National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: III. The 2020 Treatment of Chronic GVHD Report

被引：41

作者：

DeFilipp, Zachariah ^{[1
]}

Couriel, Daniel R. ^{[2
]}

Lazaryan, Aleksandr ^{[3
]}

Bhatt, Vijaya Raj ^{[4
]}

Buxbaum, Nataliya P. ^{[5
]}

Alousi, Amin M. ^{[6
]}

Olivieri, Attilio ^{[7
]}

Pulanic, Drazen ^{[8
]}

Halter, Joerg P. ^{[9
]}

Henderson, Lori A. ^{[10
]}

Zeiser, Robert ^{[11
]}

Gooley, Ted A. ^{[12
]}

MacDonald, Kelli P. A. ^{[13
]}

Wolff, Daniel ^{[14
]}

Schultz, Kirk R. ^{[15
]}

Paczesny, Sophie ^{[16
,17
]}

Inamoto, Yoshihiro ^{[18
]}

Cutler, Corey S. ^{[19
]}

Kitko, Carrie L. ^{[20
]}

Pidala, Joseph A. ^{[3
]}

Lee, Stephanie J. ^{[12
,21
]}

Socie, Gerard ^{[22
,23
]}

Sarantopoulos, Stefanie ^{[24
]}

Pavletic, Steven Z. ^{[25
]}

Martin, Paul J. ^{[12
,21
]}

Blazar, Bruce R. ^{[26
]}

Greinix, Hildegard T. ^{[27
]}

机构：

[1] Massachusetts Gen Hosp, Hematopoiet Cell Transplant & Cellular Therapy Pr, Boston, MA 02114 USA

[2] Univ Utah, Huntsman Canc Ctr, Salt Lake City, UT USA

[3] H Lee Moffitt Canc Ctr & Res Inst, Blood & Marrow Transplantat & Cellular Immunother, Tampa, FL USA

[4] Univ Nebraska Med Ctr, Fred & Pamela Buffett Canc Ctr, Omaha, NE USA

[5] Roswell Park Comprehens Canc Ctr, Dept Pediat, Buffalo, NY USA

[6] Univ Texas MD Anderson Canc Ctr, Dept Stem Cell Transplantat & Cellular Therapy, Houston, TX 77030 USA

[7] Univ Politecn Marche, Hematol & Stem Cell Transplant, Ancona, Italy

[8] Univ Zagreb, Univ Hosp Ctr Zagreb, Med Sch, Dept Internal Med,Div Hematol, Zagreb, Croatia

[9] Univ Hosp Basel, Dept Med, Div Hematol, Basel, Switzerland

[10] NCI, Clin Invest Branch, Canc Therapy Evaluat Program, Div Canc Treatment & Diag,NIH, Rockville, MD USA

[11] Univ Freiburg, Univ Clin Freiburg, Fac Med, Dept Internal Med 1,Med Ctr,Div Hematol Oncol & S, Freiburg, Germany

[12] Fred Hutchinson Canc Res Ctr, Clin Res Div, 1124 Columbia St, Seattle, WA 98104 USA

[13] Queensland Inst Med Res, Berghofer Med Res Inst, Dept Immunol, Herston, Qld, Australia

[14] Univ Hosp Regensburg, Dept Internal Med 3, Regensburg, Germany

[15] BC Childrens Hosp, Pediat Hematol Oncol BMT, Vancouver, BC, Canada

[16] Med Univ South Carolina, Dept Microbiol & Immunol, Charleston, SC 29425 USA

[17] Med Univ South Carolina, Dept Pediat, Charleston, SC 29425 USA

[18] Natl Canc Ctr, Dept Hematopoiet Stem Cell Transplantat, Tokyo, Japan

[19] Dana Farber Canc Inst, Div Hematol Malignancies, Boston, MA 02115 USA

[20] Vanderbilt Univ, Med Ctr, Pediat Blood & Marrow Transplantat Program, Nashville, TN USA

[21] Univ Washington, Dept Med, Seattle, WA USA

[22] St Louis Hosp, AP HP, Hematol Transplantat, Paris, France

[23] Univ Paris, Paris, France

[24] Duke Univ, Duke Canc Inst, Dept Med, Div Hematol Malignancies & Cellular Therapy, Durham, NC USA

[25] NCI, Ctr Canc Res, NIH, Bethesda, MD 20892 USA

[26] Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat & Cellular Therap, Minneapolis, MN USA

[27] Med Univ Graz, Clin Div Hematol, Graz, Austria

来源：

TRANSPLANTATION AND CELLULAR THERAPY | 2021年 / 27卷 / 09期

关键词：

Chronic graft-versus-host disease; Allogeneic hematopoietic cell transplantation; Consensus Initial therapy; Treatment refractory; FAILURE-FREE SURVIVAL; SYSTEMIC TREATMENT; RANDOMIZED-TRIAL; THERAPY; PREDNISONE; DIAGNOSIS; CYCLOSPORINE; THALIDOMIDE; MANAGEMENT; IBRUTINIB;

D O I：

10.1016/j.jtct.2021.05.004

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Positive results from recent clinical trials have significantly expanded current therapeutic options for patients with chronic graft-versus-host disease (GVHD). However, new insights into the associations between clinical characteristics of chronic GVHD, pathophysiologic mechanisms of disease, and the clinical and biological effects of novel therapeutic agents are required to allow for a more individualized approach to treatment. The current report is focused on setting research priorities and direction in the treatment of chronic GVHD. Detailed correlative scientific studies should be conducted in the context of clinical trials to evaluate associations between clinical outcomes and the biological effect of systemic therapeutics. For patients who require systemic therapy but not urgent initiation of glucocorticoids, clinical trials for initial systemic treatment of chronic GVHD should investigate novel agents as monotherapy without concurrently starting glucocorticoids, to avoid confounding biological, pathological, and clinical assessments. Clinical trials for treatment-refractory disease should specifically target patients with incomplete or suboptimal responses to most recent therapy who are early in their disease course. Close collaboration between academic medical centers, medical societies, and industry is needed to support an individualized, biology-based strategic approach to chronic GVHD therapy. (C) 2021 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.

引用

页码：729 / 737

页数：9

共 45 条

[1] Adaptive platform trials: definition, design, conduct and reporting considerations The Adaptive Platform Trials Coalition [J].

Angus, Derek C. ;

Alexander, Brian M. ;

Berry, Scott ;

Buxton, Meredith ;

Lewis, Roger ;

Paoloni, Melissa ;

Webb, Steven A. R. ;

Arnold, Steven ;

Barker, Anna ;

Berry, Donald A. ;

Bonten, Marc J. M. ;

Brophy, Mary ;

Butler, Christopher ;

Cloughesy, Timothy F. ;

Derde, Lennie P. G. ;

Esserman, Laura J. ;

Ferguson, Ryan ;

Fiore, Louis ;

Gaffey, Sarah C. ;

Gaziano, J. Michael ;

Giusti, Kathy ;

Goossens, Herman ;

Heritier, Stephane ;

Hyman, Bradley ;

Krams, Michael ;

Larholt, Kay ;

LaVange, Lisa M. ;

Lavori, Philip ;

Lo, Andrew W. ;

London, Alex John ;

Manax, Victoria ;

McArthur, Colin ;

O'Neill, Genevieve ;

Parmigiani, Giovanni ;

Perlmutter, Jane ;

Petzold, Elizabeth A. ;

Ritchie, Craig ;

Rowan, Kathryn M. ;

Seymour, Christopher W. ;

Shapiro, Nathan, I ;

Simeone, Diane M. ;

Smith, Bradley ;

Spellberg, Bradley ;

Stern, Ariel Dora ;

Trippa, Lorenzo ;

Trusheim, Mark ;

Viele, Kert ;

Wen, Patrick Y. ;

Woodcock, Janet .

NATURE REVIEWS DRUG DISCOVERY, 2019, 18 (10) :797-807

[2] Randomized clinical trial of thalidomide, cyclosporine, and prednisone versus cyclosporine and prednisone as initial therapy for chronic graft-versus-host disease [J].

Arora, M ;

Wagner, JE ;

Davies, SM ;

Blazar, BR ;

Defor, T ;

Enright, H ;

Miller, WF ;

Weisdorf, DJ .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2001, 7 (05) :265-273

[3] A phase II/III randomized, multicenter trial of prednisone/sirolimus versus prednisone/sirolimus/calcineurin inhibitor for the treatment of chronic graft-versus-host disease: BMT CTN 0801 [J].

Carpenter, Paul A. ;

Logan, Brent R. ;

Lee, Stephanie J. ;

Weisdorf, Daniel J. ;

Johnston, Laura ;

Costa, Luciano J. ;

Kitko, Carrie L. ;

Bolanos-Meade, Javier ;

Sarantopoulos, Stefanie ;

Alousi, Amin M. ;

Abhyankar, Sunil ;

Waller, Edmund K. ;

Mendizabal, Adam ;

Zhu, Jiaxi ;

O'Brien, Kelly A. ;

Lazaryan, Aleksandr ;

Wu, Juan ;

Nemecek, Eneida R. ;

Pavletic, Steven Z. ;

Cutler, Corey S. ;

Horowitz, Mary M. ;

Arora, Mukta .

HAEMATOLOGICA, 2018, 103 (11) :1915-1924

[4] The Biology of Chronic Graft-versus-Host Disease: A Task Force Report from the National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease [J].

Cooke, Kenneth R. ;

Luznik, Leo ;

Sarantopoulos, Stefanie ;

Hakim, Frances T. ;

Jagasia, Madan ;

Fowler, Daniel H. ;

van den Brink, Marcel R. M. ;

Hansen, John A. ;

Parkman, Robertson ;

Miklos, David B. ;

Martin, Paul J. ;

Paczesny, Sophie ;

Vogelsang, Georgia ;

Pavletic, Steven ;

Ritz, Jerome ;

Schultz, Kirk R. ;

Blazar, Bruce R. .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2017, 23 (02) :211-234

[5] A randomized phase 2 trial of pomalidomide in subjects failing prior therapy for chronic graft-versus-host disease [J].

Curtis, Lauren M. ;

Ostojic, Alen ;

Venzon, David J. ;

Holtzman, Noa G. ;

Pirsl, Filip ;

Kuzmina, Zoya J. ;

Baird, Kristin ;

Rose, Jeremy J. ;

Cowen, Edward W. ;

Mays, Jacqueline W. ;

Mitchell, Sandra A. ;

Parsons-Wandell, Laura ;

Joe, Galen O. ;

Comis, Leora E. ;

Berger, Ann ;

Pusic, Iskra ;

Peer, Cody J. ;

Figg, William D. ;

Cao, Liang ;

Gale, Robert Peter ;

Hakim, Frances T. ;

Pavletic, Steven Z. .

BLOOD, 2021, 137 (07) :896-907

[6] Belumosudil for Chronic Graft-Versus-Host Disease (cGVHD) after 2 or More Prior Lines of Therapy: The Rockstar Study (KD025-213) [J].

Cutler, Corey ;

Lee, Stephanie J. ;

Arai, Sally ;

Rotta, Marcello ;

Zoghi, Behyar ;

Ramakrishnan, Aravind ;

Eiznhamer, David ;

Schueller, Olivier ;

Yang, Zhongming ;

Green, Laurie S. ;

Aggarwal, Sanjay K. ;

Blazar, Bruce R. ;

Pavletic, Steven Z. ;

Jagasia, Madan .

BLOOD, 2020, 136

[7] Mechanistic approaches for the prevention and treatment of chronic GVHD [J].

Cutler, Corey S. ;

Koreth, John ;

Ritz, Jerome .

BLOOD, 2017, 129 (01) :22-29

[8] Benefits and challenges with diagnosing chronic and late acute GVHD in children using the NIH consensus criteria [J].

Cuvelier, Geoffrey D. E. ;

Nemecek, Eneida R. ;

Wahlstrom, Justin T. ;

Kitko, Carrie L. ;

Lewis, Victor A. ;

Schechter, Tal ;

Jacobsohn, David A. ;

Harris, Andrew C. ;

Pulsipher, Michael A. ;

Bittencourt, Henrique ;

Choi, Sung Won ;

Caywood, Emi H. ;

Kasow, Kimberly A. ;

Bhatia, Monica ;

Oshrine, Benjamin R. ;

Flower, Allyson ;

Chaudhury, Sonali ;

Coulter, Donald ;

Chewning, Joseph H. ;

Joyce, Michael ;

Savasan, Sureyya ;

Pawlowska, Anna B. ;

Megason, Gail C. ;

Mitchell, David ;

Cheerva, Alexandra C. ;

Lawitschka, Anita ;

West, Lori J. ;

Pan, Bo ;

Al Hamarneh, Yazid N. ;

Halevy, Anat ;

Schultz, Kirk R. .

BLOOD, 2019, 134 (03) :304-316

[9] Diagnosis and management of chronic graft-versus-host disease [J].

Dignan, Fiona L. ;

Amrolia, Persis ;

Clark, Andrew ;

Cornish, Jacqueline ;

Jackson, Graham ;

Mahendra, Prem ;

Scarisbrick, Julia J. ;

Taylor, Peter C. ;

Shaw, Bronwen E. ;

Potter, Michael N. .

BRITISH JOURNAL OF HAEMATOLOGY, 2012, 158 (01) :46-61

[10] National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report [J].

Filipovich, AH ;

Weisdorf, D ;

Pavletic, S ;

Socie, G ;

Wingard, JR ;

Lee, SJ ;

Martin, P ;

Chien, J ;

Przepiorka, D ;

Couriel, D ;

Cowen, EW ;

Dinndorf, P ;

Farrell, A ;

Hartzman, R ;

Henslee-Downey, J ;

Jacobsohn, D ;

McDonald, G ;

Mittleman, B ;

Rizzo, JD ;

Robinson, M ;

Schubert, M ;

Schultz, K ;

Shulman, H ;

Turner, M ;

Vogelsang, G ;

Flowers, MED .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2005, 11 (12) :945-956

← 1 2 3 4 5 →