Umbilical Cord Blood Transplantation in Children with Acute Leukemia: Impact of Conditioning on Transplantation Outcomes

Mary Eapen; Joanne Kurtzberg; Mei Jie Zhang; Gareth Hattersely; Mingwei Fei; Adam Mendizabal; Ka Wah Chan; Satiro De Oliveira; Kirk R. Schultz; Donna Wall; Mary M. Horowitz; John E. Wagner

doi:10.1016/j.bbmt.2017.06.023

Umbilical Cord Blood Transplantation in Children with Acute Leukemia: Impact of Conditioning on Transplantation Outcomes

Mary Eapen, Joanne Kurtzberg, Mei Jie Zhang, Gareth Hattersely, Mingwei Fei, Adam Mendizabal, Ka Wah Chan, Satiro De Oliveira, Kirk R. Schultz, Donna Wall, Mary M. Horowitz, John E. Wagner

Pediatrics - Blood/Marrow Transplant

Research output: Contribution to journal › Article › peer-review

20 Scopus citations

Abstract

The Blood and Marrow Transplant Clinical Trials Network (BMT CTN 0501) randomized children with hematologic malignancies to transplantation with 1 or 2 cord blood units (UCB) between 2006 and 2012. While the trial concluded that survival was similar regardless of number of units infused, survival was better than previously reported. This prompted a comparison of survival of trial versus nontrial patients to determine the generalizability of trial results and whether survival was better because of the trial treatment regimen. During the trial period, 396 recipients of a single UCB unit met trial eligibility but were not enrolled. Trial patients (n = 100) received total body irradiation (TBI) 1320 cGy, cyclophosphamide 120 mg/kg, and fludarabine 75 mg/m² (TCF). Nontrial patients either received the same regimen (n = 62; nontrial TCF) or alternative regimens (n = 334; nontrial regimens). Five-year survival between trial and nontrial patients conditioned with TCF was similar (70% versus 62%). However, 5-year survival was significantly lower with nontrial TBI-containing (47%; hazard ratio [HR], 1.97; P =.001) and chemotherapy-only regimens (49%; HR, 1.87; P =.007). The results of BMT CTN 0501 appear generalizable to the population of trial-eligible patients. The survival difference between the trial-specified regimen and other regimens indicate the importance of conditioning regimen for UCB transplantation.

Original language	English (US)
Pages (from-to)	1714-1721
Number of pages	8
Journal	Biology of Blood and Marrow Transplantation
Volume	23
Issue number	10
DOIs	https://doi.org/10.1016/j.bbmt.2017.06.023
State	Published - Oct 2017

Bibliographical note

Funding Information:
Support for BMT CTN 0501 was provided by U10-HL069294 from the National Heart, Lung, and Blood Institute and National Cancer Institute to the Blood and Marrow Transplant Clinical Trials Network and U10CA098543 from the National Cancer Institute to the Children's Oncology Group ; the Center for International Blood and Marrow Transplant Research is supported primarily by U24-CA76518 from the National Cancer Institute , the National Heart, Lung, and Blood Institute, and the National Institute of Allergy and Infectious Diseases ; HHSH250201200016C (HRSA/DHHS) to the Center for International Blood and Marrow Transplant Research. The content is solely the responsibility of the authors and do not represent the official policy of the National Institutes of Health or the Health Resources and Services Administration or any other agency of the United States Government. Financial disclosure: The authors declare they have no relevant or material financial interests that relate to the research described in this paper. Conflict of interest statement: There are no conflicts of interest to report. Authorship statement: M.E., G.H., and M.J.Z. designed the study. G.H. and M.F. prepared the dataset and M.J.Z. analyzed the data. M.E. drafted the first manuscript. J.K., M.J.Z., A.M., G.H., M.F., K.W.C., S.D.O., K.R.S., D.W., M.M.H., and J.E.W. critically reviewed the manuscript. All authors approved the final manuscript.

Funding Information:
Support for BMT CTN 0501 was provided by U10-HL069294 from the National Heart, Lung, and Blood Institute and National Cancer Institute to the Blood and Marrow Transplant Clinical Trials Network and U10CA098543 from the National Cancer Institute to the Children's Oncology Group; the Center for International Blood and Marrow Transplant Research is supported primarily by U24-CA76518 from the National Cancer Institute, the National Heart, Lung, and Blood Institute, and the National Institute of Allergy and Infectious Diseases; HHSH250201200016C (HRSA/DHHS) to the Center for International Blood and Marrow Transplant Research. The content is solely the responsibility of the authors and do not represent the official policy of the National Institutes of Health or the Health Resources and Services Administration or any other agency of the United States Government.

Publisher Copyright:
© 2017 The American Society for Blood and Marrow Transplantation

Keywords

Acute leukemia
Conditioning regimen
Cord blood transplantation
Survival

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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Eapen, M., Kurtzberg, J., Zhang, M. J., Hattersely, G., Fei, M., Mendizabal, A., Chan, K. W., De Oliveira, S., Schultz, K. R., Wall, D., Horowitz, M. M., & Wagner, J. E. (2017). Umbilical Cord Blood Transplantation in Children with Acute Leukemia: Impact of Conditioning on Transplantation Outcomes. Biology of Blood and Marrow Transplantation, 23(10), 1714-1721. https://doi.org/10.1016/j.bbmt.2017.06.023

Eapen, M, Kurtzberg, J, Zhang, MJ, Hattersely, G, Fei, M, Mendizabal, A, Chan, KW, De Oliveira, S, Schultz, KR, Wall, D, Horowitz, MM & Wagner, JE 2017, 'Umbilical Cord Blood Transplantation in Children with Acute Leukemia: Impact of Conditioning on Transplantation Outcomes', Biology of Blood and Marrow Transplantation, vol. 23, no. 10, pp. 1714-1721. https://doi.org/10.1016/j.bbmt.2017.06.023

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abstract = "The Blood and Marrow Transplant Clinical Trials Network (BMT CTN 0501) randomized children with hematologic malignancies to transplantation with 1 or 2 cord blood units (UCB) between 2006 and 2012. While the trial concluded that survival was similar regardless of number of units infused, survival was better than previously reported. This prompted a comparison of survival of trial versus nontrial patients to determine the generalizability of trial results and whether survival was better because of the trial treatment regimen. During the trial period, 396 recipients of a single UCB unit met trial eligibility but were not enrolled. Trial patients (n = 100) received total body irradiation (TBI) 1320 cGy, cyclophosphamide 120 mg/kg, and fludarabine 75 mg/m2 (TCF). Nontrial patients either received the same regimen (n = 62; nontrial TCF) or alternative regimens (n = 334; nontrial regimens). Five-year survival between trial and nontrial patients conditioned with TCF was similar (70% versus 62%). However, 5-year survival was significantly lower with nontrial TBI-containing (47%; hazard ratio [HR], 1.97; P =.001) and chemotherapy-only regimens (49%; HR, 1.87; P =.007). The results of BMT CTN 0501 appear generalizable to the population of trial-eligible patients. The survival difference between the trial-specified regimen and other regimens indicate the importance of conditioning regimen for UCB transplantation.",

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N2 - The Blood and Marrow Transplant Clinical Trials Network (BMT CTN 0501) randomized children with hematologic malignancies to transplantation with 1 or 2 cord blood units (UCB) between 2006 and 2012. While the trial concluded that survival was similar regardless of number of units infused, survival was better than previously reported. This prompted a comparison of survival of trial versus nontrial patients to determine the generalizability of trial results and whether survival was better because of the trial treatment regimen. During the trial period, 396 recipients of a single UCB unit met trial eligibility but were not enrolled. Trial patients (n = 100) received total body irradiation (TBI) 1320 cGy, cyclophosphamide 120 mg/kg, and fludarabine 75 mg/m2 (TCF). Nontrial patients either received the same regimen (n = 62; nontrial TCF) or alternative regimens (n = 334; nontrial regimens). Five-year survival between trial and nontrial patients conditioned with TCF was similar (70% versus 62%). However, 5-year survival was significantly lower with nontrial TBI-containing (47%; hazard ratio [HR], 1.97; P =.001) and chemotherapy-only regimens (49%; HR, 1.87; P =.007). The results of BMT CTN 0501 appear generalizable to the population of trial-eligible patients. The survival difference between the trial-specified regimen and other regimens indicate the importance of conditioning regimen for UCB transplantation.

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Umbilical Cord Blood Transplantation in Children with Acute Leukemia: Impact of Conditioning on Transplantation Outcomes

Abstract

Bibliographical note

Keywords

UN SDGs

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