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Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a phase 1 trial using the AastromReplicell System.

Publication ,  Journal Article
Jaroscak, J; Goltry, K; Smith, A; Waters-Pick, B; Martin, PL; Driscoll, TA; Howrey, R; Chao, N; Douville, J; Burhop, S; Fu, P; Kurtzberg, J
Published in: Blood
June 15, 2003

Allogeneic stem cell transplantation with umbilical cord blood (UCB) cells is limited by the cell dose a single unit provides recipients. Ex vivo expansion is one strategy to increase the number of cells available for transplantation. Aastrom Biosciences developed an automated continuous perfusion culture device for expansion of hematopoietic stem cells (HSCs). Cells are expanded in media supplemented with fetal bovine serum, horse serum, PIXY321, flt-3 ligand, and erythropoietin. We performed a phase 1 trial augmenting conventional UCB transplants with ex vivo-expanded cells. The 28 patients were enrolled on the trial between October 8, 1997 and September 30, 1998. UCB cells were expanded in the device, then administered as a boost to the conventional graft on posttransplantation day 12. While expansion of total cells and colony-forming units (CFUs) occurred in all cases, the magnitude of expansion varied considerably. The median fold increase was 2.4 (range, 1.0-8.5) in nucleated cells, 82 (range, 4.6-266.4) in CFU granulocyte-macrophages, and 0.5 (range, 0.09-2.45) in CD34+ lineage negative (lin-) cells. CD3+ cells did not expand under these conditions. Clinical-scale ex vivo expansion of UCB is feasible, and the administration of ex vivo-expanded cells is well tolerated. Augmentation of UCB transplants with ex vivo-expanded cells did not alter the time to myeloid, erythroid, or platelet engraftment in 21 evaluable patients. Recipients of ex vivo-expanded cells continue to have durable engraftment with a median follow-up of 47 months (range, 41-51 months). A randomized phase 2 study will determine whether augmenting UCB transplants with ex vivo-expanded UCB cells is beneficial.

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Published In

Blood

DOI

ISSN

0006-4971

Publication Date

June 15, 2003

Volume

101

Issue

12

Start / End Page

5061 / 5067

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Time Factors
  • Recurrence
  • Infant
  • Immunology
  • Humans
  • Hematopoietic Stem Cells
  • Graft vs Host Disease
  • Graft Survival
  • Fetal Blood
 

Citation

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Jaroscak, J., Goltry, K., Smith, A., Waters-Pick, B., Martin, P. L., Driscoll, T. A., … Kurtzberg, J. (2003). Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a phase 1 trial using the AastromReplicell System. Blood, 101(12), 5061–5067. https://doi.org/10.1182/blood-2001-12-0290
Jaroscak, Jennifer, Kristin Goltry, Alan Smith, Barbara Waters-Pick, Paul L. Martin, Timothy A. Driscoll, Richard Howrey, et al. “Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a phase 1 trial using the AastromReplicell System.Blood 101, no. 12 (June 15, 2003): 5061–67. https://doi.org/10.1182/blood-2001-12-0290.
Jaroscak J, Goltry K, Smith A, Waters-Pick B, Martin PL, Driscoll TA, et al. Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a phase 1 trial using the AastromReplicell System. Blood. 2003 Jun 15;101(12):5061–7.
Jaroscak, Jennifer, et al. “Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a phase 1 trial using the AastromReplicell System.Blood, vol. 101, no. 12, June 2003, pp. 5061–67. Pubmed, doi:10.1182/blood-2001-12-0290.
Jaroscak J, Goltry K, Smith A, Waters-Pick B, Martin PL, Driscoll TA, Howrey R, Chao N, Douville J, Burhop S, Fu P, Kurtzberg J. Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a phase 1 trial using the AastromReplicell System. Blood. 2003 Jun 15;101(12):5061–5067.

Published In

Blood

DOI

ISSN

0006-4971

Publication Date

June 15, 2003

Volume

101

Issue

12

Start / End Page

5061 / 5067

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Time Factors
  • Recurrence
  • Infant
  • Immunology
  • Humans
  • Hematopoietic Stem Cells
  • Graft vs Host Disease
  • Graft Survival
  • Fetal Blood