Abstract
Mutations in CSF3R (colony-stimulating factor 3 receptor) are frequent oncogenic drivers in chronic neutrophilic leukemia (CNL) and atypical chronic myeloid leukemia (aCML). Here we describe a 75 year old man who was diagnosed with CSF3R-T618I-positive atypical CML. He presented with leukocytosis, anemia, and thrombocytopenia and developed massive splenomegaly and severe constitutional symptoms. Hydroxyurea was given over a 6 month period but failed to provide any measureable clinical benefit. Eventually, he was treated with ruxolitinib, an FDA-approved JAK1/2 inhibitor, which resulted in dramatic improvement of his blood counts. He also had significant reduction of spleen volume and constitutional symptoms. This case highlights the need for a clinical trial to interrogate JAK1/2 as a potential molecular target in CNL and aCML in patients with or without CSF3R mutation. A clinical trial evaluating the safety and efficacy of ruxolitinib for this patient population is registered at ClinicalTrials.gov (NCT02092324).
Original language | English (US) |
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Pages (from-to) | 67-69 |
Number of pages | 3 |
Journal | Leukemia Research Reports |
Volume | 3 |
Issue number | 2 |
DOIs | |
State | Published - 2014 |
Keywords
- Atypical chronic myeloid leukemia
- Chronic neutrophilic leukemia
- Colony-stimulating factor 3 receptor
- Ruxolitinib
ASJC Scopus subject areas
- Hematology
- Oncology