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Chronic graft-versus-host disease (cGVHD) complicating allogeneic hematopoietic stem-cell transplantation affects the skin, gastrointestinal tract, and other organs, resulting in considerable morbidity. The disorder is often refractory to steroids, although in such cases, benefit has been reported with imatinib (NEJM JW Oncol Hematol Oct 30 2013) and ruxolitinib (NEJM JW Oncol Hematol Jun 24 2014) in small numbers of patients. Also, bortezomib has inhibitory effects on B-cells and plasma cells, and could potentially ameliorate cGVHD, a disease of B-cell dysregulation.
To examine the activity of bortezomib in cGVHD, investigators from several universities studied a mouse model of the disorder and conducted a single-arm pilot study in patients…