Systemic mastocytosis (SM) is a myeloproliferative disorder characterized by symptoms of mast cell (MC) activation and/or organ dysfuction related to MC tissue accumulation. Treatment of this condition is evolving as our understanding of the pathophysiology of the disease advances. This paper aims to highlight novel and experimental therapies for SM.
PubMed literature search and ClinicalTrials.gov.
Peer-reviewed studies involving therapies for SM was included. There was a particular focus on preclinical and clinical trial studies.
SM presents with a wide range of symptoms including symptoms of mast cell activation such as anaphylaxis, urticaria, diarrhea as well as organ failure secondary to aggressive tissue infiltration. Treatment of the disease is dependent on the variant; patients with aggressive disease warrant advanced therapies and a higher tolerance of adverse effects. As our understanding of the disease has advanced, several novel therapeutic options have emerged. These include tyrosine kinase inhibitors directed at the KIT protein and targeted monoclonal antibodies which decrease MC activation or reduce mast cell burden. There are a variety of new medications under development which will revolutionize treatment for patients with SM.
Current treatment options for SM have inherent limitations and, in many cases, unacceptable side effects. As our molecular understanding of the disease advances, novel and experimental therapies are changing treatment paradigms of the disease.

Copyright © 2021. Published by Elsevier Inc.