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Cord Blood Stem Cells

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

Bone Marrow Transplantation volume 33, pages 613–622 (2004)Cite this article

Summary:

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.

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Acknowledgements

We acknowledge the expert editorial assistance of Linda Rahl in the preparation of this manuscript. We also acknowledge the nursing staff and bone marrow transplant team at the Children's Hospital of New York-Presbyterian at Columbia University for their superb care of these patients and their families. Lastly, we acknowledge and thank the children and their families for participating in this novel investigation. This study is supported in part by grants from the Pediatric Cancer Research Foundation, Swim Across America Foundation, Triple ‘C’ Foundation, and National Cancer Institute (5P30CA13697-29).

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Authors and Affiliations

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

    G Del Toro, P Satwani, L Harrison, M Brigid Bradley, D George, D J Yamashiro, J Garvin, O Bessmertny, K Wolownik, C Wischhover, C van de Ven & M S Cairo

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

    Y-K Cheung

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

    D Skerrett

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  1. G Del Toro
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Correspondence to M S Cairo.

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Presented in part at the International Society of Experimental Hematology (ISEH), Montreal, Canada, July 2002, and the American Society of Blood and Marrow Transplantation (ASBMT), Keystone, CO, USA, February 2003.

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Del Toro, G., Satwani, P., Harrison, L. et al. 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. Bone Marrow Transplant 33, 613–622 (2004). https://doi.org/10.1038/sj.bmt.1704399

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