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

Gerard Michel 1 Claire Galambrun 2 Anne Sirvent Cécile Pochon 3 Bénédicte Bruno 4 Charlotte Jubert Anderson Loundou 5 Ibrahim Yakoub-Agha 6 Noël Milpied 7 Patrick Lutz 8 Aude Marie-Cardine Virginie Gandemer 9, 10 Didier Blaise 11 Mauricette Michallet 12 Fanny Rialland 13 Cecile Renard Claire Oudin 14 Sophie Esmiol 14 Mylène Seux 15, 16 Karine Baumstarck 17 Mohamad Mohty 18, 19, 20, 21, 22 Vanderson Rocha 23, 24, 25 Jean-Hugues Dalle 26
Abstract : Transplantation of 2 unrelated cord blood (UCB) units instead of 1 has ă been proposed to increase the cell dose. We report a prospective ă randomized study, designed to compare single-vs double-UCB ă transplantation in children and young adults with acute leukemia in ă remission or myelodysplasia. Eligible patients had at least two 4-6 ă HLA-identical UCBs with >3x10(7) nucleated cells/kg for the first and ă >1.5x10(7) for the second. The primary end point was the 2-year ă cumulative incidence of transplantation strategy failure, a composite ă end point including transplant-related mortality (TRM), engraftment ă failure, and autologous recovery. Randomized patients who did not ă proceed to transplantation due to refractory disease were considered ă transplantation failures. A total of 151 patients were randomized and ă included in the intent-to-treat analysis; 137 were transplanted. ă Double-UCB transplantation did not decrease transplantation strategy ă failure (23.4% 6 4.9% vs 14.9% +/- 4.2%). Two-year posttransplant ă survival, disease-free survival, and TRM were 68.8% +/- 6.0%, 67.6% ă +/- 6.0%, and 5.9% +/- 2.9% after single-unit transplantation ă compared with 74.8% +/- 5.5%, 68.1% +/- 6.0%, and 11.6% +/- 3.9% ă after double-unit transplantation. The final relapse risk did not ă significantly differ, but relapses were delayed after double-unit ă transplantation. Overall incidences of graft-versus-host disease (GVHD) ă were similar, but chronic GVHD was more frequently extensive after ă double-UCB transplantation (31.9% +/- 5.7% vs 14.7% +/- 4.3%, ă P=.02). In an exploratory subgroup analysis, we found a significantly ă lower relapse risk after double-unit transplantation in patients ă receiving total body irradiation without antithymocyte globulin (ATG), ă whereas the relapse risk was similar in the group treated with busulfan, ă cyclophosphamide, and ATG. Single-UCB transplantation with adequate cell ă dose remains the standard of care and leads to low TRM. Double-unit ă transplantation should be reserved for patients who lack such units. ă This trial was registered at www. clinicaltrials. gov as #NCT01067300.
Keywords : Quality
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Gerard Michel, Claire Galambrun, Anne Sirvent, Cécile Pochon, Bénédicte Bruno, et al.. Single- vs double-unit cord blood transplantation for children and young ă adults with acute leukemia or myelodysplastic syndrome. Blood, American Society of Hematology, 2016, 127 (26), pp.3450-3457. 〈10.1182/blood-2016-01-694349〉. 〈hal-01482531〉



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