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A Randomized Study of Safety and Efficacy of Two Doses of Ambrisentan to Treat Pulmonary Arterial Hypertension in Pediatric Patients Aged 8 Years up to 18 Years.
Ivy, Dunbar; Beghetti, Maurice; Juaneda-Simian, Ernesto; Miller, Diane; Lukas, Mary Ann; Ioannou, Chris; Okour, Malek; Narita, Jun; Berger, Rolf M F.
Afiliação
  • Ivy D; Pediatric Cardiology, Children's Hospital, Colorado, Aurora, CO.
  • Beghetti M; Pediatric Cardiology Unit, University Children's Hospital HUG, Pulmonary Hypertension Program HUG, Centre Universitaire Romand de Cardiologie et Chirurgie Cardiaque Pédiatrique (CURCCCP), University of Geneva, Geneva, Switzerland.
  • Juaneda-Simian E; Department of Cardiology, Hospital de Niños de la Santísma Trinidad, Córdoba, Argentina.
  • Miller D; Developmental Biostatistics, GlaxoSmithKline, Collegeville, Philadelphia, PA.
  • Lukas MA; Metabolic Pathways and Cardiovascular Therapeutic Area, GlaxoSmithKline, Collegeville, Philadelphia, PA.
  • Ioannou C; Global Development Clinical Science, GlaxoSmithKline, Middlesex, UK.
  • Okour M; Clinical Pharmacology Modeling and Simulation, GlaxoSmithKline, Collegeville, Philadelphia, PA.
  • Narita J; Pediatric Cardiology, Osaka University Hospital, Osaka, Japan.
  • Berger RMF; Center for Congenital Heart Diseases, Pediatric Cardiology, Beatrix Children's Hospital, University Medical Center Groningen, University of Groningen, the Netherlands.
J Pediatr X ; 5: 100055, 2020.
Article em En | MEDLINE | ID: mdl-37332660
Objective: To assess the safety and efficacy of the endothelin receptor antagonist ambrisentan in pediatric pulmonary arterial hypertension (PAH). Study design: In this open-label, phase IIb study, patients with PAH aged 8 to <18 years were randomized to low- or high-dose ambrisentan for 24 weeks. Most patients were receiving other PAH medication(s) that could not be changed during the trial. The primary outcome was safety (treatment-emergent adverse events [TEAEs]); secondary outcome was efficacy (including change from baseline to week 24 in 6-minute walking distance and World Health Organization functional class). Study staff were blinded to treatment. No statistical testing was performed. Results: Most of the 41 patients randomized (80%) experienced ≥1 TEAE; most were mild (22%) or moderate (49%) in severity (no difference between dose groups). Most common TEAEs were headache (24%), nausea (17%), abdominal pain (12%), and nasopharyngitis (12%). Eight patients had serious TEAEs; 2 were fatal (unrelated to study treatment). Improved 6-minute walking distance was observed from baseline to week 24: total mean (SD) change, +40.69 (84.58) meters; World Health Organization functional class was maintained or improved in 70% and 27% patients, respectively. Conclusions: Ambrisentan was well tolerated; TEAEs were consistent with the adult safety profile. Efficacy was similar to previous findings in adult PAH; however, interpretation is limited by small sample size. Findings support a potentially similar benefit:risk profile in pediatric (8 to <18 years) and adult patients with PAH. Trial registration: ClinicalTrials.gov: NCT01332331.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Clinical_trials Idioma: En Revista: J Pediatr X Ano de publicação: 2020 Tipo de documento: Article País de publicação: Estados Unidos
Texto completo
Adicionar na Minha BVS
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Clinical_trials Idioma: En Revista: J Pediatr X Ano de publicação: 2020 Tipo de documento: Article País de publicação: Estados Unidos