Titre
Riociguat in children with pulmonary arterial hypertension: The PATENT-CHILD study.
Type
article
Institution
UNIL/CHUV/Unisanté + institutions partenaires
Périodique
Auteur(s)
García Aguilar, H.
Auteure/Auteur
Gorenflo, M.
Auteure/Auteur
Ivy, D.D.
Auteure/Auteur
Moledina, S.
Auteure/Auteur
Castaldi, B.
Auteure/Auteur
Ishida, H.
Auteure/Auteur
Cześniewicz, P.
Auteure/Auteur
Kusa, J.
Auteure/Auteur
Miera, O.
Auteure/Auteur
Pattathu, J.
Auteure/Auteur
Weng, K.P.
Auteure/Auteur
Ablonczy, L.
Auteure/Auteur
Apitz, C.
Auteure/Auteur
Katona, M.
Auteure/Auteur
Kurosaki, K.
Auteure/Auteur
Pulido, T.
Auteure/Auteur
Yamagishi, H.
Auteure/Auteur
Yasuda, K.
Auteure/Auteur
Cisternas, G.
Auteure/Auteur
Goth, M.
Auteure/Auteur
Lippert, S.
Auteure/Auteur
Radomskyj, A.
Auteure/Auteur
Saleh, S.
Auteure/Auteur
Willmann, S.
Auteure/Auteur
Wirsching, G.
Auteure/Auteur
Bonnet, D.
Auteure/Auteur
Beghetti, M.
Auteure/Auteur
Liens vers les personnes
Liens vers les unités
ISSN
2045-8932
Statut éditorial
Publié
Date de publication
2022-07
Volume
12
Numéro
3
Première page
e12133
Peer-reviewed
Oui
Langue
anglais
Notes
Publication types: Journal Article
Publication Status: epublish
Publication Status: epublish
Résumé
Riociguat, a soluble guanylate cyclase stimulator, is approved for treatment of adults with pulmonary arterial hypertension (PAH). The safety, tolerability, and pharmacokinetics (PK) of oral riociguat in a pediatric population with PAH was assessed in PATENT-CHILD (NCT02562235), a multicenter, single-arm, 24-week, open-label, Phase 3 study. Patients aged 6-17 years in World Health Organization functional class (WHO-FC) I-III treated with stable endothelin receptor antagonists and/or prostacyclin analogs received riociguat equivalent to 0.5-2.5 mg three times daily in adults, as either oral pediatric suspension or tablets, based on bodyweight. Primary outcomes were safety, tolerability, and PK of riociguat. Twenty-four patients (mean age 12.8 years), 18 of whom were in WHO-FC II, were enrolled. Adverse events (AEs), mostly mild or moderate, were reported in 20 patients (83%). Four patients (17%) experienced a serious AE; all resolved by study end and two (8%) were considered study-drug related. Hypotension was reported in three patients and hemoptysis in one (all mild/moderate intensity). Riociguat plasma concentrations in pediatric patients were consistent with those published in adult patients. From baseline to Week 24, mean ± standard deviation increase in 6-minute walking distance was 23 ± 69 m (n = 19), and mean decrease in NT-proBNP was -66 ± 585 pg/ml (n = 14). There was no change in WHO-FC. Two patients experienced clinical worsening events of hospitalization for right heart failure. PK results confirmed a suitable riociguat dosing strategy for pediatric patients with PAH. The data suggest an acceptable safety profile with potential efficacy signals.
PID Serval
serval:BIB_1B8D3ADE9F89
PMID
Open Access
Oui
Date de création
2022-10-11T11:25:12.889Z
Date de création dans IRIS
2025-05-20T18:18:52Z
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Nom
36186721_BIB_1B8D3ADE9F89.pdf
Version du manuscrit
published
Licence
https://creativecommons.org/licenses/by-nc/4.0
Taille
1.17 MB
Format
Adobe PDF
PID Serval
serval:BIB_1B8D3ADE9F89.P001
URN
urn:nbn:ch:serval-BIB_1B8D3ADE9F894
Somme de contrôle
(MD5):5d75ec1cace5b61db6dfe70a41db80a8