Unexpected Toxicities When Nivolumab Was Given as Maintenance Therapy following Allogeneic Stem Cell Transplantation

Biol Blood Marrow Transplant. 2020 May;26(5):1025-1027. doi: 10.1016/j.bbmt.2020.01.021. Epub 2020 Feb 1.


Treatment options are limited for patients with hematologic malignancies who relapse after allogeneic stem cell transplantation (allo-SCT). We conducted a pilot study to assess the tolerability and efficacy of low-dose nivolumab, a PD-1 inhibitor, as maintenance therapy after allo-SCT. Of the 4 patients enrolled in the study, all rapidly developed immune-related adverse events (irAEs); 2 patients experienced serious adverse events, including grade 4 neutropenia and grade 3 autoimmune encephalopathy. As a result of these unexpected severe toxicities, the study was closed to further enrollment. Even at low doses, nivolumab maintenance in the post allo-SCT setting can cause serious irAEs beyond graft-versus-host disease, and further studies of dosage and timing after allo-SCT are needed.

Keywords: Allogeneic stem cell transplantation; Maintenance therapy; Nivolumab.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Graft vs Host Disease*
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Humans
  • Neoplasm Recurrence, Local
  • Nivolumab / adverse effects
  • Pilot Projects
  • Stem Cell Transplantation
  • Transplantation, Homologous


  • Nivolumab