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Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease.

Publication ,  Journal Article
Hunley, TE; Corzo, D; Dudek, M; Kishnani, P; Amalfitano, A; Chen, Y-T; Richards, SM; Phillips, JA; Fogo, AB; Tiller, GE
Published in: Pediatrics
October 2004
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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.

Duke Scholars

Priya Sunil Kishnani
Author Priya Sunil Kishnani Pediatrics, Medical Genetics
Yuan-Tsong Chen
Author Yuan-Tsong Chen Pediatrics, Medical Genetics
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Published In

Pediatrics

DOI

10.1542/peds.2003-0988-L

EISSN

1098-4275

Publication Date

October 2004

Volume

114

Issue

4

Start / End Page

e532 / e535

Location

United States

Related Subject Headings

  • alpha-Glucosidases
  • Pediatrics
  • Nephrotic Syndrome
  • Male
  • Kidney
  • Immune Tolerance
  • Humans
  • Glycogen Storage Disease Type II
  • Child, Preschool
  • Antibodies
 

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Hunley, T. E., Corzo, D., Dudek, M., Kishnani, P., Amalfitano, A., Chen, Y.-T., … Tiller, G. E. (2004). Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease. Pediatrics, 114(4), e532–e535. https://doi.org/10.1542/peds.2003-0988-L
Hunley, Tracy E., Deyanira Corzo, Martha Dudek, Priya Kishnani, Andrea Amalfitano, Yuan-Tsong Chen, Susan M. Richards, John A. Phillips, Agnes B. Fogo, and George E. Tiller. “Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease.” Pediatrics 114, no. 4 (October 2004): e532–35. https://doi.org/10.1542/peds.2003-0988-L.
Hunley TE, Corzo D, Dudek M, Kishnani P, Amalfitano A, Chen Y-T, et al. Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease. Pediatrics. 2004 Oct;114(4):e532–5.
Hunley, Tracy E., et al. “Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease.” Pediatrics, vol. 114, no. 4, Oct. 2004, pp. e532–35. Pubmed, doi:10.1542/peds.2003-0988-L.
Hunley TE, Corzo D, Dudek M, Kishnani P, Amalfitano A, Chen Y-T, Richards SM, Phillips JA, Fogo AB, Tiller GE. Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease. Pediatrics. 2004 Oct;114(4):e532–e535.

Published In

Pediatrics

DOI

10.1542/peds.2003-0988-L

EISSN

1098-4275

Publication Date

October 2004

Volume

114

Issue

4

Start / End Page

e532 / e535

Location

United States

Related Subject Headings

  • alpha-Glucosidases
  • Pediatrics
  • Nephrotic Syndrome
  • Male
  • Kidney
  • Immune Tolerance
  • Humans
  • Glycogen Storage Disease Type II
  • Child, Preschool
  • Antibodies