Systemic Mastocytosis Medication

Updated: Jun 21, 2021
  • Author: Devapiran Jaishankar, MBBS; Chief Editor: Emmanuel C Besa, MD  more...
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Medication

Medication Summary

The tyrosine kinase inhibitors (TKIs) midostaurin and imatinib have each been approved by the US Food and Drug Administration (FDA) for various types of mastocytosis. [12, 13, 50] In a case report, the Janus-associated kinase (JAK) inhibitor ruxolitinib was shown to improve symptoms and quality of life in a patient with systemic mastocytosis. [51] A phase I study with the TKI avapritinib has shown favorable reponses, with improvement in symptom burden and quality of life scores. [52]

 

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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 in tumor cells that express these PDGFRa and c-Kit mutants. It is indicated for the treatment of adults with AdvSM. AdvSM includes patients with ASM, SM-AHN, and MCL. Not recommended for treatment of patients with AdvSM 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.

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