Targeting CD123 in hematologic malignancies: identifying suitable patients for targeted therapy

Mrinal M. Patnaik, Tariq I. Mughal, Christopher Brooks, Ross Lindsay, Naveen Pemmaraju

Research output: Contribution to journalReview articlepeer-review


Following the observation of interleukin 3 receptor α chain (IL-3Rα; CD123) upregulation on leukemia stem cells (LSCs) almost two decades ago, targeted treatment via CD123-diptheria toxin conjugates has now been tested in patients with diverse myeloid malignancies. Targeted eradication of LSCs could result in effective treatments for many challenging diseases initiated by these cells. Consequently, considerable effort has been directed toward targeting CD123 as a potential strategy for treating patients with hematologic malignancies in which CD123 is overexpressed. However, these therapies have had limited success so far, highlighting the need for suitable criteria to identify patients who could benefit from them. Given the diversity in CD123 expression across different hematologic malignancies, understanding CD123 expression patterns and the functional pathogenetic significance is crucial. Here, we review the methodologies available for CD123 assessment and discuss the biological and clinical characteristics of patients for whom CD123-targeting therapies may have a clinical impact.

Original languageEnglish (US)
Pages (from-to)2568-2586
Number of pages19
JournalLeukemia and Lymphoma
Issue number11
StatePublished - 2021


  • CD123
  • IL-3Rα
  • hematologic malignancies
  • leukemia stem cells
  • targeted therapy

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research


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