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PEDIATRICS Vol. 114 No. 2 August 2004, pp. 417-426

Cost-Effectiveness of Inhaled Nitric Oxide for the Management of Persistent Pulmonary Hypertension of the Newborn

Scott A. Lorch, MD*,{ddagger},§, Avital Cnaan, PhD* and Kurt Barnhart, MD, MSCE§,||

* Department of Pediatrics
{ddagger} Division of Neonatology and Center for Outcomes Research, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania
§ Center for Clinical Epidemiology and Biostatistics
|| Department of Obstetrics and Gynecology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania

Objective. Inhaled nitric oxide (iNO) is a selective pulmonary vasodilator that has become part of the standard management for persistent pulmonary hypertension of the newborn (PPHN). This treatment modality, like many in neonatology, has not been well studied using quantitative economic techniques. The objective of this study was to evaluate the economic impact of adding iNO to the treatment protocol of PPHN for term infants from birth to the time of discharge from their initial hospitalization.

Methods. We used decision analysis modeling from a societal perspective to obtain an incremental cost-effectiveness ratio. Outcome probabilities were taken from the medical literature and a cohort of 123 infants who were treated with PPHN at The Children’s Hospital of Philadelphia between 1991 and 2002. Costs were estimated from daily resources used by these infants in 2001 dollars. Survival and quality-adjusted life years were used as effectiveness measures. One-way, threshold, and probabilistic sensitivity analyses were performed to assess the robustness of the base-case estimate.

Results. The addition of iNO to the treatment regimen of PPHN increased the cost of treating an infant by an average of $1141, primarily from an increased number of mechanical ventilation days. Use of iNO led to 3.4% more lives saved and a 6% increase in the average utility gained per infant. The incremental cost-effectiveness ratio was $33 234 per life saved and $19 022 per quality-adjusted life year gained. The model was robust to changes in outcome probabilities, cost, and utility variables. Only 3.6% of the trials using probabilistic sensitivity analysis found iNO to be more expensive with a worse outcome than conventional therapy alone, whereas 35.7% of the trials found iNO to be cheaper and more effective than conventional treatment alone.

Conclusions. iNO is cost-effective but not cost-saving in treating infants with PPHN from a societal perspective. There are critical time points during an infant’s hospitalization that could improve the efficiency and consequently the cost of care for this patient population.

Key Words: cost-effectiveness analysis • inhaled nitric oxide • persistent pulmonary hypertension • neonate • decision analysis

Abbreviations: NICU, neonatal intensive care unit • ECMO, extracorporeal membrane oxygenation • PPHN, persistent pulmonary hypertension of the newborn • iNO, inhaled nitric oxide • CHOP, Children’s Hospital of Philadelphia • CI, confidence interval • QALY, quality-adjusted life year • CV, conventional ventilation

Received for publication Jun 13, 2003; Accepted Dec 11, 2003.


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