A Vignette Study to Derive Health-Related Quality of Life Weights for Patients With Steroid Refractory Chronic Graft-Versus-Host Disease Receiving Third Line Therapy in the United Kingdom

OBJECTIVES: Chronic graft-versus-host disease (cGVHD) following allogeneic haematopoietic stem cell transplantation, occurs in 20-40% of patients. cGVHD can have a significant impact on health-related quality of life (HRQL) due to the involvement of multiple organs and need for multiple lines of therapy. This study generated HRQL weights (utilities) for health state vignettes describing different levels of treatment response in cGVHD patients receiving a third line therapy. Utilities for health states related to late stage cGVHD are not well documented.

METHODS: Four draft vignettes were developed through a review of previously published vignette studies which included GVHD states. The vignettes varied by level of response to treatment at third line characterised by increasing disease severity (complete response, partial response, lack of response, and recurrent cGVHD). Semi-structured interviews were conducted with five clinical experts to validate and refine the content of the draft vignettes. The finalised vignettes were hosted online and valued using the EQ-5D-5L by the United Kingdom general public (N=300).

RESULTS: The data revealed a large decline in utilities with worsening severity of cGVHD. Utilities ranged from 0.577 (complete response) to 0.061 (recurrent cGVHD). The qualitative data from interviews with clinical experts highlighted the numerous aspects of daily life that are affected by cGVHD including changes to physical function and appearance, high levels of fatigue, increased risk of infection as well as impacts to emotional wellbeing, daily activities and social functioning.

CONCLUSIONS: This study generated utilities for four health state vignettes describing different levels of treatment response in cGVHD patients receiving a third line therapy. The results demonstrate the substantial HRQL burden of cGVHD with significant differentiation associated with treatment response. The low utility values are partly a reflection of the public’s perception of the disease severity. Patients themselves may learn to cope and adjust over time.