Biomarkers for post-transplantation outcomes

Sophie Paczesny


During the last decade, the development of biomarkers for the complications seen after allogeneic hematopoietic stem cell transplantation (allo-HSCT) has expanded tremendously with the most progress been made for acute graft-versus-host disease (aGVHD), a common and often fatal complication. Although many factors are known to determine transplant outcome (including the age of the recipient, comorbidity, conditioning intensity, donor source, donor-recipient human leukocyte antigen (HLA) compatibility, conditioning regimen, post-transplant GVHD prophylaxis), they are incomplete guides for predicting outcomes. Thanks to the advances in genomics, transcriptomics, proteomics and cytomics technologies, blood biomarkers have been identified and validated for us in promising diagnostic tests, prognostic tests stratifying for future occurrence of aGVHD, and predictive tests for responsiveness to GVHD therapy and non-relapse mortality. These biomarkers may facilitate timely and selective therapeutic intervention. However, such blood tests are not yet available for routine clinical care. This Perspectives article provides an overview of the candidate biomarkers for clinical evaluation and outlines a path from biomarker discovery to first clinical correlation, to validation in independent cohorts, to a biomarker-based clinical trial, and finally to general clinical application. This Perspectives article focuses on biomarkers discovered with a large-scale proteomics platform and validated with the same reproducible assay in at least two independent cohorts with sufficient sample size according to the 2014 NIH consensus on biomarkers criteria as well as on biomarkers as tests for risk stratification of outcomes but not on their pathophysiologic contributions, which have been reviewed recently.

  • Submitted February 1, 2018.
  • Accepted April 4, 2018.