Assessing response of therapy for acute and chronic graft-versus-host disease

Graft-versus-host disease (GVHD) is a significant cause of morbidity and mortality following allogeneic hematopoietic stem cell transplantation. Currently, there are no formal guidelines for assessing the response to GVHD therapy, limiting clinical care and the development of clinical trials. There are proposals for assessing response to acute GVHD therapy. For example, response to therapy at day 28 is predictive of treatment-related mortality. Response to therapy at the same time point may also be predictive of overall survival. The NIH Response Criteria Working Group of the Consensus Conference has proposed guidelines that will help make collection of chronic GVHD (cGVHD) response data more uniform so that response assessment can be formally studied. Core measures of cGVHD including organ-specific assessments and the Lee cGVHD symptom scale were first proposed. In addition, provisional criteria for definition of response were suggested. Finally, biomarkers may provide a powerful tool for predicting response to GVHD therapy. A six-biomarker panel measured prior to the start of therapy has been described that predicts response to treatment at day 28 of therapy for acute GVHD. Further work will determine if biomarkers can be identified that can predict response to cGVHD therapy.