Drug1st line

Imatinib (Gleevec)

Tyrosine kinase inhibitor (KIT, BCR-ABL, PDGFR)

Response rate
Effective in non-D816V cases; ineffective in D816V+ SM
Onset
Weeks–months
Route
Oral 400mg daily
Line
1st
IgM effect
N/A
Evidence level
amber

Evidence summary

FDA-approved (2006) for ASM without D816V or unknown KIT status. Inhibits wild-type KIT and select non-codon 816 mutants (e.g., K509I, F522C). D816V causes conformational resistance. Effective in well-differentiated SM (WDSM), which typically lacks D816V. Long-term remissions reported in non-D816V patients.

Approved indications

Conditions for which Imatinib (Gleevec) has regulatory approval (not specific to rare diseases covered here):

ASM without D816V KIT mutationCMLGISTOther KIT-driven malignancies

Drug identifiers

DrugBankDB00619 ↗
ATC CodeL01EA01