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Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide.

Publication ,  Journal Article
Horwitz, ME; Wease, S; Blackwell, B; Valcarcel, D; Frassoni, F; Boelens, JJ; Nierkens, S; Jagasia, M; Wagner, JE; Kuball, J; Koh, LP; Hanna, R ...
Published in: J Clin Oncol
February 10, 2019

PURPOSE: Increasing the number of hematopoietic stem and progenitor cells within an umbilical cord blood (UCB) graft shortens the time to hematopoietic recovery after UCB transplantation. In this study, we assessed the safety and efficacy of a UCB graft that was expanded ex vivo in the presence of nicotinamide and transplanted after myeloablative conditioning as a stand-alone hematopoietic stem-cell graft. METHODS: Thirty-six patients with hematologic malignancies underwent transplantation at 11 sites. RESULTS: The cumulative incidence of neutrophil engraftment at day 42 was 94%. Two patients experienced secondary graft failure attributable to viral infections. Hematopoietic recovery was compared with that observed in recipients of standard UCB transplantation as reported to the Center for International Blood and Marrow Transplant Research (n = 146). The median time to neutrophil recovery was 11.5 days (95% CI, 9 to 14 days) for recipients of nicotinamide-expanded UCB and 21 days (95% CI, 20 to 23 days) for the comparator ( P < .001). The median time to platelet recovery was 34 days (95% CI, 32 to 42 days) and 46 days (95% CI, 42 to 50 days) for the expanded and the comparator cohorts, respectively ( P < .001). The cumulative incidence of grade 2 to 4 acute graft-versus-host disease (GVHD) at day 100 was 44%, and grade 3 and 4 acute GVHD at day 100 was 11%. The cumulative incidence at 2 years of all chronic GVHD was 40%, and moderate/severe chronic GVHD was 10%. The 2-year cumulative incidences of nonrelapse mortality and relapse were 24% and 33%, respectively. The 2-year probabilities of overall and disease-free survival were 51% and 43%, respectively. CONCLUSION: UCB expanded ex vivo with nicotinamide shortens median neutrophil recovery by 9.5 days (95% CI, 7 to 12 days) and median platelet recovery by 12 days (95% CI, 3 to 16.5 days). This trial establishes feasibility, safety, and efficacy of an ex vivo expanded UCB unit as a stand-alone graft.

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Published In

J Clin Oncol

DOI

EISSN

1527-7755

Publication Date

February 10, 2019

Volume

37

Issue

5

Start / End Page

367 / 374

Location

United States

Related Subject Headings

  • Young Adult
  • Transplantation Conditioning
  • Oncology & Carcinogenesis
  • Niacinamide
  • Neutrophils
  • Middle Aged
  • Male
  • Humans
  • Hematologic Neoplasms
  • Graft vs Host Disease
 

Citation

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Horwitz, M. E., Wease, S., Blackwell, B., Valcarcel, D., Frassoni, F., Boelens, J. J., … Sanz, G. (2019). Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide. J Clin Oncol, 37(5), 367–374. https://doi.org/10.1200/JCO.18.00053
Horwitz, Mitchell E., Stephen Wease, Beth Blackwell, David Valcarcel, Francesco Frassoni, Jaap Jan Boelens, Stefan Nierkens, et al. “Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide.J Clin Oncol 37, no. 5 (February 10, 2019): 367–74. https://doi.org/10.1200/JCO.18.00053.
Horwitz ME, Wease S, Blackwell B, Valcarcel D, Frassoni F, Boelens JJ, et al. Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367–74.
Horwitz, Mitchell E., et al. “Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide.J Clin Oncol, vol. 37, no. 5, Feb. 2019, pp. 367–74. Pubmed, doi:10.1200/JCO.18.00053.
Horwitz ME, Wease S, Blackwell B, Valcarcel D, Frassoni F, Boelens JJ, Nierkens S, Jagasia M, Wagner JE, Kuball J, Koh LP, Majhail NS, Stiff PJ, Hanna R, Hwang WYK, Kurtzberg J, Cilloni D, Freedman LS, Montesinos P, Sanz G. Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367–374.

Published In

J Clin Oncol

DOI

EISSN

1527-7755

Publication Date

February 10, 2019

Volume

37

Issue

5

Start / End Page

367 / 374

Location

United States

Related Subject Headings

  • Young Adult
  • Transplantation Conditioning
  • Oncology & Carcinogenesis
  • Niacinamide
  • Neutrophils
  • Middle Aged
  • Male
  • Humans
  • Hematologic Neoplasms
  • Graft vs Host Disease