title: National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: III. The 2014 Biomarker Working Group Report.
abstract: 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.
authors: Department of Pediatrics and Immunology, Indiana University of Medicine, Indianapolis, Indiana. Electronic address: sophpacz@iu.edu.
authors: Sophie
authors: S
authors: Paczesny
authors: Experimental Transplantation and Immunology Branch, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
authors: Frances T
authors: FT
authors: Hakim
authors: Blood and Marrow Transplantation, Moffitt Cancer Center, Tampa, Florida.
authors: Joseph
authors: J
authors: Pidala
authors: Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, Maryland.
authors: Kenneth R
authors: KR
authors: Cooke
authors: Office of In Vitro Diagnostics and Radiological Health, Center for Devices and Radiological Health, Food and Drug Administration, Silver Spring, Maryland.
authors: Julia
authors: J
authors: Lathrop
authors: Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.
authors: Linda M
authors: LM
authors: Griffith
authors: Clinical Research Division, Fred Hutchinson Cancer Research Center, and the Division of Medical Oncology, University of Washington School of Medicine, Seattle, Washington.
authors: John
authors: J
authors: Hansen
authors: Department of Medicine, Division of Hematology-Oncology, Vanderbilt University Medical Center, Nashville, Tennessee.
authors: Madan
authors: M
authors: Jagasia
authors: Stanford Bone Marrow Transplant-Cellular Therapy Facility, Stanford University, Stanford, California.
authors: David
authors: D
authors: Miklos
authors: Experimental Transplantation and Immunology Branch, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
authors: Steven
authors: S
authors: Pavletic
authors: Department of Pediatrics, Children's Hospital of Los Angeles, University of Southern California, Los Angeles, California.
authors: Robertson
authors: R
authors: Parkman
authors: Division of Biostatistics, Center for Biologics, Food and Drug Administration, Silver Spring, Maryland.
authors: Estelle
authors: E
authors: Russek-Cohen
authors: Clinical Research Division, Fred Hutchinson Cancer Research Center, and the Division of Medical Oncology, University of Washington School of Medicine, Seattle, Washington.
authors: Mary E D
authors: ME
authors: Flowers
authors: Clinical Research Division, Fred Hutchinson Cancer Research Center, and the Division of Medical Oncology, University of Washington School of Medicine, Seattle, Washington.
authors: Stephanie
authors: S
authors: Lee
authors: Clinical Research Division, Fred Hutchinson Cancer Research Center, and the Division of Medical Oncology, University of Washington School of Medicine, Seattle, Washington.
authors: Paul
authors: P
authors: Martin
authors: Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, Maryland.
authors: Georgia
authors: G
authors: Vogelsang
authors: Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland.
authors: Marc
authors: M
authors: Walton
authors: Michael Cuccione Childhood Cancer Research Program, BC Children's Hospital and University of British Columbia, Vancouver, British Columbia, Canada.
authors: Kirk R
authors: KR
authors: Schultz
mesh_terms: D015415:Biomarkers / Q000378:metabolism*; D002986:Clinical Trials as Topic; D006086:Graft vs Host Disease / Q000175:diagnosis* / Q000378:metabolism* / Q000628:therapy*; D006801:Humans; D011379:Prognosis; D009626:Terminology as Topic; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D015415:Biomarkers
keywords: Biomarkers; Chronic graft-versus-host disease; Consensus; National Institutes of Health
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