Medical Therapy. Efficacy or Lack of Efficacy

The Clinical and Cost Utility of Cardiac Catheterizations in Infants with Bronchopulmonary Dysplasia

Emily L. Yang, Philip T. Levy, Paul J. Critser, Dmitry Dukhovny, Patrick D. EversOregon Health and Sciences University. Harvard Medical School and Boston Children’s Hospital. Cincinnati Children’s Hospital Medical Center, Cincinnati, OH; Department of Pediatrics, University of Cincinnati College of Medicine and Cincinnati Children’s Hospital Medical Center.United States Journal of PediatricsJ Pediatr 2022; 246: 56-63DOI: 10.1016/j.jpeds.2022.04.009 […]

The Clinical and Cost Utility of Cardiac Catheterizations in Infants with Bronchopulmonary Dysplasia Read More »

Neonatal pulmonary hypertension after severe early-onset fetal growth restriction: post hoc reflections on the Dutch STRIDER study

Anouk Pels, Wes Onland, Rolf M. F. Berger, Arno F. J. van Heijst, Enrico Lopriore, Irwin K. M. Reiss, Jacqueline Limpens, Sanne J. Gordijn, Wessel GanzevoortUniversity of Amsterdam and Emma Children’s Hospital. University of Groningen, University Medical Center Groningen and Beatrix Children’s Hospital. Radboud University Medical Center and Amalia Children’s Hospital. Leiden University Medical Center. Erasmus University Medical Center. Netherlands European Journal of PediatricsEur J Pediatr 2022; 181: 1709-1718DOI: 10.1007/s00431-021-04355-x AbstractThe aim was

Neonatal pulmonary hypertension after severe early-onset fetal growth restriction: post hoc reflections on the Dutch STRIDER study Read More »

Efficacy and Safety of IV Sildenafil in the Treatment of Newborn Infants with, or at Risk of, Persistent Pulmonary Hypertension of the Newborn (PPHN): A Multicenter, Randomized, Placebo-Controlled Trial

Christine M. Pierce, Min H. Zhang, Baldvin Jonsson, Dinu Iorga, Narayan Cheruvu, Cecile C. Balagtas, Robin H. SteinhornGreat Ormond Street Hospital for Children. Pfizer Inc. Karolinska Institute and University Hospital. Rady Children’s Hospital and University of California San Diego.United States Journal of PediatricsJ Pediatr 2021; 237: 154-161DOI: 10.1016/j.jpeds.2021.05.051 AbstractObjective: To investigate the efficacy and safety of sildenafil

Efficacy and Safety of IV Sildenafil in the Treatment of Newborn Infants with, or at Risk of, Persistent Pulmonary Hypertension of the Newborn (PPHN): A Multicenter, Randomized, Placebo-Controlled Trial Read More »

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

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

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),

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

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

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

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

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

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

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

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

Inpatient Transition From Intravenous to Inhaled Treprostinil in a Pediatric Patient Read More »

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

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

Scroll to Top