PNH: Real-world Experience
Jointly Provided by


Supported by an educational grant from Alexion Pharmaceuticals, Inc.
Start date: July 15, 2022
End date: July 15, 2023
Estimated time to complete: 0.25 hours
Activity Description
This accredited CME activity, led by Satheesh Chonat, MD, Assistant Professor at Emory University School of Medicine and hematologist-oncologists at the Pediatric Hematology Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, highlights the latest real world data focused on paroxysmal nocturnal hemoglobinuria (PNH). Dr Chonat also provides expert analysis of the data’s clinical relevance for members of the care team to help them manage patients with PNH they may encounter with this rare condition. PNH is a rare, acquired blood disease characterized by hemolytic anemia, bone marrow failure, thrombosis, and fatigue.
Activity Faculty
Satheesh Chonat, MD
Assistant Professor
Emory University School of Medicine
Hematologist-oncologist
Pediatric Hematology Aflac Cancer and Blood Disorders Center
Children’s Healthcare of Atlanta
Target Audience
This activity has been designed to meet the educational needs of physicians specializing in hematology, neurology, nephrology, immunology, gastroenterology, respiratory, and cardiology. Other members of the care team may also participate.
After participating in the activity, learners should be better able to:
- Appraise the latest real-world data presented to better manage patients with PNH and how it can improve clinical care.

Accreditation and Credit Designation
In support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. 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.
Physicians
American Academy of CME, Inc., designates this enduring material for a maximum of 0.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Disclosure Statement
According to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated.
Disclosure of relevant financial relationships are as follows:
Faculty Educators
Dr. Chonat discloses the following relevant financial relationships with ineligible companies:
- Advisory Board/Consultant: Agios, Alexion, Novartis, Takeda, Forma, Daiichi Sankyo
- Grant/Research Support: Global Blood Therapeutics, Novartis
Planners for this activity have no relevant financial relationships with any ineligible companies.
This activity will review off-label or investigational information.
The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information.
Method of Participation
There are no fees to participate in the activity. Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the pre and post-program assessments. Your certificate will be emailed to you in within 30 days.
Hardware/Software Requirements
Windows Requirements: • Operating system: Windows XP Service Pack 2 or later • Browser: Internet Explorer 7 or later, Mozilla Firefox 2.5 or later • Internet connection: DSL, cable modem, or other high-speed connection
Macintosh Requirements: • Operating system: Mac OS X v10.3 or later • Browser: Mozilla Firefox 2.5 or later • Internet connection: DSL, cable modem, or other high-speed connection
Privacy
For more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm. For more information about CheckRare’s privacy policy, please access https://checkrare.com/privacy/
Contact
For any questions, please contact: CEServices@academycme.org
Copyright
© 2022. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare CE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s)
PNH: Real-world Experience
References
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Socie G, et al. Changing prognosis in paroxysmal nocturnal haemoglobinuria disease subcategories: an analysis of the International PNH Registry. Intern Med J. 2016; 46: 1044-1053.
Hill A, et al. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primers. 2017; 3: 17028.
Ware, et al. Paroxysmal nocturnal hemoglobinuria with onset in childhood and adolescence. New Engl J Med. 1991; 325: 991-996.
Young NS, Maciejewski JP. Genetic and environmental effects in paroxysmal nocturnal hemoglobinuria: this little PIG-A goes "Why? Why? Why? J Clin Investig. 2000; 106: 637-641.
Karadimitris A, et al. Abnormal T-cell repertoire is consistent with immune process underlying the pathogenesis of paroxysmal nocturnal hemoglobinuria. Blood. 2000; 96: 2613-2620.
Urbano-Ispizua A, et al. Different clinical characteristics of paroxysmal nocturnal hemoglobinuria in pediatric and adult patients. Haematologica 2017; 102: e76-e79.
Parker C, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005; 106: 3699-3709.
Parker CJ. Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program. 2016; 2016: 208-216.
Mercuri A, et al. A retrospective study of paroxysmal nocturnal hemoglobinuria in pediatric and adolescent patients. Blood Cells Mol Dis. 2017; 64: 45–50.
Reiss UM, et al. Efficacy and safety of eculizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria. Pediatr Blood Cancer 2014;61:1544–1550.
Schrezenmeier H et al. Baseline clinical characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry. Ann Hematol. 2020;99(7):1505-1514.
Cella D et al. Clinically Important Difference for the FACIT-Fatigue Scale in Paroxysmal Nocturnal Hemoglobinuria: A Derivation from International PNH Registry Patient Data. Blood. 2021; 138 (supply 1): Abstract 1952.
Terriou L et al. Long-Term Survival Benefit of Eculizumab Treatment in Patients with Paroxysmal Nocturnal Hemoglobinuria: Data from the International PNH Registry. Blood. 2021; 138 (supply 1): Abstract 2188.
Yeh M et al. Changes in Hemoglobin Measures Observed in PNH Patients Treated with Both C5 Inhibitors Ravulizumab and Eculizumab: Real-World Evidence from a US-Based EMR Network. Blood. 2021; 138 (supply 1): Abstract 1112.
Chavez EA et al. Total Oral Anticoagulation for Classic PNH As Primary Prophylaxis Strategy in a Complement Inhibitor Restricted Scenario: A Retrospective Analysis. Blood. 2021; 138 (supply 1): Abstract 1118.