Original ArticleAdd-On Therapy with Subcutaneous Treprostinil for Refractory Pediatric Pulmonary Hypertension
Section snippets
Methods
Eight children with severe PAH (median age, 4 years; range, 1.5-10 years) were admitted to our institution to begin SC treprostinil therapy. Two patients had IPAH, one patient had heritable PAH, and 5 patients had PAH associated with a congenital heart defect (Table I). Three of these latter patients had PAH after correction of a cardiac defect. Patient 1 was diagnosed with PAH at 3 months after undergoing an arterial switch operation for transposition of the great arteries. Patient 2, with
Results
The duration of follow-up after initiation of SC treprostinil therapy ranged from 6-18 months (median, 15 months). At their last assessment, 6 patients exhibited clinical improvement and were in WHO functional class I or II (Figure). The 6MWT could be performed in only the 4 oldest children, and was improved in all of these children (by a mean of 144 m; range, 10-220 m). The youngest two patients (patients 2 and 6) demonstrated improved oxygen saturation and functional status. Seven patients
Discussion
In this observational study concerning the particularly difficult clinical situation of refractory pediatric PAH despite dual oral therapy or stable PAH on triple therapy but with complications from IV lines, we found that 6 of 8 such children had a symptomatic and hemodynamic response to the addition of SC treprostinil, with manageable control of local injection site discomfort. Thus, treprostinil may be a useful adjunct in the therapeutic armamentarium in this potentially devastating
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2018, Seminars in PerinatologyCitation Excerpt :Alternatively, this drug can be delivered via continuous subcutaneous injection with bioavailability that is equivalent to IV delivery, and without safety concerns or significant problems with site pain.90 Subcutaneous treprostinil has been used to transition some children who were chronically stable on IV epoprostenol,91 and may be effective when added to other vasodilator therapies.92 Preliminary studies of treprostinil imply an acceptable safety profile and clinical benefit in pediatric patients with PH,93 and case series describe dramatic improvement in premature infants who were non-responsive to nitric oxide.90,94
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The authors declare no conflicts of interest.