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Original Investigation
August 12, 2024

Apixaban vs Aspirin in Patients With Cancer and Cryptogenic Stroke: A Post Hoc Analysis of the ARCADIA Randomized Clinical Trial

Author Affiliations
  • 1Clinical and Translational Neuroscience Unit, Feil Family Brain and Mind Research Institute and Department of Neurology, Weill Cornell Medicine, New York, New York
  • 2Department of Neurology, Memorial Sloan Kettering Cancer Center, New York, New York
  • 3Department of Neurology, University of Minnesota, Minneapolis
  • 4Division of Hematology and Oncology, Department of Medicine, University of Vermont Larner College of Medicine, Burlington
  • 5Department of Neurology, University of Pennsylvania School of Medicine, Philadelphia
  • 6Department of Neurology, Vagelos College of Physicians and Surgeons, Columbia University, New York, New York
  • 7Department of Epidemiology, Mailman School of Public Health, Columbia University, New York, New York
  • 8Department of Neurology, University of Washington, Seattle
  • 9Department of Epidemiology, University of Washington, Seattle
  • 10Department of Biostatistics, University of Washington, Seattle
  • 11Department of Neurology, Inselspital, Bern University Hospital and University of Bern, Bern, Switzerland
  • 12Department of Biostatistics, Medical University of South Carolina, Charleston
  • 13Ochsner Neuroscience Institute, Ochsner Health, New Orleans, Louisiana
  • 14Department of Neurology, Neurocenter of Southern Switzerland EOC, Lugano, Switzerland
  • 15Stroke Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
  • 16Neurology Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
  • 17Department of Clinical Neurosciences, University of Lausanne, Lausanne, Switzerland
  • 18Department of Neurology and Rehabilitation Medicine, University of Cincinnati College of Medicine, Cincinnati, Ohio
  • 19Sunnybrook Research Institute, Hurvitz Brain Sciences Program, Sunnybrook Health Sciences Centre, and Division of Neurology, Department of Medicine, University of Toronto, Toronto, Ontario, Canada
  • 20Deputy Editor, JAMA Neurology
JAMA Neurol. 2024;81(9):958-965. doi:10.1001/jamaneurol.2024.2404
Key Points

Question  What is the optimal antithrombotic strategy for cancer-associated cryptogenic stroke?

Findings  In a secondary analysis of 1015 participants with cryptogenic stroke and atrial cardiopathy in the Atrial Cardiopathy and Antithrombotic Drugs in Prevention After Cryptogenic Stroke (ARCADIA) randomized clinical trial, 137 participants had history of cancer. Among this subgroup, the risk of major ischemic or major hemorrhagic events was nominally but not significantly lower with apixaban compared with aspirin, but analyses were underpowered given small numbers.

Meaning  Among ARCADIA trial participants with history of cancer, the risk of major ischemic or hemorrhagic events did not differ significantly with apixaban vs aspirin, but a clinically important benefit of apixaban could not be ruled out.

Abstract

Importance  Approximately 10% to 15% of ischemic strokes are associated with cancer; cancer-associated stroke, particularly when cryptogenic, is associated with high rates of recurrent stroke and major bleeding. Limited data exist on the safety and efficacy of different antithrombotic strategies in patients with cancer and cryptogenic stroke.

Objective  To compare apixaban vs aspirin for the prevention of adverse clinical outcomes in patients with history of cancer and cryptogenic stroke.

Design, Setting, and Participants  Post hoc analysis of data from 1015 patients with a recent cryptogenic stroke and biomarker evidence of atrial cardiopathy in the Atrial Cardiopathy and Antithrombotic Drugs in Prevention After Cryptogenic Stroke (ARCADIA) trial, a multicenter, randomized, double-blind clinical trial conducted from 2018 to 2023 at 185 stroke centers in North America. Data analysis was performed from October 15, 2023, to May 23, 2024.

Exposures  Oral apixaban, 5 mg (or 2.5 mg if criteria met), twice daily vs oral aspirin, 81 mg, once daily. Subgroups of patients with and without cancer at baseline were examined.

Main Outcomes and Measures  The primary outcome for this post hoc analysis was a composite of major ischemic or major hemorrhagic events. Major ischemic events were recurrent ischemic stroke, myocardial infarction, systemic embolism, and symptomatic deep vein thrombosis or pulmonary embolism. Major hemorrhagic events included symptomatic intracranial hemorrhage and any major extracranial hemorrhage.

Results  Among 1015 participants (median [IQR] age, 68 [60-76] years; 551 [54.3%] female), 137 (13.5%) had a history of cancer. The median (IQR) follow-up was 1.5 (0.6-2.5) years for patients with history of cancer and 1.5 (0.6-3.0) years for those without history of cancer. Participants with history of cancer, compared with those without history of cancer, had a higher risk of major ischemic or major hemorrhagic events (hazard ratio [HR], 1.73; 95% CI, 1.10-2.71). Among those with history of cancer, 8 of 61 participants (13.1%) randomized to apixaban and 16 of 76 participants (21.1%) randomized to aspirin had a major ischemic or major hemorrhagic event; however, the risk was not significantly different between groups (HR, 0.61; 95% CI, 0.26-1.43). Comparing participants randomized to apixaban vs aspirin among those with cancer, events included recurrent stroke (5 [8.2%] vs 9 [11.8%]), major ischemic events (7 [11.5%] vs 14 [18.4%]), and major hemorrhagic events (1 [1.6%] vs 2 [2.6%]).

Conclusions and Relevance  Among participants in the ARCADIA trial with history of cancer, the risk of major ischemic and hemorrhagic events did not differ significantly with apixaban compared with aspirin.

Trial Registration  ClinicalTrials.gov Identifier: NCT03192215

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1 Comment for this article
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Considerations and Reflections on Antithrombotic Management in Cancer-Associated Cryptogenic Stroke: Insights from the ARCADIA Trial
Ching-Mao Chang, Assistant professor | Center for Traditional Medicine, Taipei Veterans General Hospital, Taipei; Institute of Traditional Medicine, and School of Chinese Medicine, National Yang Ming Chiao Tung University, Taipei, Taiwan
Prof. Navi and his colleagues' work 1 offers valuable insights into the antithrombotic management of patients with cancer and cryptogenic stroke, a population with significant unmet needs. However, I would like to highlight several limitations that I believe should be considered when interpreting the findings.
Firstly, the study’s focus on comparing apixaban with aspirin does not allow for the generalization of results to other anticoagulants or dosing regimens. Given the diverse pharmacological profiles of different anticoagulants, the findings might not extend to agents like rivaroxaban or low-molecular-weight heparins. Comparative studies involving multiple anticoagulants are necessary to determine the optimal strategy
for this patient population.
Secondly, the ARCADIA trial’s specific inclusion of patients with atrial cardiopathy and cryptogenic stroke may limit the external validity of the results for broader cancer-related stroke populations 2. These patients often have different stroke mechanisms, which may not be adequately represented in the study cohort.
Thirdly, the study did not explore the differential effects of various anticoagulants and antiplatelets 3. Given the distinct mechanisms by which these drugs act, further research is needed to understand their specific efficacy and safety in patients with cancer and stroke.
Additionally, the absence of D-dimer data is a significant limitation 4. D-dimer is a crucial biomarker in cancer-related thromboembolism, and its inclusion could have provided more context for interpreting the study’s results.
Moreover, the potential for detection bias, given the post hoc nature of the analysis and the combined primary endpoint of ischemic and hemorrhagic events, may obscure important differences in drug efficacy and safety.
Lastly, the post hoc analysis is limited by its exploratory nature, being underpowered to draw definitive conclusions. Therefore, these findings should be viewed as hypothesis-generating, emphasizing the need for robust prospective trials to establish clear clinical guidelines.
In conclusion, while the study adds valuable preliminary data, the limitations underscore the need for further research to clarify the best treatment strategies for this high-risk population.

References
1. Navi BB, Zhang C, Miller B, et al. Apixaban vs Aspirin in Patients With Cancer and Cryptogenic Stroke: A Post Hoc Analysis of the ARCADIA Randomized Clinical Trial. JAMA Neurol. 2024.
2. Bang OY, Chung JW, Lee MJ, et al. Cancer-Related Stroke: An Emerging Subtype of Ischemic Stroke with Unique Pathomechanisms. J Stroke. 2020;22(1):1-10.
3. Ageno W, Gallus AS, Wittkowsky A, Crowther M, Hylek EM, Palareti G. Oral anticoagulant therapy: Antithrombotic Therapy and Prevention of Thrombosis, 9th ed: American College of Chest Physicians Evidence-Based Clinical Practice Guidelines. Chest. 2012;141(2 Suppl):e44S-e88S.
4. Ay C, Dunkler D, Pirker R, et al. High D-dimer levels are associated with poor prognosis in cancer patients. Haematologica. 2012;97(8):1158-1164.

Chun-Pai Yang, M.D., Ph.D. 1, Chung-Chih Liao, M.D., Ph.D. 2, Ching-Mao Chang, M.D., Ph.D. 3,*

1 Department of Neurology, Kuang Tien General Hospital, Taichung, Taiwan
2 Department of Post-Baccalaureate Veterinary Medicine, College of Medical and Health Science, Asia University, Taichung, Taiwan
3 Center for Traditional Medicine, Taipei Veterans General Hospital, Taipei, Taiwan; and Institute of Traditional Medicine, and School of Chinese Medicine, Colledge of Medicine, National Yang Ming Chiao Tung Unive
CONFLICT OF INTEREST: None Reported
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