Medical Therapy. Efficacy or Lack of Efficacy

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

Shinichi Takatsuki, Tomotaka Nakayama, Yurika Shimizu, Reiko Kawai, Hiroyuki MatsuuraToho University Omori Medical Center. Kochi Medical School Hospital.Japan Cardiology in the YoungCardiol Young 2023; 33: 196-200DOI: 10.1017/S1047951122000415 AbstractObjective: 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 […]

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Safety and Feasibility of Riociguat Therapy for the Treatment of Chronic Pulmonary Arterial Hypertension in Infancy

Regan E. Geisinger, Amy H. Stanford, Brady Thomas, Steven H. Abman, Patrick J. McNammaraUniversity of Iowa. University of Colorado.United States Journal of PediatricsJ Pediatr 2022; DOI: 10.1016/j.jpeds.2022.11.026 AbstractThe effects of riociguat, an oral-soluble guanylate-cyclase stimulator, were studied in 10 infants with chronic pulmonary arterial hypertension. Respiratory status (n = 8/10), right heart dilation (n = 7/10),

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Riociguat in children with pulmonary arterial hypertension: The PATENT-CHILD study

Humberto García Aguilar, Matthias Gorenflo, D. Dunbar Ivy, Shahin Moledina, Biagio Castaldi, Hidekazu Ishida, Paweł Cześniewicz, Jacek Kusa, Oliver Miera, Joseph Pattathu, Ken‐Pen Weng, Laszlo Ablonczy, Christian Apitz, Marta Katona, Kenichi Kurosaki, Tomas Pulido, Hiroyuki Yamagishi, Kazushi Yasuda, Galia Cisternas, Melanie Goth, Susanne Lippert, Anna Radomskyj, Soundos Saleh, Stefan Willmann, Gabriela Wirsching, Damien Bonnet, Maurice

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Safety and tolerability of combination therapy with ambrisentan and tadalafil for the treatment of pulmonary arterial hypertension in children: Real-world experience

Azadeh Issapour, Benjamin Frank, Sarah Crook, Michelle D. Hite, Michelle L. Dorn, Erika B. Rosenzweig, D. Dunbar Ivy, Usha S. KrishnanColumbia University Irving Medical Center. University of Colorado and Children’s Hospital Colorado.United States Pediatric PulmonologyPediatr Pulmonol 2022; 57: 724-733DOI: 10.1002/ppul.25796 AbstractObjective: To describe the safety and tolerability of treatment with ambrisentan and tadalafil in pediatric pulmonary hypertension

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Parenteral Prostanoids in Pediatric Pulmonary Arterial Hypertension: Start Early, Dose High, Combine

Johannes M. Douwes, Willemijn M. H. Zijlstra, Erika B. Rosenzweig, Mark-Jan Ploegstra, Usha S. Krishnan, Meindina G. Haarman, Marcus T. R. Roofthooft, Douwe Postmus, Hans L. Hillege, D. Dunbar Ivy, Rolf M. F. BergerUniversity of Groningen, University Medical Center Groningen and Beatrix Children’s Hospital. Columbia University. University of Colorado, Children’s Hospital Colorado.Netherlands and United States

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Inpatient Transition From Intravenous to Inhaled Treprostinil in a Pediatric Patient

David Procaccini, Dennis Delany, Abigail Self, Patricia Lawrence Kane, John D. CoulsonJohns Hopkins HospitalUnited States Journal of Pediatric Pharmacology and TherapeuticsJ Pediatr Pharmacol Ther 2023; 28: 102-107DOI: 10.5863/1551-6776-28.1.102 AbstractWe report a case of a 7-year old male with idiopathic pulmonary arterial hypertension, successfully transitioned from an intravenous infusion to inhaled treprostinil during inpatient admission, after his

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Innovations in Pediatric Therapeutics Development: Principles for the Use of Bridging Biomarkers in Pediatric Extrapolation

Thomas R. Fleming, Christine E. Garnett, Laurie S. Conklin, Solange Corriol‑Rohou, Sudharshan Hariharan, Daphne Hsu, Guenther Mueller‑Velten, Yeruk Mulugeta, Ronald Portman, Mark D. Rothmann, Norman L. Stockbridge, Simon Wandel, Jialu Zhang, Lynne YaoUniversity of Washington. U.S. Food and Drug Administration. Janssen Research and Development. AstraZeneca. Children’s Hospital at Montefiore and Albert Einstein College of Medicine. Novartis Pharma AG and Novartis Pharmaceuticals.United States Therapeutic Innovation and Regulatory ScienceTher Innov Regul Sci 2023; 57: 109-120DOI: 10.1007/s43441-022-00445-6 AbstractEven with recent substantive improvements in health care in pediatric populations, considerable need remains

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Ambrisentan for in pediatric pulmonary arterial hypertension: a cost-utility analysis

Jefferson Antonio Buendia, Diana Guerrero Patino, Erika Fernanda LindarteUniversity of Antioquia.Columbia Pediatric PulmonologyPediatr Pulmonol 2023; DOI: 10.1002/ppul.26361 AbstractIntroduction: Despite the growing evidence of efficacy, little is known regarding the efficiency of ambrisentan to decrease cost and improve the functional classes of pediatric patients with Pulmonary arterial hypertension. This study aims to determine the cost-utility of ambrisentan regarding

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Multicenter review of a tadalafil suspension formulation for infants and children with pulmonary hypertension: A North American experience

David Edward Youssef, Stephanie S. Handler, Susan Marjorie Richards, Catherine Anne Sheppard, Jenna Smith, Kathryn Tillman, Matthew Pietrosanu, Edward Kirkpatrick, Angela Bates University of Alberta and Stollery Children’s Hospital. Medical College of Wisconsin.Canada and United States Frontiers in PediatricsFront Pediatr 2023; DOI: 10.3389/fped.2023.1055131 AbstractIntroduction: Phosphodiesterase type 5 (PDE5) inhibitors, with sildenafil the earliest among them, are widely used

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Sotatercept for the Treatment of Pulmonary Arterial Hypertension

Marc Humbert, Vallerie McLaughlin, J. Simon R. Gibbs, Mardi Gomberg-Maitland, Marius M. Hoeper, Ioana R. Preston, Rogerio Souza, Aaron B. Waxman, Hossein-Ardeschir Ghofrani, Pilar Escribano Subias, Jeremy Feldman, Gisela Meyer, David Montani, Karen M. Olsson, Solaiappan Manimaran, Jennifer Barnes, Peter G. Linde, Janethe de Oliveira Pena, David B. BadeschPULSAR Trial Investigators.France, United States, United Kingdom,

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