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Oral treprostinil Use in the Management of a Pediatric Pulmonary Hypertension: A Case Report

Michelle Cash

Jenna Faircloth

S Melissa Magness

Russel Hirsch


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Conference: 2017 PH Professional Network Symposium

Release Date: 10.06.2017

Presentation Type: Abstracts

File Download: 2017 PHPN Abstract 1040

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Abstract presented at the 2017 PH Professional Network Symposium held in Bethesda, MD on October 5-7, 2017


The use of oral treprostinil in the management of four pediatric WHO Group I pulmonary arterial hypertension (PAH) patients will be described.


Symptoms and hemodynamics measured by repeated right heart catheterization indicated the need for prostacyclin therapy in all 4, and all had been treated with other routes of prostacyclin therapy. Although limited data is available to define the role of oral treprostinil in pediatric PH patients, it was selected as the most appropriate prostacyclin formulation in these patients. This abstract will discuss the method of titration and the initial response to oral treprostinil.


The model for initiation of oral treprostinil was developed within our PH service using a review of available clinical trials, our current practice, input from the manufacturer, and discussion with the families. The goal was a safe and tolerable commencement of oral prostacyclin therapy in an intensive care setting, with achievement of comparative dose administration. The patients were 4-15 years old, weighing 19-70kg. Two patients were simultaneously transitioned off SQ therapy, 1 off IV and 1 patient a new start having previously failed SQ therapy. A detailed titration plan (considering oral versus parenteral dose equivalency) was derived for each patient. Oral treprostinil was started at 0.125-0.5mg and increased at 0.125-0.25mg increments with goal doses of 1.5-6.5mg TID. Simultaneously, the parenteral administration in those three patients on SQ and IV therapy was accordingly down-titrated. The transitions were completed in 6-13-days.


Three of the 4 patients had minimal to no side effects, with 1 patient experiencing GI symptoms. That patient had their oral up-titration slowed, with alleviation of symptoms, but with the ultimate dose attained prior to discharge. We believe the experience gained with the commencement of this therapy will further expand the utility for oral prostacyclin use in other pediatric patients. Based on the clinical responses and family reports, the oral treprostinil has provided the clinical benefit of parenteral therapy, but without infection risk, impact on quality of life and a diminution in the risk of medication errors 


Oral treprostinil was relatively well tolerated in this patient group. These cases illustrate various clinical scenarios in which oral treprostinil provided an opportunity to maximize medical therapy and deliver prostacyclin treatment in a broad range of pediatric patients. Additionally, this report emphasizes that institution of all prostacyclin agents requires close collaboration with the health care team and family members to ensure safe and effective administration.