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, Frances T. Hakim, Joseph Pidala, Kenneth R. Cooke, Julia Lathrop, Linda M. Griffith, John Hansen, Madan Jagasia, David Miklos, Steven Pavletic, Robertson Parkman, Estelle Russek-Cohen, Mary E.D. Flowers, Stephanie Lee, Paul Martin, Georgia Vogelsang, Marc Walton, Kirk R. Schultz

Research output: Contribution to journalArticle

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.

Original languageEnglish (US)
Pages (from-to)780-792
Number of pages13
JournalBiology of Blood and Marrow Transplantation
Volume21
Issue number5
DOIs
StatePublished - May 1 2015

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Keywords

  • Biomarkers
  • Chronic graft-versus-host disease
  • Consensus
  • National institutes of health

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

Paczesny, S., Hakim, F. T., Pidala, J., Cooke, K. R., Lathrop, J., Griffith, L. M., Hansen, J., Jagasia, M., Miklos, D., Pavletic, S., Parkman, R., Russek-Cohen, E., Flowers, M. E. D., Lee, S., Martin, P., Vogelsang, G., Walton, M., & Schultz, K. R. (2015). 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. Biology of Blood and Marrow Transplantation, 21(5), 780-792. https://doi.org/10.1016/j.bbmt.2015.01.003