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Patients with hematologic malignancy who relapse after allogeneic stem-cell transplantation have limited treatment options and, in most cases, short survival.
To test whether graft-versus-tumor activity could be regenerated via an immune checkpoint inhibitor, investigators conducted a multicenter, phase I/Ib trial in which 28 relapsing patients were treated with ipilimumab (3 mg/kg or 10 mg/kg × 4 doses) followed by ongoing treatment for responders.
Among the six patients treated at the lower dose, no responses were observed. Among the 22 patients treated at the higher dose, 5 achieved complete remission (including all 3 with leukemia cutis, 1 with myeloid sarcoma, and 1 with myelodysplastic syndrome/acute myeloid leukemia), and 2 achieved pa…