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

Sophie Paczesny¹, Frances T Hakim², Joseph Pidala³, Kenneth R Cooke⁴, Julia Lathrop⁵, Linda M Griffith⁶, John Hansen⁷, Madan Jagasia⁸, David Miklos⁹, Steven Pavletic², Robertson Parkman¹⁰, Estelle Russek-Cohen¹¹, Mary E D Flowers⁷, Stephanie Lee⁷, Paul Martin⁷, Georgia Vogelsang⁴, Marc Walton¹², Kirk R Schultz¹³

Affiliations

¹ Department of Pediatrics and Immunology, Indiana University of Medicine, Indianapolis, Indiana. Electronic address: sophpacz@iu.edu.
² Experimental Transplantation and Immunology Branch, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
³ Blood and Marrow Transplantation, Moffitt Cancer Center, Tampa, Florida.
⁴ Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, Maryland.
⁵ Office of In Vitro Diagnostics and Radiological Health, Center for Devices and Radiological Health, Food and Drug Administration, Silver Spring, Maryland.
⁶ Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.
⁷ Clinical Research Division, Fred Hutchinson Cancer Research Center, and the Division of Medical Oncology, University of Washington School of Medicine, Seattle, Washington.
⁸ Department of Medicine, Division of Hematology-Oncology, Vanderbilt University Medical Center, Nashville, Tennessee.
⁹ Stanford Bone Marrow Transplant-Cellular Therapy Facility, Stanford University, Stanford, California.
¹⁰ Department of Pediatrics, Children's Hospital of Los Angeles, University of Southern California, Los Angeles, California.
¹¹ Division of Biostatistics, Center for Biologics, Food and Drug Administration, Silver Spring, Maryland.
¹² Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland.
¹³ Michael Cuccione Childhood Cancer Research Program, BC Children's Hospital and University of British Columbia, Vancouver, British Columbia, Canada.

PMID: 25644957
PMCID: PMC4408233
DOI: 10.1016/j.bbmt.2015.01.003

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

Sophie Paczesny et al. Biol Blood Marrow Transplant. 2015 May.

. 2015 May;21(5):780-92.

doi: 10.1016/j.bbmt.2015.01.003. Epub 2015 Jan 30.

Authors

Affiliations

¹ Department of Pediatrics and Immunology, Indiana University of Medicine, Indianapolis, Indiana. Electronic address: sophpacz@iu.edu.
² Experimental Transplantation and Immunology Branch, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
³ Blood and Marrow Transplantation, Moffitt Cancer Center, Tampa, Florida.
⁴ Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, Maryland.
⁵ Office of In Vitro Diagnostics and Radiological Health, Center for Devices and Radiological Health, Food and Drug Administration, Silver Spring, Maryland.
⁶ Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.
⁷ Clinical Research Division, Fred Hutchinson Cancer Research Center, and the Division of Medical Oncology, University of Washington School of Medicine, Seattle, Washington.
⁸ Department of Medicine, Division of Hematology-Oncology, Vanderbilt University Medical Center, Nashville, Tennessee.
⁹ Stanford Bone Marrow Transplant-Cellular Therapy Facility, Stanford University, Stanford, California.
¹⁰ Department of Pediatrics, Children's Hospital of Los Angeles, University of Southern California, Los Angeles, California.
¹¹ Division of Biostatistics, Center for Biologics, Food and Drug Administration, Silver Spring, Maryland.
¹² Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland.
¹³ Michael Cuccione Childhood Cancer Research Program, BC Children's Hospital and University of British Columbia, Vancouver, British Columbia, Canada.

PMID: 25644957
PMCID: PMC4408233
DOI: 10.1016/j.bbmt.2015.01.003

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.

Keywords: Biomarkers; Chronic graft-versus-host disease; Consensus; National Institutes of Health.

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Figures

**Figure 1**
Example of a predictive biomarker or response biomarker development project

See this image and copyright information in PMC

References

1. Wingard JR, Majhail NS, Brazauskas R, Wang Z, Sobocinski KA, Jacobsohn D, et al. Long-term survival and late deaths after allogeneic hematopoietic cell transplantation. Journal of clinical oncology : official journal of the American Society of Clinical Oncology. 2011;29:2230–9. - PMC - PubMed
1. Filipovich AH, Weisdorf D, Pavletic S, Socie G, Wingard JR, Lee SJ, et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2005;11:945–56. - PubMed
1. Inamoto Y, Jagasia M, Wood WA, Pidala J, Palmer J, Khera N, et al. Investigator feedback about the 2005 NIH diagnostic and scoring criteria for chronic GVHD. Bone marrow transplantation. 2014;49:532–8. - PMC - PubMed
1. Schultz KR, Miklos DB, Fowler D, Cooke K, Shizuru J, Zorn E, et al. Toward biomarkers for chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: III. Biomarker Working Group Report. Biol Blood Marrow Transpl. 2006;12:126–37. - PubMed
1. Biomarkers Definitions Working G. Biomarkers and surrogate endpoints: preferred definitions and conceptual framework. Clinical pharmacology and therapeutics. 2001;69:89–95. - PubMed

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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

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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

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