Table 6.
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.