Safety and Effectiveness of Selexipag in Pediatric Pulmonary Hypertension: A Retrospective Multicenter Cohort Study.

Autor: Frank BS; Section of Cardiology, Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO. Electronic address: Benjamin.Frank@childrenscolorado.org., Gentzler ER; Section of Cardiology, Department of Pediatrics, Columbia University Irving Medical Center, Morgan Stanley Childrens Hospital of New York Presbyterian Hospital, New York, NY., Avitabile CM; Division of Cardiology, Department of Pediatrics, Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, PA., Miller-Reed K; Section of Cardiology, Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO., Pan Z; Department of Biostatistics, Children's Hospital Colorado, Aurora, CO., Rosenzweig EB; Section of Cardiology, Department of Pediatrics, Columbia University Irving Medical Center, Morgan Stanley Childrens Hospital of New York Presbyterian Hospital, New York, NY., Ivy DD; Section of Cardiology, Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO., Krishnan US; Section of Cardiology, Department of Pediatrics, Columbia University Irving Medical Center, Morgan Stanley Childrens Hospital of New York Presbyterian Hospital, New York, NY.
Jazyk: angličtina
Zdroj: The Journal of pediatrics [J Pediatr] 2024 Aug 02; Vol. 275, pp. 114221. Date of Electronic Publication: 2024 Aug 02.
DOI: 10.1016/j.jpeds.2024.114221
Abstrakt: Objective: To describe the safety and effectiveness of treating pediatric patients who have pulmonary arterial hypertension (PAH) with selexipag in a real-world, multicenter cohort, given that data supporting its use in pediatric PAH are sparse.
Study Design: We report a multicenter, retrospective, cohort study of children with PAH treated with selexipag. Demographic and clinical variables were extracted from the medical records. Clinical parameters were analyzed at 3 timepoints: before selexipag, 3-12 months after selexipag, and >12 months follow-up.
Results: Eighty-seven patients were included, 32 received selexipag as add-on to background therapy, and 55 transitioned from another prostanoid. The median starting and final doses were 4.7 and 28.5 μg/kg/dose twice daily, respectively. Add-on patients demonstrated improved indexed pulmonary to systemic vascular resistance ratio after selexipag initiation (PVRi/SVRi, 0.62v0.53; P = .034) with a lower average mean pulmonary artery pressure (46 vs 39 mm Hg; P = NS), and oxygen consumption (maximal oxygen consumption during cardiopulmonary exercise testing [VO 2 max] 27.8 mL/kg/min vs 30.9 mL/kg/min; P = NS). Transition patients demonstrated stable mean pulmonary artery pressure (47 mm Hg vs 45 mm Hg; P = NS) and a lower mean indexed pulmonary vascular resistance (10.9 Wood units∗m 2 vs 8.2 Wood units∗m 2 ; P = NS) but late functional worsening in some with VO 2 max decreased at follow-up (26.0 mL/kg/min vs 19.5 mL/kg/min). Side effects were noted in 40% of the cohort, but prompted discontinuation in only 2%.
Conclusions: In a large, multicenter cohort, the oral prostacyclin agonist selexipag demonstrates favorable tolerability and effectiveness. Add-on patients demonstrated early hemodynamic improvement. Transition patients demonstrated early stability with risk of late functional worsening, highlighting the importance of ongoing monitoring.
Competing Interests: Declaration of Competing Interest The authors report no conflicts of interest.
(Copyright © 2024 Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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