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Blood, 15 May 2001, Vol. 97, No. 10, pp. 2962-2971
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Comparison of outcomes of unrelated bone marrow and
umbilical cord blood transplants in children with acute
leukemia
Vanderson Rocha,
Jacqueline Cornish,
Eric L. Sievers,
Alexandra Filipovich,
Franco Locatelli,
Cristina Peters,
Mats Remberger,
Gérard Michel,
William Arcese,
Sandro Dallorso,
Karin Tiedemann,
Alessandro Busca,
Ka-Wah Chan,
Shunichi Kato,
Juan Ortega,
Marcus Vowels,
Axel Zander,
Gérard Souillet,
Anthony Oakill,
Ann Woolfrey,
Andrea L. Pay,
Ann Green,
Federico Garnier,
Irina Ionescu,
Peter Wernet,
Girolamo Sirchia,
Pablo Rubinstein,
Sylvie Chevret, and
Eliane Gluckman
From Eurocord Cord Blood Transplant Group (CBTG) and
Biostatistics Department, Saint Louis Hospital AP-HP, University of
Paris 7, France; Hospital for Sick Children, Bristol, United Kingdom;
Fred Hutchinson Cancer Research Center, Seattle, WA; Children's
Hospital Medical Center, Cincinnati, OH; Clinica Pediatrica, IRCCS
Policlinico San Matteo, Pavia, Italy; St Anna Kinderspital, Vienna,
Austria; Huddinge University Hospital, Huddinge, Sweden; Hospital La
Timone, Marseille, France; University La Sapienza, Rome, Italy;
Institute G. Gaslini, Genoa, Italy; Royal Children's Hospital,
Melbourne, Australia; Ospedale Regina Margherita, Turin, Italy; MD
Anderson Cancer Center, Houston, TX; Tokai University School of
Medicine, Isehara, Japan; Hospital Infantil Vall d'Hebron, Barcelona,
Spain; Sydney Children's Hospital, Sydney, Australia; University
Hospital Eppendorf, Hamburg, Germany; Hospital Debrousse, Lyon, France;
Anthony Nolan Bone Marrow Trust Registry, London, United Kingdom;
National Blood Service, Bristol, United Kingdom; Duesseldorf Cord Blood
Bank, Duesseldorf, Germany; Milano Cord Blood Bank, Milano, Italy; New
York Cord Blood Bank, New York, NY.
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.
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