A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency.

Abstract:

: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.

journal_name

Pediatr Transplant

authors

Dvorak CC,Horn BN,Puck JM,Czechowicz A,Shizuru JA,Ko RM,Cowan MJ

doi

10.1111/petr.12309

subject

Has Abstract

pub_date

2014-09-01 00:00:00

pages

602-8

issue

6

eissn

1397-3142

issn

1399-3046

journal_volume

18

pub_type

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