Hematological Adverse Events in Immune Checkpoint Therapy

Immune checkpoint inhibitors have transformed oncology by harnessing T-cell responses against malignancies, yet they may provoke a spectrum of hematological adverse events. These include thrombocytopenia, autoimmune haemolytic anaemia, neutropenia, aplastic anaemia and thrombotic microangiopathies. Although each manifestation is uncommon relative to more familiar dermatological or gastrointestinal toxicities, their potential severity demands vigilant surveillance. Pathogenic mechanisms encompass off-target T-cell activation, break-down of self-tolerance, cytokine-driven suppression of haematopoietic precursors and dysregulation of megakaryocyte development. Clinical features vary from asymptomatic cytopenias to life-threatening bleeding, infection or haemolysis. Management generally involves prompt immunosuppression with corticosteroids, intravenous immunoglobulin or second-line agents such as rituximab, alongside supportive measures including transfusions and growth-factor support. Risk stratification and early detection are central to preserving the benefits of immunotherapy while minimising interruption of cancer treatment. Emerging predictive models and multidisciplinary care pathways aim to guide personalised surveillance and intervention strategies.

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Hematological Adverse Events in Immune Checkpoint Therapy publication trend

The graph below shows the total number of articles in hematological adverse events in immune checkpoint therapy across all publications each year (not limited to Nature Index journals).

Technical terms

Immune checkpoint inhibitors (ICIs): Monoclonal antibodies that block regulatory receptors such as CTLA-4 or PD-1/PD-L1 to enhance T-cell–mediated antitumour immunity.

Immune-related adverse events (irAEs): Inflammatory toxicities arising from heightened immune activation against non-tumour tissues.

Thrombocytopenia: A reduction in circulating platelet count below normal thresholds, increasing bleeding risk.

Autoimmune haemolytic anaemia: Destruction of red blood cells by autoantibodies, leading to anaemia and haemolytic markers.

Neutropenia: Abnormally low neutrophil count, heightening susceptibility to infections.

References

1. Association of thrombocytopenia with immune checkpoint inhibitors: a large-scale pharmacovigilance analysis based on the data from FDA adverse event reporting system database. Frontiers in Pharmacology (2024).
2. Immunotherapy-associated autoimmune hemolytic anemia. Journal for ImmunoTherapy of Cancer (2017).
3. Isolated neutropenia as a rare but serious adverse event secondary to immune checkpoint inhibition. Journal for ImmunoTherapy of Cancer (2019).