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Blood, Vol. 93 No. 11 (June 1), 1999:
pp. 3662-3671
Factors Associated With Outcome After Cord Blood Transplantation in
Children With Acute Leukemia
Franco Locatelli,
Vanderson Rocha,
Claude Chastang,
William Arcese,
Gerard Michel,
Manoel Abecasis,
Chiara Messina,
Juan Ortega,
Isabel Badell-Serra,
Emanuel Plouvier,
Gerard Souillet,
Jean-Pierre Jouet,
Ricardo Pasquini,
Euripedes Ferreira,
Federico Garnier, and
Eliane Gluckman on behalf of Eurocord-Cord Blood Transplant Group
From the Clinica Pediatrica, IRCCS Policlinico San Matteo, University
of Pavia, Italy; Hôpital Saint-Louis, Paris, France; University
La Sapienza, Rome, Italy; Hôpital La Timone, Marseille, France;
Instituto Portugues de Oncologia, Lisboa, Portugal; Clinica
Oncoematologica Pediatrica e Centro delle Leucemie Infantili, Padova,
Italy; Hospital Infantil Vall d'Hebron, Barcelona, Spain; Hospital
Santa Creu i Sant Pau, Barcelona, Spain; Hôpital Saint Jacques,
Besançon, Hôpital Debrousse, Lyon, and Hôpital Claude
Huriez, Lille, France; Hospital de Clinicas, Curitiba, and Hospital
Albert Einstein, Sao Paulo, Brazil.
We have analyzed factors influencing the outcome of 102 children
with acute leukemia given a cord blood transplantation (CBT) and
reported to the Eurocord Registry. Seventy patients with acute lymphoblastic and 32 with acute myeloid leukemia were given either a
related (n = 42) or an unrelated (n = 60) CBT. Children given CBT during first or second complete remission were considered as
belonging to the good-risk group (n = 66), whereas those who received a transplant in a more advanced stage of disease were assigned
to the poor-risk group (n = 36). In the related group (RCBT), 12 of
42 patients received transplantation from an HLA-disparate donor,
whereas in the unrelated group (UCBT) 54 of 60 received an HLA
mismatched CBT. Kaplan-Meier estimates for neutrophil recovery at day
60 were 84% ± 7% in RCBT and 79 ± 6% in UCBT
(P = .16). In multivariate analysis, the most important
factor influencing neutrophil engraftment in UCBT was a nucleated cell
dose infused greater than 3.7 × 107/kg
(P = .05, relative risk [RR] of 1.85, 95% confidence
interval [CI]: 0.98-3.4). The incidence of grade II through IV acute
graft-versus-host disease was 41% ± 8% in the RCBT group and 37% ± 6% in the UCBT group (P = .59). Kaplan-Meier estimates
of 2-year event-free survival (EFS) after RCBT or UCBT were 39% ± 8% and 30% ± 7%, respectively (P = .19). In
multivariate analysis, the most important factor influencing EFS was
disease status at time of transplantation: good-risk patients had a
2-year EFS of 49% ± 7% as compared to 8% ± 5% in patients with
more advanced disease (P = .0003, RR: 0.40, 95% CI: 0.24 to 0.65). This was a consequence of both an increased 1-year transplant
related mortality and a higher 2-year relapse rate in the poor-risk
group (65% ± 9% and 77% ± 14%, respectively), as compared
with good risk patients (34% ± 6% and 31% ± 9%,
respectively). These data confirm that allogeneic CBT from either a
related or an unrelated donor is a feasible procedure able to cure a
significant proportion of children with acute leukemia, especially if
transplanted in a favorable phase of disease.
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