title: 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.
abstract: BACKGROUND
  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.
  
  
  METHODS
  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).
  
  
  RESULTS
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Blood and Marrow Transplantation, Moffitt Cancer Center, Tampa, FL 33612, USA. joseph.pidala@moffitt.org
authors.forename: Joseph
authors.initials: J
authors.lastname: Pidala
authors.forename: Brenda F
authors.initials: BF
authors.lastname: Kurland
authors.forename: Xiaoyu
authors.initials: X
authors.lastname: Chai
authors.forename: Georgia
authors.initials: G
authors.lastname: Vogelsang
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: Weisdorf
authors.forename: Steven
authors.initials: S
authors.lastname: Pavletic
authors.forename: Corey
authors.initials: C
authors.lastname: Cutler
authors.forename: Navneet
authors.initials: N
authors.lastname: Majhail
authors.forename: Stephanie J
authors.initials: SJ
authors.lastname: Lee
mesh_terms: D000328:Adult; D000368:Aged; D002908:Chronic Disease; D015331:Cohort Studies; D059026:Diagnostic Self Evaluation; D005260:Female; D006086:Graft vs Host Disease / Q000175:diagnosis*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D008297:Male; D008875:Middle Aged; D017063:Outcome Assessment, Health Care; D011788:Quality of Life; D012720:Severity of Illness Index; D055815:Young Adult