Efficacy and limitations of continuous intravenous epoprostenol therapy for idiopathic pulmonary arterial hypertension in Japanese children

Circ J. 2007 Nov;71(11):1785-90. doi: 10.1253/circj.71.1785.


Background: There is little data on the long-term effects of continuous intravenous epoprostenol for children with idiopathic pulmonary arterial hypertension (IPAH) in Japan.

Methods and results: Thirty-one IPAH patients younger than 18 years old who had begun epoprostenol therapy at Toho University Omori Medical Center between January 1999 and June 2004 were reviewed. During a mean follow up of 3.4 years, the rate of those who survived or did not undergo a lung transplantation among the 27 patients who received home infusion therapy of epoprostenol was 100% at 1 year, 96.3% at 2 years, and 79.4% at 3 years. In 82% of survivors, the World Health Organization functional class was changed from III or IV to II according to improvements in the plasma brain natriuretic peptide level and the distance walked in 6 min during the follow-up period. In most cases, mean pulmonary artery pressure and the ratio of pulmonary to systemic vascular resistance remained high, although the cardiac index had improved to within a normal range 1 year after the initiation of epoprostenol. Therefore, sildenafil was administered as an additional therapy to 16 patients who presented with sustained severe PAH.

Conclusions: Continuous IV epoprostenol certainly improves survival and exercise tolerance in childhood IPAH, although the improvement of pulmonary vascular resistance regardless of long-term epoprostenol therapy is insufficient. Therefore, the addition of a new drug, such as sildenafil, is recommended to be administered in adjunction with epoprostenol.

MeSH terms

  • Adolescent
  • Antihypertensive Agents / administration & dosage
  • Antihypertensive Agents / therapeutic use*
  • Blood Pressure / physiology
  • Child
  • Child, Preschool
  • Dose-Response Relationship, Drug
  • Epoprostenol / administration & dosage
  • Epoprostenol / therapeutic use*
  • Humans
  • Hypertension, Pulmonary / blood
  • Hypertension, Pulmonary / drug therapy*
  • Hypertension, Pulmonary / physiopathology
  • Infant
  • Infant, Newborn
  • Infusions, Intravenous
  • Japan
  • Natriuretic Peptide, Brain / blood
  • Retrospective Studies
  • Treatment Outcome


  • Antihypertensive Agents
  • Natriuretic Peptide, Brain
  • Epoprostenol