We report a patient with Pompe disease who developed reversible nephrotic syndrome
during prolonged, high-dose, experimental, enzyme replacement therapy with recombinant
human acid α-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 …