دورية أكاديمية
Long-term safety and tolerability of ambrisentan treatment for pediatric patients with pulmonary arterial hypertension:An open-label extension study
| العنوان: | Long-term safety and tolerability of ambrisentan treatment for pediatric patients with pulmonary arterial hypertension:An open-label extension study |
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| المؤلفون: | Ivy, Dunbar, Beghetti, Maurice, Juaneda-Simian, Ernesto, Ravindranath, Ramiya, Lukas, Mary Ann, Machlitt-Northen, Sandra, Scott, Nicola, Narita, Jun, Berger, Rolf M F |
| المصدر: | Ivy , D , Beghetti , M , Juaneda-Simian , E , Ravindranath , R , Lukas , M A , Machlitt-Northen , S , Scott , N , Narita , J & Berger , R M F 2024 , ' Long-term safety and tolerability of ambrisentan treatment for pediatric patients with pulmonary arterial hypertension : An open-label extension study ' , European Journal of Pediatrics , vol. 183 , no. 5 , pp. 2141-2153 . https://doi.org/10.1007/s00431-024-05446-1Test |
| سنة النشر: | 2024 |
| المجموعة: | University of Groningen research database |
| مصطلحات موضوعية: | Humans, Pyridazines/adverse effects, Phenylpropionates/administration & dosage, Male, Child, Female, Adolescent, Treatment Outcome, Pulmonary Arterial Hypertension/drug therapy, Antihypertensive Agents/adverse effects, Dose-Response Relationship, Drug, Walk Test, Hypertension, Pulmonary/drug therapy |
| الوصف: | This open-label, extension study assessed long-term safety, tolerability, and efficacy of ambrisentan in a pediatric population (age 8- < 18 years) with pulmonary arterial hypertension (PAH). Following completion of a 6-month, randomized study, participants entered the long-term extension at individualized ambrisentan dosages (2.5/5/7.5 or 10 mg/day). Safety assessments included adverse events (AEs), AEs of special interest, and serious AEs (SAEs); efficacy outcomes included 6-min walking distance (6MWD) and World Health Organization functional class (WHO FC). Thirty-eight of 41 (93%) randomized study participants entered the extension; 21 (55%) completed (reaching age 18 years). Most participants received concomitant phosphodiesterase-5 inhibitors (n = 25/38, 66%). Median ambrisentan exposure was 3.5 years. Most participants experienced ≥ 1 AE (n = 34/38, 89%), and 21 (55%) experienced SAEs, most commonly worsening PAH (n = 3/38, 8%), acute cardiac failure, pneumonia, or anemia (n = 2/38; 5% each); none considered ambrisentan-related. Seven participants (18%) died, with recorded reasons (MedDRA preferred term): cardiac failure (n = 2), PAH (n = 2), COVID-19 (n = 1), acute right ventricular failure (n = 1), and failure to thrive (n = 1); median time to death: 5.2 years. Anemia and hepatotoxicity AEs were generally mild to moderate and did not require ambrisentan dose adjustment. Assessed at study end in 29 participants (76%), mean 6MWD improved by 17% (standard deviation: 34.3%), and all (29/29, 100%) had improved or unchanged WHO FC. Conclusion: Long-term weight-based ambrisentan dosing, alone or combined with other PAH therapies in children with PAH aged 8- < 18 years, exhibited tolerability and clinical improvements consistent with prior randomized study results. Trial registration: NCT01342952, April 27, 2011. What is Known: • The endothelin receptor antagonist, ambrisentan, is indicated for treatment of pulmonary arterial hypertension (PAH). Previous studies have shown similar efficacy and ... |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| العلاقة: | https://research.rug.nl/en/publications/3da88bcd-7ae7-42f1-9697-2a93acc9accaTest |
| DOI: | 10.1007/s00431-024-05446-1 |
| الإتاحة: | https://doi.org/10.1007/s00431-024-05446-1Test https://hdl.handle.net/11370/3da88bcd-7ae7-42f1-9697-2a93acc9accaTest https://research.rug.nl/en/publications/3da88bcd-7ae7-42f1-9697-2a93acc9accaTest |
| حقوق: | info:eu-repo/semantics/restrictedAccess |
| رقم الانضمام: | edsbas.CA702628 |
| قاعدة البيانات: | BASE |
| DOI: | 10.1007/s00431-024-05446-1 |
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