Real-world evidence of incidence and outcomes of aplastic anaemia following administration of immune checkpoint inhibitors

Br J Haematol. 2023 Sep;202(6):1205-1208. doi: 10.1111/bjh.18985. Epub 2023 Jul 16.

Abstract

Aplastic anaemia (AA) is a rare immune-related adverse events (irAEs) after immune checkpoint inhibitors (ICIs) administration with poorly understood incidence and outcomes. We analysed an electronic health record database of 52 303 ICI-treated patients and found 77 (0.15%) cases of AA, with a median onset of 126 days (interquartile range, 58-363 days). The most used treatment for AA was systemic glucocorticoids 60 (77.9%) and 32 (41.6%) patients were able to resume ICI within 1 year. Patients diagnosed with AA had a steep decline in overall survival (OS) within the first 120 days; when compared to propensity score-matched patients without AA, they had a significantly worse OS (hazard ratio 1.72, 95% confidence interval 1.19-2.50; p = 0.003).

Keywords: aplastic anaemia; bone marrow failure; immune checkpoint inhibitors; immune-related adverse events.

MeSH terms

  • Anemia, Aplastic* / chemically induced
  • Anemia, Aplastic* / epidemiology
  • Databases, Factual
  • Electronic Health Records
  • Humans
  • Immune Checkpoint Inhibitors / adverse effects
  • Incidence
  • Retrospective Studies

Substances

  • Immune Checkpoint Inhibitors