Treatment of systemic mastocytosis: Novel and emerging therapies.

OBJECTIVE

Systemic mastocytosis (SM) is a myeloproliferative disorder characterized by symptoms of mast cell (MC) activation and/or organ dysfunction related to MC tissue accumulation. Treatment of this condition is evolving as our understanding of the pathophysiology of the disease advances. This article aims to highlight novel and experimental therapies for SM.

DATA SOURCES: PubMed literature search and ClinicalTrials.gov.

STUDY SELECTIONS: Peer-reviewed studies involving therapies for SM were included. There was a particular focus on preclinical and clinical trial studies.

RESULTS

SM presents with a wide range of symptoms including symptoms of MC activation such as anaphylaxis, urticaria, diarrhea, and organ failure secondary to aggressive tissue infiltration. The treatment of the disease is dependent on the variant; patients with aggressive disease warrant advanced therapies and 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 that will revolutionize the treatment for patients with SM.

CONCLUSION

Current treatment options for SM have inherent limitations and, in many cases, unacceptable adverse effects. As our molecular understanding of the disease advances, novel, and experimental therapies are changing treatment paradigms of the disease.