Michael E. Williams, MD, ScM reviewing Davids MS et al. N Engl J Med 2016 Jul 14.
Patients with recurrent myeloid and lymphoid malignancies responded to the immune checkpoint inhibitor ipilimumab.
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 partial remission (1 with Hodgkin lymphoma and 1 with pulmonary plasmacytoma). Six additional patients showed a decrease in tumor burden. Two patients developed liver graft-versus-host disease (GVHD), and one developed gut GVHD that responded to glucocorticoid therapy. Immune-related adverse events included immune thrombocytopenia in one patient, inflammatory colitis in one, and pneumonitis in one that proved fatal. Overall, five patients discontinued ipilimumab due to toxicity.
Davids MS et al. Ipilimumab for patients with relapse after allogeneic transplantation. N Engl J Med 2016 Jul 14; 375:143.