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September 23, 2025DrugsOpen Access

Pharmacological Therapies in Paroxysmal Nocturnal Haemoglobinuria: Focus on Complement Inhibition

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Authors

RKRichard J. KellyMHMatthew T. HoltJSJeff Szer

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Overview

This review highlights pharmacological interventions reducing thrombosis and improving quality of life in patients with PNH.

Key Points

  • Complement inhibitors like eculizumab and ravulizumab significantly reduce thrombotic events in PNH.
  • Ravulizumab offers advantages over eculizumab with its longer-acting properties, reducing transfusion dependence.
  • Proximal complement inhibitors have been effective in managing complications like extravascular haemolysis, improving patient outcomes.
  • Breakthrough intravascular haemolysis remains a risk with complement therapies, necessitating ongoing development of management strategies.

Cite This Study

Kelly et al. (2025) studied this question.

synapsesocial.com/papers/68d44098713b0b5dfea7f7b7https://doi.org/10.1007/s40265-025-02235-4
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