Acquired hemophilia due to immune checkpoint inhibitors: A case series introducing emicizumab treatment

Wolff L, Ertl C, Heinzerling L, Aspeslagh S, Verhaert M, Lattenist R, Confente C, Lambert C, Ilzkovitz M (2025)

Publication Type: Journal article

Publication year: 2025

Journal

Oncologist Oxford University Press

Book Volume: 30

Article Number: oyaf260

Journal Issue: 10

DOI: 10.1093/oncolo/oyaf260

Abstract

Background Acquired hemophilia A (AHA) is a rare but potentially life-threatening autoimmune bleeding disorder and immune-related adverse event (irAE) associated with immune checkpoint inhibitors (ICI). This study discusses 3 new cases of ICI-induced AHA from Belgium and the SERIO-Side Effect Registry Immuno-Oncology (www.serio-registry.org), placing them in the context of existing literature. Methods One case was encountered at a tertiary care center in Belgium. SERIO was queried and yielded another 2 cases. A comprehensive literature review using PubMed identified 8 additional cases. Results A total of 11 patients were analyzed, with a median age of 68 years (range: 56-71), 10 were male. Most (9/11) were treated with anti-PD-1 monotherapy. In 5 cases, toxicity appeared before the fourth cycle. Immunosuppressive therapy successfully achieved complete resolution of AHA in 9 of 11 patients. For the first time, one patient was successfully treated with emicizumab, a monoclonal bispecific antibody, which bridges activated factor IX and factor X. Conclusions Clinicians should be vigilant about ICI-induced AHA as a potentially severe irAE. Prolonged aPTT requires thorough evaluation. Upon AHA confirmation, eradication of anti-FVIII antibodies with corticosteroids, rituximab, or other immunosuppressant should be attempted. Emicizumab offers advantages over traditional replacement therapy, including ease of use and a potential reduction in immunosuppressive drug requirements - an important consideration in ICI-treated patients. Further research is needed to fully understand the pathophysiology and optimize treatment strategy for AHA.

Involved external institutions

Institut Jules Bordet Klinikum der Universität München (LMU Klinikum) Belgian Society of Medical Oncology - BSMO BE Belgium (BE) Hôpital de Jolimont Cliniques universitaires Saint-Luc (CHU St-Luc)

How to cite

APA:

Wolff, L., Ertl, C., Heinzerling, L., Aspeslagh, S., Verhaert, M., Lattenist, R.,... Ilzkovitz, M. (2025). Acquired hemophilia due to immune checkpoint inhibitors: A case series introducing emicizumab treatment. Oncologist, 30(10). https://doi.org/10.1093/oncolo/oyaf260

MLA:

Wolff, Louis, et al. "Acquired hemophilia due to immune checkpoint inhibitors: A case series introducing emicizumab treatment." Oncologist 30.10 (2025).

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