Are We Making PROGRESS in Preventing Graft-versus-Host Disease and Improving Clinical Outcomes? Impact of BMT CTN 1301 Study Results on Clinical Practice

Betty K. Hamilton; Corey Cutler; Clint Divine; Mark Juckett; Charles LeMaistre; Susan Stewart; Jennifer Wilder; Mary Horowitz; Nandita Khera; Linda J. Burns

doi:10.1016/j.jtct.2022.05.002

Are We Making PROGRESS in Preventing Graft-versus-Host Disease and Improving Clinical Outcomes? Impact of BMT CTN 1301 Study Results on Clinical Practice

Betty K. Hamilton, Corey Cutler, Clint Divine, Mark Juckett, Charles LeMaistre, Susan Stewart, Jennifer Wilder, Mary Horowitz, Nandita Khera, Linda J. Burns

Research output: Contribution to journal › Comment/debate › peer-review

2 Scopus citations

Abstract

The need for prospective randomized clinical trials investigating novel graft-versus-host disease (GVHD) prevention strategies that include other clinical outcomes impacted by GVHD has been highlighted as a priority for the field of hematopoietic cell transplantation. A recently completed study through the Blood and Marrow Transplant Clinical Trials Network (BMT CTN 1301) comparing CD34⁺ selection and post-transplantation cyclophosphamide with tacrolimus/methotrexate (Tac/MTX) for GVHD prevention demonstrated no significant differences in the primary endpoint of chronic GVHD relapse-free survival among the 3 approaches. The trial did not demonstrate a superior approach compared with Tac/MTX; however, it did highlight several challenges in determining the best and most relevant approaches to clinical trial design, particularly in the context of current and ongoing changes in real-world practices. Here we review the results of BMT CTN 1301 and their implications for clinical practice and future clinical trial design.

Original language	English (US)
Pages (from-to)	419-425
Number of pages	7
Journal	Transplantation and Cellular Therapy
Volume	28
Issue number	8
DOIs	https://doi.org/10.1016/j.jtct.2022.05.002
State	Published - Aug 2022

Bibliographical note

Funding Information:
Financial disclosure: Support for this study was provided by Grants U10 HL069294 and U24 HL138660 to the BMT CTN from the National Heart, Lung, and Blood Institute and the National Cancer Institute, along with contributions by Miltenyi Biotec. The content is solely the responsibility of the authors and does not necessarily represent the official views of the aforementioned parties. The CIBMTR registry is supported primarily by Grant U24 CA76518 from the National Cancer Institute, the National Heart, Lung, and Blood Institute, and the National Institute of Allergy and Infectious Diseases and Grant HHSH234200637015C from the Health Resources and Services Administration to the Center for International Blood and Marrow Transplant Research.

Funding Information:
The authors thank Amy Foley for administrative support of the BMT CTN Task Force on Evidence into Practice. They also acknowledge the BMT CTN 1301 protocol team that developed this important study and published the seminal results in their original manuscript: Leo Luznik, MD, Marcelo C. Pasquini, MD, Brent Logan, PhD, Robert Soiffer, MD, Juan Wu, MS, Steven M. Devine, MD, Nancy Geller, PhD, Sergio Giralt, MD, Helen E. Heslop, MD, Mary M. Horowitz, MD, Richard J. Jones, MD, Mark R. Litzow MD, Adam Mendizabal, PhD, Lori Muffly, MD, Eneida R. Nemecek, MD, Lynn O'Donnell, MD, Richard J. O'Reilly, MD, Raquel Palencia, PharmD, Johannes Schetelig, MD, Leyla Shune, MD, Scott R. Solomon, MD, Sumithira Vasu, MBBS, Vincent T. Ho, MD, and Miguel-Angel Perales, MD. Financial disclosure: Support for this study was provided by Grants U10 HL069294 and U24 HL138660 to the BMT CTN from the National Heart, Lung, and Blood Institute and the National Cancer Institute, along with contributions by Miltenyi Biotec. The content is solely the responsibility of the authors and does not necessarily represent the official views of the aforementioned parties. The CIBMTR registry is supported primarily by Grant U24 CA76518 from the National Cancer Institute, the National Heart, Lung, and Blood Institute, and the National Institute of Allergy and Infectious Diseases and Grant HHSH234200637015C from the Health Resources and Services Administration to the Center for International Blood and Marrow Transplant Research. Conflict of interest statement: There are no conflicts of interest to disclose. Financial disclosure: See Acknowledgments on page XXX.

Publisher Copyright:
© 2022 The American Society for Transplantation and Cellular Therapy

Keywords

Allogeneic hematopoietic cell transplantation
CD34 selection
Graft-versus-host disease
Post-transplantation cyclophosphamide

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10.1016/j.jtct.2022.05.002

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Hamilton, B. K., Cutler, C., Divine, C., Juckett, M., LeMaistre, C., Stewart, S., Wilder, J., Horowitz, M., Khera, N., & Burns, L. J. (2022). Are We Making PROGRESS in Preventing Graft-versus-Host Disease and Improving Clinical Outcomes? Impact of BMT CTN 1301 Study Results on Clinical Practice. Transplantation and Cellular Therapy, 28(8), 419-425. https://doi.org/10.1016/j.jtct.2022.05.002

Hamilton, BK, Cutler, C, Divine, C, Juckett, M, LeMaistre, C, Stewart, S, Wilder, J, Horowitz, M, Khera, N & Burns, LJ 2022, 'Are We Making PROGRESS in Preventing Graft-versus-Host Disease and Improving Clinical Outcomes? Impact of BMT CTN 1301 Study Results on Clinical Practice', Transplantation and Cellular Therapy, vol. 28, no. 8, pp. 419-425. https://doi.org/10.1016/j.jtct.2022.05.002

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abstract = "The need for prospective randomized clinical trials investigating novel graft-versus-host disease (GVHD) prevention strategies that include other clinical outcomes impacted by GVHD has been highlighted as a priority for the field of hematopoietic cell transplantation. A recently completed study through the Blood and Marrow Transplant Clinical Trials Network (BMT CTN 1301) comparing CD34+ selection and post-transplantation cyclophosphamide with tacrolimus/methotrexate (Tac/MTX) for GVHD prevention demonstrated no significant differences in the primary endpoint of chronic GVHD relapse-free survival among the 3 approaches. The trial did not demonstrate a superior approach compared with Tac/MTX; however, it did highlight several challenges in determining the best and most relevant approaches to clinical trial design, particularly in the context of current and ongoing changes in real-world practices. Here we review the results of BMT CTN 1301 and their implications for clinical practice and future clinical trial design.",

keywords = "Allogeneic hematopoietic cell transplantation, CD34 selection, Graft-versus-host disease, Post-transplantation cyclophosphamide",

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note = "Funding Information: Financial disclosure: Support for this study was provided by Grants U10 HL069294 and U24 HL138660 to the BMT CTN from the National Heart, Lung, and Blood Institute and the National Cancer Institute, along with contributions by Miltenyi Biotec. The content is solely the responsibility of the authors and does not necessarily represent the official views of the aforementioned parties. The CIBMTR registry is supported primarily by Grant U24 CA76518 from the National Cancer Institute, the National Heart, Lung, and Blood Institute, and the National Institute of Allergy and Infectious Diseases and Grant HHSH234200637015C from the Health Resources and Services Administration to the Center for International Blood and Marrow Transplant Research. Funding Information: The authors thank Amy Foley for administrative support of the BMT CTN Task Force on Evidence into Practice. They also acknowledge the BMT CTN 1301 protocol team that developed this important study and published the seminal results in their original manuscript: Leo Luznik, MD, Marcelo C. Pasquini, MD, Brent Logan, PhD, Robert Soiffer, MD, Juan Wu, MS, Steven M. Devine, MD, Nancy Geller, PhD, Sergio Giralt, MD, Helen E. Heslop, MD, Mary M. Horowitz, MD, Richard J. Jones, MD, Mark R. Litzow MD, Adam Mendizabal, PhD, Lori Muffly, MD, Eneida R. Nemecek, MD, Lynn O'Donnell, MD, Richard J. O'Reilly, MD, Raquel Palencia, PharmD, Johannes Schetelig, MD, Leyla Shune, MD, Scott R. Solomon, MD, Sumithira Vasu, MBBS, Vincent T. Ho, MD, and Miguel-Angel Perales, MD. Financial disclosure: Support for this study was provided by Grants U10 HL069294 and U24 HL138660 to the BMT CTN from the National Heart, Lung, and Blood Institute and the National Cancer Institute, along with contributions by Miltenyi Biotec. The content is solely the responsibility of the authors and does not necessarily represent the official views of the aforementioned parties. The CIBMTR registry is supported primarily by Grant U24 CA76518 from the National Cancer Institute, the National Heart, Lung, and Blood Institute, and the National Institute of Allergy and Infectious Diseases and Grant HHSH234200637015C from the Health Resources and Services Administration to the Center for International Blood and Marrow Transplant Research. Conflict of interest statement: There are no conflicts of interest to disclose. Financial disclosure: See Acknowledgments on page XXX. Publisher Copyright: {\textcopyright} 2022 The American Society for Transplantation and Cellular Therapy",

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Are We Making PROGRESS in Preventing Graft-versus-Host Disease and Improving Clinical Outcomes? Impact of BMT CTN 1301 Study Results on Clinical Practice

Abstract

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