Plasma biomarkers represent potentially noninvasive, objective, and cost-efficient risk stratification of patients with graft-versus-host disease (GVHD).
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Use of the correct biomarker National Institutes of Health-Food and Drug Administration (FDA) BEST terminology will support the GVHD biomarker effort as it will enable physicians in our field and other fields and regulatory authorities to speak the same language.
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Given that no GVHD biomarkers have yet completed the FDA 4-step framework, additional studies are needed before GVHD biomarkers qualify for clinical use.
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No risk biomarkers for acute GVHD or predictive biomarkers for chronic GVHD have been identified to date.
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A randomized phase 2 clinical trial successfully used biomarkers to guide steroid-free GVHD treatment.
Abstract
Hematopoietic cell transplantation (HCT) is a potentially curative treatment for many hematologic and nonhematologic disorders. Graft-versus-host-disease (GVHD) in its acute or chronic form remains the most important nonrelapse post-HCT complication. Biomarkers offer objective, unbiased information on systemic disorders, and significant attention has focused on identifying biomarkers for GVHD. Ideally, a GVHD biomarker is actionable, with the results of biomarker testing used to guide clinical management of disease and clinical trial design. Although many GVHD biomarkers have been identified, none have been properly qualified for clinical use. The National Institutes of Health (NIH) and Food and Drug Administration (FDA) have provided biomarker subtype definitions; however, confusion remains about the proper definition and application of these subtypes in the HCT field. The 2014 NIH consensus development project provided a framework for the development of biomarkers for clinical practice. This review aims to clarify the biomarker subtype definitions and reemphasize the developmental framework. Armed with this knowledge, clinicians can properly translate GVHD biomarkers for clinical use.
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Financial disclosure: See Acknowledgments on page 665.
