Maternal and Fetal Outcomes in Imiglucerase-Treated Patients With Gaucher Disease: Real-World Evidence From the International Collaborative Gaucher Group (ICGG) Gaucher Registry Pregnancy Sub-Registry.

Untreated women with Gaucher disease (GD) are at an increased risk of GD-related complications during pregnancy. Enzyme replacement therapy with imiglucerase is effective at improving hematologic, visceral, and bone manifestations of GD, and the Food & Drug Administration prescribing information supports that imiglucerase is not associated with adverse maternal or fetal outcomes when used during pregnancy. This study population included women with GD enrolled in the International Collaborative Gaucher Group Gaucher Registry (NCT00358943) Pregnancy Sub-Registry who were treated with imiglucerase during at least one pregnancy as of October 2023. We describe frequency of pregnancy outcomes, birth outcomes, and maternal and neonatal complications. Imiglucerase exposure was reported in 110 pregnancies in 68 women with GD type 1; 68% of pregnancies were exposed during all three trimesters. Of 104 fetuses with reported data, 92 were live births (88.5%), eight were spontaneous abortions (7.7%), and four were elective/therapeutic terminations (3.8%); no stillbirths (> 20 weeks gestation) were reported. The majority of infants (80 of 85 [94.1%]) were born at term. Among 108 pregnancies with data, maternal pregnancy, labor and delivery, and post-partum complications were reported for 33 (30.6%), 26 (24.1%), and 24 (22.2%) pregnancies, respectively, with anemia, thrombocytopenia, and vaginal bleeding among the most prevalent complications. Among 74 infants with data, neonatal complications were reported for seven infants (9.5%). Most pregnancies to women with GD treated with imiglucerase resulted in live births and healthy infants, with risk of spontaneous abortions similar to that of the general population (12%-18%).