Overview
How I Treat Paroxysmal Nocturnal Hemoglobinuria
Point Counterpoint Digital Activity (Clinical Debate)
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In this point-counterpoint digital activity, two expert faculty discuss and debate treatment selection for patients with PNH through case-based scenarios. Faculty review mechanisms of action, clinical trial data, and real-world evidence supporting divergent treatment strategies for the presented cases. How do the experts make their initial treatment selections? Why might they choose a C5 inhibitor or a C3 inhibitor? How do they assess response to treatment, and what do they do when treatment response is suboptimal? View the activity and find out!
This educational activity is designed for hematologists, oncologists, hospital system pharmacists, nurses, and the multidisciplinary healthcare team who are involved in the care of patients with PNH.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic stem cell disorder that renders blood cells more sensitive to the complement system and is characterized by a variety of symptoms, including hemolytic anemia, hemoglobinuria, thrombosis, and bone marrow failure. PNH has a significant clinical burden and impact on patient quality of life (QoL) due to transfusion dependence and hemolysis, a well-recognized risk factor for thrombosis that can lead to organ failure and insufficiency, and is the primary cause of mortality; however, therapeutic advances have improved survival rates. Indeed, though PNH is rare, there is extensive knowledge regarding its pathophysiology and underlying molecular defects, which supported the development of efficacious mechanism-based therapies.
Until recently, only two treatment strategies—complement component 5 (C5) blockade with either eculizumab or ravulizumab and allogeneic hematopoietic stem cell transplantation—were available for treating PNH. In 2021, the complement protein C3 inhibitor pegcetacoplan was approved to treat PNH. Eculizumab is associated with a greater than 50% reduction in transfusions and a close to 70% reduction in risk of thrombotic events and significant adverse vascular event rates, but nearly 30% of patients may experience breakthrough hemolysis due to insufficient complement inhibition. Ravulizumab has a similar safety and efficacy profile to eculizumab with fewer breakthrough hemolysis episodes and less-frequent dosing, which may contribute to greater patient satisfaction and improved QoL. The complement protein C3 inhibitor pegcetacoplan significantly improved hemoglobin levels and reduced the need for transfusion in a randomized comparison with eculizumab, creating another therapeutic option for patients with PNH. However, formal recommendations for treatment selection are lacking, and clinicians could benefit from expert guidance in this area to assist in the optimal therapeutic strategy for individualized patient care.
Upon completion of this activity, learners will be able to:
• Outline the clinical manifestations of PNH and their role in guiding initial treatment selection and changes in therapy Review the pharmacologic profiles, efficacy, and safety of available complement inhibitors and summarize their use in clinical practice
• Discuss recent advances in PNH therapeutic approaches
• Formulate personalized treatment strategies considerate of patient risks, characteristics, complications, and preferences
• Outline the clinical manifestations of PNH and their role in guiding initial treatment selection and changes in therapy Review the pharmacologic profiles, efficacy, and safety of available complement inhibitors and summarize their use in clinical practice
• Discuss recent advances in PNH therapeutic approaches
• Formulate personalized treatment strategies considerate of patient risks, characteristics, complications, and preferences
This educational activity is presented in collaboration with Rush University Medical Center and Academy for Continued Healthcare Learning (ACHL).
Supported educational grants from Alexion Pharmaceuticals and Apellis Pharmaceuticals, Inc.
Jamile Shammo, MD, FASCP, FACP
Marjorie C. Barnett-Dr. Hau C. Kwaan Professor and Chair
Division of Hematology/Oncology
Northwestern University Feinberg School of Medicine
Chicago, IL
Marjorie C. Barnett-Dr. Hau C. Kwaan Professor and Chair
Division of Hematology/Oncology
Northwestern University Feinberg School of Medicine
Chicago, IL
Carlos de Castro, MD
Professor of Medicine
Duke University School of Medicine and Duke Cancer Institute
Durham, NC
It is the policy of Rush University Medical Center and the Office of Interprofessional Continuing Education to ensure that its CE activities are independent, free of commercial bias and beyond the control of persons or organizations with an economic interest in influencing the content of CE. All individuals in control of educational content must disclose all financial relationships with ineligible companies in the past 24 months. An ineligible company is an entity whose primary business is producing, marketing, selling, re-selling or distributing healthcare products used by or on patients. All conflicts of interest, including potential ones, for individuals in control of educational content are mitigated prior to the planning, implementation, or evaluation of the continuing education activity.
The IPCE identifies the presence or absence of relevant financial relationships for all individuals in control of content including but not limited to planning committee members, course directors, invited presenters/authors, and staff through a standardized disclosure form. If a financial relationship is identified for the person in control of content, conflict mitigation strategies will be used to mitigate the financial relationship before they assume their role.
Jamile Shammo, MD
Research grant from: Abbvie, Alexion-Astra Zeneca, Bristol Meyers Squibb, Incyte, CTI BioPharma, Kartos Therapeutics
Consulting to: Alexion-Astra Zeneca, Bristol Meyers Squibb, Sanofi, Incyte, MJH life sciences,
Member, Speakers' Bureau for: Alexion-Astra Zeneca, Bristol Meyers Squibb, Incyte
Carlos de Castro, MD
Member, Speakers Bureau for: Alexion, Appellis Pharmaceuticals, Bristol Myers Suibb
Consultant to: Novartis, Regeneron
Shivi Jain, MD
No financial relationships to disclose
Kai Ting Wu, MSN
No financial relationships to disclose
Peter Wu, PharmD
No financial relationships to disclose
Discussion of Off-Label, Investigational, or Experimental Drug/Device Use: Danicopan and iptacopan are investigational therapies for the treatment of PNH and are not FDA-approved.
ACHL staff members, Rush Interprofessional Continuing Education staff members and others involved with the planning, development, and review of the content for this activity have no relevant affiliations or financial relationships with ineligible companies to disclose.
The content for this activity was developed independently of any ineligible companies. All materials are included with permission. The opinions expressed are those of the faculty and are not to be construed as those of the publisher or grantor(s).
This educational activity was planned and produced in accordance with the ACCME Standards for Integrity and Independence in Accredited Continuing Education. Recommendations involving clinical medicine in a continuing medical education (CME/CE) activity must be based on evidence that is accepted within the profession of medicine as adequate justification for their indications and contraindications in the care of patients. All scientific research referred to, reported, or used in CME/CE in support or justification of a patient care recommendation must conform to the generally accepted standards of experimental design, data collection, and analysis.
Unapproved Uses of Drugs/Devices: In accordance with requirements of the FDA, the audience is advised that information presented in this continuing medical education activity may contain references to unlabeled or unapproved uses of drugs or devices. Please refer to the FDA approved package insert for each drug/device for full prescribing/utilization information.
Shivi Jain, MD
Rush University Medical Center
Rush University Medical Center
Kait Ting Wu, NP
Rush University Medical Center
Peter Wu, PharmD
Rush University Medical Center
This activity will take approximately 60 minutes to complete. To receive credit, learners are required to complete the pretest, view the online activity, and complete the posttest and evaluation. To receive credit, 75% must be achieved on the posttest. A certificate will be immediately available. There is no fee to participate in the activity or for the generation of the certificate.
CPE credit will be submitted to the CPE Monitor® on the first business day of each month.
For questions, contact Karen Catino at kcatino@achlcme.org
Rush University Medical Center is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.
This activity is being presented without bias and with commercial support.
Rush University Medical Center designates this internet enduring material for a maximum of 1.0 AMA PRA Category 1 Credit™. Physicians should claim only credit commensurate with the extent of their participation in the activity.
Rush University Medical Center designates this internet enduring material for a maximum of (1) nursing contact hour.
Rush University Medical Center designates this knowledge-based Internet Enduring Material for a maximum of 1.0 contact hour for pharmacists.
Rush University Medical Center designates this knowledge-based Internet Enduring Material for a maximum of 1.0 contact hour for pharmacists. ACPE Universal Activity Number: JA0000275-0000-23-073-HO1-P
1. Active Internet connection (DSL or Cable). Dial-up connection will have constant buffering problem.
2. Compatible with Windows PC and MAC (256 MB of RAM or higher)
3. Activity is best viewed on Internet Explorer 9.0 or higher, Safari 5.0 or higher and Firefox 29.0 or higher
4. Adobe Flash Player 12.0 (or higher). Click here to download Adobe Flash Player for free.
5. Adobe Reader to print certificate. Click here to download Adobe Reader for free.
6. Allow ActiveX controls to run on your computer: If the yellow strip appears on the top of your web browser while running the Webcast, right click on it and select Allow blocked contents to run.
7. Turn the Pop-up blocker off: On the Tools menu, point to Pop-up Blocker, and then click Turn Off Pop-up Blocker
2. Compatible with Windows PC and MAC (256 MB of RAM or higher)
3. Activity is best viewed on Internet Explorer 9.0 or higher, Safari 5.0 or higher and Firefox 29.0 or higher
4. Adobe Flash Player 12.0 (or higher). Click here to download Adobe Flash Player for free.
5. Adobe Reader to print certificate. Click here to download Adobe Reader for free.
6. Allow ActiveX controls to run on your computer: If the yellow strip appears on the top of your web browser while running the Webcast, right click on it and select Allow blocked contents to run.
7. Turn the Pop-up blocker off: On the Tools menu, point to Pop-up Blocker, and then click Turn Off Pop-up Blocker
