Original Article
Add-On Therapy with Subcutaneous Treprostinil for Refractory Pediatric Pulmonary Hypertension

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Objective

To evaluate the efficacy and tolerability of subcutaneous (SC) treprostinil, a prostacyclin analogue, in young children with refractory pulmonary arterial hypertension.

Study design

Eight children (median age, 4 years) received SC treprostinil therapy after failure of combined oral treatment (n = 7) or because of severe complications with intravenous epoprostenol (n = 1). Treprostinil was delivered through an SC catheter at gradually increasing doses to an average of 40 ng/kg/min, depending on the presence of adverse effects.

Results

Seven patients demonstrated early significant improvement (in functional class, hemodynamics, and/or 6-minute walk distance; P <.05), and 6 had a sustained good response. Site pain could be effectively managed in all but one child.

Conclusions

Treprostinil may be a potentially valuable rescue therapy in children with refractory pulmonary arterial hypertension, but further study in a larger number of patients is needed.

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

References (20)

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The authors declare no conflicts of interest.

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