Extracorporeal photopheresis as second-line treatment for acute graft-versus-host disease: impact on six-month freedom from treatment failure.

Das-Gupta E, Greinix H, Jacobs R, Zhou L, Savani BN, Engelhardt BG, Kassim A, Worel N, Knobler R, Russell N, Jagasia M
Haematologica. 2014 99 (11): 1746-52

PMID: 25150260 · PMCID: PMC4222474 · DOI:10.3324/haematol.2014.108217

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.

MeSH Terms (22)

Acute Disease Adolescent Adrenal Cortex Hormones Adult Aged Cause of Death Chronic Disease Female Graft vs Host Disease Hematopoietic Stem Cell Transplantation Humans Immunosuppressive Agents Male Middle Aged Photopheresis Recurrence Retreatment Time Factors Transplantation, Homologous Treatment Failure Treatment Outcome Young Adult

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