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Reported outcomes of 453 pregnancies in patients with Gaucher disease: An analysis from the Gaucher outcome survey.
- Source :
-
Blood cells, molecules & diseases [Blood Cells Mol Dis] 2018 Feb; Vol. 68, pp. 226-231. Date of Electronic Publication: 2016 Oct 20. - Publication Year :
- 2018
-
Abstract
- Gaucher disease (GD) may worsen during pregnancy, leading to the discussion of continuing treatment during pregnancy. We examined fetal outcomes of pregnancies reported in the Gaucher Outcome Survey, an international GD-specific registry established in 2010. A total of 453 pregnancies were reported. Most pregnancies (336/453, 74.2%) were in women who did not receive GD-specific treatment during pregnancy, while enzyme replacement therapy (ERT) was received during 117/453 (25.8%) pregnancies. No pregnancies exposed to substrate reduction therapy were reported. The percentage of normal outcomes (live birth delivered at term with no congenital abnormalities) was similar in untreated and treated pregnancies (92.9% vs. 91.4%). The percentage of spontaneous abortions in untreated pregnancies was 3.6% (95% CI, 1.9%- 6.2%) compared with 6.9% (95% CI, 3.0%-13.1%) in treated pregnancies (p=0.1866). In women who received velaglucerase alfa <1month prior to conception and/or during pregnancy, 34/36 (94.4%) pregnancies had normal outcomes and 2 (5.6%) ended in spontaneous abortion. Normal outcomes were observed in the 20 pregnancies with velaglucerase alfa exposure starting <1month prior to conception and continuing through all trimesters. These observations, in addition to information in the literature, suggest that continuation of ERT during pregnancy may be appropriate for GD patients.<br /> (Copyright © 2016 Shire Human Genetic Therapies, Inc. Published by Elsevier Inc. All rights reserved.)
- Subjects :
- Adolescent
Adult
Child
Female
Gaucher Disease epidemiology
Humans
Pregnancy
Pregnancy Complications epidemiology
Pregnancy Outcome
Registries
Surveys and Questionnaires
Treatment Outcome
Young Adult
Enzyme Replacement Therapy methods
Gaucher Disease complications
Gaucher Disease drug therapy
Glucosylceramidase therapeutic use
Pregnancy Complications drug therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1096-0961
- Volume :
- 68
- Database :
- MEDLINE
- Journal :
- Blood cells, molecules & diseases
- Publication Type :
- Academic Journal
- Accession number :
- 27839985
- Full Text :
- https://doi.org/10.1016/j.bcmd.2016.10.003