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Advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, mast cell leukaemia): lines of therapy

Cancer page →

5 standard-of-care settings across 5 lines and 2 biomarker subgroups. Rows come from the cancer page's standard of care; the grid places each on its line and subgroup.

LineAll comersRisk group
Screening, prevention and diagnosis1·
Early / localised·1
Advanced, first line1·
Second line1·
Other settings1·

Screening, prevention and diagnosis

SubgroupSettingApproachProducts and trialsEvidence
All comersDiagnosis and risk assessmentBone marrow biopsy and aspirate, KIT D816V allele burden, myeloid mutation panel, tryptase, imaging for organomegaly and bone lesions, MARS or IPSM score.95

Early / localised

SubgroupSettingApproachProducts and trialsEvidence
Risk groupFit patients, especially mast cell leukaemia or high-risk SM-AHNAllogeneic haematopoietic cell transplantation after response to a KIT inhibitor.92

Advanced, first line

SubgroupSettingApproachProducts and trialsEvidence
All comersFirst lineAvapritinib 200 mg daily when platelets are 50 x 10^9/L or more (EXPLORER, PATHFINDER, approved 2021); midostaurin as the alternative (approved 2017).97

Second line

SubgroupSettingApproachProducts and trialsEvidence
All comersRelapsed or intolerantSwitch between avapritinib and midostaurin; cladribine; interferon alfa; bezuclastinib in the Apex trial.43

Other settings

SubgroupSettingApproachProducts and trialsEvidence
All comersAssociated haematological neoplasmTreat the neoplasm on its own merits (azacitidine for CMML or MDS, intensive chemotherapy for AML) alongside or after KIT inhibition.89

Lines and subgroups are parsed from the setting text of each standard-of-care row and can misclassify an unusual phrasing; the row’s own setting is always shown. Guideline chips reflect the NCCN category and ESMO-MCBS grade recorded on the cancer page, checked on its stated date. Not medical advice.