Mastocytosis: update on pharmacotherapy and future directions

Expert Opin Pharmacother. 2013 Oct;14(15):2033-45. doi: 10.1517/14656566.2013.824424.


Introduction: Mastocytosis is a disorder characterized by abnormal mast cell (MC) accumulation in skin and internal organs such as bone marrow. The disease follows a benign course in most patients with cutaneous and indolent systemic mastocytosis (SM); however, advanced variants associated with decreased life expectancy also exist. Pharmacotherapy of mastocytosis is aimed at the control of symptoms caused by MC mediator release, treatment of comorbidities and cytoreductive therapies in advanced variants.

Areas covered: This article will cover the general treatment principles of anti-MC mediator and cytoreductive therapies of mastocytosis. The literature discussed was retrieved with PubMed using the search terms 'treatment of mastocytosis,' 'mastocytosis antimediator therapy' and looking for important cross-references.

Expert opinion: Pharmacotherapy of mastocytosis should be individualized for each patient considering the category of disease, reduction of risk of anaphylaxis, constitutional symptoms and comorbidities including osteoporosis. Cytoreductive therapies are generally reserved for patients with aggressive mastocytosis (ASM), MC leukemia (MCL) and MC sarcoma (MCS); however, some patients with indolent disease and recurrent anaphylactic episodes not responsive to antimediator therapies may also be considered for cytoreduction on a case-by-case basis.

Publication types

  • Review

MeSH terms

  • Humans
  • Immunologic Factors / therapeutic use
  • Mastocytosis / drug therapy*
  • Protein Kinase Inhibitors / therapeutic use


  • Immunologic Factors
  • Protein Kinase Inhibitors