The Role of Avapritinib for the Treatment of Systemic Mastocytosis

Systemic mastocytosis is a rare hematologic disorder characterized by the clonal proliferation of mast cells in extra-cutaneous organs. This disease can be further subdivided into five different phenotypes: indolent systemic mastocytosis (ISM), smoldering systemic mastocytosis (SSM), aggressive syst...

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Bibliographic Details
Published in:Curēus (Palo Alto, CA) CA), 2021-09, Vol.13 (9), p.e18385
Main Authors: Sumbly, Vikram, Landry, Ian, Iqbal, Saba, Bhatti, Zamaraq, Alshamam, Mohsen S, Ashfaq, Salman, Rizzo, Vincent
Format: Article
Language:English
Subjects:
Biopsy
Bone marrow
Cell cycle
Fractures
Gastrointestinal cancer
Genes
Hematology
Keywords
Kinases
Leukemia
Mutation
Oncology
Stem cells
Therapeutics
Tumors
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Summary:Systemic mastocytosis is a rare hematologic disorder characterized by the clonal proliferation of mast cells in extra-cutaneous organs. This disease can be further subdivided into five different phenotypes: indolent systemic mastocytosis (ISM), smoldering systemic mastocytosis (SSM), aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). The tyrosine kinase inhibitor (and also potent KIT D816V inhibitor) avapritinib, initially approved for the treatment of gastrointestinal stromal tumors (GISTs) bearing a PDGFRA exon 18 mutation, also showed great promise in patients with systemic mastocytosis, a disease known to be driven by a mutation in KIT (D816V). We present an overview of this rare disorder, including a review of the current understanding of the genetic mechanisms which lead to the disease state, the action of the tyrosine kinase inhibitors, as well as the latest clinical trial data which led to the current recommendations for the use of avapritinib.
ISSN:2168-8184
2168-8184
DOI:10.7759/cureus.18385