1.
Unrelated cord blood transplantation in patients with acquired refractory aplastic anemia: a nationwide phase II study
Peffault de Latour, R., Chevret, S., Jubert, C., Sirvent, A., Galambrun, C., Ruggeri, A., Gandemer, V., Cornillon, J., Rialland, F., Dalle, J. H., et al
Blood. 2018
Abstract
Outcomes remain poor for patients with refractory severe aplastic anemia (SAA). Alternative donor transplantation may be considered, but results from previous studies have not been encouraging. We conducted a prospective nationwide phase II study to assess the efficacy and safety of unrelated cord blood transplantation (CBT) in patients with refractory SAA (APCORD protocol, NCT 01343953). To demonstrate a significant difference in one-year survival from 20% (null hypothesis) to 50% (alternative hypothesis), we needed to include 25 transplanted patients. Twenty-six patients (median age: 16 years) were therefore included. Eligibility criteria required one or two unrelated CB units, containing separately or together more than 4x10(7) frozen nucleated cells/kg recipient body weight. The conditioning regimen comprised fludarabine, cyclophosphamide, thymoglobulin and 2-Gy total body irradiation. With a median follow-up of 38.8 months, engraftment occurred in 23 patients (88%); cumulative incidences of grade II-IV acute and chronic GvHD were 45.8% and 36%, respectively. Twenty-three patients were alive at one year, with an overall survival rate of 88.5%, differing significantly from the expected 20% (p<0.0001) (84% overall survival at 2 years). CBT with units containing at least 4x10(7) frozen nucleated cells/kg is therefore a valuable curative option for young adults with refractory SAA and no available matched unrelated donors.
2.
RIC versus MAC UCBT in adults with AML: A report from Eurocord, the ALWP and the CTIWP of the EBMT
Baron, F., Ruggeri, A., Beohou, E., Labopin, M., Sanz, G., Milpied, N., Michallet, M., Bacigalupo, A., Blaise, D., Sierra, J., et al
Oncotarget. 2016;7(28):43027-43038
Abstract
Nonrelapse mortality (NRM) is the first cause of treatment failure after unrelated cord blood transplantation (UCBT) following myeloablative conditioning (MAC). In the last decade, reduced-intensity conditioning (RIC) regimens have been developed with the aim of reducing NRM and allowing older patients and those with medical comorbidities to benefit from UCBT. The aim of the current retrospective study was to compare transplantation outcomes of acute myeloid leukemia (AML) patients given UCBT after either RIC or MAC. Data from 894 adults with AML receiving a single or double UCBT as first allograft from 2004 to 2013 at EBMT centers were included in this study. 415 patients were given UCBT after RIC while 479 patients following a MAC. In comparison to MAC recipients, RIC recipients had a similar incidence of neutrophil engraftment and of acute and chronic graft-versus-host disease (GVHD). However, RIC recipients had a higher incidence of disease relapse and a lower NRM, translating to comparable leukemia-free (LFS), GVHD-free, relapse-free survival (GRFS) and overall survival (OS). These observations remained qualitatively similar after adjusting for differences between groups in multivariate analyses. In conclusion, these data suggest that LFS and OS are similar with RIC or with MAC in adults AML patients transplanted with UCBT. These observations could serve as basis for a future prospective randomized study.