title: Safe and efficacious allogeneic bone marrow transplantation for nonmalignant disorders using partial T cell depletion and no posttransplantation graft-versus-host-disease prophylaxis.
abstract: In an attempt to abrogate the deleterious effects of graft-versus-host disease (GVHD), allogeneic transplantation for nonmalignant diseases was performed using high-dose CD34-cell infusion, partial T cell depletion, and no posttransplantation GVHD prophylaxis. Between 1998 and 2004, 16 patients with matched related donors were treated. Median age was 1.5 years (range, 5 months-18 years). The conditioning regimen consisted of busulphan 16 mg/kg, cyclophosphamide 200 mg/kg, antithymocyte globulin (ATG) 25 mg/kg, and fludarabine 200 mg/m(2). No GVHD prophylaxis was given. High doses of CD34 cells, positively selected by immunomagnetic beads, were infused at a median dose of 10.7 x 10(6) CD34/kg (range, 7.4-50 x 10(6)). A total of 1 x 10(5)/kg T cells were given. All patients engrafted, with no graft rejections. All were alive and well at a median of 37 months posttransplantation (range, 18-89 months). Only 1 patient developed chronic GVHD. No episodes of severe infection occurred during or after transplantation. Immunologic reconstitution with CD3/CD4 T cells > 200/microL was observed at a median of 117 days and that with naive T cells (CD4/CD45RA) at a median of 188 days posttransplantation. Our findings suggest that allogeneic transplantation from a matched family donor for nonmalignant disorders can be successfully performed using high doses of CD34 cells, moderate T cell depletion, and no posttransplantation immunosuppression.
authors.affiliation: Department of Pediatric Hemato-Oncology, Meyer Children Hospital, Haifa, Israel. r_elhasid@rambam.health.gov.il <r_elhasid@rambam.health.gov.il>
authors.forename: Ronit
authors.initials: R
authors.lastname: Elhasid
authors.forename: Myriam Ben
authors.initials: MB
authors.lastname: Arush
authors.forename: Irena
authors.initials: I
authors.lastname: Zaidman
authors.forename: Ronit
authors.initials: R
authors.lastname: Leiba
authors.forename: Ayelet Ben
authors.initials: AB
authors.lastname: Barak
authors.forename: Sergey
authors.initials: S
authors.lastname: Postovsky
authors.forename: Nuhad
authors.initials: N
authors.lastname: Haddad
authors.forename: Tami
authors.initials: T
authors.lastname: Katz
authors.forename: Shimon
authors.initials: S
authors.lastname: Pollack
authors.forename: Ivanka
authors.initials: I
authors.lastname: Sami
authors.forename: Osnat
authors.initials: O
authors.lastname: Gidoni
authors.forename: Dina
authors.initials: D
authors.lastname: Rubin
authors.forename: Hanna
authors.initials: H
authors.lastname: Mandel
authors.forename: Dina
authors.initials: D
authors.lastname: Attias
authors.forename: Yair
authors.initials: Y
authors.lastname: Reisner
authors.forename: Amos
authors.initials: A
authors.lastname: Etzioni
authors.forename: Jacob M
authors.initials: JM
authors.lastname: Rowe
mesh_terms: D000293:Adolescent; D018952:Antigens, CD34; D016026:Bone Marrow Transplantation / Q000379:methods*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000517:prevention & control*; D006801:Humans; D007107:Immune System / Q000166:cytology; D007223:Infant; D008212:Lymphocyte Depletion / Q000379:methods*; D008297:Male; D012189:Retrospective Studies; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D018952:Antigens, CD34