Treatment of Systemic Mastocytosis with an Associated Myeloid Neoplasm and KIT D816V Mutation

This protocol addresses patients with systemic mastocytosis who also carry an associated myeloid neoplasm (AMN) — such as acute leukemia — and harbour the KIT D816V mutation. The co-occurrence of SM and a myeloid neoplasm defines a distinct, high-risk clinical entity.

Treatment of SM-AMN primarily targets the AMN component, particularly when an aggressive disease such as acute leukemia is present. The nature and severity of the myeloid neoplasm drives the treatment strategy.

A transplant-based approach is considered in eligible patients when the associated myeloid neoplasm warrants it. Further eligibility criteria, sequencing, and the complete evidence-based regimen are detailed in the full protocol…