Caution is recommended prior to sildenafil use in vascular anomalies

Pediatr Blood Cancer. 2015 Nov;62(11):2015-7. doi: 10.1002/pbc.25600. Epub 2015 May 15.


Since publication of a single case report of lymphatic malformation improvement during sildenafil therapy for pulmonary hypertension, sildenafil use has propagated across multiple vascular anomalies diagnoses. Vascular anomalies are rare conditions, often with poor long-term outcomes from available therapies, making these patients vulnerable to novel therapy use. We have retrospectively reviewed 14 children with vascular anomalies treated with sildenafil. None of these patients reported improvement of disease while on treatment and some reported side effects including infections and bleeding. Pending more convincing prospective data, we recommend caution prior to sildenafil use for vascular anomalies.

Keywords: lymphatic malformation; medical therapy; natural history; sildenafil; vascular anomalies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Female
  • Hemorrhage / chemically induced*
  • Humans
  • Infant
  • Infections / chemically induced*
  • Lymphatic Abnormalities / drug therapy*
  • Male
  • Phosphodiesterase 5 Inhibitors / administration & dosage
  • Phosphodiesterase 5 Inhibitors / adverse effects*
  • Retrospective Studies
  • Sildenafil Citrate / administration & dosage
  • Sildenafil Citrate / adverse effects*


  • Phosphodiesterase 5 Inhibitors
  • Sildenafil Citrate