Medical Therapy. Efficacy or Lack of Efficacy

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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Sotatercept for the treatment of pulmonary arterial hypertension: PULSAR open-label extension

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 Subias1, Jeremy Feldman, Gisela Meyer, David Montani, Karen M. Olsson, Solaiappan Manimaran, Janethe de Oliveira Pena, David B. BadeschHôpital Bicêtre, Assistance Publique-Hôpitaux de Paris. University of Michigan Health System. Imperial

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Management of Complex Pulmonary Vein Stenosis at Altitude Combining Comprehensive Percutaneous Interventional Treatment with Sirolimus, Pulmonary Hypertension Medications and Intraluminal Imaging with Optical Coherence Tomography

Michael J. Shorofsky, Gareth J. Morgan, Ernesto Mejia, Salvador A. Rodriguez, Megan Greene, Paige Sheaks, Dunbar Ivy, Jenny E. ZablahUniversity of Colorado and Children’s Hospital Colorado.United States Pediatric CardiologyPediatr Cardiol 2023; DOI: 10.1007/s00246-023-03102-3 AbstractBackground: Pulmonary vein stenosis (PVS) is a growing problem for the pediatric congenital heart population. Sirolimus has previously been shown to improve survival and

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Novel use of riociguat in infants with severe pulmonary arterial hypertension unable to wean from inhaled nitric oxide

Liezl T. Domingo, D. Dunbar Ivy, Steven H. Abman, Alicia M. Grenolds, James T. MacLean, J. A. Breaux, Katie J. Minford, Benjamin S. FrankUniversity of Utah. University of Colorado.United States Frontiers in PediatricsFront Pediatr 2022; 10: DOI: 10.3389/fped.2022.1014922 AbstractIntroduction: Riociguat, an oral soluble guanylate cyclase stimulator, has been approved for use in adults with pulmonary arterial hypertension

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High-dose epoprostenol therapy in pediatric patients with pulmonary hypertension and developmental lung disease: A report of two cases

Yoshie Fukusawa, Hidenori Yamamoto, Miharu Ito, Akiko Saito, Kiyotaka Go, Yoshihito Morimoto, Kazushi Yasuda, Yoshiaki Sato, Masahiro Hayakawa, Taichi KatoNagoya University and Nagoya University Hospital. Ogaki Municipal Hospital. Japanese Red Cross Nagoya Daichi Hospital. Aichi Child Health and Medical General Center.Japan Frontiers in Pediatrics. Pediatric Cardiology SectionFront Pediatr 2023; 11: DOI: 10.3389/fped.2023.1116434 AbstractPulmonary hypertension (PH) with

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Hemodynamic assessment of transitioning from parenteral prostacyclin to selexipag in pediatric pulmonary hypertension

Elizabeth Colglazier, Leah Stevens, Claire Parker, Hythem M. Nawaytou, Elena K. Amin, Jasmine Becerra, Martina Steurer, Jeffrey R. FinemanUniversity of California San FranciscoUnited States Pulmonary CirculationPulm Circ 2022; 12: DOI: 10.1002/pul2.12159 AbstractDespite the increase in therapeutic options, parenteral prostacyclins remain the cornerstone in the medical management of pulmonary arterial hypertension (PAH). While the use of parenteral

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Systemic Sirolimus Therapy for Infants and Children With Pulmonary Vein Stenosis

Jay D. Patel, Michael Briones, Mansi Mandhani, Shannon Jones, Divya Suthar, Rosemary Gray, Joelle Pettus, Courtney McCraken, Amanda Thomas, Christopher J. PetitEmory University and Children’s Healthcare of AtlantaUnited States Journal of the American College of CardiologyJ Am Coll Cardiol 2021; 77: 2807-2818DOI: 10.1016/j.jacc.2021.04.013 AbstractBackground: Anatomic interventions for pulmonary vein stenosis (PVS) in infants and children have been

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