Glanzmann thrombasthenia is a serious inherited platelet bleeding disorder.

Platelets for bleed treatment/prevention may result in antibodies/refractoriness.

rFVIIa is effective whether antibodies/refractoriness to platelets is present.

rFVIIa’s mechanisms of action in Glanzmann thrombasthenia are now well understood.

A few patients have been cured by transplant; gene therapy remains a prospect.