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Paroxysmal Nocturnal Hemoglobinuria in Brief: Clinical Management Today

Clinical Thought

Read this commentary to learn how experts are using complement inhibitor therapies to treat patients with paroxysmal nocturnal hemoglobinuria. Key topics discussed are diagnostic insights to facilitate faster identification, treatment options and their differences, and important points for patient education and shared decision-making. 

Released: March 12, 2026

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Provided by Clinical Care Options, LLC dba Decera Clinical Education

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Supporters

Supported by an educational grant from Novartis Pharmaceuticals Corporation.

Novartis Pharmaceuticals Corporation

Target Audience

This educational activity is intended for hematologists and other healthcare professionals who diagnose and care for patients with paroxysmal nocturnal hemoglobinuria.

Learning Objectives

Upon completion of this activity, participants should be able to:

  • Evaluate current recommendations for managing PNH, including challenges to optimal outcomes, potential therapeutic targets in the complement cascade, and evidence for emerging complement-targeted therapies

  • Differentiate the mechanisms of action for conventional and emerging complement-targeted therapies in PNH

  • Select appropriate complement-targeted therapies for treating patients with PNH by analyzing efficacy and safety evidence, indications, disease-related factors, and patient needs and preferences

  • Implement recommended patient-centered strategies to identify and manage complications associated with PNH 

Disclosure

Primary Author

Carlos M. De Castro, MD: consultant/advisor/speaker: Alexion/AstraZeneca, Apellis, BioCryst, Genentech, Novartis, Omeros; data and safety monitoring board: Regeneron.

David Dingli, MD, PhD, FRCP, FRCPEd, FACP, FRCPath: consultant/advisor/speaker: Alexion, Bristol Myers Squibb, Johnson & Johnson, Merck Sharp & Dohme, Novartis, Regeneron.