Systemic Mastocytosis with Associated Myeloid Neoplasm and KIT D816V Mutation When Avapritinib Has Not Achieved Goals

This protocol covers the next clinical step for patients with systemic mastocytosis with an associated myeloid neoplasm (SM-AMN) carrying a KIT D816V mutation, after first-line therapy has failed to reach its intended endpoints.

Clinical Scenario

Systemic mastocytosis co-occurring with an associated myeloid neoplasm (SM-AMN) and a confirmed KIT D816V mutation. Treatment of SM-AMN primarily targets the AMN component, particularly when an aggressive disease such as acute leukemia is present.

Previous Treatment & Why Escalation Is Needed

The prior line consisted of Avapritinib, the FDA-approved first-line treatment for advanced SM including SM-AMN. This protocol applies when Avapritinib has not achieved reversion of disease-related organ damage or a marked decrease in bone marrow mast cell burden — the two primary endpoints expected of that line.

Treatment Direction (Partial)

The next step centres on cytoreductive therapy directed at the AMN component. This approach uses established cytoreductive agents active in this setting.

Specific agents, sequencing, eligibility criteria, and the full structured regimen are available via the protocol below.

Instant Access to Structured Evidence-Based Regimens

References

DOI: 10.1002/ajh.26962

Treatment of SM-AMN primarily targets the AMN component, particularly if an aggressive disease such as acute leukemia is present.

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