TY - JOUR
T1 - No evidence for cell activation or brain vaso-occlusion with plerixafor mobilization in sickle cell mice
AU - Choi, Erika
AU - Branch, Craig
AU - Cui, Min Hui
AU - Yazdanbakhsh, Karina
AU - Mohandas, Narla
AU - Billett, Henny H.
AU - Shi, Patricia A.
N1 - Publisher Copyright:
© 2015 Elsevier Inc.
PY - 2016/3/1
Y1 - 2016/3/1
N2 - 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.
AB - 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.
KW - Endothelial cell activation
KW - Hematopoietic progenitor cell mobilization
KW - Neutrophil activation
KW - Platelet activation
KW - Plerixafor
KW - Sickle cell disease
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U2 - 10.1016/j.bcmd.2015.12.008
DO - 10.1016/j.bcmd.2015.12.008
M3 - Article
C2 - 26852658
AN - SCOPUS:84959186003
SN - 1079-9796
VL - 57
SP - 67
EP - 70
JO - Blood Cells, Molecules, and Diseases
JF - Blood Cells, Molecules, and Diseases
ER -