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Updated: May 5, 2026

Comprehensive Evaluation of the Effectiveness and Safety of Placenta-Targeted Drug Delivery Using Three Complementary Methods
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Paroxysmal Nocturnal Hemoglobinuria in Pregnancy Treated With Pegcetacoplan: Case Report and Pharmacokinetic Analysis

Benjamin Chin-Yee1,2,3,4, Benjamin D Hedley1,3, Rui Su3

  • 1Department of Pathology and Laboratory Medicine Western University London Ontario Canada.

Ejhaem
|November 21, 2025
PubMed

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.

Keywords:
case reportparoxysmal nocturnal hemoglobinuriapegcetacoplanpharmacokineticpregnancy

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