Failure-free survival after initial systemic treatment of chronic graft-versus host disease.

Publication Type:

Journal Article


Blood, Volume 124, Issue 8, p.1363-71 (2014)


2014, Center-Authored Paper, Clinical Research Division, June 2014, Research Trials Office Core Facility - Biostatistics Service, Shared Resources


This study was designed to characterize failure-free survival (FFS) as a novel endpoint for clinical trials of chronic graft-versus-host disease (GVHD). The study cohort included 400 consecutive patients who received initial systemic treatment for chronic GVHD at our center. Failure-free survival was defined by the absence of second-line treatment, nonrelapse mortality and recurrent malignancy during initial treatment. The FFS rate was 68% at 6 months and 54% at 12 months after initial treatment. Multivariate analysis identified four risk factors associated with treatment failure: time interval <12 months from transplantation to initial treatment, patient age ≥60 years, severe involvement of the gastrointestinal tract, liver or lungs and Karnofsky score <80% at initial treatment. Initial steroid doses and the type of initial treatment were not associated with risk of treatment failure. Lower steroid doses after 12 months of initial treatment were associated with long-term success in withdrawing all systemic treatment. Failure-free survival offers a potentially useful basis for interpreting results of initial treatment for chronic GVHD. Incorporation of steroid doses at 12 months would increase clinical benefit associated with the endpoint. Studies using FFS as the primary endpoint should measure changes in GVHD-related symptoms, activity, damage and disability as secondary endpoints.