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Pulmonary hypertension (PH) is a complex condition that encompasses an array of underlying disease processes and affects a diverse population of infants, including those with congenital heart disease, congenital diaphragmatic hernia, persistent PH of the newborn, and those with lung disease such as bronchopulmonary dysplasia. While there are treatments available to adults with PH, limited data exists for infants, especially for the newer medications. Therapies that target the three main pathophysiologic pathways of pulmonary hypertension appear to benefit infants, but which are best for each individual disease process is unclear. A review of the therapies to treat pulmonary hypertension is covered in this article including the prostacyclin pathway, endothelin pathway, and the nitric oxide pathway. Other adjunctive treatments are also discussed. Findings are based on a PubMed literature search of research papers spanning 1990–2023 and a search of ongoing trials registered with clinicaltrials.gov. Overall therapies seem to improve outcomes with most infants with PH. However, given the diverse population of infants with PH, it is imperative to understand the basis for the PH in individual patients and understand which therapies can be applicable. Further research into tailored therapy for the specific populations is warranted.

Current and emerging pharmacotherapies for the treatment of pulmonary arterial hypertension in infants