Predictors of survival, non-relapse mortality and failure-free survival in patients treated for chronic graft-versus-host disease.

Publication Type:

Journal Article


Blood (2015)


Chronic graft-versus-host disease (GVHD) is a pleotropic syndrome that lacks validated methods of measuring response in clinical trials, although several endpoints have been proposed. To investigate the prognostic significance of these proposed endpoints, such as the 2005 NIH response measures, 2014 NIH response measures, clinician-reported response and patient-reported response, we tested their ability to predict subsequent overall survival (OS), non-relapse mortality (NRM), and failure free survival (FFS). Patients (n=575) were enrolled on a prospective chronic GVHD observational trial. At 6 months, clinician-reported response (p=0.004) and 2014 NIH calculated response (p=0.001) correlated with subsequent FFS, and clinician-reported response predicted OS (p=0.007). Multivariate models were used to identify changes in organ involvement, laboratory values and patient-reported outcomes that were associated with long-term outcomes. At 6 months, a change in the 2005 NIH 0-3 clinician-reported skin score and 0-10 patient-reported itching score predicted subsequent FFS. Change in the Lee skin symptom score and FACT BMT score predicted subsequent OS. Change in the Lee skin symptom score predicted subsequent NRM. This study provides evidence that clinician-reported response and patient-reported outcomes are predictive of long term survival. The study is registered to as NCT00637689.