No evidence for cell activation or brain vaso-occlusion with plerixafor mobilization in sickle cell mice

Erika Choi, Craig A. Branch, Min-Hui Cui, Karina Yazdanbakhsh, Narla Mohandas, Henny H. Billett, Patricia A. Shi

Research output: Contribution to journalArticle

8 Scopus citations

Abstract

Gene therapy for sickle cell disease is currently in active trials. Collecting hematopoietic progenitor cells safely and effectively is challenging, however, because granulocyte colony stimulating factor, the drug used most commonly for mobilization, can cause life-threatening vaso-occlusion in patients with sickle cell disease, and bone marrow harvest requires general anesthesia and multiple hip bone punctures. Plerixafor is an inhibitor of the CXCR4 chemokine receptor on hematopoietic progenitor cells, blocking its binding to SDF-1 (CXCL12) on bone marrow stroma. In support of a clinical trial in patients with sickle cell disease of plerixafor mobilization (NCT02193191), we administered plerixafor to sickle cell mice and found that it mobilizes hematopoietic progenitor cells without evidence of concomitant cell activation or brain vaso-occlusion.

Original languageEnglish (US)
Pages (from-to)67-70
Number of pages4
JournalBlood Cells, Molecules, and Diseases
Volume57
DOIs
StatePublished - Mar 1 2016

Keywords

  • Endothelial cell activation
  • Hematopoietic progenitor cell mobilization
  • Neutrophil activation
  • Platelet activation
  • Plerixafor
  • Sickle cell disease

ASJC Scopus subject areas

  • Molecular Biology
  • Molecular Medicine
  • Hematology
  • Cell Biology

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