Systemic Mastocytosis Medication

Updated: May 30, 2023
  • Author: Devapiran Jaishankar, MBBS; Chief Editor: Emmanuel C Besa, MD  more...
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Medication

Medication Summary

The tyrosine kinase inhibitors (TKIs) midostaurin, imatinib, and avapritinib have each been approved by the US Food and Drug Administration (FDA) for various types of mastocytosis. [16, 13, 63] Other agents are used for symptomatic relief, including prevention and treatment of anaphylaxis.

 

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Antineoplastics, Tyrosine Kinase Inhibitor

Class Summary

The tyrosine kinase inhibitors midostaurin and imatinib have each been approved by the FDA for various types of mastocytosis. A case report of ruxolitinib, a Janus Associated Kinase (JAK) inhibitor, was shown to improve symptoms and quality of life a patient with systemic mastocytosis.

Midostaurin (Rydapt)

Midostaurin has demonstrated the ability to inhibit KIT signaling, cell proliferation, and histamine release and induce apoptosis in mast cells. It is indicated for adults with advanced mastocytosis which includes aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).

Imatinib (Gleevec)

Imatinib is a protein-tyrosine kinase inhibitor that inhibits the BCR-ABL tyrosine kinase. It is also an inhibitor of the receptor tyrosine kinases for platelet-derived growth factor (PDGF) and stem cell factor (SCF), c-kit, and inhibits PDGF-and SCF-mediated cellular events. In vitro, imatinib inhibits proliferation and induces apoptosis in GIST cells, which express an activating c-kit mutation. It is indicated for adults with aggressive systemic mastocytosis without the D816V c-Kit mutation as determined with an FDA-approved test.

Avapritinib (Ayvakit)

Avapritinib is a tyrosine kinase inhibitor that binds to and inhibits specific mutant forms of PDGFRα and c-Kit, including the PDGFRα D842V mutant and various KIT exon 17 mutants. This results in the inhibition of PDGFRa- and c-Kit–mediated signal transduction pathways and the inhibition of proliferation of tumor cells that express these PDGFRa and c-Kit mutants. It is indicated for treatment of adults with advanced systemic mastocytosis (AdvSM), which includes aggressive systemic mastocytosis, systemic mastocytosis with an associated hematologic neoplasm, and mast cell leukemia. It is also indicated for adults with indolent systemic mastocytosis (ISM). Not recommended for treatment of patients with AdvSM or ISM with platelet counts 9</sup>/L.

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Sympathomimetics

Class Summary

Sympathomimetic agents are used in the treatment of anaphylaxis.

Epinephrine (Adrenalin, Bronitin, EpiPen)

Drug of choice for treating anaphylactoid reactions. Has alpha-agonist effects that include increased peripheral vascular resistance, reversed peripheral vasodilatation, systemic hypotension, and vascular permeability. Beta-agonist effects of epinephrine include bronchodilatation, chronotropic cardiac activity, and positive inotropic effects.

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Mast Cell Stabilizers

Class Summary

Mast cell stabilizers prevent release of mediators from mast cells, which cause airway inflammation and bronchospasm.

Cromolyn sodium (Intal, NasalCrom)

Inhibits degranulation of sensitized mast cells following their exposure to specific antigens.

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Corticosteroids

Class Summary

Have anti-inflammatory properties and cause profound and varied metabolic effects. Corticosteroids modify body's immune response to diverse stimuli.

Prednisone (Deltasone, Orasone, Meticorten)

Immunosuppressant for the treatment of autoimmune disorders. May decrease inflammation by reversing increased capillary permeability and suppressing polymorphonuclear neutrophil activity. Has been used in patients with systemic mastocytosis to control malabsorption, ascites, and bone pain and to prevent anaphylaxis

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