Measuring therapeutic response in chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IV. Response criteria working group report

Steven Z. Pavletic; Paul Martin; Stephanie J. Lee; Sandra Mitchell; David Jacobsohn; Edward W. Cowen; Maria L. Turner; Gorgun Akpek; Andrew Gilman; George McDonald; Mark Schubert; Ann Berger; Peter Bross; Jason W. Chien; Daniel Couriel; J. P. Dunn; Jane Fall-Dickson; Ann Farrell; Mary E D Flowers; Hildegard Greinix; Steven Hirschfeld; Lynn Gerber; Stella Kim; Robert Knobler; Peter A. Lachenbruch; Frederick W. Miller; Barbara Mittleman; Esperanza Papadopoulos; Susan K. Parsons; Donna Przepiorka; Michael Robinson; Michael Ward; Bryce Reeve; Lisa G. Rider; Howard Shulman; Kirk R. Schultz; Daniel Weisdorf; Georgia B. Vogelsang

doi:10.1016/j.bbmt.2006.01.008

Measuring therapeutic response in chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IV. Response criteria working group report

Steven Z. Pavletic, Paul Martin, Stephanie J. Lee, Sandra Mitchell, David Jacobsohn, Edward W. Cowen, Maria L. Turner, Gorgun Akpek, Andrew Gilman, George McDonald, Mark Schubert, Ann Berger, Peter Bross, Jason W. Chien, Daniel Couriel, J. P. Dunn, Jane Fall-Dickson, Ann Farrell, Mary E D Flowers, Hildegard GreinixSteven Hirschfeld, Lynn Gerber, Stella Kim, Robert Knobler, Peter A. Lachenbruch, Frederick W. Miller, Barbara Mittleman, Esperanza Papadopoulos, Susan K. Parsons, Donna Przepiorka, Michael Robinson, Michael Ward, Bryce Reeve, Lisa G. Rider, Howard Shulman, Kirk R. Schultz, Daniel Weisdorf, Georgia B. Vogelsang

Medicine - Hematology, Oncology, Transplant

Research output: Contribution to journal › Review article › peer-review

367 Scopus citations

Abstract

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.

Original language	English (US)
Pages (from-to)	252-266
Number of pages	15
Journal	Biology of Blood and Marrow Transplantation
Volume	12
Issue number	3
DOIs	https://doi.org/10.1016/j.bbmt.2006.01.008
State	Published - Mar 2006

Bibliographical note

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.

Keywords

Allogeneic cell transplantation
Chronic graft-versus-host disease
Consensus
Response criteria

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Pavletic, S. Z., Martin, P., Lee, S. J., Mitchell, S., Jacobsohn, D., Cowen, E. W., Turner, M. L., Akpek, G., Gilman, A., McDonald, G., Schubert, M., Berger, A., Bross, P., Chien, J. W., Couriel, D., Dunn, J. P., Fall-Dickson, J., Farrell, A., Flowers, M. E. D., ... Vogelsang, G. B. (2006). Measuring therapeutic response in chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IV. Response criteria working group report. Biology of Blood and Marrow Transplantation, 12(3), 252-266. https://doi.org/10.1016/j.bbmt.2006.01.008

Measuring therapeutic response in chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IV. Response criteria working group report. / Pavletic, Steven Z.; Martin, Paul; Lee, Stephanie J. et al.
In: Biology of Blood and Marrow Transplantation, Vol. 12, No. 3, 03.2006, p. 252-266.

Research output: Contribution to journal › Review article › peer-review

Pavletic, SZ, Martin, P, Lee, SJ, Mitchell, S, Jacobsohn, D, Cowen, EW, Turner, ML, Akpek, G, Gilman, A, McDonald, G, Schubert, M, Berger, A, Bross, P, Chien, JW, Couriel, D, Dunn, JP, Fall-Dickson, J, Farrell, A, Flowers, MED, Greinix, H, Hirschfeld, S, Gerber, L, Kim, S, Knobler, R, Lachenbruch, PA, Miller, FW, Mittleman, B, Papadopoulos, E, Parsons, SK, Przepiorka, D, Robinson, M, Ward, M, Reeve, B, Rider, LG, Shulman, H, Schultz, KR, Weisdorf, D & Vogelsang, GB 2006, 'Measuring therapeutic response in chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IV. Response criteria working group report', Biology of Blood and Marrow Transplantation, vol. 12, no. 3, pp. 252-266. https://doi.org/10.1016/j.bbmt.2006.01.008

Pavletic SZ, Martin P, Lee SJ, Mitchell S, Jacobsohn D, Cowen EW et al. Measuring therapeutic response in chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IV. Response criteria working group report. Biology of Blood and Marrow Transplantation. 2006 Mar;12(3):252-266. doi: 10.1016/j.bbmt.2006.01.008

Pavletic, Steven Z. ; Martin, Paul ; Lee, Stephanie J. et al. / Measuring therapeutic response in chronic graft-versus-host disease : National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IV. Response criteria working group report. In: Biology of Blood and Marrow Transplantation. 2006 ; Vol. 12, No. 3. pp. 252-266.

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abstract = "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.",

keywords = "Allogeneic cell transplantation, Chronic graft-versus-host disease, Consensus, Response criteria",

author = "Pavletic, {Steven Z.} and Paul Martin and Lee, {Stephanie J.} and Sandra Mitchell and David Jacobsohn and Cowen, {Edward W.} and Turner, {Maria L.} and Gorgun Akpek and Andrew Gilman and George McDonald and Mark Schubert and Ann Berger and Peter Bross and Chien, {Jason W.} and Daniel Couriel and Dunn, {J. P.} and Jane Fall-Dickson and Ann Farrell and Flowers, {Mary E D} and Hildegard Greinix and Steven Hirschfeld and Lynn Gerber and Stella Kim and Robert Knobler and Lachenbruch, {Peter A.} and Miller, {Frederick W.} and Barbara Mittleman and Esperanza Papadopoulos and Parsons, {Susan K.} and Donna Przepiorka and Michael Robinson and Michael Ward and Bryce Reeve and Rider, {Lisa G.} and Howard Shulman and Schultz, {Kirk R.} and Daniel Weisdorf and Vogelsang, {Georgia B.}",

note = "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.",

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AU - Martin, Paul

AU - Lee, Stephanie J.

AU - Mitchell, Sandra

AU - Jacobsohn, David

AU - Cowen, Edward W.

AU - Turner, Maria L.

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AU - Bross, Peter

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AU - Couriel, Daniel

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AU - Fall-Dickson, Jane

AU - Farrell, Ann

AU - Flowers, Mary E D

AU - Greinix, Hildegard

AU - Hirschfeld, Steven

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AU - Ward, Michael

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Measuring therapeutic response in chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IV. Response criteria working group report

Abstract

Bibliographical note

Keywords

Access

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