What is the role of umbilical cord blood transplantation (CBT) for the treatment of aplastic anemia?

Updated: Jan 29, 2021
  • Author: Sameer Bakhshi, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Early studies of unrelated umbilical cord blood transplantation (UCBT) reported high graft failure rates. However, subsequent studies of UCBT using conditioning regimens that included low-dose total-body irradiation (TBI) yielded improvements in engraftment and overall survival. [87]

A nationwide prospective phase II study from France concluded that UCBT is a valuable option for young adults with refractory severe aplastic anemia (SAA) and no available matched unrelated donors. The conditioning regimen comprised fludarabine, cyclophosphamide, antithymocyte globulin (ATG), and 2-Gy TBI. Engraftment occurred in 23 of the 26 patients in the study, and all 23 were alive at 1 year, for an overall survival rate of 88.5%. The cumulative incidences of grade II-IV acute and chronic graft-versus-host disease were 45.8% and 36%, respectively. [88]

Du et al reported successful use of an immunoablative conditioning regimen for UCBT that does not include TBI and does not include ATG, as previous studies have found reduced engraftment and survival rates with conditioning regimens containing ATG. In their 15 patients, the first 10 received fludarabine and cyclophosphamide, to which busulfan was added in the subsequent 5 patients. Sustained engraftment was observed in 13 patients and after median follow-up of 33.8 months, 12 patients remained alive. [87]


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