Blood and Marrow Transplant Clinical Trials Network Report on the Development of Novel Endpoints and Selection of Promising Approaches for Graft-versus-Host Disease Prevention Trials

Marcelo C. Pasquini; Brent Logan; Richard J. Jones; Amin M. Alousi; Frederick R. Appelbaum; Javier Bolaños-Meade; Mary E.D. Flowers; Sergio Giralt; Mary M. Horowitz; David Jacobsohn; John Koreth; John E. Levine; Leo Luznik; Richard Maziarz; Adam Mendizabal; Steven Pavletic; Miguel Angel Perales; David Porter; Ran Reshef; Daniel Weisdorf; Joseph H. Antin

doi:10.1016/j.bbmt.2018.01.002

Blood and Marrow Transplant Clinical Trials Network Report on the Development of Novel Endpoints and Selection of Promising Approaches for Graft-versus-Host Disease Prevention Trials

Marcelo C. Pasquini, Brent Logan, Richard J. Jones, Amin M. Alousi, Frederick R. Appelbaum, Javier Bolaños-Meade, Mary E.D. Flowers, Sergio Giralt, Mary M. Horowitz, David Jacobsohn, John Koreth, John E. Levine, Leo Luznik, Richard Maziarz, Adam Mendizabal, Steven Pavletic, Miguel Angel Perales, David Porter, Ran Reshef, Daniel WeisdorfJoseph H. Antin

School of Medicine

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

21 Scopus citations

Abstract

Graft-versus-host disease (GVHD) is a common complication after hematopoietic cell transplantation (HCT) and associated with significant morbidity and mortality. Preventing GVHD without chronic therapy or increasing relapse is a desired goal. Here we report a benchmark analysis to evaluate the performance of 6 GVHD prevention strategies tested at single institutions compared with a large multicenter outcomes database as a control. Each intervention was compared with the control for the incidence of acute and chronic GVHD and overall survival and against novel composite endpoints: acute and chronic GVHD, relapse-free survival (GRFS), and chronic GVHD, relapse-free survival (CRFS). Modeling GRFS and CRFS using the benchmark analysis further informed the design of 2 clinical trials testing GVHD prophylaxis interventions. This study demonstrates the potential benefit of using an outcomes database to select promising interventions for multicenter clinical trials and proposes novel composite endpoints for use in GVHD prevention trials.

Original language	English (US)
Pages (from-to)	1274-1280
Number of pages	7
Journal	Biology of Blood and Marrow Transplantation
Volume	24
Issue number	6
DOIs	https://doi.org/10.1016/j.bbmt.2018.01.002
State	Published - Jun 2018

Keywords

Clinical trials
GVHD
Hematopoietic cell transplantation

ASJC Scopus subject areas

Hematology
Transplantation

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10.1016/j.bbmt.2018.01.002

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Pasquini, M. C., Logan, B., Jones, R. J., Alousi, A. M., Appelbaum, F. R., Bolaños-Meade, J., Flowers, M. E. D., Giralt, S., Horowitz, M. M., Jacobsohn, D., Koreth, J., Levine, J. E., Luznik, L., Maziarz, R., Mendizabal, A., Pavletic, S., Perales, M. A., Porter, D., Reshef, R., ... Antin, J. H. (2018). Blood and Marrow Transplant Clinical Trials Network Report on the Development of Novel Endpoints and Selection of Promising Approaches for Graft-versus-Host Disease Prevention Trials. Biology of Blood and Marrow Transplantation, 24(6), 1274-1280. https://doi.org/10.1016/j.bbmt.2018.01.002

Blood and Marrow Transplant Clinical Trials Network Report on the Development of Novel Endpoints and Selection of Promising Approaches for Graft-versus-Host Disease Prevention Trials. / Pasquini, Marcelo C.; Logan, Brent; Jones, Richard J. et al.
In: Biology of Blood and Marrow Transplantation, Vol. 24, No. 6, 06.2018, p. 1274-1280.

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

Pasquini, MC, Logan, B, Jones, RJ, Alousi, AM, Appelbaum, FR, Bolaños-Meade, J, Flowers, MED, Giralt, S, Horowitz, MM, Jacobsohn, D, Koreth, J, Levine, JE, Luznik, L, Maziarz, R, Mendizabal, A, Pavletic, S, Perales, MA, Porter, D, Reshef, R, Weisdorf, D & Antin, JH 2018, 'Blood and Marrow Transplant Clinical Trials Network Report on the Development of Novel Endpoints and Selection of Promising Approaches for Graft-versus-Host Disease Prevention Trials', Biology of Blood and Marrow Transplantation, vol. 24, no. 6, pp. 1274-1280. https://doi.org/10.1016/j.bbmt.2018.01.002

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abstract = "Graft-versus-host disease (GVHD) is a common complication after hematopoietic cell transplantation (HCT) and associated with significant morbidity and mortality. Preventing GVHD without chronic therapy or increasing relapse is a desired goal. Here we report a benchmark analysis to evaluate the performance of 6 GVHD prevention strategies tested at single institutions compared with a large multicenter outcomes database as a control. Each intervention was compared with the control for the incidence of acute and chronic GVHD and overall survival and against novel composite endpoints: acute and chronic GVHD, relapse-free survival (GRFS), and chronic GVHD, relapse-free survival (CRFS). Modeling GRFS and CRFS using the benchmark analysis further informed the design of 2 clinical trials testing GVHD prophylaxis interventions. This study demonstrates the potential benefit of using an outcomes database to select promising interventions for multicenter clinical trials and proposes novel composite endpoints for use in GVHD prevention trials.",

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author = "Pasquini, {Marcelo C.} and Brent Logan and Jones, {Richard J.} and Alousi, {Amin M.} and Appelbaum, {Frederick R.} and Javier Bola{\~n}os-Meade and Flowers, {Mary E.D.} and Sergio Giralt and Horowitz, {Mary M.} and David Jacobsohn and John Koreth and Levine, {John E.} and Leo Luznik and Richard Maziarz and Adam Mendizabal and Steven Pavletic and Perales, {Miguel Angel} and David Porter and Ran Reshef and Daniel Weisdorf and Antin, {Joseph H.}",

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doi = "10.1016/j.bbmt.2018.01.002",

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AU - Koreth, John

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AU - Maziarz, Richard

AU - Mendizabal, Adam

AU - Pavletic, Steven

AU - Perales, Miguel Angel

AU - Porter, David

AU - Reshef, Ran

AU - Weisdorf, Daniel

AU - Antin, Joseph H.

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AB - Graft-versus-host disease (GVHD) is a common complication after hematopoietic cell transplantation (HCT) and associated with significant morbidity and mortality. Preventing GVHD without chronic therapy or increasing relapse is a desired goal. Here we report a benchmark analysis to evaluate the performance of 6 GVHD prevention strategies tested at single institutions compared with a large multicenter outcomes database as a control. Each intervention was compared with the control for the incidence of acute and chronic GVHD and overall survival and against novel composite endpoints: acute and chronic GVHD, relapse-free survival (GRFS), and chronic GVHD, relapse-free survival (CRFS). Modeling GRFS and CRFS using the benchmark analysis further informed the design of 2 clinical trials testing GVHD prophylaxis interventions. This study demonstrates the potential benefit of using an outcomes database to select promising interventions for multicenter clinical trials and proposes novel composite endpoints for use in GVHD prevention trials.

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Blood and Marrow Transplant Clinical Trials Network Report on the Development of Novel Endpoints and Selection of Promising Approaches for Graft-versus-Host Disease Prevention Trials

Abstract

Keywords

ASJC Scopus subject areas

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