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

被引：14

作者：

Dvorak, Christopher C. ^{[1
]}

Horn, Biljana N. ^{[1
]}

Puck, Jennifer M. ^{[1
]}

Czechowicz, Agnieszka ^{[2
]}

Shizuru, Judy A. ^{[2
]}

Ko, Rose M. ^{[2
]}

Cowan, Morton J. ^{[1
]}

机构：

[1] Univ Calif San Francisco, Div Pediat Allergy Immunol & Blood & Marrow Trans, Benioff Childrens Hosp, San Francisco, CA 94143 USA

[2] Stanford Univ, Div Blood & Marrow Transplantat, Sch Med, Stanford, CA 94305 USA

来源：

PEDIATRIC TRANSPLANTATION | 2014年 / 18卷 / 06期

关键词：

severe combined immunodeficiency; plerixafor; haploidentical; engraftment; BONE-MARROW TRANSPLANTATION; REFERENCE VALUES; CD34(+) CELLS; DISEASE; CHILDREN; MOBILIZATION; ENGRAFTMENT; CHILDHOOD; DIAGNOSIS; RECOVERY;

D O I：

10.1111/petr.12309

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

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.

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收藏

页码：602 / 608

页数：7

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