title: Failure-free survival after initial systemic treatment of chronic graft-versus-host disease.
abstract: This study was designed to characterize failure-free survival (FFS) as a novel end point for clinical trials of chronic graft-versus-host disease (GVHD). The study cohort included 400 consecutive patients who received initial systemic treatment of chronic GVHD at our center. FFS 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 4 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. FFS offers a potentially useful basis for interpreting results of initial treatment of chronic GVHD. Incorporation of steroid doses at 12 months would increase clinical benefit associated with the end point. Studies using FFS as the primary end point should measure changes in GVHD-related symptoms, activity, damage, and disability as secondary end points.
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA; and.
authors.forename: Yoshihiro
authors.identifier: http://orcid.org/0000-0003-4881-0427
authors.initials: Y
authors.lastname: Inamoto
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA; and Departments of Medicine.
authors.forename: Mary E D
authors.identifier: http://orcid.org/0000-0003-1631-0911
authors.initials: ME
authors.lastname: Flowers
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA; and Departments of Medicine.
authors.forename: Brenda M
authors.identifier: http://orcid.org/0000-0002-9767-9739
authors.initials: BM
authors.lastname: Sandmaier
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA; and.
authors.forename: Sahika Z
authors.initials: SZ
authors.lastname: Aki
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA; and Pediatrics, and.
authors.forename: Paul A
authors.identifier: http://orcid.org/0000-0001-9810-1798
authors.initials: PA
authors.lastname: Carpenter
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA; and Departments of Medicine.
authors.forename: Stephanie J
authors.identifier: http://orcid.org/0000-0003-2600-6390
authors.initials: SJ
authors.lastname: Lee
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA; and Biostatistics, University of Washington School of Medicine, Seattle, WA.
authors.forename: Barry E
authors.identifier: http://orcid.org/0000-0003-1565-435X
authors.initials: BE
authors.lastname: Storer
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA; and Departments of Medicine.
authors.forename: Paul J
authors.identifier: http://orcid.org/0000-0001-9051-1215
authors.initials: PJ
authors.lastname: Martin
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D064591:Allografts; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000188:drug therapy* / Q000401:mortality*; D019337:Hematologic Neoplasms / Q000401:mortality / Q000628:therapy; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D012307:Risk Factors; D033581:Stem Cell Transplantation; D013256:Steroids / Q000008:administration & dosage*; D015996:Survival Rate; D013997:Time Factors
chemical_list: D013256:Steroids