Comparison of outcomes of unrelated bone marrow and umbilical cord blood transplants in children with acute leukemia

• Published in: Blood Vol. 97; no. 10; pp. 2962 - 2971
• Main Authors: Rocha, Vanderson, Cornish, Jacqueline, Sievers, Eric L., Filipovich, Alexandra, Locatelli, Franco, Peters, Cristina, Remberger, Mats, Michel, Gérard, Arcese, William, Dallorso, Sandro, Tiedemann, Karin, Busca, Alessandro, Chan, Ka-Wah, Kato, Shunichi, Ortega, Juan, Vowels, Marcus, Zander, Axel, Souillet, Gérard, Oakill, Anthony, Woolfrey, Ann, Pay, Andrea L., Green, Ann, Garnier, Federico, Ionescu, Irina, Wernet, Peter, Sirchia, Girolamo, Rubinstein, Pablo, Chevret, Sylvie, Gluckman, Eliane
• Format: Journal Article
• Language: English
• Published: Washington, DC Elsevier Inc 15.05.2001; The Americain Society of Hematology
• Subjects: Analysis of Variance; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Biological and medical sciences; Bone Marrow Transplantation; Bone marrow, stem cells transplantation. Graft versus host reaction; Child; Child, Preschool; Disease-Free Survival; Female; Fetal Blood - cytology; Graft vs Host Disease; Hematopoietic Stem Cell Transplantation; Humans; Leukemia - surgery; Leukemia, Myeloid, Acute - mortality; Leukemia, Myeloid, Acute - surgery; Male; Medical sciences; Medicin och hälsovetenskap; Precursor Cell Lymphoblastic Leukemia-Lymphoma - mortality; Precursor Cell Lymphoblastic Leukemia-Lymphoma - surgery; Recurrence; Registries; Retrospective Studies; Tissue Donors; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Transplantation Conditioning; Treatment Outcome; Human; Immunopathology; Prognosis; Acute; Stem cell; Hematopoietic cell; Homograft; Graft versus host reaction; Malignant hemopathy; Blood; Depletion; Treatment; Lymphoproliferative syndrome; T-Lymphocyte; Bone marrow; Graft; Umbilical cord; Acute lymphocytic leukemia; Child; Comparative study; Acute myelocytic leukemia
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood.V97.10.2962

In order to compare the outcomes of unrelated umbilical cord blood transplants (UCBTs) or bone marrow transplants, 541 children with acute leukemia (AL) transplanted with umbilical cord blood (n = 99), T-cell–depleted unrelated bone marrow transplants (T-UBMT) (n = 180), or nonmanipulated (UBMT) (n = 262), were analyzed in a retrospective multicenter study. Comparisons were performed after adjustment for patient, disease, and transplant variables. The major difference between the 3 groups was the higher number in the UCBT group of HLA mismatches (defined by serology for class I and molecular typing for DRB1). The donor was HLA mismatched in 92% of UCBTs, in 18% of UBMTs, and in 43% of T-UBMTs (P < .001). Other significant differences were observed in pretransplant disease characteristics, preparative regimens, graft-versus-host disease (GVHD) prophylaxis, and number of cells infused. Nonadjusted estimates of 2-year survival and event-free survival rates were 49% and 43%, respectively, in the UBMT group, 41% and 37% in the T-UBMT group, and 35% and 31% in the UCBT group. After adjustment, differences in outcomes appeared in the first 100 days after the transplantation. Compared with UBMT recipients, UCBT recipients had delayed hematopoietic recovery (Hazard ratio [HR] = 0.37; 95% confidence interval [95CI]: 0.27-0.52; P < .001), increased 100 day transplant-related mortality (HR = 2.13; 95CI: 1.20-3.76;P < .01) and decreased acute graft-versus-host disease (aGVHD) (HR = 0.50; 95CI: 0.34-0.73; P < .001). T-UBMT recipients had decreased aGVHD (HR = 0.25; 95CI: 0.17-0.36;P < .0001) and increased risk of relapse (HR = 1.96; 95CI: 1.11-3.45; P = .02). After day 100 posttransplant, the 3 groups achieved similar results in terms of relapse. Chronic GVHD was decreased after T-UBMT (HR = 0.21; 95CI: 0.11-0.37;P < .0001) and UCBT (HR = 0.24; 95CI: 0.01-0.66;P = .002), and overall mortality was higher in T-UBMT recipients (HR = 1.39; 95CI: 0.97-1.99; P < .07). In conclusion, the use of UCBT, as a source of hematopoietic stem cells, is a reasonable option for children with AL lacking an acceptably matched unrelated marrow donor.