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Oral Tresprostinil in Transition or as Add-on Therapy in Pediatric Pulmonary Arterial hypertension

dc.contributor.authorIvy, D. Dunbar
dc.contributor.authorFeinstein, Jeffrey A.
dc.contributor.authorYung, Delphine
dc.contributor.authorMullen, Mary P.
dc.contributor.authorKirkpatrick, Edward C.
dc.contributor.authorHirsch, Russel
dc.contributor.authorAustin, Eric D.
dc.contributor.authorFineman, Jeffrey
dc.contributor.authorTryong, Uyen
dc.contributor.authorSolum, Derek
dc.contributor.authorDeng, C.Q.
dc.contributor.authorHopper, Rachel K.
dc.identifier.citationVolume: 9 issue: 3, Article first published online: June 19, 2019; Issue published: July 1, 2019 Received: February 06, 2019; Accepted: May 15, 2019en_US
dc.description.abstractTreprostinil, a prostacyclin analogue, is approved for the treatment of pulmonary arterial hypertension (PAH) in adults. Transition from parenteral to oral treprostinil has been successfully accomplished in adults with PAH but not in children. In this multicenter study, pediatric patients treated with parenteral (Cohort 1) or inhaled (Cohort 2) treprostinil were transitioned to oral treprostinil. Prostacyclin-naïve individuals on background oral PAH therapy received oral treprostinil as add-on therapy (Cohort 3). Successful transition was oral treprostinil dose maintenance through week 24. Patients were monitored for adverse events (AEs), 6-min walk distance (6MWD), PAH symptoms, World Health Organization (WHO) Functional Class (FC), cardiac magnetic resonance imaging (cMRI), cardiopulmonary exercise testing (CPET), and quality of life through 24 weeks. A total of 32 patients were enrolled in the study; 23 (72%) were girls (mean age = 12.2 years). All patients were on background oral PAH therapy. Overall, patients (96.9%) maintained transition to oral treprostinil; one patient (Cohort 1) transitioned to oral treprostinil, then back to parenteral after experiencing syncope and WHO FC change from II to III. Cohorts 1, 2, and 3 received a final mean oral treprostinil dose of 5.6, 3.3, and 4.5 mg t.i.d., respectively. All cohorts had variable changes in 6MWD, cMRI, and CPET. Overall, 12 serious AEs were reported. All patients had drug-related AEs including headache (81%), diarrhea (69%), nausea (66%), vomiting (66%), and flushing (56%). Pediatric patients maintained transition to oral treprostinil with preservation of exercise capacity and WHO FC. Prostanoid-related AEs were most common and similar to those reported in adults.en_US
dc.publisherPulmonary Circulationen_US
dc.subjectpulmonary arterial hypertensionen_US
dc.subject.lcshPulmonary Hypertensionen_US
dc.titleOral Tresprostinil in Transition or as Add-on Therapy in Pediatric Pulmonary Arterial hypertensionen_US

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