title: A pilot study of reduced intensity conditioning and allogeneic stem cell transplantation from unrelated cord blood and matched family donors in children and adolescent recipients.
abstract: Reduced intensity (RI) allogeneic stem cell transplantation (AlloSCT) was initially demonstrated in adults following HLA-matched family and unrelated adult donor AlloSCT. There is little information about RI AlloSCT in children. We report results of a pilot study of RI AlloSCT in 21 recipients (< or =21 years). Age: median 13 (0.5-21) years, 8F:13M, 14 unrelated cord blood units (UCB) (10 4/6, 4 5/6), two related BM (6/6, 5/6), four related PBSC (2 6/6, 2 5/6), and one related BM+PBSC (6/6). RI: fludarabine, busulfan (n=14); fludarabine, cyclophosphamide (n=4); fludarabine, melphalan (n=1); total body irradiation, fludarabine, cyclophosphamide (n=1); or fludarabine, cyclophosphamide, and etoposide (n=1). Graft-versus-host disease prophylaxis: FK506 0.03 mg/kg/day and mycophenolate mofetil 15 mg/kg/q 12 h. UCB median nuc/kg and CD34/kg was 4.3 x 10(7)/kg (0.9-10.8) and 1.9 x 10(5)/kg (0.3-6.9), and related BM/PBSC median nuc/kg and CD34/kg was 8.3 x 10(8) (4.7-18.9) and 5.0 x 10(6)/kg (4.6-6.4). Maximal chimerism following unrelated cord blood transplantation, 100% x 7, 98% x 1, 95% x 2, 55% x 1, and 0% x 3; related PBSC/BM, 100% x 5, 65% x 1, and 55% x 1. Graft failure occurred in 5/21 (24%). In summary, RI AlloSCT in children is feasible and tolerable (< or =25% GF) and results in > or =85% of recipients initially achieving > or =50% donor chimerism.
authors.affiliation: Department of Pediatrics, Children's Hospital New York-Presbyterian, Herbert Irving Comprehensive Cancer Center, Columbia University, New York, NY 10032, USA.
authors.forename: G
authors.initials: G
authors.lastname: Del Toro
authors.forename: P
authors.initials: P
authors.lastname: Satwani
authors.forename: L
authors.initials: L
authors.lastname: Harrison
authors.forename: Y-K
authors.initials: YK
authors.lastname: Cheung
authors.forename: M
authors.initials: M
authors.lastname: Brigid Bradley
authors.forename: D
authors.initials: D
authors.lastname: George
authors.forename: D J
authors.initials: DJ
authors.lastname: Yamashiro
authors.forename: J
authors.initials: J
authors.lastname: Garvin
authors.forename: D
authors.initials: D
authors.lastname: Skerrett
authors.forename: O
authors.initials: O
authors.lastname: Bessmertny
authors.forename: K
authors.initials: K
authors.lastname: Wolownik
authors.forename: C
authors.initials: C
authors.lastname: Wischhover
authors.forename: C
authors.initials: C
authors.lastname: van de Ven
authors.forename: M S
authors.initials: MS
authors.lastname: Cairo
mesh_terms: D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D005190:Family; D005260:Female; D005312:Fetal Blood / Q000166:cytology*; D006086:Graft vs Host Disease / Q000517:prevention & control*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D009369:Neoplasms / Q000188:drug therapy / Q000401:mortality / Q000628:therapy; D033581:Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D016019:Survival Analysis; D013997:Time Factors; D014019:Tissue Donors / Q000706:statistics & numerical data; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous
chemical_list: D007166:Immunosuppressive Agents