High success rate of hematopoietic cell transplantation regardless of donor source in children with very high-risk leukemia

Wing Leung, Dario Campana, Jie Yang, Deqing Pei, Elaine Coustan-Smith, Kwan Gan, Jeffrey E. Rubnitz, John T. Sandlund, Raul C. Ribeiro, Ashok Srinivasan, Christine Hartford, Brandon M. Triplett, Mari Dallas, Asha Pillai, Rupert Handgretinger, Joseph H. Laver, Ching Hon Pui

Research output: Contribution to journalArticle

114 Scopus citations


We evaluated 190 children with very high-risk leukemia, who underwent allogeneic hematopoietic cell transplantation in 2 sequential treatment eras, to determine whether those treated with contemporary protocols had a high risk of relapse or toxic death, and whether non-HLA-identical transplantations yielded poor outcomes. For the recent cohorts, the 5-year overall survival rates were65%for the 37 patients with acute lymphoblastic leukemia and 74% for the 46 with acute myeloid leukemia; these rates compared favorably with those of earlier cohorts (28%, n = 57; and 34%, n = 50, respectively). Improvement in the recent cohorts was observed regardless of donor type (sibling, 70% vs 24%; unrelated, 61% vs 37%; and haploidentical,88%vs 19%), attributable to less infection (hazard ratio [HR] = 0.12; P = .005), regimen-related toxicity (HR = 0.25; P = .002), and leukemiarelated death (HR = 0.40; P = .01). Survival probability was dependent on leukemia status (first remission vs more advanced disease; HR = 0.63; P = .03) or minimal residual disease (positive vs negative; HR = 2.10; P = .01) at the time of transplantation. We concluded that transplantation has improved over time and should be considered for all children with very high-risk leukemia, regardless of matched donor availability.

Original languageEnglish (US)
Pages (from-to)223-230
Number of pages8
Issue number2
StatePublished - Jul 14 2011


ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

Cite this

Leung, W., Campana, D., Yang, J., Pei, D., Coustan-Smith, E., Gan, K., Rubnitz, J. E., Sandlund, J. T., Ribeiro, R. C., Srinivasan, A., Hartford, C., Triplett, B. M., Dallas, M., Pillai, A., Handgretinger, R., Laver, J. H., & Pui, C. H. (2011). High success rate of hematopoietic cell transplantation regardless of donor source in children with very high-risk leukemia. Blood, 118(2), 223-230. https://doi.org/10.1182/blood-2011-01-333070