Clinical Scenario

This protocol applies to patients with aggressive systemic mastocytosis (ASM) presenting with one or more C-findings per WHO criteria, or eligible organ damage findings per IWG-MRT-ECNM criteria, who do not meet the diagnostic threshold for mast cell leukemia (MCL).

Defining Conditions

The ASM designation requires the presence of at least one C-finding reflecting significant mast cell–mediated organ damage. The diagnosis does not meet the criteria for mast cell leukemia. Accurately characterising this level of organ involvement is essential to appropriate treatment selection.

Treatment Approach — Partial Overview

Management is built around cytoreductive therapy, with agent selection guided by clinical factors — including mutation status — as well as eligibility for clinical trial enrolment. For selected patients, allogeneic hematopoietic cell transplantation represents an additional option.

The complete evidence-based regimen — including full agent selection criteria, sequencing, and monitoring guidance — is available in the structured protocol below.

References

DOI: 10.6004/jnccn.2018.0088

The diagnosis of ASM requires the presence of one or more C-findings, but does not meet the criteria for MCL.
Midostaurin, an oral multikinase inhibitor, has shown activity for the treatment of advanced SM (ASM, SM-AHN, and MCL).
Imatinib is approved by the FDA for the treatment of adult patients with ASM without the KIT D816V mutation (including wild-type) or with unknown mutational status.
Allogeneic HCT can be considered as an initial treatment option for patients with ASM and acute MCL.
Clinical improvement is defined as the resolution of ≥1 findings of nonhematologic or hematologic organ damage without concomitant worsening of other eligible organ damage.
Responses (resolution of findings of organ damage as well as reduction in bone marrow mast cell burden and serum tryptase level) should be maintained or confirmed for a period of at least 12 weeks to fulfill the criteria for clinical improvement, CR, and PR.

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What Is the Treatment for Aggressive Systemic Mastocytosis (ASM) with C-Findings?

References