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. 2025 Oct;58(7):955-961.
doi: 10.1007/s11239-025-03124-z. Epub 2025 Jun 9.

Direct oral anticoagulants as secondary prophylaxis of venous thromboembolism in paroxysmal nocturnal hemoglobinuria: an Italian monocentric experience

M Biglietto  1 , A L Faccini  1 , J Micozzi  1 , M Salvatori  1 , M Antonacci  1 , U La Rocca  1 , A P Iori  1 , Antonio Chistolini  2
Affiliations

Direct oral anticoagulants as secondary prophylaxis of venous thromboembolism in paroxysmal nocturnal hemoglobinuria: an Italian monocentric experience

M Biglietto et al. J Thromb Thrombolysis. 2025 Oct.

Abstract

In 2011 Luzzatto et al. stated that "Paroxysmal nocturnal hemoglobinuria (PNH) is the most vicious acquired thrombophilic state known in medicine". Fourteen years later, although anti-complement therapy reduced the incidence of thrombotic events, their management remains an unmet clinical need. Historically Vitamin K Antagonists were the first-choice medications for anticoagulation in this setting. Nowadays, Direct Oral Anticoagulants (DOACs) are the standard anticoagulant therapy in most settings due to their predictable pharmacokinetics, fixed dosing, and no need for laboratory monitoring. Poor data is available on their use in paroxysmal nocturnal hemoglobinuria patients in the treatment of the acute-phase of venous thromboembolism (VTE), while no data is available on their use in secondary prophylaxis of VTE. We describe our monocentric experience on the management of thrombotic events in PNH patients and on the use of DOACs as secondary prophylaxis medication. Our retrospective monocentric analysis shows that DOACs could be an effective and safe choice in this setting.

Keywords: Direct oral anticoagulants; Paroxysmal nocturnal hemoglobinuria; Secondary prophylaxis of VTE; Venous thromboembolism.

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Conflict of interest statement

Declarations. Ethical approval: The study was conducted in accordance with the Declaration of Helsinki and its later amendments or comparable ethical standards. Competing interests: The authors declare no competing interests.

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