Failure-free survival in a prospective cohort of patients with chronic graft-versus-host disease

Jeanne Palmer, Xiaoyu Chai, Paul J. Martin, Daniel Weisdorf, Yoshihiro Inamoto, Joseph Pidala, Madan Jagasia, Steven Pavletic, Corey Cutler, Georgia Vogelsang, Sally Arai, Mary E.D. Flowers, Stephanie J. Lee

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Abstract

Failure-free survival, defined as the absence of relapse, non-relapse mortality or addition of another systemic therapy, has been proposed as a potential endpoint for clinical trials, but its use has only been reported for single-center studies. We measured failure-free survival in a prospective observational cohort of patients (n=575) with both newly diagnosed and existing chronic graft-versus-host disease from nine centers. Failure was observed in 389 (68%) patients during the observation period. The median follow up of all patients was 30.9 months, and the median failure-free survival was 9.8 months (63% at 6 months, 45% at 1 year, and 29% at 2 years). Of the variables measured at enrollment, ten were associated with shorter failure-free survival: higher National Institutes of Health 0-3 skin score, higher National Institutes of Health 0-3 gastrointestinal score, worse range of motion summary score, lower forced vital capacity (%), bronchiolitis obliterans syndrome, worse quality of life, moderate to severe hepatic dysfunction, absence of treatment for gastric acid, female donor for male recipient, and prior grade II-IV acute graft-versus-host disease. Addition of a new systemic treatment, the major cause of failure, was associated with an increased risk of subsequent non-relapse mortality (hazard ratio=2.06, 95% confidence interval: 1.29- 3.32; P<0.003) and decreased survival (hazard ratio=1.51, 95% confidence interval: 1.04-2.18; P<0.03). These results show that fewer than half of patients on systemic treatment will be failure-free survivors at 1 year, and fewer than a third will reach 2 years without experiencing failure. Better treatments are needed for chronic graftversus- host disease. Clinicaltrials.gov identifier: NCT00637689.

Original languageEnglish (US)
Pages (from-to)690-695
Number of pages6
JournalHaematologica
Volume100
Issue number5
DOIs
StatePublished - Jan 1 2015
Externally publishedYes

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Graft vs Host Disease
Survival
National Institutes of Health (U.S.)
Confidence Intervals
Bronchiolitis Obliterans
Therapeutics
Mortality
Gastric Acid
Vital Capacity
Articular Range of Motion
Survivors
Quality of Life
Observation
Tissue Donors
Clinical Trials
Recurrence
Skin
Liver

ASJC Scopus subject areas

  • Hematology

Cite this

Palmer, J., Chai, X., Martin, P. J., Weisdorf, D., Inamoto, Y., Pidala, J., ... Lee, S. J. (2015). Failure-free survival in a prospective cohort of patients with chronic graft-versus-host disease. Haematologica, 100(5), 690-695. https://doi.org/10.3324/haematol.2014.117283

Failure-free survival in a prospective cohort of patients with chronic graft-versus-host disease. / Palmer, Jeanne; Chai, Xiaoyu; Martin, Paul J.; Weisdorf, Daniel; Inamoto, Yoshihiro; Pidala, Joseph; Jagasia, Madan; Pavletic, Steven; Cutler, Corey; Vogelsang, Georgia; Arai, Sally; Flowers, Mary E.D.; Lee, Stephanie J.

In: Haematologica, Vol. 100, No. 5, 01.01.2015, p. 690-695.

Research output: Contribution to journalArticle

Palmer, J, Chai, X, Martin, PJ, Weisdorf, D, Inamoto, Y, Pidala, J, Jagasia, M, Pavletic, S, Cutler, C, Vogelsang, G, Arai, S, Flowers, MED & Lee, SJ 2015, 'Failure-free survival in a prospective cohort of patients with chronic graft-versus-host disease', Haematologica, vol. 100, no. 5, pp. 690-695. https://doi.org/10.3324/haematol.2014.117283
Palmer, Jeanne ; Chai, Xiaoyu ; Martin, Paul J. ; Weisdorf, Daniel ; Inamoto, Yoshihiro ; Pidala, Joseph ; Jagasia, Madan ; Pavletic, Steven ; Cutler, Corey ; Vogelsang, Georgia ; Arai, Sally ; Flowers, Mary E.D. ; Lee, Stephanie J. / Failure-free survival in a prospective cohort of patients with chronic graft-versus-host disease. In: Haematologica. 2015 ; Vol. 100, No. 5. pp. 690-695.
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