The Lynx Group

ASH 2021 Systemic Mastocytosis Wrap-Up

Criteria to assess presenting patterns of organ damage have not been extensively evaluated. Preliminary evidence suggests identifying these patterns could be useful in profiling organ dysfunction in avapritinib clinical trials.
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Cladribine has long been established as an efficient therapy in systemic mastocytosis and remains a valuable treatment option even as new therapies emerge.
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Hereditary alpha-tryptasemia may promote development of systemic mastocytosis. Screening for variations in copy number of the alpha tryptase gene may provide early evidence for diagnosis of mastocytosis.
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CAR T-cell immunotherapy is highly efficient because of its ability to target specific tumor antigens. Preliminary evidence suggests that CAR T-cells directed against CD117, the KIT receptor, may emerge as a therapeutic option for advanced systemic mastocytosis.
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Cytogenetic aberrations are rarely found by cytogenetics in systemic mastocytosis but are associated with advanced disease. Whole genome sequencing detects both chromosomal aberrations and non-KIT gene mutations and can be used as an alternative to cytogenetics for assessing disease risk.
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Avapritinib was recently approved for treatment in advanced systemic mastocytosis after demonstrating in clinicals trials the ability to improve hematologic and bone marrow responses in patients.
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KIT D816V mutations are highly associated with most cases of systemic mastocytosis. Avapritinib demonstrates efficacy in controlling disease progression in patients with KIT D816V mutations.
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Advanced systemic mastocytosis often develops in patients aged >60 years but some younger patients qualify for Medicare due to preexisting disability. All patients with advanced systemic mastocytosis require more healthcare resources and have greater medical costs.
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Clinical trials in systemic mastocytosis have led to the development of new prognostic scoring systems. Although effective in a controlled study, their usefulness in identifying high-risk patients in a real-world clinical setting requires further evaluation.
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Bezuclastinib treatment has shown clinical activity in advanced solid tumors with little toxicity. A phase 2 investigation seeks to determine its potential as a safe and effective option for KIT D816V–driven advanced systemic mastocytosis.
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