Mastocytosis: Update on pharmacotherapy and future directions

Juan Carlos Cardet, Cem Akin, Min Jung Lee

Research output: Contribution to journalReview articlepeer-review

38 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)2033-2045
Number of pages13
JournalExpert Opinion on Pharmacotherapy
Issue number15
StatePublished - Oct 2013


  • Antihistamines
  • Mastocytosis
  • Tyrosine kinase inhibitors

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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