Treatment of paroxysmal nocturnal hemoglobinuria in pregnancy with eculizumab: A case report.

Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease caused by mutations in hematopoietic stem cells leading to pancytopenia and a predisposition for thromboembolic events. In pregnancy, these manifestations can be amplified, leading to increased neonatal and maternal morbidity and mortality. Although data are limited, eculizumab has emerged as a potential treatment of PNH in pregnancy. This report describes a case of a woman with PNH successfully treated with eculizumab during two pregnancies. Although during both pregnancies she experienced breakthrough hemolysis requiring intermittent blood transfusions, she had no thromboembolic events and had term vaginal births. Granted more research is needed, eculizumab may be an acceptable therapy for PNH in pregnancy.