This protocol addresses patients with systemic mastocytosis presenting as mast cell leukemia (MCL) driven by the KIT D816V mutation, in whom first-line therapy has not achieved adequate disease control.
Mast cell leukemia with the KIT D816V mutation represents an aggressive presentation of systemic mastocytosis. The c-KIT D816V driver mutation shapes both the disease course and the selection of therapeutic approaches across treatment lines.
When prior therapy with avapritinib or midostaurin has failed to produce reversion of disease-related organ damage and a marked decrease in bone marrow mast cell burden, escalation to a next-line approach is indicated.
In this salvage setting, a nucleoside analogue with demonstrated activity across systemic mastocytosis subtypes — including mast cell leukemia — is employed. The agent is delivered intravenously over repeated treatment cycles. Full regimen details, including sequencing and clinical criteria, are available via the link below.
DOI: 10.1002/ajh.26962