This protocol addresses patients with aggressive systemic mastocytosis (ASM) who carry the KIT D816V mutation. ASM is defined by the presence of one or more C-findings reflecting organ dysfunction driven by mast cell infiltration, as characterised in the 2022 WHO classification.
Patients in this setting frequently require mast cell cytoreductive therapy aimed at reversing disease-related organ dysfunction.
The structured regimen for this scenario involves interferon-based cytoreductive therapy, which may be combined with a corticosteroid in the initial phase to support tolerability and response.
The complete sequencing, dosing schedule, escalation steps, and management of response are detailed in the full protocol.
DOI: 10.1002/ajh.26962
Patients with advanced SM frequently need MC cytoreductive therapy to reverse disease-related organ dysfunction.
ASM is characterized by one or more C-findings (Table 5); of note, the definition of C-findings has undergone revision in the 2022 WHO classification.
Interferon-α Starting dose: 1–3 MU SQ three times per week Target dose: 3–5 MU SQ 3–5 times per week
Prednisone (30–60 mg/day) can be added at the start of treatment to improve tolerability and response, and is tapered over a 2–3 month period.
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