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J Pediatr. 2011 Apr;158(4):584-8. doi: 10.1016/j.jpeds.2010.09.025. Epub 2010 Oct 30.

Add-on therapy with subcutaneous treprostinil for refractory pediatric pulmonary hypertension.

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1
Paris-Descartes University Paris, France. marilyne.levy@nck.aphp.fr

Abstract

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.

PMID:
21035821
DOI:
10.1016/j.jpeds.2010.09.025
[Indexed for MEDLINE]
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