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Clinical efficacy and safety of selexipag in children and young adults with idiopathic and heritable pulmonary arterial hypertension

Published online by Cambridge University Press:  06 April 2022

Shinichi Takatsuki Open the ORCID record for Shinichi Takatsuki [Opens in a new window] ,
Tomotaka Nakayama ,
Yurika Shimizu ,
Reiko Kawai  and
Hiroyuki Matsuura
Shinichi Takatsuki*
Affiliation:
Department of Pediatrics, Toho University Omori Medical Center, Tokyo, Japan
Tomotaka Nakayama
Affiliation:
Department of Pediatrics, Kochi Medical School Hospital, Kochi, Japan
Yurika Shimizu
Affiliation:
Department of Pediatrics, Toho University Omori Medical Center, Tokyo, Japan
Reiko Kawai
Affiliation:
Department of Pediatrics, Toho University Omori Medical Center, Tokyo, Japan
Hiroyuki Matsuura
Affiliation:
Department of Pediatrics, Toho University Omori Medical Center, Tokyo, Japan
*
Author for correspondence: S Takatsuki, Department of Pediatrics, Toho University Omori Medical Center, 6-11-1 Omori-nishi, Ota-ku, Tokyo, 1438541, Japan. Tel: +81-3-3762-4151, Fax: +81-3-3298-8217. E-mail: s-taka@med.toho-u.ac.jp
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Abstract

Objective:

This study aimed to investigate the safety, tolerability, and efficacy of selexipag in children and young adults with idiopathic and heritable pulmonary arterial hypertension.

Methods:

This retrospective cohort study included clinical data from five children and six young adults with pulmonary arterial hypertension receiving selexipag as add-on therapy or as a transition from beraprost sodium or epoprostenol infusion therapy. Clinical efficacy was evaluated by measuring improvement in clinical variables from baseline, including hemodynamic parameters.

Results:

Of the 11 patients, 6 were switched from beraprost sodium to selexipag and one paediatric patient transitioned from epoprostenol to selexipag. The median maintenance dose of selexipag in children was 80 μg/kg/day. In nine patients undergoing repeat catheterisation, statistically significant improvements were observed after the initiation of selexipag in terms of mean pulmonary arterial pressure (p < 0.01), pulmonary vascular resistance index (p < 0.05), and cardiac index (p < 0.01). None of the patients had clinical worsening after selexipag during follow-up, but one young adult patient discontinued treatment due to severe headache. The most common side effect profiles were headache, nausea, abdominal pain, jaw pain, myalgia, and diarrhoea.

Conclusions:

Selexipag may have a favourable safety profile and potential efficacy in children and young adults with pulmonary arterial hypertension.

Keywords

Selective IP2-receptor agonistcombination therapyprognosis
Type
Original Article
Information
Cardiology in the Young , Volume 33 , Issue 2 , February 2023 , pp. 196 - 200
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Copyright
© The Author(s), 2022. Published by Cambridge University Press

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