Cladribine (2-CdA)

Purine nucleoside analog — cytoreductive chemotherapy

Response rate
50-60% in advanced SM
Onset
Weeks–months
Route
IV 5mg/m2 daily x5 days, every 4-8 weeks
Line
2nd
IgM effect
N/A

Evidence summary

Cytoreductive purine analog with significant activity against monocytic lineage cells that share a common progenitor with mast cells. Used in advanced SM when KIT inhibitors are insufficient. Major limitations include myelosuppression and immunosuppression. May be combined with other agents.

Sources (2)

DetailsCastells M et al. (2022) Treatment of Indolent and Advanced Systemic Mastocytosis · J Allergy Clin Immunol PractDOI
DetailsPardanani A (2021) Systemic mastocytosis in adults: 2021 Update on diagnosis, risk stratification and management · Am J HematolDOI