Pre-stem cell transplantation enzyme replacement therapy in Hurler syndrome does not lead to significant antibody formation or delayed recovery of the endogenous enzyme post-transplant: a case report.

Abstract:

:Combined enzyme replacement therapy (ERT) and stem cell transplant (SCT) were done for a two year old boy with severe Hurler syndrome(HS) with the aim to decrease transplant related complications. He tolerated both the procedures well without any major complications. Urine glycosaminoglycans (GAGs) decreased post-transplant and child has improved clinically and neurologically. Insignificant titers of the anti-iduronidase antibodies which developed post-transplant did not affect the transplant outcome or the endogenous recovery of the alpha-L-iduronidase enzyme.

journal_name

Pediatr Transplant

authors

Soni S,Hente M,Breslin N,Hersh J,Whitley C,Cheerva A,Bertolone S

doi

10.1111/j.1399-3046.2007.00720.x

subject

Has Abstract

pub_date

2007-08-01 00:00:00

pages

563-7

issue

5

eissn

1397-3142

issn

1399-3046

pii

PTR720

journal_volume

11

pub_type

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