Paroxysmal Nocturnal Hemoglobinuria: Managed Care Strategies to Mitigate Burden and Enhance Outcomes

Program Description

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic hematologic disorder caused by a genetic mutation that leads to complement-mediated destruction of red blood cells, resulting in hemolysis, thrombosis, bone marrow failure, and a range of debilitating symptoms caused by hemolytic anemia. Current management of PNH includes supportive care, hematopoietic cell transplantation, and novel biologic treatments. PNH presents a significant health and economic burden, with patients experiencing frequent health care utilization and reduced quality of life primarily due to chronic hemolytic anemia and its associated symptoms of fatigue, weakness, and the frequent need for blood transfusions. With current and emerging treatments for PNH, managed care professionals are poised to play a vital role in facilitating patient access to newer biologic therapies by establishing effective medication management systems designed to improve quality of care and reduce economic burdens associated with PNH. This activity will explore the role of the complement pathway and the pathophysiology of PNH while assessing the impact and clinical burden of PNH on patients. In addition to analyzing clinical data and guidelines surrounding current treatments and emerging biologics, this supplement will equip managed care professionals with the knowledge to navigate access barriers, reduce patient burden, and improve overall outcomes.

Target audience: Managed care pharmacists
Type of activity: Application
Release date: August 29, 2025
Expiration date: August 29, 2026
Learner level: Foundational, Intermediate
Time to complete activity: 2.0 hours
Fee: Free

Educational Objectives

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

• Explore the role of the complement pathway in the pathophysiology of paroxysmal nocturnal hemoglobinuria (PNH) and the clinical burdens associated with this disease.
• Analyze the clinical data for current and emerging treatments and evidence-based recommendations for the therapeutic approaches for the treatment of PNH.
• Apply best practices and effective management strategies in the treatment of PNH to mitigate the health and economic burdens while facilitating appropriate access to patient-centered clinical interventions.