Abstract |
We describe our single institution experience with cladribine therapy in 42 patients with systemic mastocytosis (SM): 22 advanced (adv-SM; median age 65 years, 68% males) and 20 indolent/smouldering SM (ISM/SSM; median age 56 years, 45% males); subcategories included eight aggressive, 13 associated with another haematological neoplasm, one mast cell leukaemia, 17 ISM and three SSM. Overall/major response rates were 77%/45% for adv-SM and 70%/60% for ISM/SSM, and median (range) duration of response 10 (4-75) and 46 (4-140) months respectively. A >50% reduction in bone marrow mast cell burden and serum tryptase level was documented in 63% and 67% of patients with adv-SM and 50% and 46% with ISM/SSM respectively. The presence of KIT proto-oncogene, receptor tyrosine kinase (KIT)D816V predicted response in adv-SM: 17 (90%) of 19 with and none of three without the mutation responded (P < 0·01). Treatment-emergent adverse events were mostly limited to transient cytopenias: Grade 3/4 neutropenia, thrombocytopenia, or lymphopenia occurred in 27%, 27% and 27% of patients with adv-SM, and 5%, 5% and 30% with ISM/SSM respectively. The present study provides practical information that might be considered when making treatment choices between cladribine and newer KIT-targeted therapies and identifies the absence of KITD816V as a potential marker of cladribine resistance in advanced SM; the latter observation needs confirmation in a larger study.
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Authors | Ayalew Tefferi, Jaya Kittur, Faiqa Farrukh, Kebede H Begna, Mrinal M Patnaik, Aref Al-Kali, Michelle A Elliott, Kaaren K Reichard, Naseema Gangat, Animesh Pardanani |
Journal | British journal of haematology
(Br J Haematol)
Vol. 196
Issue 4
Pg. 975-983
(02 2022)
ISSN: 1365-2141 [Electronic] England |
PMID | 34729775
(Publication Type: Journal Article)
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Copyright | © 2021 British Society for Haematology and John Wiley & Sons Ltd. |
Chemical References |
- Antineoplastic Agents
- Cladribine
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Topics |
- Adolescent
- Adult
- Aged
- Aged, 80 and over
- Antineoplastic Agents
(pharmacology, therapeutic use)
- Child
- Cladribine
(pharmacology, therapeutic use)
- Female
- Humans
- Male
- Mastocytosis, Systemic
(drug therapy)
- Middle Aged
- United States
- Young Adult
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