Baby born healthy after exposure to Ultomiris for mother’s PNH
Case report sheds light on drug's potential safety during pregnancy
A healthy baby was born after exposure to Ultomiris (ravulizumab-cwvz) during the first trimester of pregnancy in a 25-year-old woman with paroxysmal nocturnal hemoglobinuria (PNH), according to a new case report.
The woman was on Ultomiris when she was found to be pregnant. Because little is known about the drug’s safety during pregnancy, treatment was stopped, and she was switched to Soliris (eculizumab), a PNH therapy with more established safety data on pregnancy.
The pregnancy and postpartum period were completed without serious complications for either the mother or the baby, researchers reported.
“This case represents one of the earliest reports of [Ultomiris] use in pregnancy, suggesting potential safety,” researchers wrote. “Further data are needed, and multidisciplinary management remains essential for pregnant patients with PNH.”
The study, “Successful Birth After First-Trimester Ravulizumab Exposure in a Patient With Paroxysmal Nocturnal Hemoglobinuria: A Case Report,” was published in eJHaem by researchers in Japan.
Pregnancy in PNH carries increased risks of hemolysis, thrombosis
PNH is a rare blood disorder in which overactivation of the complement system, a component of the immune system that normally helps fight infections, leads to the destruction of blood cells, known as hemolysis. This results in PNH symptoms such as low red blood cell levels (anemia), blood-clotting complications (thrombosis), and reduced blood cell counts (cytopenia).
Pregnancy in people with PNH carries increased risks of hemolysis and thrombosis, as well as a higher likelihood of serious complications for both the mother and the fetus.
Soliris, developed by Alexion Pharmaceuticals, now part of AstraZeneca, was the first therapy approved to lessen hemolysis in patients with PNH. Given by infusions into the bloodstream, it works by blocking a complement protein called C5.
Ultomiris, also developed by Alexion, is a newer approved treatment for PNH that targets the same complement protein as Soliris but remains active in the body for a longer period, allowing for less frequent dosing.
“Before [Soliris], pregnancy was generally discouraged; however, multiple reports describe successful pregnancies under [Soliris],” the researchers wrote. “In contrast, evidence regarding [Ultomiris] use in pregnancy remains extremely limited. Given that [Ultomiris] has largely replaced [Soliris] in non-pregnant PNH patients due to its longer dosing interval and sustained complement [blocking], the importance of establishing its safety in pregnancy cannot be overstated.”
Woman received Ultomiris during first trimester of pregnancy
In this study, a team of researchers described the case of a woman with PNH who was inadvertently exposed to Ultomiris during the first trimester of her pregnancy before switching to Soliris.
The woman, who had no prior medical history, was diagnosed with PNH during her first pregnancy at 21 weeks of gestation after a blood test revealed anemia and signs of ongoing hemolysis.
She was started on Soliris but continued to require red blood cell and platelet transfusions despite treatment. Platelets are blood components that help control bleeding.
At 39 weeks of gestation, she gave birth to a healthy baby. The child showed normal development during follow-up, and the woman continued treatment with Soliris after delivery.
Nine months later, the woman experienced another pregnancy that ended in a spontaneous miscarriage at nine weeks of gestation, after which she decided to switch from Soliris to Ultomiris.
On the day she received her third dose, she was found to be about eight weeks pregnant. Because data on Ultomiris use during pregnancy are limited, the treatment was discontinued, and Soliris was restarted eight weeks after the last dose of Ultomiris.
Although our patient received only a single dose of [Ultomiris] at [eight] weeks of pregnancy, this case, together with the previously reported cases, suggests that [Ultomiris] may be a feasible and potentially safe option during pregnancy.
Despite requiring repeated red blood cell transfusions during pregnancy and a platelet transfusion shortly before delivery, she gave birth to a healthy baby at 39 weeks and two days of gestation.
After delivery, she developed mastitis, a mild breast infection that triggered a temporary episode of hemolysis, which resolved with antibiotic treatment. She continued Soliris while breastfeeding.
“Although our patient received only a single dose of [Ultomiris] at [eight] weeks of pregnancy, this case, together with the previously reported cases, suggests that [Ultomiris] may be a feasible and potentially safe option during pregnancy,” the researchers wrote.
They noted that as newer treatments targeting the complement system become more widely used, outcomes for people with PNH are improving, and pregnancies in this population are likely to become more common. As a result, they emphasized the need to collect more safety data on these therapies to help guide care during pregnancy.
“The importance of close collaboration between hematologists and obstetricians during the perinatal period cannot be overemphasized,” they concluded.
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