Systematic review and cost‐effectiveness of bosentan and sildenafil as therapeutic drugs for pediatric pulmonary arterial hypertension

Abstract

Background Pulmonary arterial hypertension (PAH) is a rare disease in children, with significant mortality. Because of the limited research on pediatric PAH, first, systematic review of related drugs is conducted, and then economic evaluation of PAH drug treatment programs is conducted, which to provide a reference for the choice of more cost‐effective treatment options. Methods The search includes electronic databases such as Pubmed, ScienceDirect, and Embase. Through inclusion and exclusion criteria, screen high‐quality randomized controlled trials. We used TreeAge Pro 2011 software to construct the markov model, that to simulate the total medical cost and quality‐adjusted life years (QALYs), and to calculate the incremental cost‐effectiveness ratio. Sensitivity analysis of transfer probability, utility, and cost was carried out. Results Incorporate two studies that meet the criteria, one compared the therapeutic effects of bosentan and placebo on pediatric PAH, the other compared therapeutic effects of sildenafil and placebo on pediatric PAH, both articles were of good quality. Compared with the sildenafil group (3.38QALYs and $161,120.14), the QALY of the bosentan treatment group (3.33QALYs and $257,411.29) was reduced by 0.05, and the cost increased by $96,291.15. The estimated improvement to quality of life and reduced costs result in an estimate of economic dominance for sildenafil over bosentan. This dominant result persisted probabilistic analyses. Conclusions Based on this model, a more cost‐effective treatment drug for PAH in children is sildenafil.

Ambrisentan for in pediatric pulmonary arterial hypertension: A cost‐utility analysis

“…Our result is consistent with economic results in adults where sildenafil was always the most cost‐effective treatment option regarding other endothelin‐receptor antagonists 20 . Chen et al recently show in pediatric patients that bosentan (3.33 QALYs and $257,411.29) was related to fewer QALYs and higher cost than sildenafil (3.38QALYs and $161,120.14) 9 . Sildenafil demonstrated economic dominance over bosentan, and this conclusion was robust during the probabilistic sensitivity analysis 9 …”

“…and $161,120.14). 9 Sildenafil demonstrated economic dominance over bosentan, and this conclusion was robust during the probabilistic sensitivity analysis. 9 PAH is a chronic progressive devastating disease with no cure, which may bring a significant medical and financial burden to patients' families.…”

Long-Term Study on Therapeutic Strategy for Treatment of Eisenmenger Syndrome Patients: A Case Series Study

“…In addition, our health care team includes cardiologists, nephrologists, hematologists, endocrinologists, dietitians, and psychiatrists. During the long-term therapeutic strategy, sildenafil was given as first-line treatment, except for case 4 [ 37 , 38 ]; later, the add-on was treated based on individualized need. Although genetic association with PAH has been proposed [ 5 , 39 ], none of our patients received genetic tests.…”

Bosentan Is Associated with a Reduction in Right Ventricular Systolic Pressure N-Terminal Pro-Hormone B-Type Natriuretic Peptide Levels in Young Patients with Pulmonary Hypertension

“…Pulmonary hypertension is a rare disease in children that is characterized by a series of vascular changes that result in increased pulmonary vascular resistance and eventually right heart failure [1] [2] [3]. Despite varying etiologies, pulmonary hypertension in the pediatric age group carries a poor prognosis if left untreated [2] [4] [5] [6] [7]. Significant therapeutic advances have been made over the past several decades; this is due in part to an improved understanding of the mechanisms involved in the pathogenesis of this disease.…”

“…Significant therapeutic advances have been made over the past several decades; this is due in part to an improved understanding of the mechanisms involved in the pathogenesis of this disease. Current treatment options target one of three pathways: the nitric oxide pathway, the endothelin pathway, or the prostacyclin pathway [2] [3] [8] [9] [10].…”