Successful Birth After First-Trimester Ravulizumab Exposure in a Patient With Paroxysmal Nocturnal Hemoglobinuria: A...

Area of Science:

• Hematology
• Pharmacology
• Reproductive Medicine

Background:

• Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired blood disorder.
• Complement C5 inhibitors like eculizumab (Ecu) and ravulizumab (Rav) are standard PNH treatments.
• Safety data for ravulizumab (Rav) in pregnancy are limited compared to eculizumab (Ecu).

Purpose of the Study:

• To report on the safety and outcomes of inadvertent ravulizumab (Rav) exposure during early pregnancy in a patient with paroxysmal nocturnal hemoglobinuria (PNH).
• To contribute to the scarce data on ravulizumab (Rav) use in pregnant PNH patients.

Main Methods:

• Case report of a 25-year-old woman diagnosed with PNH.
• Documented inadvertent exposure to ravulizumab (Rav) at 8 weeks gestation.
• Transition of care to eculizumab (Ecu) for PNH management during pregnancy.
• Monitoring of maternal and fetal outcomes throughout pregnancy and postpartum.

Main Results:

• The patient required transfusions during pregnancy.
• She delivered a healthy infant at term without congenital anomalies.
• No instances of thrombosis were observed in the mother or infant.
• This case represents one of the earliest documented instances of ravulizumab (Rav) exposure in pregnancy.

Conclusions:

• Inadvertent early pregnancy exposure to ravulizumab (Rav) in a PNH patient was associated with a healthy infant delivery.
• Ravulizumab (Rav) may have potential safety in pregnancy, but more data are essential.
• Multidisciplinary management is crucial for pregnant patients with PNH receiving complement inhibitors.