Treatment of Paroxysmal Nocturnal Hemoglobinuria with Venous Thromboembolism
Clinical Scenario
This protocol covers patients with Paroxysmal nocturnal hemoglobinuria (PNH) who have active venous thromboembolism, a history of previous venous thromboembolism, or are at increased risk of venous thromboembolism. A thromboembolic event may be the first clinical manifestation of PNH, and its occurrence defines a specific, urgent management threshold.
Why This Situation Requires Specific Management
In PNH, venous thromboembolism — whether active, prior, or at elevated risk — requires simultaneous attention to intravascular hemolysis and thrombotic risk. Immediate intervention is necessary when a thromboembolic event presents as the first sign of PNH.
Treatment Approach (Overview)
Management involves rapid control of intravascular hemolysis through a terminal complement inhibitor, together with long-term anticoagulation. The complete regimen — including agent selection, sequencing, and duration — is available in the full protocol.
References
Venous thromboembolism or previous venous thromboembolism or increased risk (see chapter 4.1.2)
In the event of a thromboembolic event as the first manifestation of PNH, immediate interruption of intravascular haemolysis (IVH) is necessary.
In the event of a thromboembolic event as the first manifestation of PNH, immediate interruption of intravascular haemolysis (IVH) is necessary. According to current data, this is most reliably achieved by administering an effective terminal complement inhibitor (e.g. ravulizumab), as thrombin can directly activate C3 and C5 and plasmin also activates C5, making rapid C5 blockade useful.
Long-term or lifelong anticoagulation after a thrombosis is recommended.
Both coumarins and heparins can be used therapeutically and prophylactically.
Published data on the use of DOACs are only available for very small groups of patients, but their use, especially with simultaneous administration of complement inhibitors, seems to lead to comparable results based on experience in centers.
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