Sensitivity of changes in chronic graft-versus-host disease activity to changes in patient-reported quality of life: results from the Chronic Graft-versus-Host Disease Consortium

• Published in: Haematologica (Roma) 2011-10, Vol.96 (10), p.1528-1535
• Main Authors: PIDALA, Joseph, KURLAND, Brenda F, XIAOYU CHAI, VOGELSANG, Georgia, WEISDORF, Daniel J, PAVLETIC, Steven, CUTLER, Corey, MAJHAIL, Navneet, LEE, Stephanie J
• Format: Article
• Language: English
• Subjects: Adult; Aged; Biological and medical sciences; Chronic Disease; Cohort Studies; Diagnostic Self Evaluation; Female; Graft vs Host Disease - diagnosis; Hematologic and hematopoietic diseases; Hematopoietic Stem Cell Transplantation - adverse effects; Humans; Male; Medical sciences; Middle Aged; Original; Outcome Assessment (Health Care); Quality of Life; Severity of Illness Index; Young Adult
• ISSN: 0390-6078; 1592-8721
• DOI: 10.3324/haematol.2011.046367

The 2005 National Institute of Health Chronic Graft-versus-Host Disease Consensus Conference recommended collection of patient-reported outcomes in clinical trials on chronic graft-versus-host disease. We assessed whether changes in chronic graft-versus-host disease severity, determined using National Institute of Health criteria, clinicians' assessment or patients' self-evaluation, correlated with patient-reported quality of life as measured by the Short Form-36 and Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) instruments. Three-hundred and thirty-six adult patients (median age 52 years; range, 19-79) with chronic graft-versus-host disease from six transplant centers contributed baseline and follow-up data (from 936 visits overall). While the majority of the patients had stable chronic graft-versus-host disease, improvement or worsening was noted in approximately 40% of follow-up visits. Multivariable analysis demonstrated no association between change in chronic graft-versus-host disease severity evaluated by National Institute of Health criteria and change in quality of life, while clinician-reported changes in severity were associated with changes in some quality of life measures. Patient-reported changes in the severity of chronic graft-versus-host disease were associated with changes in all quality of life measures. Comparison of the Short Form-36 and the FACT-BMT suggested that the data collected in the Functional Assessment of Cancer Therapy-General (FACT-G) core survey are sufficient without the need for the Short Form-36 or the FACT-BMT subscale. We conclude that serial National Institute of Health and clinician-reported chronic graft-versus-host disease severity assessments cannot substitute for patient-reported outcomes in clinical trials. Collection of just the FACT-G instead of the Short Form-36 and the full FACT-BMT will decrease respondent burden without compromising quality of life assessment.