Presenter Status
Fellow
Abstract Type
Case Report
Primary Mentor or Principal Investigator
John Daniel MD
Presentation Type
Oral Presentation
Start Date
12-5-2026 12:15 PM
End Date
12-5-2026 12:30 PM
Abstract Text
Background: Severe neonatal hemolysis can result in multiorgan dysfunction requiring advanced extracorporeal support. While plasma exchange and extracorporeal membrane oxygenation (ECMO) are effective supportive therapies, persistent hemolysis despite these interventions poses significant therapeutic challenges. Eculizumab, a monoclonal antibody targeting terminal complement protein C5, is approved for complement-mediated hemolytic disorders but has not been described in neonates receiving ECMO or concurrent plasma exchange. The pharmacokinetics and efficacy of monoclonal antibody therapies in extracorporeal circuits remain poorly characterized.
Objectives/Goal: To describe the clinical response and pharmacokinetic profile of eculizumab in a neonate with refractory hemolysis requiring ECMO, continuous renal replacement therapy (CRRT), and plasma exchange.
Methods/Design: We report a term neonate with severe hemolytic disease of the newborn complicated by high-output cardiac failure, acute kidney injury, and cutaneous erythroderma who required VA-ECMO, CRRT, and repeated plasma exchange. Despite immunomodulatory therapy and daily plasmapheresis, plasma free hemoglobin remained persistently elevated. After multidisciplinary consultation, a single dose of eculizumab (300 mg, approximately 100 mg/kg) was administered. Serial eculizumab drug levels were obtained to assess pharmacokinetics while on extracorporeal support. Volume of distribution and estimated half-life were calculated and compared with published pediatric data.
Results: Following eculizumab administration, plasma free hemoglobin levels declined, cutaneous findings rapidly improved, and urine output returned within 24 hours. Over subsequent days, renal function continued to recover, allowing decannulation from VA-ECMO and eventual discontinuation of CRRT. No additional doses of eculizumab were required. Pharmacokinetic analysis demonstrated a calculated volume of distribution of 0.25 L/kg and an estimated half-life of approximately 290 hours (12 days), values comparable to those reported in infants not receiving ECMO. These findings suggest minimal sequestration or clearance of eculizumab by ECMO circuitry.
Conclusions: In this neonate with severe, refractory hemolysis requiring multiple extracorporeal modalities, eculizumab administration was associated with rapid clinical improvement and favorable pharmacokinetic characteristics despite ECMO support. This case suggests that monoclonal antibody therapy may retain efficacy in neonatal extracorporeal circuits and supports further investigation into complement inhibition for select cases of severe neonatal hemolysis.
Treatment of severe hemolysis in a baby requiring multiple tandem extracorporeal support modalities and experimental monoclonal antibody therapy
Background: Severe neonatal hemolysis can result in multiorgan dysfunction requiring advanced extracorporeal support. While plasma exchange and extracorporeal membrane oxygenation (ECMO) are effective supportive therapies, persistent hemolysis despite these interventions poses significant therapeutic challenges. Eculizumab, a monoclonal antibody targeting terminal complement protein C5, is approved for complement-mediated hemolytic disorders but has not been described in neonates receiving ECMO or concurrent plasma exchange. The pharmacokinetics and efficacy of monoclonal antibody therapies in extracorporeal circuits remain poorly characterized.
Objectives/Goal: To describe the clinical response and pharmacokinetic profile of eculizumab in a neonate with refractory hemolysis requiring ECMO, continuous renal replacement therapy (CRRT), and plasma exchange.
Methods/Design: We report a term neonate with severe hemolytic disease of the newborn complicated by high-output cardiac failure, acute kidney injury, and cutaneous erythroderma who required VA-ECMO, CRRT, and repeated plasma exchange. Despite immunomodulatory therapy and daily plasmapheresis, plasma free hemoglobin remained persistently elevated. After multidisciplinary consultation, a single dose of eculizumab (300 mg, approximately 100 mg/kg) was administered. Serial eculizumab drug levels were obtained to assess pharmacokinetics while on extracorporeal support. Volume of distribution and estimated half-life were calculated and compared with published pediatric data.
Results: Following eculizumab administration, plasma free hemoglobin levels declined, cutaneous findings rapidly improved, and urine output returned within 24 hours. Over subsequent days, renal function continued to recover, allowing decannulation from VA-ECMO and eventual discontinuation of CRRT. No additional doses of eculizumab were required. Pharmacokinetic analysis demonstrated a calculated volume of distribution of 0.25 L/kg and an estimated half-life of approximately 290 hours (12 days), values comparable to those reported in infants not receiving ECMO. These findings suggest minimal sequestration or clearance of eculizumab by ECMO circuitry.
Conclusions: In this neonate with severe, refractory hemolysis requiring multiple extracorporeal modalities, eculizumab administration was associated with rapid clinical improvement and favorable pharmacokinetic characteristics despite ECMO support. This case suggests that monoclonal antibody therapy may retain efficacy in neonatal extracorporeal circuits and supports further investigation into complement inhibition for select cases of severe neonatal hemolysis.
