TY - JOUR
T1 - National institutes of health consensus development project on criteria for clinical trials in chronic graft-versus-host disease
T2 - III: The 2014 biomarker working group report
AU - Paczesny, Sophie
AU - Hakim, Frances T.
AU - Pidala, Joseph
AU - Cooke, Kenneth R.
AU - Lathrop, Julia
AU - Griffith, Linda M.
AU - Hansen, John
AU - Jagasia, Madan
AU - Miklos, David
AU - Pavletic, Steven
AU - Parkman, Robertson
AU - Russek-Cohen, Estelle
AU - Flowers, Mary E.D.
AU - Lee, Stephanie
AU - Martin, Paul
AU - Vogelsang, Georgia
AU - Walton, Marc
AU - Schultz, Kirk R.
N1 - Funding Information:
This project was supported by the National Institutes of Health's (NIH's) National Cancer Institute , Center for Cancer Research, Intramural Research Program and Division of Cancer Treatment and Diagnosis, Cancer Therapy Evaluation Program; Office of Rare Disease Research, National Center for Advancing Translational Sciences ; Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases ; National Heart, Lung, and Blood Institute , Division of Blood Diseases and Resources. The authors 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, US Chronic GVHD Consortium (supported by Office of Rare Diseases Research/National Center for Advancing Translational Sciences and National Cancer Institute), German-Austrian-Swiss chronic GVHD Consortium, National Marrow Donor Program, the Health Resources and Services Administration, Division of Transplantation, US Department of Human Health and Services, Canadian Blood and Marrow Transplant Group, European Group for Blood and Marrow Transplantation, Pediatric Blood and Marrow Transplant Consortium, and the representatives of the Brazilian Chronic GVHD consortium (Drs. Maria Claudia Moreira, Márcia de Matos Silva and Vaneuza Funke) and Deutsche José Carreras Leukämie-Stiftung. The authors thank Dr. Joseph Antin (Dana Farber Cancer Center, Boston, MA), Dr. Hildegard Greinix (Medical University of Vienna, Vienna, Austria), and Dr. Gerard Socie (University Paris VII & AP-HP, Hospital Saint Louis, Paris, France) for their critical review of the manuscript. The organizers are in debt to patients and patient and research advocacy groups, who made this process much more meaningful by their engagement. Acknowledgement goes to the Meredith Cowden GVHD foundation for facilitating the initial planning meeting in Cleveland in November of 2013 in conjunction with the National GVHD Symposium. The project group also recognizes the contributions of numerous colleagues in the field of blood and marrow transplantation in the United States and internationally, medical specialists and consultants, the pharmaceutical industry, and the NIH and US Food and Drug Administration professional staff for their intellectual input, dedication, and enthusiasm on the road to completion of these documents. For their expert contributions to this 2014 NIH Biomarkers Consensus Working Group document, special acknowledgements to Ms. Licia Masuch, Ms. Kristie Bradley, and Ms. Linda Henson for assistance in the manuscript preparation. The opinions expressed are those of the authors and do not represent the position of the National Cancer Institute; the National Heart, Lung and Blood Institute; the National Institute of Allergy and Infectious Diseases; the National Institutes of Health; the Food and Drug Administration; or the United States government.
Publisher Copyright:
© 2015 American Society for Blood and Marrow Transplantation.
Copyright:
Copyright 2015 Elsevier B.V., All rights reserved.
PY - 2015/5/1
Y1 - 2015/5/1
N2 - Biology-based markers to confirm or aid in the diagnosis or prognosis of chronic graft-versus-host disease (GVHD) after allogeneic hematopoietic cell transplantation or monitor its progression are critically needed to facilitate evaluation of new therapies. Biomarkers have been defined as any characteristic that is objectively measured and evaluated as an indicator of a normal biological or pathogenic process, or of a pharmacologic response to a therapeutic intervention. Applications of biomarkers in chronic GVHD clinical trials or patient management include the following: (1) diagnosis and assessment of chronic GVHD disease activity, including distinguishing irreversible damage from continued disease activity; (2) prognostic risk to develop chronic GVHD; and (3) prediction of response to therapy. Sample collection for chronic GVHD biomarkers studies should be well documented following established quality control guidelines for sample acquisition, processing, preservation, and testing, at intervals that are both calendar and event driven. The consistent therapeutic treatment of subjects and standardized documentation needed to support biomarker studies are most likely to be provided in prospective clinical trials. To date, no chronic GVHD biomarkers have been qualified for use in clinical applications. Since our previous chronic GVHD Biomarkers Working Group report in 2005, an increasing number of chronic GVHD candidate biomarkers are available for further investigation. This paper provides a 4-part framework for biomarker investigations: identification, verification, qualification, and application with terminology based on Food and Drug Administration and European Medicines Agency guidelines.
AB - Biology-based markers to confirm or aid in the diagnosis or prognosis of chronic graft-versus-host disease (GVHD) after allogeneic hematopoietic cell transplantation or monitor its progression are critically needed to facilitate evaluation of new therapies. Biomarkers have been defined as any characteristic that is objectively measured and evaluated as an indicator of a normal biological or pathogenic process, or of a pharmacologic response to a therapeutic intervention. Applications of biomarkers in chronic GVHD clinical trials or patient management include the following: (1) diagnosis and assessment of chronic GVHD disease activity, including distinguishing irreversible damage from continued disease activity; (2) prognostic risk to develop chronic GVHD; and (3) prediction of response to therapy. Sample collection for chronic GVHD biomarkers studies should be well documented following established quality control guidelines for sample acquisition, processing, preservation, and testing, at intervals that are both calendar and event driven. The consistent therapeutic treatment of subjects and standardized documentation needed to support biomarker studies are most likely to be provided in prospective clinical trials. To date, no chronic GVHD biomarkers have been qualified for use in clinical applications. Since our previous chronic GVHD Biomarkers Working Group report in 2005, an increasing number of chronic GVHD candidate biomarkers are available for further investigation. This paper provides a 4-part framework for biomarker investigations: identification, verification, qualification, and application with terminology based on Food and Drug Administration and European Medicines Agency guidelines.
KW - Biomarkers
KW - Chronic graft-versus-host disease
KW - Consensus
KW - National institutes of health
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UR - http://www.scopus.com/inward/citedby.url?scp=84928103474&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2015.01.003
DO - 10.1016/j.bbmt.2015.01.003
M3 - Article
C2 - 25644957
AN - SCOPUS:84928103474
VL - 21
SP - 780
EP - 792
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
SN - 1083-8791
IS - 5
ER -