TY - JOUR
T1 - National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease
T2 - IIb. The 2020 Preemptive Therapy Working Group Report
AU - Pidala, Joseph
AU - Kitko, Carrie
AU - Lee, Stephanie J.
AU - Carpenter, Paul
AU - Cuvelier, Geoffrey D.E.
AU - Holtan, Shernan
AU - Flowers, Mary E.
AU - Cutler, Corey
AU - Jagasia, Madan
AU - Gooley, Ted
AU - Palmer, Joycelynne
AU - Randolph, Tim
AU - Levine, John E.
AU - Ayuk, Francis
AU - Dignan, Fiona
AU - Schoemans, Helene
AU - Tkaczyk, Eric
AU - Farhadfar, Nosha
AU - Lawitschka, Anita
AU - Schultz, Kirk R.
AU - Martin, Paul J.
AU - Sarantopoulos, Stefanie
AU - Inamoto, Yoshihiro
AU - Socie, Gerard
AU - Wolff, Daniel
AU - Blazar, Bruce
AU - Greinix, Hildegard
AU - Paczesny, Sophie
AU - Pavletic, Steven
AU - Hill, Geoffrey
N1 - Publisher Copyright:
© 2021
PY - 2021/8
Y1 - 2021/8
N2 - Chronic graft-versus-host disease (GVHD) commonly occurs after allogeneic hematopoietic cell transplantation (HCT) despite standard prophylactic immune suppression. Intensified universal prophylaxis approaches are effective but risk possible overtreatment and may interfere with the graft-versus-malignancy immune response. Here we summarize conceptual and practical considerations regarding preemptive therapy of chronic GVHD, namely interventions applied after HCT based on evidence that the risk of developing chronic GVHD is higher than previously appreciated. This risk may be anticipated by clinical factors or risk assignment biomarkers or may be indicated by early signs and symptoms of chronic GVHD that do not fully meet National Institutes of Health diagnostic criteria. However, truly preemptive, individualized, and targeted chronic GVHD therapies currently do not exist. In this report, we (1) review current knowledge regarding clinical risk factors for chronic GVHD, (2) review what is known about chronic GVHD risk assignment biomarkers, (3) examine how chronic GVHD pathogenesis intersects with available targeted therapeutic agents, and (4) summarize considerations for preemptive therapy for chronic GVHD, emphasizing trial development, including trial design and statistical considerations. We conclude that robust risk assignment models that accurately predict chronic GVHD after HCT and early-phase preemptive therapy trials represent the most urgent priorities for advancing this novel area of research.
AB - Chronic graft-versus-host disease (GVHD) commonly occurs after allogeneic hematopoietic cell transplantation (HCT) despite standard prophylactic immune suppression. Intensified universal prophylaxis approaches are effective but risk possible overtreatment and may interfere with the graft-versus-malignancy immune response. Here we summarize conceptual and practical considerations regarding preemptive therapy of chronic GVHD, namely interventions applied after HCT based on evidence that the risk of developing chronic GVHD is higher than previously appreciated. This risk may be anticipated by clinical factors or risk assignment biomarkers or may be indicated by early signs and symptoms of chronic GVHD that do not fully meet National Institutes of Health diagnostic criteria. However, truly preemptive, individualized, and targeted chronic GVHD therapies currently do not exist. In this report, we (1) review current knowledge regarding clinical risk factors for chronic GVHD, (2) review what is known about chronic GVHD risk assignment biomarkers, (3) examine how chronic GVHD pathogenesis intersects with available targeted therapeutic agents, and (4) summarize considerations for preemptive therapy for chronic GVHD, emphasizing trial development, including trial design and statistical considerations. We conclude that robust risk assignment models that accurately predict chronic GVHD after HCT and early-phase preemptive therapy trials represent the most urgent priorities for advancing this novel area of research.
KW - Allogeneic hematopoietic cell transplantation
KW - Chronic graft-versus-host disease
KW - Consensus
KW - Preemptive therapy
KW - Risk assignment biomarkers
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UR - http://www.scopus.com/inward/citedby.url?scp=85107954199&partnerID=8YFLogxK
U2 - 10.1016/j.jtct.2021.03.029
DO - 10.1016/j.jtct.2021.03.029
M3 - Article
C2 - 33836313
AN - SCOPUS:85107954199
SN - 2666-6367
VL - 27
SP - 632
EP - 641
JO - Transplantation and Cellular Therapy
JF - Transplantation and Cellular Therapy
IS - 8
ER -