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Oral Treprostinil Use in the Management of a Pediatric Patient Diagnosed with Severe Late Presenting Pulmonary Hypertension: A Case Report

Alison Cress

Michelle Cash

Jenna Faircloth

Russel Hirsch


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Conference: 2015 PH Professional Network Symposium

Release Date: 09.17.2015

Presentation Type: Abstracts

File Download: 2015 Symposium Abstract - 1007

File Size: (241 kb)

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This abstract will discuss the titration model utilized and response to oral treprostinil in a pediatric patient presenting with severe pulmonary hypertension and Trisomy 21.


The management of a 16-year-old female with Trisomy 21 and repaired congenital heart disease, diagnosed with late presenting severe pulmonary hypertension (PH) will be described. The hemodynamics measured by right heart catheterization warranted the need for prostacyclin therapy. Due to significant developmental and social circumstances, this patient was not a candidate for an intravenous or subcutaneous infusion. 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 for this situation.


The model for initiation of oral treprostinil was developed within our PH service using a critical review of available clinical trials, our practices, input from the manufacturer and discussion with the family. The goal was a safe and tolerable commencement of prostacyclin therapy in an intensive care setting. Using a detailed titration plan guided by previous studies, oral treprostinil was initiated at 0.125mg BID (0.008mg/kg/day) and increased by 0.125mg increments daily, to a goal dose of 1mg BID over 6 days. She experienced nausea, vomiting, and diarrhea post-discharge. After 2 days of continued side effects, the dose was decreased to 0.875mg BID. On therapy day 15, she was readmitted for diuretic manipulation due to fluid retention and the dose was adjusted to 0.75mg BID. On therapy day 17, dose and frequency were adjusted to 0.5mg TID to improve side effect tolerance based on pharmacokinetic profile. Over a period of months, oral treprostinil was then slowly up-titrated to a well-tolerated dose of 1mg TID (0.1mg/kg/day).


The initiation of oral treprostinil therapy was the first of its kind in a pediatric PH patient at our institution. The experience gained with the commencement of this therapy will further expand the utility for oral treprostinil in future pediatric patients. Based on her clinical response and family report, the convenience of oral treprostinil provided the benefit of prostacyclin therapy without infection risk and adverse quality of life issues associated with parenteral therapy. Lengthening time between titration steps and initiating at TID frequency may have limited some of the early gastro-intestinal side effects.


Despite the initial adverse effects, oral treprostinil was relatively well tolerated in this patient with Trisomy 21 and severe PH. The case illustrates a complex clinical scenario in which oral treprostinil provided an opportunity to maximize medical therapy and deliver prostacyclin treatment in a patient unable to take other formulations. Additionally, this case emphasizes that institution of all prostacyclin agents requires close collaboration with the health care team and family members to ensure safe and effective administration.