Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease.
Pediatrics
; 114(4): e532-5, 2004 Oct.
Article
in En
| MEDLINE
| ID: mdl-15466083
ABSTRACT
We report a patient with Pompe disease who developed reversible nephrotic syndrome during prolonged, high-dose, experimental, enzyme replacement therapy with recombinant human acid alpha-glucosidase (rhGAA). Because of the development of antibodies to rhGAA and concomitant clinical decline, escalating doses of rhGAA were administered as part of an experimental immune tolerance regimen. Histologic evaluation of kidney tissue revealed glomerular deposition of immune complexes containing rhGAA itself, in a pattern of membranous nephropathy. To our knowledge, this is the first reported case of nephrotic syndrome occurring during enzyme replacement therapy. The nephrotic syndrome gradually resolved after the rhGAA dose was decreased, indicating that decreasing the antigenic load can ameliorate glomerular immune complex deposition associated with enzyme replacement in a highly sensitized patient.
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Collection:
01-internacional
Database:
MEDLINE
Main subject:
Glycogen Storage Disease Type II
/
Alpha-Glucosidases
/
Nephrotic Syndrome
Limits:
Child, preschool
/
Humans
/
Male
Language:
En
Journal:
Pediatrics
Year:
2004
Document type:
Article
Affiliation country:
United States
Country of publication:
EEUU
/
ESTADOS UNIDOS
/
ESTADOS UNIDOS DA AMERICA
/
EUA
/
UNITED STATES
/
UNITED STATES OF AMERICA
/
US
/
USA