Immune Thrombocytopenia (ITP) Medication

Updated: Jul 20, 2023
  • Author: Craig M Kessler, MD, MACP; Chief Editor: Srikanth Nagalla, MD, MS, FACP  more...
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Medication

Medication Summary

The treatment of immune thrombocytopenia (ITP) requires considerable individualization. [100] Medications used for ITP include the following:

  • Corticosteroids
  • Immunoglobulins
  • Thrombopoieting receptor agonists (TPO-RAs)
  • Spleen tyrosine kinase (SYK) inhibitor
  • Rituximab
  • Immunosuppressive agents
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Corticosteroids

Prednisone (Deltasone, Orasone, Sterapred)

Oral corticosteroid that is used most frequently because of its relatively low cost, known adverse effects, and long-term clinical record. DOC for initial treatment of ITP in children and adults. For aggressive treatment, may be combined with IV RhIG or IVIG. In emergency, replace PO prednisone with IV methylprednisolone.

Methylprednisolone (Solu-Medrol)

DOC for the initial management of severe bleeding tendency in ITP. IV is recommended when the most rapid and reliable treatment of ITP is required. In this situation, combine with IV RhIG in qualified Rh(D)-positive patients or IVIG in Rh(D)-negative patients or unqualified Rh(D)-positive patients.

Dexamethasone (Baycadron, Decadron DSC, Dexamethasone Intensol)

Alternative corticosteroid for the initial management of severe bleeding tendency in ITP. IV is recommended when the most rapid and reliable treatment of ITP is required.

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Blood Products

IV RhIG (WinRho SDF)

Specialized immunoglobulin product manufactured from pools of plasma from Rh(D)-negative persons and alloimmunized to D blood group antigen. Subjected to anion-exchange column chromatography to permit IV infusion and solvent-detergent treatment and nanofiltration to reduce infectivity by lipid-enveloped viruses. Induces immune RBC hemolysis in Rh(D)-positive recipients, decreasing function of mononuclear macrophages (reticuloendothelial blockade) and sparing immunoglobulin-coated platelets from splenic destruction.

IVIG (Gamimune, Gammagard, Sandoglobulin)

Large dose of 1 g/kg induces decreased function of mononuclear macrophages (reticuloendothelial blockade), sparing immunoglobulin-coated platelets from splenic destruction. Used with IV methylprednisolone to manage acute ITP in children. Decreased time to an increased platelet count compared with IV RhIG, but the difference does not appear to be clinically significant. Compared with IV RhIG, associated with more adverse effects, longer infusions, and increased cost, causing many hematologists to prefer IV RhIG as a supplement to corticosteroids, at least for Rh(D)-positive patients.

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Immunosuppressive Antimetabolites

Azathioprine (Imuran)

May be effective in some patients with ITP whose conditions do not or no longer have response to corticosteroids, IV RhIG, or IVIG. May be used with prednisone to reduce dose of prednisone or as another PO medication to delay splenectomy.

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Synthetic Antineoplastic Drugs

Cyclophosphamide (Cytoxan)

May be useful in some patients whose conditions do not or no longer have a response to corticosteroids, IV RhIG, IVIG, or splenectomy. Induces less of a decrease in platelet count than other immunosuppressive alkylating agents.

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Androgens

Danazol (Danocrine)

May impair the clearance of immunoglobulin-coated platelets and decreases autoantibody production. Increased platelet counts in 40-50% of patients, particularly postmenopausal women.

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Monoclonal Antibodies

Rituximab (Rituxan)

Chimeric monoclonal antibody directed against the CD20 antigen on the surface of normal and malignant B lymphocytes. Antibody is IgG kappa immunoglobulin with murine light- and heavy-chain variable sequences and human constant region sequences.

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Thrombopoietic Agents

Avatrombopag (Doptelet)

Second-generation orally administered thrombopoietin receptor agonist (TPO-RA). Stimulates proliferation and differentiation of megakaryocytes from bone marrow progenitor cells, resulting in an increased production of platelets. It is indicated for thrombocytopenia in adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Romiplostim (Nplate)

An Fc-peptide fusion protein (peptibody) that increases platelet production through binding and activation of the thrombopoietin (TPO) receptor, a mechanism similar to endogenous TPO. Indicated for chronic immune thrombocytopenia (ITP) in adults who are newly diagnosed or those who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

It is also indicated in children aged ≥1 year with ITP for ≥6 months who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

Eltrombopag (Promacta)

Oral thrombopoietin (TPO) receptor agonist. Interacts with transmembrane domain of human TPO receptor and induces megakaryocyte proliferation and differentiation from bone marrow progenitor cells. Indicated for thrombocytopenia associated with chronic idiopathic thrombocytopenic purpura in patients experiencing inadequate response to corticosteroids, immunoglobulins, or splenectomy. Not for use to normalize platelet counts, but used when clinical condition increases bleeding risk.

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SYK Inhibitors

Fostamatinib (Tavalisse)

Fostamatinib is a spleen tyrosine kinase (SYK) inhibitor. The major active metabolite of fostamatinib (ie, R406) inhibits signal transduction of Fc-activating receptors and B-cell receptor, thereby reducing antibody-mediated destruction of platelets. It is indicated for thrombocytopenia in patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

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