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Am J Perinatol 2021; 38(14): 1505-1512
DOI: 10.1055/s-0040-1713819
Original Article

Cost-Utility of Sildenafil for Persistent Pulmonary Hypertension of the Newborn

Patrick D. Evers 
1   Division of Pediatric Cardiology, Oregon Health and Sciences University, Portland, Oregon
,
Paul J. Critser
2   Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio
,
Michelle Cash
2   Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio
,
Melissa Magness
2   Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio
,
Susan Hoelle
2   Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio
,
Russel Hirsch
2   Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio
3   Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio
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Abstract

Objective While advanced therapies for severe persistent pulmonary hypertension of the newborn (PPHN) such as inhaled nitric oxide (iNO) and extracorporeal membrane oxygenation (ECMO) are standard treatments in high-income countries, these therapies are often unavailable in resource-limited settings such as middle-income countries. However, there are small clinical trials illustrating the efficacy of sildenafil at reducing mortality in PPHN. This analysis sought to determine the cost-utility of enteral sildenafil for the treatment of severe PPHN.

Study Design A Markov-state transition model was constructed for the two clinical approaches to compare costs, clinical outcomes, and quality of life: (1) “conventional,” (2) “sildenafil.” The impact of sildenafil was modeled as a relative risk modifier of the conventional strategy's mortality risk. Transitional probabilities, costs, and utility metrics were extracted from the literature. Sensitivity analyses for each model input as well as 100-patient Monte Carlo simulations were used to test the durability of the model conclusion.

Results The sildenafil strategy was cost-effective for upper but not lower middle-income countries with an incremental cost-effectiveness ratio of $2,339 per quality-adjusted life year. This conclusion was durable across a wide-range of model assumptions; the sildenafil strategy only failed to meet criteria for cost-effectiveness when sildenafil therapy had a mortality relative risk efficacy of >0.89, if life expectancy in that country is <40 years, or if the lifetime forecasted costs of a survivor's life was quite high.

Conclusion Enteral sildenafil is a cost-effective intervention for severe PPHN for upper middle-income countries where ECMO and iNO are not available.

Key Points

  • PPHN is a common life-threatening condition in newborns.

  • Sildenafil improves survival of PPHN.

  • Sildenafil is cost-effective for upper-middle income countries.

Keywords

pediatrics - pulmonary hypertension - cost-effectiveness - global health

Authors' Contributions

P.D.E. conceptualized and designed the study, assisted in data acquisition, performed the data analysis, and drafted portions of the initial manuscript. P.J.C. conceptualized and designed the study, drafted portions of the initial manuscript, and critically reviewed the manuscript for important intellectual content. M.C., M.M., and S.H. conceptualized and designed the study and critically reviewed the manuscript for important intellectual content. R.H. conceptualized and designed the study, interpreted the data analysis, and critically reviewed the manuscript for important intellectual content. All authors approved the final manuscript as submitted and agreed to be accountable for all aspects of the work.




Publication History

Received: 09 January 2020

Accepted: 26 May 2020

Article published online:
02 July 2020

© 2020. Thieme. All rights reserved.

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