Why the study?
Pulmonary arterial hypertension occurs in children, but therapeutic options are limited and infrequently described, including newer agents like oral treprostinil.
Is oral treprostinil safe and tolerable for initiation in pediatric patients with pulmonary arterial hypertension?
Is oral treprostinil safe and tolerable for initiation in pediatric patients with pulmonary arterial hypertension?
While oral treprostinil can be successfully initiated in pediatric patients with pulmonary arterial hypertension, gastrointestinal adverse effects frequently lead to discontinuation.
Initial pediatric experience supports oral treprostinil feasibility in PAH; hypothesis-generating and should not yet change practice.
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.
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Kanaan et al. (2019) studied this question.