Published online October 1, 2004
PEDIATRICS Vol. 114 No. 4 October 2004, pp. e532-e535 (doi:10.1542/peds.2003-0988-L)
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ELECTRONIC ARTICLE

Nephrotic Syndrome Complicating {alpha}-Glucosidase Replacement Therapy for Pompe Disease

Tracy E. Hunley, MD*, Deyanira Corzo, MD§,{ddagger}, Martha Dudek, MS||, Priya Kishnani, MD, Andrea Amalfitano, DO, PhD, Yuan-Tsong Chen, MD, PhD, Susan M. Richards, PhD{ddagger}, John A. Phillips, III, MD||, Agnes B. Fogo, MD#,* and George E. Tiller, MD, PhD||

* Divisions of Pediatric Nephrology
|| Medical Genetics, Department of Pediatrics
# Department of Pathology, Vanderbilt University School of Medicine, Nashville, Tennessee
{ddagger} Genzyme Corp, Cambridge, Massachusetts
§ Division of Clinical Genetics, Children’s Hospital, Boston, Massachusetts
Division of Medical Genetics, Department of Pediatrics, Duke University, Durham, North Carolina

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.

Key Words: enzyme replacement therapy • glycogen storage disease type II • rhGAA • membranous nephropathy • nephrotic syndrome

Abbreviations: rhGAA, recombinant human acid {alpha}-glucosidase • C3, complement component 3

Accepted May 6, 2004.


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