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

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


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.

MeSH terms

  • Antihypertensive Agents / administration & dosage*
  • Antihypertensive Agents / therapeutic use
  • Drug Therapy, Combination
  • Epoprostenol / administration & dosage
  • Epoprostenol / analogs & derivatives*
  • Epoprostenol / therapeutic use
  • Humans
  • Hypertension, Pulmonary
  • Infusions, Subcutaneous
  • Treatment Outcome


  • Antihypertensive Agents
  • Epoprostenol
  • treprostinil