Selexipag use for paediatric pulmonary hypertension: a single centre report focussed on congenital heart disease patients

Abstract

Pulmonary hypertension is a rare and complex disease with poor prognosis. Paediatric cases are infrequent and usually associated with congenital heart disease. Management is problematical due to the limited therapy available and poor evidence of efficacy. Recently a new medication, selexipag (UptraviR), a prostacyclin receptor agonist, has been approved for the treatment of pulmonary artery hypertension in adults. We report our experience using selexipag in four paediatric patients with pulmonary hypertension associated with congenital heart disease.

Hemodynamic assessment of transitioning from parenteral prostacyclin to selexipag in pediatric pulmonary hypertension

“…Available data are limited by the small number of patients, lack of hemodynamic confirmation, long‐term follow‐up, and sparse pediatric data. 12 , 13 , 14 , 15 , 16 , 17 , 18 , 19 , 20 The largest series to date is presented by Frost et al In this series the authors present 34 adult patients that underwent a transition from inhaled treprostinil to oral selexipag with successful transition in 32 patients (94%) as assessed by changes in functional class, BNP, and 6MWT over a 16‐week period. No hemodynamic assessments were made, and these patients were long‐term survivors with a mean time from diagnosis to transition of 8 years.…”

“… 9 , 10 Despite its increasing use in both the adult and pediatric PAH populations, there is limited data on the hemodynamic efficacy of transitioning from parenteral prostacyclins to selexipag, particularly in the pediatric population. 11 , 12 , 13 , 14 , 15 , 16 , 17 , 18 , 19 , 20 Therefore, the objective of this study was to characterize the hemodynamic changes associated with transitioning from parenteral prostacyclins to enteral selexipag in children. To this end, we retrospectively collected hemodynamic data from 11 children who had undergone a transition from parenteral prostacyclin ( n = 7 SC treprostinil; n = 1 IV treprostinil; and n = 3 inhaled tyvaso) to enteral selexipag from October 2016 to May 2022.…”

“…9,10 Despite its increasing use in both the adult and pediatric PAH populations, there is limited data on the hemodynamic efficacy of transitioning from parenteral prostacyclins to selexipag, particularly in the pediatric population. [11][12][13][14][15][16][17][18][19][20] Therefore, the objective of this study was to characterize the hemodynamic changes associated with transitioning from parenteral prostacyclins to enteral selexipag in children.…”

“…Following large, randomized trials in adult PAH that demonstrated a significant decrease in mortality and PAH complications, selexipag (Uptravi®) was approved for the treatment of adults with PAH in 2015 9,10 . Despite its increasing use in both the adult and pediatric PAH populations, there is limited data on the hemodynamic efficacy of transitioning from parenteral prostacyclins to selexipag, particularly in the pediatric population 11–20 . Therefore, the objective of this study was to characterize the hemodynamic changes associated with transitioning from parenteral prostacyclins to enteral selexipag in children.…”

A Canadian, retrospective, multicenter experience with selexipag for a heterogeneous group of pediatric pulmonary hypertension patients

“…Despite the growing collective experience with selexipag for treating pediatric PH (10)(11)(12)(13)(14), present literature on the clinical benefits of this drug is scarce, especially with regards to the younger population (Supplementary Table S1). The largest study of pediatric PH is a multicenter case report series from Germany that describes experiences with selexipag in patients with WHO functional classifications of III and IV.…”