Taking a Deep Data Dive into PNH: Focus on New and Emerging Agents


This activity is provided by Integrity Continuing Education, Inc.


This activity is supported by an educational grant from Apellis Pharmaceuticals, Inc.


If you’ve never heard of paroxysmal nocturnal hemoglobinuria (PNH), you’re not alone.  It’s a rare disease that results in the destruction of red blood cells, resulting in severe anemia and high risk for lethal thrombotic events. Started by a mutation in a single gene, PNH is activated through the complement cascade, a less-understood part of the innate immune system. Until recently, the pathogenesis of PNH was poorly understood and there were no effective treatments. Patients had to rely on frequent transfusions of red blood cells and 50% died within 10 years of diagnosis, but the last few years have transformed this orphan disease state into a hotbed of therapeutic activity. A new treatment targeting a different level of the complement cascade was approved in 2021 and numerous other therapies are now in late-phase development.

Join PNH expert Dr. Ilene Weitz as she takes a deep dive into very recently published insights into the pathogenesis, treatment options, and management of this poorly understood hematologic disorder.


Ilene Ceil Weitz, MD
Professor of Clinical Medicine
Keck School of Medicine
University of Southern California
Los Angeles, California

Release date: June 15, 2023
Expiration date: June 15, 2024
Estimated time to complete activity: 45 minutes

This educational activity is designed for hematologists, oncologists, and other healthcare providers involved in managing care for patients with PNH, including primary care physicians, physician assistants, and nurse practitioners.


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

  • Explain the pathogenesis of PNH and the importance of targeting the proximal as well as terminal complement system pathways
  • Evaluate recently released efficacy and safety findings from clinical trials of new and emerging treatments for PNH
  • Integrate a patient-focused strategy when selecting and monitoring treatments for PNH

There are no fees for participating and receiving CME credit for this activity. During the period of June 15, 2023 through June 15, 2024 participants must:

  • Read the learning objectives
  • Complete the pretest
  • Study the educational activity
  • Complete the posttest and the evaluation form

A statement of credit will be issued only upon receipt of a completed activity evaluation form and a completed posttest with a score of 60% or better.




Integrity Continuing Education, Inc. is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.


Integrity Continuing Education, Inc. designates this enduring material for a maximum of 0.75 AMA PRA Category 1 CreditTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity.


For information about ACCME accreditation of this activity, please contact Integrity Continuing Education, Inc. at (855) 835-4004 or cme@integrityce.com.


Integrity Continuing Education, Inc. requires instructors, planners, managers, and other individuals who are in a position to control the content of this activity to disclose any financial relationships with ineligible companies. All identified relevant financial relationships are thoroughly vetted by Integrity Continuing Education, Inc. for fair balance, scientific objectivity of studies mentioned in the materials or used as the basis for content, and appropriateness of patient care recommendations. All relevant financial relationships have been mitigated.

The following faculty/planners reported the financial relationships or relationships to products or devices they have with ineligible companies related to the content of this CME activity:

Ilene Ceil Weitz, MD
Consultant: Alexion Pharmaceuticals, Apellis-Sobi Pharmaceuticals, Novartis, Sanofi Pharmaceuticals
Speakers’ Bureau: Alexion Pharmaceuticals

The Integrity Continuing Education, Inc. planners and managers do not have any financial relationships or relationships to products or devices with ineligible companies.


This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. Integrity Continuing Education, Inc. does not recommend the use of any agent outside of the labeled indications.

The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of any organization associated with this activity. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.


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The information provided at this activity is for continuing education purposes only and is not meant to substitute for the independent medical judgment of a physician relative to diagnostic and treatment options of a specific patient’s medical condition.

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