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Blood. 2016 Jun 30;127(26):3450-7. doi: 10.1182/blood-2016-01-694349. Epub 2016 Apr 20.

Single- vs double-unit cord blood transplantation for children and young adults with acute leukemia or myelodysplastic syndrome.

Author information

1
Department of Pediatric Hematology-Oncology, Assistance Publique-Hôpitaux de Marseille, La Timone Hospital, Aix-Marseille University, Marseille, France; Department of Public Health, Laboratoire de Santé Publique (EA 3279) Research Unit, Marseille University Hospital, Aix-Marseille University, Marseille, France;
2
Department of Pediatric Hematology-Oncology, Assistance Publique-Hôpitaux de Marseille, La Timone Hospital, Aix-Marseille University, Marseille, France;
3
Department of Pediatric Hematology-Oncology, Montpellier University Hospital, Montpellier, France;
4
Department of Pediatric Hematology-Oncology, Nancy University Hospital, Nancy, France;
5
Department of Pediatric Hematology, Jeanne de Flandre Hospital, Lille Nord de France University, Lille, France;
6
Department of Pediatric Hematology-Oncology, Bordeaux University Hospital, Bordeaux, France;
7
Department of Public Health, Laboratoire de Santé Publique (EA 3279) Research Unit, Marseille University Hospital, Aix-Marseille University, Marseille, France;
8
Department of Hematology, Huriez Hospital, Lille Nord de France University, Lille, France;
9
Department of Hematology, Haut-Leveque Hospital, Bordeaux University Hospital, Pessac, France;
10
Department of Pediatric Hematology-Oncology, Strasbourg University Hospital, Strasbourg, France;
11
Department of Pediatric Hematology-Oncology, Rouen University Hospital, Rouen, France;
12
Department of Pediatric Hematology-Oncology, Rennes University Hospital, Rennes, France;
13
Department of Hematology-Oncology, Transplant and Cellular Therapy Unit, Paoli-Calmettes Institute, Centre de Recherche en Cancérologie de Marseille and Aix-Marseille University, Marseille, France;
14
Department of Hematology, Edouard Herriot Hospital, Lyon University Hospital, Lyon, France;
15
Department of Pediatric Hematology-Oncology, Nantes University Hospital, Nantes, France;
16
Institute of Pediatric Hematology-Oncology, Lyon University Hospital, Lyon, France;
17
Clinical Research and Innovation Direction, Assistance Publique-Hôpitaux de Marseille, Marseille, France;
18
Department of Hematology, Assistance Publique-Hôpitaux de Paris (AP-HP), Hôpital Saint-Antoine, Université Pierre and Marie Curie, Inserm Unité 938, Paris, France;
19
Eurocord, Hôpital Saint Louis AP-HP, University Paris VII, Paris, France; and.
20
Department of Pediatric Hematology, AP-HP, Robert Debré Hospital, Paris Diderot University, Paris, France.

Abstract

Transplantation of 2 unrelated cord blood (UCB) units instead of 1 has been proposed to increase the cell dose. We report a prospective randomized study, designed to compare single- vs double-UCB transplantation in children and young adults with acute leukemia in remission or myelodysplasia. Eligible patients had at least two 4-6 HLA-identical UCBs with >3 × 10(7) nucleated cells/kg for the first and >1.5 × 10(7) for the second. The primary end point was the 2-year cumulative incidence of transplantation strategy failure, a composite end point including transplant-related mortality (TRM), engraftment failure, and autologous recovery. Randomized patients who did not proceed to transplantation due to refractory disease were considered transplantation failures. A total of 151 patients were randomized and included in the intent-to-treat analysis; 137 were transplanted. Double-UCB transplantation did not decrease transplantation strategy failure (23.4% ± 4.9% vs 14.9% ± 4.2%). Two-year posttransplant survival, disease-free survival, and TRM were 68.8% ± 6.0%, 67.6% ± 6.0%, and 5.9% ± 2.9% after single-unit transplantation compared with 74.8% ± 5.5%, 68.1% ± 6.0%, and 11.6% ± 3.9% after double-unit transplantation. The final relapse risk did not significantly differ, but relapses were delayed after double-unit transplantation. Overall incidences of graft-versus-host disease (GVHD) were similar, but chronic GVHD was more frequently extensive after double-UCB transplantation (31.9% ± 5.7% vs 14.7% ± 4.3%, P = .02). In an exploratory subgroup analysis, we found a significantly lower relapse risk after double-unit transplantation in patients receiving total body irradiation without antithymocyte globulin (ATG), whereas the relapse risk was similar in the group treated with busulfan, cyclophosphamide, and ATG. Single-UCB transplantation with adequate cell dose remains the standard of care and leads to low TRM. Double-unit transplantation should be reserved for patients who lack such units. This trial was registered at www.clinicaltrials.gov as #NCT01067300.

Comment in

PMID:
27099151
DOI:
10.1182/blood-2016-01-694349
[Indexed for MEDLINE]
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