Paroxysmal Nocturnal Hemoglobinuria in Pregnancy Treated With Pegcetacoplan: Case Report and Pharmacokinetic Analysis.

There is an unmet clinical need for effective treatment of paroxysmal nocturnal hemoglobinuria (PNH) in pregnancy for patients with inadequate response to C5 inhibitors. We report the first case of pegcetacoplan use in pregnancy with accompanying pharmacokinetic analysis. In a patient with transfusion-dependent anemia on eculizumab, third-trimester initiation of pegcetacoplan led to hematological stabilization, transfusion independence, and an uncomplicated term cesarean delivery of a healthy infant. Pegcetacoplan was undetectable in cord blood and breast milk despite therapeutic maternal levels, suggesting fetal and neonatal safety due to lack of significant placental and lactational transfer, and advancing evidence for pegcetacoplan use in pregnancy. Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.