Aplastic Anemia Medication

Updated: Mar 15, 2023
  • Author: Sameer Bakhshi, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Medication Summary

The goals of pharmacotherapy in cases of aplastic anemia are to reduce morbidity and prevent complications. Chelating agents are used to reduce iron overload in patients undergoing repeated transfusions. Immunosuppressive agents are used in conditioning regimens for hematopoietic stem cell transplantation; immunosuppression is also used for non-transplant therapy, most often with the combination of antithymocyte globulin (ATG) and cyclosporine.

Hematopoietic support with eltrombopag, granulocyte colony-stimulating factor (G-CSF) and granulocyte-macrophage colony-stimulating factor (GM-CSF) may be considered in refractory infections, although the cost and efficacy of this therapy should be weighed. [10, 76, 77, 78, 112, 113]


Immunosuppressive Agents

Class Summary

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.


Hematopoietic Growth Factors

Class Summary

Eltrombopag has gained FDA approval for severe aplastic anemia. It is indicated for first-line treatment, in combination with standard immunosuppressive therapy, and as second-line treatment in patients who fail to respond adequately to at least 1 prior immunosuppressive therapy.

Several preliminary studies have demonstrated that the addition of cytokines (eg, granulocyte colony-stimulating factor [G-CSF], granulocyte-macrophage colony-stimulating factor [GM-CSF]) may hasten the neutrophil recovery and that these agents may improve response rate and survival, although long-term use may increase the risk of clonal evolution.

Eltrombopag (Promacta)

Eltrombopag is a thrombopoietin (TPO)-receptor agonist that interacts with human TPO receptor transmembrane domain of human TPO-receptor. It initiates signaling cascades that induce proliferation and differentiation of megakaryocytes from bone marrow progenitor cells. It is indicated for severe aplastic anemia in patients who fail to respond adequately to at least 1 prior immunosuppressive therapy.

Sargramostim (Leukine)

A recombinant human GM-CSF, sargramostim can stimulate production of neutrophils and activate mature granulocytes and macrophages. The dose and frequency of administration vary with the investigational protocol.

Filgrastim (Neupogen)

Filgrastim is a G-CSF that activates and stimulates the production, maturation, migration, and cytotoxicity of neutrophils.


Antineoplastic Agent, Antimetabolite (purine)

Class Summary

Antimetabolites are antineoplastic agent that inhibit cell growth and proliferation.

Fludarabine (Fludara)

Fludarabine contains fludarabine phosphate, a fluorinated nucleotide analogue of the antiviral agent vidarabine, 9-b-D-arabinofuranosyladenine (ara-A) that enters the cell and is phosphorylated to form the active metabolite 2-fluoro-ara-adenosine triphosphate, which inhibits deoxyribonucleic acid (DNA) synthesis. Specifically, this agent inhibits DNA polymerase, DNA primase, DNA ligase, and ribonucleotide reductase, as well as ribonucleic acid (RNA) function, RNA processing, and mRNA translation. Fludarabine also activates apoptosis.



Class Summary

Chelating agents eliminate iron overload from transfusions.

Deferoxamine (Desferal)

Deferoxamine chelates iron by forming a stable complex that prevents the iron from entering into further chemical reactions; it also chelates iron readily from ferritin and hemosiderin but not readily from transferrin. Desferoxamine does not combine with the iron from cytochromes and hemoglobin. The chelate is readily soluble and is renally excreted.

Deferasirox (Exjade)

Deferasirox chelates trivalent iron. This agent is used to treat chronic iron overload due to blood transfusions. Monitor patients' renal and hepatic function.