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Cord Blood Stem Cells

Reduced intensity allogeneic umbilical cord blood transplantation in children and adolescent recipients with malignant and non-malignant diseases

Bone Marrow Transplantation volume 40, pages 621–631 (2007)Cite this article

Abstract

There is a significant amount of morbidity and mortality following myeloablative umbilical cord blood transplantation (UCBT). Reduced intensity (RI) conditioning offers an alternative to myeloablative conditioning before UCBT. We investigated RI-UCBT in 21 children and adolescents with malignant (n=14), and non-malignant diseases (n=7). RI conditioning consisted of fludarabine (150–180 mg/m2) with either busulfan (⩽8 mg/kg)+rabbit antithymocyte globulin (R-ATG; n=16) or cyclophosphamide+R-ATG±etoposide (n=5). Human leukocyte antigen match: 4/6 (n=13), 5/6 (n=5) and 6/6 (n=3). The median total nucleated cell and CD34+ cell dose per kilogram were 3.58 × 107 and 2.54 × 105, respectively. The median time for neutrophil and platelet engraftment was 17.5 and 52 days, respectively. There were six primary graft failures (chronic myelogenous leukemia (CML), β-thalassemia, hemophagocytic lymphohistiocytosis (HLH) and myelodysplastic syndrome (MDS)). The probability of developing grade II to grade IV acute graft-versus-host disease (GVHD) and chronic GVHD was 28.6 and 16.7%, respectively. Incidence of transplant-related mortality (TRM) was 14%. The 5 years overall survival (OS) in all patients was 59.8%. The 5 years OS for patients with average versus poor-risk malignancy was 77.8 versus 22.2% (P=0.03). RI-UCBT may result in graft failure in specific high-risk chemo-naïve patients (CML, β-thalassemia, HLH and MDS), but in more heavily pretreated pediatric and adolescent recipients results in rapid engraftment and may be associated with decreased severe GVHD and TRM.

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Acknowledgements

We thank Leslie Disla for her assistance with the preparation of this manuscript. We also thank the nurses of the inpatient and outpatient units for their expert care and most importantly to the patients and their families who participated in these clinical research studies. This paper was presented in part at the American Society of Blood and Marrow Transplant, 2007 Annual Meeting, Keystone, Colorado. This work was supported in part by the Pediatric Cancer Research Foundation, National Institute of Arthritis and Musculoskeletal and Skin Diseases (R21AR49330) (MSC), National Cancer Institute (5P30CA13697) (MSC), Marisa Fund, Sonia Scaramella Fund, Bevanmar Foundation and Brittany Barron Fund.

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  1. M B Bradley and P Satwani: These authors contributed equally to this work and should be considered co-primary or first authors.

Authors and Affiliations

  1. Department of Pediatrics, Columbia University, New York, NY, USA

    M B Bradley, P Satwani, L Baldinger, E Morris, C van de Ven, J Garvin, D George, M Bhatia, E Roman, E Qualter, R Hawks, K Wolownik, S Foley, O Militano, J Leclere & M S Cairo

  2. Department of Pediatric Hematology/Oncology, Mount Sinai Medical Center, New York, NY, USA

    G Del Toro

  3. Department of Surgery, Immunogenetics and Transplantation Laboratory, University of California, San Francisco, CA, USA

    L A Baxter-Lowe

  4. Department of Pathology, Columbia University, New York, NY, USA

    J Schwartz & M S Cairo

  5. Department of Biostatistics, Columbia University, New York, NY, USA

    Y-K Cheung

  6. Department of Medicine, Columbia University, New York, NY, USA

    M S Cairo

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  1. M B Bradley
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Correspondence to M S Cairo.

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Bradley, M., Satwani, P., Baldinger, L. et al. Reduced intensity allogeneic umbilical cord blood transplantation in children and adolescent recipients with malignant and non-malignant diseases. Bone Marrow Transplant 40, 621–631 (2007). https://doi.org/10.1038/sj.bmt.1705785

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