First experience with enzyme replacement therapy during pregnancy and lactation in Pompe disease

Molecular Genetics and Metabolism

Volumn 104, Issue 4, 2011, Pages 552-555

  de Vries, Juna M ^a   Brugma, Jan Dietert C ^a   Özkan, Lale ^a   Steegers, Eric A P ^a   Reuser, Arnold J J ^a   van Doorn, Pieter A ^a   van der Ploeg, Ans T ^a  

^a ERASMUS MEDICAL CENTER   (Netherlands)

Author keywords

Acid glucosidase; Enzyme replacement therapy; Lactation; Lysosomal storage disorder; Pompe disease; Pregnancy

Indexed keywords

ALPHA GLUCOSIDASE;

ARTICLE; BREAST MILK; CASE REPORT; CESAREAN SECTION; DRUG BLOOD LEVEL; DRUG INFUSION; DRUG METABOLISM; DYSPNEA; ENZYME ACTIVITY; ENZYME REPLACEMENT; FEMALE; FETUS GROWTH; GESTATIONAL AGE; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; LACTATION; MOBILIZATION; PREGNANCY; PRIORITY JOURNAL; RESPIRATORY TRACT DISEASE; SAFETY;

ADULT; ALPHA-GLUCOSIDASES; ENZYME REPLACEMENT THERAPY; FEMALE; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; INFANT, NEWBORN; MALE; MILK, HUMAN; PREGNANCY; PREGNANCY COMPLICATIONS; TREATMENT OUTCOME;

EID: 82255175863     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2011.09.012     Document Type: Article

References (10)

1. Hirschhorn R., Reuser A.J.J. Glycogen storage disease type II; acid alpha-glucosidase (acid maltase) deficiency. The Metabolic and Molecular Bases of Inherited Disease 2001, 3389-3420. McGraw-Hill, New York. C.R. Scriver, A.L. Beaudet, W.S. Sly, D. Valle (Eds.).
2. Myozyme: European public assessment report - product information: Annex 1 Summary of product characteristics, in: European Medicines Agency/EMA [online] Available at:, Accessed March 21, 2011. http://www.elsevier.com/xml/linking-roles/text/html.
3. Elstein Y., Eisenberg V., Granovsky-Grisaru S., Rabinowitz R., Samueloff A., Zimran A., Elstein D. Pregnancies in Gaucher disease: a 5-year study. Am. J. Obstet. Gynecol. 2004, 190:435-441.
4. Granovsky-Grisaru S., Belmatoug N., Vom Dahl S., Mengel E., Morris E., Zimran A. The management of pregnancy in Gaucher disease. Eur. J. Obstet. Gynecol. Reprod. Biol. 2011, 156:3-8.
5. Kalkum G., Macchiella D., Reinke J., Kolbl H., Beck M. Enzyme replacement therapy with agalsidase alfa in pregnant women with Fabry disease. Eur. J. Obstet. Gynecol. Reprod. Biol. 2009, 144:92-93.
6. Politei J.M. Treatment with agalsidase beta during pregnancy in Fabry disease. J. Obstet. Gynaecol. Res. 2010, 36:428-429.
7. Cilliers H.J., Yeo S.T., Salmon N.P. Anaesthetic management of an obstetric patient with Pompe disease. Int. J. Obstet. Anesth. 2008, 17:170-173.
8. de Vries J.M., van der Beek N.A., Kroos M.A., Ozkan L., van Doorn P.A., Richards S.M., Sung C.C., Brugma J.D., Zandbergen A.A., van der Ploeg A.T., Reuser A.J. High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa. Mol. Genet. Metab. 2010, 101:338-345.
9. van der Ploeg A.T., Clemens P.R., Corzo D., Escolar D.M., Florence J., Groeneveld G.J., Herson S., Kishnani P.S., Laforet P., Lake S.L., Lange D.J., Leshner R.T., Mayhew J.E., Morgan C., Nozaki K., Park D.J., Pestronk A., Rosenbloom B., Skrinar A., van Capelle C.I., van der Beek N.A., Wasserstein M., Zivkovic S.A. A randomized study of alglucosidase alfa in late-onset Pompe's disease. N. Engl. J. Med. 2010, 362:1396-1406.
10. Sekijima Y., Ohashi T., Ohira S., Kosho T., Fukushima Y. Successful pregnancy and lactation outcome in a patient with Gaucher disease receiving enzyme replacement therapy, and the subsequent distribution and excretion of imiglucerase in human breast milk. Clin. Ther. 2010, 32:2048-2052.