Addressing the Burden of Hemolysis in Paroxysmal Nocturnal Hemoglobinuria: The Pharmacist's Contribution to Patient Care

Program Description
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening blood disorder characterized by the destruction of red blood cells, leading to anemia, thrombosis, and impaired quality of life. Given its significant clinical and economic burden, effective treatment strategies are crucial. To support patients with PNH, pharmacists must be familiar with current standard treatments, including supportive care, as well as the mechanisms of action, efficacy, safety, and real-world data for approved and investigational complement-targeting therapies. Experts leading this panel discussion will provide a review of PNH pathophysiology, clinical presentation, and the evolving landscape of complement-directed therapies. The discussion will emphasize the crucial role of the pharmacist in the multidisciplinary management of PNH to reduce disease burden for patients through approaches that focus on optimizing care, educating patients and prescribers, addressing medication adherence, and navigating safety considerations. This program will feature patient case discussions and a direct interview with an individual living with PNH to provide invaluable insights into the realities of the disease and the impact of treatment on their daily lives.

Target Audience: Health system pharmacists and specialty pharmacists

Type of Activity: Application
Release date: August 7, 2025
Expiration date: August 7, 2026
Learner level: Foundational, Intermediate
Time to complete activity: 1.5 hours
Fee: Free

Educational Objectives

At the completion of this activity, participants will be able to:

• Examine the physical, emotional, and financial impacts of paroxysmal nocturnal hemoglobinuria (PNH) on patients, including complications from hemolysis, thrombosis, and fatigue
• Explore the mechanisms of action of complement inhibitors and their effects on preventing hemolysis and thrombosis in PNH
• Identify best practices for pharmacists in optimizing medication regimens, including dosing, administration, and monitoring of complement inhibitors