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

• Published in: Blood 2011-07, Vol.118 (2), p.223-230
• Main Authors: Leung, Wing, Campana, Dario, Yang, Jie, Pei, Deqing, Coustan-Smith, Elaine, Gan, Kwan, Rubnitz, Jeffrey E., Sandlund, John T., Ribeiro, Raul C., Srinivasan, Ashok, Hartford, Christine, Triplett, Brandon M., Dallas, Mari, Pillai, Asha, Handgretinger, Rupert, Laver, Joseph H., Pui, Ching-Hon
• Format: Article
• Language: English
• Subjects: Biological and medical sciences; Child; Clinical Trials and Observations; Cohort Studies; Female; Follow-Up Studies; Hematologic and hematopoietic diseases; Hematopoietic Stem Cell Transplantation - statistics & numerical data; Humans; Leukemia - epidemiology; Leukemia - mortality; Leukemia - pathology; Leukemia - therapy; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis; Lymphoid Neoplasia; Male; Medical sciences; Myeloid Neoplasia; Neoplasm Staging; Neoplasm, Residual - epidemiology; Plenary Paper; Retrospective Studies; Risk Factors; Survival Analysis; Tissue Donors; Transplantation; Treatment Outcome
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2011-01-333070

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 were 65% 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 leukemia-related 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.