A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency PEDIATRIC TRANSPLANTATION Dvorak, C. C., Horn, B. N., Puck, J. M., Czechowicz, A., Shizuru, J. A., Ko, R. M., Cowan, M. J. 2014; 18 (6): 602-608


For infants with SCID, the ideal conditioning regimen before allogeneic HCT would omit cytotoxic chemotherapy to minimize short- and long-term complications. We performed a prospective pilot trial with G-CSF plus plerixafor given to the host to mobilize HSC from their niches. We enrolled six patients who received CD34-selected haploidentical cells and one who received T-replete matched unrelated BM. All patients receiving G-CSF and plerixafor had generally poor CD34(+) cell and Lin(-) CD34(+) CD38(-) CD90(+) CD45RA(-) HSC mobilization, and developed donor T cells, but no donor myeloid or B-cell engraftment. Although well tolerated, G-CSF plus plerixafor alone failed to overcome physical barriers to donor engraftment.

View details for DOI 10.1111/petr.12309

View details for Web of Science ID 000340530800017

View details for PubMedCentralID PMC5413354