Premium
A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency
Author(s) -
Dvorak Christopher C.,
Horn Bilja.,
Puck Jennifer M.,
Czechowicz Agnieszka,
Shizuru Judy A.,
Ko Rose M.,
Cowan Morton J.
Publication year - 2014
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.12309
Subject(s) - plerixafor , medicine , transplantation , hematopoietic stem cell transplantation , regimen , chemotherapy , stem cell , oncology , immunology , surgery , immune system , cxcr4 , chemokine , biology , genetics
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 CD 34‐selected haploidentical cells and one who received T‐replete matched unrelated BM . All patients receiving G‐CSF and plerixafor had generally poor CD 34 + cell and Lin ‐ CD 34 + CD 38 − CD 90 + CD 45 RA − 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.