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Das-Gupta, E; Greinix, H; Jacobs, R; Zhou, L; Savani, BN; Engelhardt, BG; Kassim, A; Worel, N; Knobler, R; Russell, N; Jagasia, M.
Extracorporeal photopheresis as second-line treatment for acute graft-versus-host disease: impact on six-month freedom from treatment failure.
Haematologica. 2014; 99(11):1746-1752
Doi: 10.3324/haematol.2014.108217
[OPEN ACCESS]
Web of Science
PubMed
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- Co-authors Med Uni Graz
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Greinix Hildegard
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Second-line therapy for corticosteroid-refractory or -dependent acute graft-versus-host disease remains ill-defined, due to limited efficacy of drugs and evolving clinical trial endpoints. Six-month freedom from treatment failure has been proposed as a novel clinical trial endpoint and is defined by the absence of death, malignancy relapse/progression, or addition of a next line of systemic immunosuppressive therapy within 6 months of intervention and prior to diagnosis of chronic graft-versus-host disease. We analyzed the 6-month freedom from treatment failure endpoint in 128 patients enrolled from three centers who were treated with extracorporeal photopheresis as second-line therapy for acute graft-versus-host disease. The incidence of 6-month freedom from treatment failure was 77.3% with a 2-year survival rate of 56%. Corticosteroid dose or response status at onset of second-line therapy did not influence outcome. Higher grade of acute graft-versus-host disease (grade 2 versus grades 3-4) at onset of photopheresis predicted for poor outcome as measured by survival (hazard ratio 2.78, P<0.001), non-relapse mortality (hazard ratio 2.78, P=0.001) and 6-month freedom from treatment failure (hazard ratio 3.05, P<0.001). For the 91 patients who achieved 6-month freedom from treatment failure, 1-year, 2-year and 3-year survival rates were 78.9%, 70.8% and 69.5%, respectively. Six-month freedom from treatment failure is a reasonable early surrogate for outcome and should be considered as a clinical trial endpoint. This study demonstrates the durable effect of photopheresis as second-line therapy for corticosteroid-refractory or -dependent acute graft-versus-host disease using 6-month freedom from treatment failure as the primary endpoint.
Copyright© Ferrata Storti Foundation.
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Acute Disease -
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Adolescent -
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Adrenal Cortex Hormones - therapeutic use
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Adult -
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Aged -
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Cause of Death -
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Chronic Disease -
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Female -
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Graft vs Host Disease - diagnosis
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Graft vs Host Disease - etiology
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Graft vs Host Disease - mortality
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Graft vs Host Disease - therapy
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Hematopoietic Stem Cell Transplantation - adverse effects
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Humans -
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Immunosuppressive Agents - therapeutic use
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Male -
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Middle Aged -
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Photopheresis - methods
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Recurrence -
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Retreatment -
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Time Factors -
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Transplantation, Homologous -
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Treatment Failure -
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Treatment Outcome -
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Young Adult -