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.
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.
Soni
Sandeep
S
Division of Pediatric BMT, Columbus Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA. sonis@pediatrics.ohio-state.edu
Hente
Monica
M
Breslin
Nancy
N
Hersh
Joseph
J
Whitley
Chester
C
Cheerva
Alexandra
A
Bertolone
Salvatore
S
D000917:Antibody Formation / Q000276:immunology*; D002675:Child, Preschool; D005500:Follow-Up Studies; D006801:Humans; D007068:Iduronidase / Q000097:blood / Q000276:immunology / Q000627:therapeutic use*; D008297:Male; D008059:Mucopolysaccharidosis I / Q000201:enzymology / Q000276:immunology / Q000628:therapy*; D011300:Preoperative Care / Q000379:methods*; D011994:Recombinant Proteins; D020127:Recovery of Function; D033581:Stem Cell Transplantation
D011994:Recombinant Proteins; D007068:Iduronidase