1. ¹Department of Pediatrics, Children's Hospital New York-Presbyterian, Herbert Irving Comprehensive Cancer Center, Columbia University, New York, NY, USA
2. ²Department of Biostatistics, Children's Hospital New York-Presbyterian, Herbert Irving Comprehensive Cancer Center, Columbia University, New York, NY, USA
3. ³Department of Transfusion Medicine, Children's Hospital New York-Presbyterian, Herbert Irving Comprehensive Cancer Center, Columbia University, New York, NY, USA

Correspondence: Dr MS Cairo, Pediatrics, Medicine and Pathology (Professor), Blood and Marrow Transplantation (Director), Children's Hospital New York-Presbyterian, Columbia University, 161 Fort Washington, Irving 7, New York, NY 10032 USA. E-mail: mc1310@columbia.edu

Received 11 April 2003; Accepted 21 September 2003; Published online 19 January 2004.

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 (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 4.3 10⁷/kg (0.9–10.8) and 1.9 10⁵/kg (0.3–6.9), and related BM/PBSC median nuc/kg and CD34/kg was 8.3 10⁸ (4.7–18.9) and 5.0 10⁶/kg (4.6–6.4). Maximal chimerism following unrelated cord blood transplantation, 100% 7, 98% 1, 95% 2, 55% 1, and 0% 3; related PBSC/BM, 100% 5, 65% 1, and 55% 1. Graft failure occurred in 5/21 (24%). In summary, RI AlloSCT in children is feasible and tolerable (25% GF) and results in 85% of recipients initially achieving 50% donor chimerism.