With close monitoring, the use of oral add-on treatment with selexipag in children with pulmonary arterial hypertension (PAH) was safe and well tolerated, according to the results of a prospective, observational study published in The Journal of Heart and Lung Transplantation.  

Researchers from the European Pediatric Pulmonary Vascular Disease Network (EPPVDN) conducted in consecutive children with PAH who were treated with oral selexipag at 3 tertiary pulmonary hypertension (PH) centers.  The researchers sought to evaluate the efficacy and safety of the oral prostacyclin receptor agonist selexipag, which has been approved for use in adult patients with PAH. The current analysis is the largest, exploratory pediatric cohort to date.

A total of 15 children from the 3 PH centers were assessed: 7 from Hannover, Germany; 6 from Graz, Austria; and 2 from Gothenburg, Sweden. The age range of the participants was 7 months to 17 years. Patients’ body weight ranged from 5 kg to 73 kg. All of the patients underwent cardiac catheterization at baseline and at a median of 8 months’ follow-up. Moreover, the participants all received clinical, echocardiographic, and N-terminal pro B-type natriuretic peptide evaluations, including EPPVDN pediatric PH risk score.

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No deaths were reported with the use of selexipag. Of the 15 patients, 2 ultimately received a lung transplantation. There was 1 death reported in a patient with heritable PAH who had previously received selexipag therapy and was being treated with intravenous treprostinil. Of the 10 participants who received comparable sedation at both times of cardiac catheterization, the use of selexipag was associated with a significant improvement in mean right arterial pressure (-2 mm Hg), mean pulmonary arterial pressure to mean systemic arterial pressure ratio (-17%; P <.05), mean transpulmonary pressure gradient (-17%; P <.01), and diastolic TPG (-5.8 mm Hg; P <.05) at 5 to 18 months of follow-up vs baseline.

Additionally, at follow-up, patients treated with selexipag tended to have a lower pulmonary vascular resistance (PVR) index (-13%; P =.131) and PVR to systemic vascular resistance ratio (-20%; P =.097), as well as a higher cardiac index (Qsi, +18%; P =.322). On the whole, the efficacy of selexipag on invasive hemodynamics was variable and was frequently better in those patients who were less ill.

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The investigators concluded that the results from this study need to be confirmed in larger prospective studies of PAH to clarify the broader applicability and utility of selexipag in clinical care.

Disclosure: Several study authors declared affiliations with the pharmaceutical industry. Please see the original reference for a full list of authors’ disclosures.


Hansmann G, Meinel K, Bukova M, Chouvarine P, Wåhlander H, Koestenberger M; on behalf of the European Pediatric Pulmonary Vascular Disease Network (EPPVDN). Selexipag for the treatment of children with pulmonary arterial hypertension: first multicenter experience in drug safety and efficacy [published online April 7, 2020]. J Heart Lung Transplant. doi:10.1016/j.healun.2020.03.029