Single- vs double-unit cord blood transplantation for children and young adults with acute leukemia or myelodysplastic syndrome
Gérard Michel, Claire Galambrun, Anne Sirvent, Cecile Pochon, Benedicte Bruno, Charlotte Jubert, Anderson Loundou, Ibrahim Yakoub-Agha, Noel Milpied, Patrick Lutz, Aude Marie-Cardine, Virginie Gandemer, Didier Blaise, Mauricette Michallet, Fanny Rialland, Cecile Renard, Claire Oudin, Sophie Esmiol, Mylene Seux, Karine Baumstarck, Mohamad Mohty, Vanderson Rocha, Jean-Hugues Dalle, Gérard Michel, Claire Galambrun, Anne Sirvent, Cecile Pochon, Benedicte Bruno, Charlotte Jubert, Anderson Loundou, Ibrahim Yakoub-Agha, Noel Milpied, Patrick Lutz, Aude Marie-Cardine, Virginie Gandemer, Didier Blaise, Mauricette Michallet, Fanny Rialland, Cecile Renard, Claire Oudin, Sophie Esmiol, Mylene Seux, Karine Baumstarck, Mohamad Mohty, Vanderson Rocha, Jean-Hugues Dalle
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.
© 2016 by The American Society of Hematology.
Source: PubMed