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. 2018 Aug 9;29(9):2265–2278. doi: 10.1681/ASN.2018030329

Table 6.

Overview of successful therapy strategies for prevention or elimination of ADAs in patients with LSDs

Disease Target Drug Components Reference
Immune tolerance induction in ERT-naïve patients
 FD Immunosuppression due to transplantations Methylprednisolone with azathioprine, calcineurin inhibitors, mycophenolate-mofetil/mycophenolate acid 115
 PD B cell depletion Rituximab, MTX, IVIG 118
 PD Immunosuppression, B cell depletion, mTOR inhibition Methylprednisolone, rituximab, rapamycin, IVIG 112
 PD B cell depletion, immunomodulation Rituximab, MTX, IVIG 117
 PD B cell depletion, immunomodulation Rituximab, MTX, IVIG 114
Acute reduction of neutralizing ADAs
 FD Short-term ADA reduction by immunosuppression due to transplantations Prednisolone, tacrolimus, mycophenolate-mofetil/mycophenolate acid 115
 GD Acute ADA reduction, immunosuppression Plasma exchange (one plasma volume), cyclophosphamide, IVIG 116
 PD B cell depletion Rituximab, MTX, IVIG 111
 PD Immunosuppression proteasome inhibition, B cell/plasma cell depletion Cyclophosphamide, rituximab, bortezomib, MTX, IVIG 113
 PD Acute ADA reduction, immunosuppression Cyclophosphamide, IVIG, plasma exchange 120
 PD B cell depletion Rituximab, MTX, IVIG 119

PD, Pompe disease; IVIG, intravenous immunoglobulin; mTOR, mechanistic target of rapamycin; GD, Gaucher disease.