A phase II study of reduced intensity double umbilical cord blood transplantation using fludarabine, melphalan, and low dose total body irradiation.

Abstract

In this multicenter Phase 2 single arm study, we substituted low dose total body irradiation (TBI) for antithymocyte globulin (ATG) in a reduced intensity conditioning regimen with the intent to lower the risk for viral infections after double umbilical cord blood (UCB) transplantation. The conditioning regimen consisted of fludarabine (30 mg/m2/day, Day -7 to -2), melphalan (100 mg/m2/day, Day -1), and TBI (200cGy, Day 0). Graft-versus-host disease prophylaxis was sirolimus and tacrolimus. Thirty-one patients were treated on the protocol. The median time of follow-up for survivors was 24 months (range, 3.3-55.1). Nineteen patients experienced a total of 24 clinically significant viral reactivations or infections, with 1-year cumulative incidence rate of first significant viral event as 64% (95% CI, 43-79%), compared with our historical control of 53%. Within the context of these 24 clinically significant viral reactivations, there were a total of 10 infections with organ involvement. Nonrelapse mortality was 28% (95% CI 13-45%) at 2 years. The 2-year overall and progression-free survivals were 53% (95% CI 33-69%) and 47% (95% CI 28-64%), respectively. In conclusion, the substitution of low dose TBI for ATG did not decrease the incidence of significant viral events after UCB transplantation.

• Authors: Zachariah DeFilipp, Shuli Li, David Avigan, Philippe Armand, Vincent T Ho, John Koreth, Sarah Nikiforow, Edwin P Alyea, Jerome Ritz, Vassiliki Boussiotis, Jacalyn Rosenblatt, Jami Brown, Steven McAfee, Bimalangshu R Dey, Areej El-Jawahri, Thomas R Spitzer, Yi-Bin Chen, Robert J Soiffer, Joseph H Antin, Karen K Ballen, Corey S Cutler
• Journal: Bone marrow transplantation (Bone Marrow Transplant) Vol. 55 Issue 4 Pg. 804-810 (04 2020) ISSN: 1476-5365 [Electronic] England
• PMID: 31616065 (Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)

Chemical References

• Vidarabine
• fludarabine
• Melphalan

Topics

• Cord Blood Stem Cell Transplantation
• Graft vs Host Disease (prevention & control)
• Hematopoietic Stem Cell Transplantation
• Humans
• Melphalan
• Transplantation Conditioning
• Vidarabine (analogs & derivatives)
• Whole-Body Irradiation