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Table 5 Drugs commonly used for immune toleration

From: Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel

Drug Mechanism Adverse events
Bortezomib Proteasome inhibition: blocks protein recycle and production of antibodies in plasma cellsa [55, 56] Peripheral neuropathy, anemia, neutropenia, thrombocytopenia, gastrointestinal and cardiac side effects [23]
IVIG Binding to the neonatal Fc receptor (FcRn) which is responsible for recycling of antibodies thus downregulating antibody responses [53, 57, 58]
Passive immunity during the period of immune suppression due to other drugs (specially rituximab) [23]
Infusion-associated reactions [59]
Methotrexate Inhibits folic acid metabolism (which blocks de novo DNA synthesis), thus eliminating dividing B and T cells.
Low dose: Induces regulatory B cells rather than cell depletion [60, 61]
Bone marrow and gastrointestinal toxicities, rarely acute pneumonitis, pulmonary fibrosis and renal function impairment [12, 62]
Mycophenolate mofetil Inhibition of proliferative responses of T and B lymphocytes [12] Leukopenia, anemia and thrombocytopenia
Rapamycin Mammalian target of rapamycin (mTOR), which inhibits cell survival and proliferation of B and T lymphocytes, but selectively promotes regulatory T – Treg – cells b [63,64,65,66,67]  
Rituximab Monoclonal antibody against CD20 molecule expressed on B cells [12] Infusion-associated reactions, lymphocytopenia, progressive multifocal leukoencephalopathy [62]
  1. a In muscle, the ubiquitin-proteasome system is believed to degrade contractile skeletal muscle proteins and may play a critical role in muscle wasting [23]
  2. b May have an impact on glycogen storage in muscle by influencing the mTor pathway and inhibiting glycogen storage [68]