Conference Correspondent

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.

Cladribine has long been established as an efficient therapy in systemic mastocytosis and remains a valuable treatment option even as new therapies emerge.

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.

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.

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.

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.

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.

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.

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.

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.