title: 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.
authors: Division of Pediatric Allergy, Immunology and Blood and Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, CA, USA.
authors: Christopher C
authors: CC
authors: Dvorak
authors: Biljana N
authors: BN
authors: Horn
authors: Jennifer M
authors: JM
authors: Puck
authors: Agnieszka
authors: A
authors: Czechowicz
authors: Judy A
authors: JA
authors: Shizuru
authors: Rose M
authors: RM
authors: Ko
authors: Morton J
authors: MJ
authors: Cowan
mesh_terms: D000293:Adolescent; D001596:Benzylamines; D002140:California; D017024:Chemotherapy, Adjuvant; D002648:Child; D003131:Combined Modality Therapy; D000080027:Cyclams; D004359:Drug Therapy, Combination; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use; D019650:Hematopoietic Stem Cell Mobilization; D018380:Hematopoietic Stem Cell Transplantation; D006571:Heterocyclic Compounds / Q000627:therapeutic use*; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D008297:Male; D010865:Pilot Projects; D011446:Prospective Studies; D016511:Severe Combined Immunodeficiency / Q000188:drug therapy / Q000276:immunology / Q000628:therapy*; D019172:Transplantation Conditioning; D016896:Treatment Outcome
chemical_list: D001596:Benzylamines; D000080027:Cyclams; D006571:Heterocyclic Compounds; D016179:Granulocyte Colony-Stimulating Factor; C088327:plerixafor
keywords: engraftment; haploidentical; plerixafor; severe combined immunodeficiency