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CME: Paroxysmal nocturnal hemoglobinuria (PNH): Best practices and effective management


Activity Description / Statement of Need:

In this online, self-learning activity:

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired, hematopoietic stem cell disorder characterized by complement-mediated destruction and loss of erythrocytes and the eponymous clinical manifestation of pink-red discoloration of the urine due to presence of hemoglobin. PNH is a rare disorder with a reported incidence of clinically significant disease of between 1 to 2 cases per million. However, it is possible that this range may be underestimated, as a subset of patients remain undiagnosed. PNH occurs worldwide with no known specific ethnic or geographic distribution patterns.

Approximately 40% of patients with PNH saw at least five or more specialists before receiving a diagnosis. PNH diagnosis is complicated by the rarity of the disorder and the nonspecific findings, meriting continuing healthcare educational programming.

Target Audience:

The following healthcare professionals: Hematologists; physician assistants, nurse practitioners, and pharmacists specializing in hematology and transplant medicine; and any other healthcare professionals with an interest in or who may clinically encounter patients with PNH.

Commercial Support Disclosure: This program is supported by educational grants from Biocryst and Apellis.

This activity is free of charge.

Release Date: January 28, 2022 -- Expiration Date: January 28, 2024

Faculty: Carlos De Castro, MD


PNH Overview

  • Epidemiology: Prevalence and incidence
  • Clinical presentation: Physical exam, laboratory evidence
  • Differential diagnosis, diagnosis, classification
    • Common presenting symptoms
    • Risks of short- and long-term complications
    • Prognosis


  • Currently available therapy
    • Complement inhibitors: Available agents; mechanism of action; dosing and formulations; evidence
    • HSCT: When to consider
  • Investigational therapies: Additional complement inhibitors and targets in the complement cascade; factors B and D inhibitors
  • Supportive care
  • Patient case(s)


Learning Objectives

By the end of the session the participant will be able to:

  • Describe the common clinical presentation(s) of PNH.
  • Describe challenges to the successful identification of patients with PNH and solutions to overcoming diagnostic barriers.
  • Identify the treatment modalities presently available and emerging therapies for management of PNH and formulate a treatment plan based on patient case details.
  • Compare the mechanisms of action, dose schedules, and efficacy data for novel and emerging treatment options for PNH.


ACCME Activity #201824508


Faculty Disclosure and Resolution of COI

As a provider of continuing medical education, it is the policy of ScientiaCME to ensure balance, independence, objectivity, and scientific rigor in all of its educational activities. In accordance with this policy, faculty and educational planners must disclose any significant relationships with commercial interests whose products or devices may be mentioned in faculty presentations, and any relationships with the commercial supporter of the activity. The intent of this disclosure is to provide the intended audience with information on which they can make their own judgments. Additionally, in the event a conflict of interest (COI) does exist, it is the policy of ScientiaCME to ensure that the COI is resolved in order to ensure the integrity of the CME activity. For this CME activity, any COI has been resolved thru content review ScientiaCME.

Faculty Disclosure:

Carlos M. De Castro, MD, Professor of Medicine, Duke University, has received financial compensation as a speaker and / or consultant for Alexion, Apellis, Novartis and Biocryst.

Faculty DOES plan on discussing off-label uses of a commercial product.

Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, President of ScientiaCME, has no relevant financial disclosures.

Commercial Support Disclosure: This program is supported by educational grants from Biocryst and Apellis.

All relevant financial relationships have been mitigated.

ScientiaCME adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CE activity, including faculty, planners, reviewers or others are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity.


  • Read the learning objectives above
  • Take the Pre-Test (optional). Completion of the pre-test will help us evaluate the knowledge gained by participating in this CME activity.
  • View the online activity. You may view this is in more than one session, and may pause or repeat any portion of the presentation if you need to.
  • Minimum participation threshold: Take the post-test. A score of 70% or higher is required to pass and proceed to the activity evaluation.
  • Complete the activity evaluation and CME registration. A CE certificate will be emailed to you immediately.

Cultural/Linguistic Competence & Health Disparities

System Requirements

Windows 7 or above
Internet Explorer 8
*Adobe Acrobat Reader
Mac OS 10.2.8
Safari or Chrome or Firefox
*Adobe Acrobat Reader
Internet Explorer is not supported on the Macintosh

*Required to view Printable PDF Version

Perform Pre-Test (optional)

Please take a few minutes to participate in the optional pre-test. It will help us measure the knowledge gained by participating in this activity.

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