A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency
Corresponding Author
Christopher C. Dvorak
Division of Pediatric Allergy, Immunology and Blood and Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, CA, USA
Christopher C. Dvorak, Division of Pediatric Allergy, Immunology, & Blood and Marrow Transplantation, University of California San Francisco, 505 Parnassus Ave., M-659, San Francisco, CA 94143-1278, USA
Tel.: 415 476 2188
Fax: 415 502 4867
E-mail: [email protected]
Search for more papers by this authorBiljana N. Horn
Division of Pediatric Allergy, Immunology and Blood and Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, CA, USA
Search for more papers by this authorJennifer M. Puck
Division of Pediatric Allergy, Immunology and Blood and Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, CA, USA
Search for more papers by this authorAgnieszka Czechowicz
Division of Blood & Marrow Transplantation, Stanford University School of Medicine, Stanford, CA, USA
Search for more papers by this authorJudy A. Shizuru
Division of Blood & Marrow Transplantation, Stanford University School of Medicine, Stanford, CA, USA
Search for more papers by this authorRose M. Ko
Division of Blood & Marrow Transplantation, Stanford University School of Medicine, Stanford, CA, USA
Search for more papers by this authorMorton J. Cowan
Division of Pediatric Allergy, Immunology and Blood and Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, CA, USA
Search for more papers by this authorCorresponding Author
Christopher C. Dvorak
Division of Pediatric Allergy, Immunology and Blood and Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, CA, USA
Christopher C. Dvorak, Division of Pediatric Allergy, Immunology, & Blood and Marrow Transplantation, University of California San Francisco, 505 Parnassus Ave., M-659, San Francisco, CA 94143-1278, USA
Tel.: 415 476 2188
Fax: 415 502 4867
E-mail: [email protected]
Search for more papers by this authorBiljana N. Horn
Division of Pediatric Allergy, Immunology and Blood and Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, CA, USA
Search for more papers by this authorJennifer M. Puck
Division of Pediatric Allergy, Immunology and Blood and Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, CA, USA
Search for more papers by this authorAgnieszka Czechowicz
Division of Blood & Marrow Transplantation, Stanford University School of Medicine, Stanford, CA, USA
Search for more papers by this authorJudy A. Shizuru
Division of Blood & Marrow Transplantation, Stanford University School of Medicine, Stanford, CA, USA
Search for more papers by this authorRose M. Ko
Division of Blood & Marrow Transplantation, Stanford University School of Medicine, Stanford, CA, USA
Search for more papers by this authorMorton J. Cowan
Division of Pediatric Allergy, Immunology and Blood and Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, CA, USA
Search for more papers by this authorAbstract
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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