TY - JOUR
T1 - Measuring therapeutic response in chronic graft-versus-host disease
T2 - National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IV. Response criteria working group report
AU - Pavletic, Steven Z.
AU - Martin, Paul
AU - Lee, Stephanie J.
AU - Mitchell, Sandra
AU - Jacobsohn, David
AU - Cowen, Edward W.
AU - Turner, Maria L.
AU - Akpek, Gorgun
AU - Gilman, Andrew
AU - McDonald, George
AU - Schubert, Mark
AU - Berger, Ann
AU - Bross, Peter
AU - Chien, Jason W.
AU - Couriel, Daniel
AU - Dunn, J. P.
AU - Fall-Dickson, Jane
AU - Farrell, Ann
AU - Flowers, Mary E.D.
AU - Greinix, Hildegard
AU - Hirschfeld, Steven
AU - Gerber, Lynn
AU - Kim, Stella
AU - Knobler, Robert
AU - Lachenbruch, Peter A.
AU - Miller, Frederick W.
AU - Mittleman, Barbara
AU - Papadopoulos, Esperanza
AU - Parsons, Susan K.
AU - Przepiorka, Donna
AU - Robinson, Michael
AU - Ward, Michael
AU - Reeve, Bryce
AU - Rider, Lisa G.
AU - Shulman, Howard
AU - Schultz, Kirk R.
AU - Weisdorf, Daniel
AU - Vogelsang, Georgia B.
N1 - Funding Information:
This project was supported by the National Institutes of Health (NIH), National Cancer Institute, Office of the Director, Cancer Therapy Evaluation Program, Intramural Research Program, and Center for Cancer Research; National Heart Lung and Blood Institute, Division of Blood Diseases and Resources; Office of Rare Diseases, NIH, Office of the Director; National Institute of Allergy and Infectious Disease, Transplantation Immunology Branch; and the Health Resources and Services Administration, Division of Transplantation and the Naval Medical Research Center, C. W. Bill Young/Department of Defense Marrow Donor Recruitment and Research Program. The authors would also like to acknowledge the following individuals and organizations that by their participation made this project possible: American Society for Blood and Marrow Transplantation, Center for International Bone and Marrow Transplant Research, Blood and Marrow Transplant Clinical Trials Network, Canadian Blood and Marrow Transplant Group, European Group for Blood and Marrow Transplantation, Pediatric Blood and Marrow Transplant Consortium, and the representatives of the South American transplant centers (Drs Luis F. Bouzas and Vaneuza Funke). This project was conducted in coordination with the American Society for Clinical Oncology and American Society of Hematology (liaisons, Dr Michael Bishop and Mr Jeff Coughlin). The organizers are also indebted to patients and patient and research advocacy groups who made this process much more meaningful by their engagement. Special thanks also to Ms Paula Kim who coordinated these efforts.
PY - 2006/3
Y1 - 2006/3
N2 - The lack of standardized criteria for quantitative measurement of therapeutic response in clinical trials poses a major obstacle for the development of new agents in chronic graft-versus-host disease (GVHD). This consensus document was developed to address several objectives for response criteria to be used in chronic GVHD-related clinical trials. The proposed measures should be practical for use both by transplantation and nontransplantation medical providers, adaptable for use in adults and in children, and focused on the most important chronic GVHD manifestations. The measures should also give preference to quantitative, rather than semiquantitative, measures; capture information regarding signs, symptoms, and function separately from each other; and use validated scales whenever possible to demonstrate improved patient outcomes and meet requirements for regulatory approval of novel agents. Based on these criteria, we propose a set of measures to be considered for use in clinical trials, and forms for data collection are provided (http://www.asbmt.org/GvHDForms). Measures should be made at 3-month intervals and whenever major changes are made in treatment. Provisional definitions of complete response, partial response, and progression are proposed for each organ and for overall outcomes. The proposed response criteria are based on current expert consensus opinion and are intended to improve consistency in the conduct and reporting of chronic GVHD trials, but their use remains to be demonstrated in practice.
AB - The lack of standardized criteria for quantitative measurement of therapeutic response in clinical trials poses a major obstacle for the development of new agents in chronic graft-versus-host disease (GVHD). This consensus document was developed to address several objectives for response criteria to be used in chronic GVHD-related clinical trials. The proposed measures should be practical for use both by transplantation and nontransplantation medical providers, adaptable for use in adults and in children, and focused on the most important chronic GVHD manifestations. The measures should also give preference to quantitative, rather than semiquantitative, measures; capture information regarding signs, symptoms, and function separately from each other; and use validated scales whenever possible to demonstrate improved patient outcomes and meet requirements for regulatory approval of novel agents. Based on these criteria, we propose a set of measures to be considered for use in clinical trials, and forms for data collection are provided (http://www.asbmt.org/GvHDForms). Measures should be made at 3-month intervals and whenever major changes are made in treatment. Provisional definitions of complete response, partial response, and progression are proposed for each organ and for overall outcomes. The proposed response criteria are based on current expert consensus opinion and are intended to improve consistency in the conduct and reporting of chronic GVHD trials, but their use remains to be demonstrated in practice.
KW - Allogeneic cell transplantation
KW - Chronic graft-versus-host disease
KW - Consensus
KW - Response criteria
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UR - http://www.scopus.com/inward/citedby.url?scp=33244456998&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2006.01.008
DO - 10.1016/j.bbmt.2006.01.008
M3 - Review article
C2 - 16503494
AN - SCOPUS:33244456998
SN - 1083-8791
VL - 12
SP - 252
EP - 266
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
IS - 3
ER -