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Treatment change as a predictor of outcome among patients with classic chronic graft-versus-host disease

Flowers, Mary E.D. and Storer, Barry and Carpenter, Paul and Rezvani, Andrew R. and Vigorito, Afonso C. and Campregher, Paulo V. and Moravec, Carina and Kiem, Hans-Peter and Fero , Matthew and Georges, George and Warren, Edus and Lee, Stephanie and Sanders, Jean E. and Appelbaum, Fred and Martin, Paul J. (2008) Treatment change as a predictor of outcome among patients with classic chronic graft-versus-host disease. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 14 (12). pp. 1380-1384. ISSN 1083-8791

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We analyzed outcomes for 668 patients who had systemic treatment for chronic graft-versus-host disease (GVHD) to assess the utility of early treatment change for exacerbation of chronic GVHD as a surrogate for survival endpoints in clinical trials. Fifty-six percent of patients had treatment change within 2 years after diagnosis of chronic GVHD. The median onset of treatment change was 4.4 months (range, 0.3 – 50 months). The cumulative incidence of non-relapse mortality (NRM) at 2 years was 16%, and overall survival at 2 years was 74%. In time-dependent Cox models, treatment change was associated with an increase in risk of NRM (hazard ratio, 2.53; 95% CI, 1.7-3.7; p < .0001). The hazard ratio was attenuated by 6% per month of delay in treatment change. Our results confirm that exacerbation of chronic GVHD is associated with an increased risk of NRM and with decreased survival, but the strength of this association is not large enough to allow the use of early exacerbation as a surrogate for survival endpoints in clinical trials. Other measures of clinical benefit, such as response, will need to be developed as endpoints in phase II trials for patients with chronic GVHD.

Item Type: Article or Abstract
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DOI: 10.1016/j.bbmt.2008.09.017
PubMed ID: 19041060
PMCID: PMC2810860
Grant Numbers: CA 118953-01A1, CA78902
Keywords or MeSH Headings: * Adolescent * Adult * Aged * Child * Child, Preschool * Chronic Disease * Cohort Studies * Disease-Free Survival * Female * Graft vs Host Disease/mortality* * Graft vs Host Disease/therapy* * Humans * Incidence * Infant * Male * Middle Aged * Stem Cell Transplantation* * Survival Rate
Subjects: Diseases > Graft vs Host
Therapeutics > Drug Therapy
Depositing User: authors@FHCRC Administrator-Import
Date Deposited: 06 Nov 2008 00:41
Last Modified: 14 Feb 2012 14:43

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