Pharmacotherapy in Mast Cell Leukemia.

Research output: Contribution to journalReview articlepeer-review


INTRODUCTION: Mast cell leukemia (MCL) is one of the most aggressive forms of Systemic Mastocytosis (SM), a complex family of rare diseases, for which standard therapies are very few. MCL represents only <1% cases of SM and this is the reason why there are no specific clinical trials to better explore this disease. As a consequence, MCL is treated and grouped within other forms of SM, being all KIT-driven diseases; however, its KIT dysregulation leads to uncontrolled activation of mast cells (MCs), which correlates with forms of myeloid acute leukemia (AML).

AREAS COVERED: Different therapeutic approaches can be followed in the treatment of MCL. The authors look at both symptomatic therapies along with other approaches including targeted therapy. Further, the authors provide their expert opinion.

EXPERT OPINION: In the scenario of mast cell leukemia treatment, the key approach to achieve clinical results is, more than other similar pathologies, personalizing the therapy. It could be interesting or desirable to introduce for instance KIT mutant forms as minor criteria for the diagnosis of advanced SM, considering the small patient population with MCL and the relatively large panel of activating mutations for KIT and other important proteins involved in MCs' regulation.

Original languageEnglish
Pages (from-to)1059-1069
Number of pages11
JournalExpert Opinion on Pharmacotherapy
Issue number9
Publication statusPublished - Jun 2020


  • Humans
  • Leukemia, Mast-Cell/drug therapy


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