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Review

New Insights into the Pathogenesis of Systemic Mastocytosis

Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, 30625 Hannover, Germany
Academic Editor: Margarita Martín
Int. J. Mol. Sci. 2021, 22(9), 4900; https://doi.org/10.3390/ijms22094900
Received: 31 March 2021 / Revised: 29 April 2021 / Accepted: 30 April 2021 / Published: 5 May 2021
(This article belongs to the Special Issue Mast Cells: From Host Defense to Pathology)
Mastocytosis is a type of myeloid neoplasm characterized by the clonal, neoplastic proliferation of morphologically and immunophenotypically abnormal mast cells that infiltrate one or more organ systems. Systemic mastocytosis (SM) is a more aggressive variant of mastocytosis with extracutaneous involvement, which might be associated with multi-organ dysfunction or failure and shortened survival. Over 80% of patients with SM carry the KIT D816V mutation. However, the KIT D816V mutation serves as a weak oncogene and appears to be a late event in the pathogenesis of mastocytosis. The management of SM is highly individualized and was largely palliative for patients without a targeted form of therapy in past decades. Targeted therapy with midostaurin, a multiple kinase inhibitor that inhibits KIT, has demonstrated efficacy in patients with advanced SM. This led to the recent approval of midostaurin by the United States Food and Drug Administration and European Medicines Agency. However, the overall survival of patients treated with midostaurin remains unsatisfactory. The identification of genetic and epigenetic alterations and understanding their interactions and the molecular mechanisms involved in mastocytosis is necessary to develop rationally targeted therapeutic strategies. This review briefly summarizes recent developments in the understanding of SM pathogenesis and potential treatment strategies for patients with SM. View Full-Text
Keywords: systemic mastocytosis; KIT D816V mutation; pathogenesis; targeted therapy; TRK systemic mastocytosis; KIT D816V mutation; pathogenesis; targeted therapy; TRK
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MDPI and ACS Style

Li, Z. New Insights into the Pathogenesis of Systemic Mastocytosis. Int. J. Mol. Sci. 2021, 22, 4900. https://doi.org/10.3390/ijms22094900

AMA Style

Li Z. New Insights into the Pathogenesis of Systemic Mastocytosis. International Journal of Molecular Sciences. 2021; 22(9):4900. https://doi.org/10.3390/ijms22094900

Chicago/Turabian Style

Li, Zhixiong. 2021. "New Insights into the Pathogenesis of Systemic Mastocytosis" International Journal of Molecular Sciences 22, no. 9: 4900. https://doi.org/10.3390/ijms22094900

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