[HTML][HTML] Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease
YH Messinger, NJ Mendelsohn, W Rhead… - Genetics in …, 2012 - Elsevier
Abstract Purpose Infantile Pompe disease resulting from a deficiency of lysosomal acid α-
glucosidase (GAA) requires enzyme replacement therapy (ERT) with recombinant human
GAA (rhGAA). Cross-reactive immunologic material negative (CRIM-negative) Pompe
patients develop high-titer antibody to the rhGAA and do poorly. We describe successful
tolerance induction in CRIM-negative patients. Methods Two CRIM-negative patients with
preexisting anti-GAA antibodies were treated therapeutically with rituximab, methotrexate …
glucosidase (GAA) requires enzyme replacement therapy (ERT) with recombinant human
GAA (rhGAA). Cross-reactive immunologic material negative (CRIM-negative) Pompe
patients develop high-titer antibody to the rhGAA and do poorly. We describe successful
tolerance induction in CRIM-negative patients. Methods Two CRIM-negative patients with
preexisting anti-GAA antibodies were treated therapeutically with rituximab, methotrexate …