Oral treprostinil use in children: a multicenter, observational experience

• Type of Material: Article
• Authors: Usama Kanaan, Emory University; Nidhy P. Varghese, Texas Children’s Hospital; Ryan D. Coleman, Texas Children’s Hospital; Jeryl Huckaby, Children's Healthcare Atlanta; Patricia Lawrence, Children's Healthcare Atlanta; Lynda O. Jorgensen, Primary Children's Hospital; Kathleen Miller-Reed, Children's Hospital Colorado; Ronald W. Day, Primary Children's Hospital; George B. Mallory, Texas Children’s Hospital; David D. Ivy, Children's Hospital Colorado
• Language: English
• Date: 2019-07-01
• Publisher: SAGE Publications (UK and US): Creative Commons Attribution
• Publication Version: Final Published Version
• Copyright Statement: © The Author(s) 2019.
• License: Creative Commons Attribution-NonCommercial 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1177/2045894019862138
• Title of Journal or Parent Work: Pulmonary Circulation
• ISSN: 2045-8932
• Volume: 9
• Issue: 3
• Start Page: 2045894019862138
• End Page: 2045894019862138
• Grant/Funding Information: This research received no specific grant from any funding body in the public, commercial, or not-for-profit sectors.
• Keywords: pediatric pulmonary hypertension; Life Sciences & Biomedicine; Cardiac & Cardiovascular Systems; Science & Technology; Cardiovascular System & Cardiology; pulmonary hypertension; Respiratory System; pulmonary arterial hypertension
• Research Categories: Health Sciences, Medicine and Surgery

Abstract

Pulmonary arterial hypertension is a progressive, incurable disease that occurs in adults and children alike. Therapeutic options for children are limited and infrequently described, including newer agents such as treprostinil, an oral prostanoid. Herein, we describe the pooled pediatric experience in 28 patients from four pediatric pulmonary hypertension programs over two years. This descriptive, observational study describes the various methods of initiation of oral treprostinil in both prostanoid-naïve patients and those transitioning from parenteral or inhaled prostanoids. The youngest patient was four years old and the smallest weighed 16 kg. We describe adverse reactions and their management. Most patients in this study (27/28) were able to successfully initiate therapy. However, gastrointestinal adverse reactions were common; half of the patients started on this therapy had discontinued it within the two-year study period.