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Balancing the benefits and risks of rebalancing coagulation in haemophilia - PubMed

4 hours ago
  • #coagulation
  • #haemophilia
  • #rebalancing agents
  • Rebalancing agents are a new therapeutic class in haemophilia aimed at restoring deficient thrombin generation.
  • They work by downregulating natural anticoagulants, inducing a controlled procoagulant shift.
  • These agents have shown meaningful reductions in bleeding rates in haemophilia A and B, including in patients with inhibitors.
  • The therapeutic window is narrow; excessive thrombin generation may cause thrombosis, while conservative dosing may not prevent bleeding.
  • Potential indications include haemophilia B with inhibitors, young children, patients with suboptimal bleed control, and other bleeding disorders.
  • Tissue factor pathway inhibitor inhibition may reduce joint bleeding and synovitis due to local tissue expression.
  • Patient selection, individualized monitoring, and long-term safety evaluation are crucial for these agents.