Significant clinical response to JAK1/2 inhibition in a patient with CSF3R-T618I-positive atypical chronic myeloid leukemia

Kim Hien T. Dao, Magdolna B. Solti, Julia E. Maxson, Elliott F. Winton, Richard D. Press, Brian J. Druker, Jeffrey W. Tyner

Research output: Contribution to journalArticle

50 Scopus citations

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 languageEnglish (US)
Pages (from-to)67-69
Number of pages3
JournalLeukemia Research Reports
Volume3
Issue number2
DOIs
StatePublished - 2014

Keywords

  • Atypical chronic myeloid leukemia
  • Chronic neutrophilic leukemia
  • Colony-stimulating factor 3 receptor
  • Ruxolitinib

ASJC Scopus subject areas

  • Hematology
  • Oncology

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