biostudies-literatureUnknown6Masat EImmunogenicity of recombinant human acid-alpha glucosidase (rhGAA) in enzyme replacement therapy (ERT) is a safety and efficacy concern in the management of late-onset Pompe disease (LOPD). However, long-term effects of ERT on humoral and cellular responses to rhGAA are still poorly understood. To better understand the impact of immunogenicity of rhGAA on the efficacy of ERT, clinical data and blood samples from LOPD patients undergoing ERT for >4 years (n = 28) or untreated (n = 10) were collected and analyzed. In treated LOPD patients, anti-rhGAA antibodies peaked within the first 1000 days of ERT, while long-term exposure to rhGAA resulted in clearance of antibodies with residual production of non-neutralizing IgG. Analysis of  T cell responses to rhGAA showed detectable T cell reactiviScientific reports36182https://www.ebi.ac.uk/biostudies/studies/S-EPMC5096052biostudies-literatureLong-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients.PMC5096052Kuranda KFeasson LLabauge PLeonard-Louis SDurieu IFurby ACintas PLaforet PPrigent HBassez GTiffreau VJournel HDesnuelle CFroissart RTaouagh NPuzzo FHogrel JYMasat EPereon YBoyer FZagnoli FCorre GDecostre VRenard DSole GGermain DEymard BCaillaud CNadaj-Pakleza APouget JBenistan KOrlikowski DPetiot PCanal ANathier CRonzitti GBenveniste OPellegrini NGuffon-Fouilhoux NSacconi SColella PMallone RLevy ADe Antonio MAmelin DMinot-Myhie MCMingozzi FCastaing YPerniconi BMagot AHamroun DFrench Pompe Registry Study GroupChapon FFerrer XPiraud MLacour ALofaso FMauhin WDutry AEchaniz-Laguna AStojkovic TBehin APraline JBouhour FMariampillai KBedat-Millet ALSalort-Campana EVincent DfalseLong-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients.Immunogenicity of recombinant human acid-alpha glucosidase (rhGAA) in enzyme replacement therapy (ERT) is a safety and efficacy concern in the management of late-onset Pompe disease (LOPD). However, long-term effects of ERT on humoral and cellular responses to rhGAA are still poorly understood. To better understand the impact of immunogenicity of rhGAA on the efficacy of ERT, clinical data and blood samples from LOPD patients undergoing ERT for >4 years (n = 28) or untreated (n = 10) were collected and analyzed. In treated LOPD patients, anti-rhGAA antibodies peaked within the first 1000 days of ERT, while long-term exposure to rhGAA resulted in clearance of antibodies with residual production of non-neutralizing IgG. Analysis of  T cell responses to rhGAA showed detectable T cell reactivi2016-01-01T00:00:00Z2016 Nov2025-04-04T11:18:11.823Z2019-03-27T02:28:16ZS-EPMC50960522781202510.1038/srep36182