Which medications in the drug class Immunosuppressive Agents are used in the treatment of Aplastic Anemia?

Updated: Jan 29, 2021
  • Author: Sameer Bakhshi, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Immunosuppressive Agents

Immunosuppressive agents are used in the conditioning regimen for patients undergoing hematopoietic stem cell transplantation for aplastic anemia, and as therapy in patients with severe disease who are not eligible for transplantation or lack a suitable donor, as well as in patients with nonsevere disease who are transfusion dependent.

Cyclosporine (Sandimmune, Neoral)

Cyclosporine is a cyclic polypeptide that suppresses some humoral immunity and, to a greater extent, cell-mediated immune reactions (eg, delayed hypersensitivity, allograft rejection, experimental allergic encephalomyelitis, graft versus host disease) for a variety of organs.

For children and adults, base the initial dosing on the ideal body weight and subsequently adjust for levels. Frequent monitoring of drug levels is needed. To convert to the oral dose, use an intravenous (IV)-to-oral correction factor of 1:4. The dosage and duration of therapy may vary with different protocols. When used without hematopoietic growth factor in children, ATG and cyclosporine-based immunosuppressive therapy has been shown to lead to an excellent response and survival rate with low incidence of clonal evolution.

Methylprednisolone (Medrol, Solu-Medrol)

Steroids ameliorate the delayed effects of anaphylactoid reactions and may limit biphasic anaphylaxis. In severe serum sickness (mediated by immune complexes), parenteral steroids may reduce the inflammatory effects. Hence, methylprednisolone is used with antithymocyte globulin (ATG) to decrease the adverse effects (eg, allergic reactions, serum sickness). Also, this agent has an additional immunosuppressive effect. High doses or long duration may be needed if serum sickness occurs with ATG. The doses and duration may vary with different protocols.

Alemtuzumab (Campath, MabCampath)

Alemtuzumab is a recombinant monoclonal antibody against CD52 (lymphocyte antigen). This agent promotes antibody-dependent lysis.

Lymphocyte immune globulin, equine (Atgam)

Lymphocyte immune globulin inhibits the cell-mediated immune response by altering T-cell function or by eliminating antigen-reactive cells. There are few prospective, randomized data to suggest a single schedule that is superior, but experience suggests that a 4- to 5-day infusion is associated with less toxicity than older 7- to 10-day schedules.

Cyclophosphamide (Cytoxan)

Cyclophosphamide is chemically related to nitrogen mustards. As an alkylating agent, the mechanism of action of the active metabolites may involve cross-linking of DNA, which may interfere with the growth of normal and neoplastic cells. Monitor carefully; used only on an investigational basis.

Antithymocyte globulin, rabbit (Thymoglobulin)

Antithymocyte globulin (ATG) may modify T-cell function. The dose and duration of therapy vary with the investigational protocols.

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