title: Predictors of survival, nonrelapse mortality, and failure-free survival in patients treated for chronic graft-versus-host disease.
abstract: Chronic graft-versus-host disease (GVHD) is a pleotropic syndrome that lacks validated methods of measuring response in clinical trials, although several end points have been proposed. To investigate the prognostic significance of these proposed end points, such as the 2005 National Institutes of Health (NIH) response measures, 2014 NIH response measures, clinician-reported response, and patient-reported response, we tested their ability to predict subsequent overall survival (OS), nonrelapse 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 = .004) and 2014 NIH-calculated response (P = .001) correlated with subsequent FFS, and clinician-reported response predicted OS (P = .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 to 3 clinician-reported skin score and 0 to 10 patient-reported itching score predicted subsequent FFS. Change in the Lee skin symptom score and Functional Assessment of Cancer Therapy-Bone Marrow Transplant 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 trial was registered at www.clinicaltrials.gov as #NCT00637689.
authors.affiliation: Division of Hematology/Oncology, Mayo Clinic, Phoenix, AZ;
authors.forename: Jeanne
authors.identifier: http://orcid.org/0000-0002-2185-1504
authors.initials: J
authors.lastname: Palmer
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA;
authors.forename: Xiaoyu
authors.initials: X
authors.lastname: Chai
authors.affiliation: Blood and Marrow Transplantation, Moffitt Cancer Center, Tampa, FL;
authors.forename: Joseph
authors.initials: J
authors.lastname: Pidala
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA; Division of Hematopoietic Stem Cell Transplantation, National Cancer Center Hospital, Tokyo, Japan;
authors.forename: Yoshihiro
authors.initials: Y
authors.lastname: Inamoto
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA;
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Martin
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA;
authors.forename: Barry
authors.initials: B
authors.lastname: Storer
authors.affiliation: Division of Hematology/Oncology, Washington University, St. Louis, MO;
authors.forename: Iskra
authors.initials: I
authors.lastname: Pusic
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA;
authors.forename: Mary E D
authors.initials: ME
authors.lastname: Flowers
authors.affiliation: Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, MN; and.
authors.forename: Mukta
authors.initials: M
authors.lastname: Arora
authors.affiliation: National Cancer Institute, National Institutes of Health, Bethesda, MD.
authors.forename: Steven Z
authors.initials: SZ
authors.lastname: Pavletic
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA;
authors.forename: Stephanie J
authors.initials: SJ
authors.lastname: Lee
mesh_terms: D000328:Adult; D000368:Aged; D002908:Chronic Disease; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000209:etiology / Q000401:mortality*; D019337:Hematologic Neoplasms / Q000150:complications / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D008297:Male; D008875:Middle Aged; D009316:National Institutes of Health (U.S.); D063868:Patient Outcome Assessment; D011379:Prognosis; D012307:Risk Factors; D057566:Self Report; D012720:Severity of Illness Index; D015996:Survival Rate; D014481:United States; D055815:Young Adult