Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: need for …
SG Banugaria, TT Patel, J Mackey, S Das… - Molecular genetics and …, 2012 - Elsevier
With the advent of enzyme replacement therapy (ERT) with alglucosidase alfa (rhGAA,
Myozyme®) for Pompe disease, the clinical course of the disease has changed. We have
previously described the poor outcome in cross reactive immunologic material (CRIM)-
negative and high-titer CRIM-positive (HTCP) patients secondary to high sustained antibody
titers (HSAT) which effectively neutralize ERT efficacy. Various immunomodulation
strategies are being explored to diminish the immune response to ERT. However, once …
Myozyme®) for Pompe disease, the clinical course of the disease has changed. We have
previously described the poor outcome in cross reactive immunologic material (CRIM)-
negative and high-titer CRIM-positive (HTCP) patients secondary to high sustained antibody
titers (HSAT) which effectively neutralize ERT efficacy. Various immunomodulation
strategies are being explored to diminish the immune response to ERT. However, once …