PH associated with BPD (BPD-PH) and without congenital heart disease under consideration for the initiation of pulmonary vasodilator therapy via one of two modeled treatment strategies: (1) empiric or (2) catheterization-obligate. Transitional probabilities, costs and utilities were extracted from the literature. Forecast quality-adjusted life years (QALY) was the metric for strategy effectiveness. Sensitivity analyses for each variable were performed. A 1,000-patient Monte Carlo microsimulation was used to test the durability of our findings.

Results: The catheterization-obligate strategy resulted in an increased cost of $10,778 and 0.02 fewer QALY compared to the empiric treatment strategy. Empiric treatment remained the more cost-effective paradigm across all scenarios modeled through one-way sensitivity analyses and the Monte Carlo microsimulation (cost-effective in 98% of cases). In varying the mortality of cardiac catheterization, the catheterization-obligate paradigm does become less costly than the empiric strategy when the probability of a catheterization-related mortality exceeds 2.9%, though remains less cost effective. A three-way sensitivity analysis varying the prevalence and clinical significance of “alternative diagnoses” when starting empiric sildenafil did not alter our findings.

Conclusion: Empiric treatment with sildenafil in infants with BPD-PH is a superior strategy with both decreased costs and increased effectiveness when compared to catheterization-obligate treatment. These findings suggest that foregoing catheterization prior to initiation of sildenafil in “straight-forward” BPD-PH may be warranted.