Elimination of antibodies to recombinant enzyme in Pompe's disease

NJ Mendelsohn, YH Messinger… - … England Journal of …, 2009 - Mass Medical Soc
NJ Mendelsohn, YH Messinger, AS Rosenberg, PS Kishnani
New England Journal of Medicine, 2009Mass Medical Soc
To the Editor: Infantile Pompe's disease is due to a deficiency of lysosomal acid alpha
glucosidase (GAA). In patients in whom GAA is not produced, a status called cross-reacting
immunologic material (CRIM)–negative, enzyme-replacement therapy with recombinant
human GAA (rhGAA) has uniformly led to high titers of anti-rhGAA antibody, with an
ultimately fatal outcome. 1 Previous attempts at eliminating rhGAA antibodies in these
patients have failed. 1–3 We report the successful induction of immune modulation in a …
To the Editor: Infantile Pompe's disease is due to a deficiency of lysosomal acid alpha glucosidase (GAA). In patients in whom GAA is not produced, a status called cross-reacting immunologic material (CRIM)–negative, enzyme-replacement therapy with recombinant human GAA (rhGAA) has uniformly led to high titers of anti-rhGAA antibody, with an ultimately fatal outcome.1 Previous attempts at eliminating rhGAA antibodies in these patients have failed.13 We report the successful induction of immune modulation in a CRIM-negative patient with Pompe's disease who continues to be antibody-free at 24 months of age and continues to gain motor milestones.
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The New England Journal Of Medicine