title: Outcome of pregnancies in women receiving velaglucerase alfa for Gaucher disease.
abstract: OBJECTIVE
  Pregnancy and delivery are affected by and - in turn - impact signs and symptoms of Gaucher disease (GD). Prior to enzyme replacement therapy (ERT), the reported missed abortions rate was 25%, with worsening of anemia and thrombocytopenia, with increased frequency of post-partum hemorrhage, puerperal fever and bone crises during pregnancy. ERT with imiglucerase reduced these adverse events. Velaglucerase alfa (VPRIV), an ERT approved commercially in February 2010, had undergone preclinical reproductive toxicity testing and proven to be safe and effective in phase I/II and III clinical trials. The objective of this study was to ascertain pregnancy outcome in women receiving VPRIV.
  
  
  METHODS
  Among records collected from six multinational clinical sites, 21 females (mean age, 32.0 years) with GD received VPRIV.
  
  
  RESULTS
  There were 25 singleton pregnancies (mean gravidity, 2.7; mean parity, 2.0; mean months VPRIV, 31.2). Two primiparous women suffered three first trimester abortions and one missed abortion occurred in a multigravida female. Live birth rate was 84% (mean gestational age, 39.7 weeks). Mean birthweight was 3234.4 g, with APGAR scores above 9. All but three were vaginal deliveries; elective cesarean sections were performed in two patients with hip arthroplasty and one after previous cesarean. Nine patients received regional analgesia/anesthesia. Post-partum complications were rare, with only one post-partum (placental) bleed which resolved without intervention. Mean hemoglobin and platelet counts improved during pregnancy (9.45% and 26.0%, respectively).
  
  
  CONCLUSIONS
  VPRIV is safe for conception and pregnancy with good maternal and neonatal outcomes.
authors.affiliation: Gaucher Clinic, Shaare Zedek Medical Center, affiliated with the Hadassah Medical Center-Hebrew University Medical School, Jerusalem, Israel.
authors.forename: Deborah
authors.initials: D
authors.lastname: Elstein
authors.forename: Derralynn
authors.initials: D
authors.lastname: Hughes
authors.forename: Ozlem
authors.initials: O
authors.lastname: Goker-Alpan
authors.forename: Miriam
authors.initials: M
authors.lastname: Stivel
authors.forename: Hagit N
authors.initials: HN
authors.lastname: Baris
authors.forename: Ian J
authors.initials: IJ
authors.lastname: Cohen
authors.forename: Sorina
authors.initials: S
authors.lastname: Granovsky-Grisaru
authors.forename: Arnon
authors.initials: A
authors.lastname: Samueloff
authors.forename: Atul
authors.initials: A
authors.lastname: Mehta
authors.forename: Ari
authors.initials: A
authors.lastname: Zimran
mesh_terms: D015331:Cohort Studies; D056947:Enzyme Replacement Therapy / Q000009:adverse effects; D005260:Female; D005500:Follow-Up Studies; D005776:Gaucher Disease / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000009:adverse effects / Q000235:genetics / Q000378:metabolism / Q000627:therapeutic use*; D006801:Humans; D008499:Medical Records; D011247:Pregnancy; D011248:Pregnancy Complications / Q000188:drug therapy*; D011256:Pregnancy Outcome; D011994:Recombinant Proteins / Q000009:adverse effects / Q000378:metabolism / Q000627:therapeutic use
chemical_list: D011994:Recombinant Proteins; D005962:Glucosylceramidase; C550184:Velaglucerase alfa, human
keywords: Gaucher disease; enzyme replacement therapy; neonatal outcome; pregnancy; velaglucerase alfa