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CME: Setting it straight: transformative therapeutics in the management of sickle cell disease

Activity Description / Statement of Need:

In this online, self-learning activity:

Sickle cell disease (SCD) is the most common monogenic blood disorder, affecting millions of people worldwide and approximately 100,000 Americans. Although it may be found in various areas of the world, SCD predominantly affects individuals of African or Hispanic heritage. It is caused by the inheritance of b-globin alleles that code for hemoglobin S, resulting in an amino acid substitution in hemoglobin’s b chain and clinical disease. Patients with SCD have impaired circulation, and lysis of the erythrocytes contributes to a chronic inflammatory response, causing severe pain and less efficient oxygen delivery. The hallmark clinical features of SCD are hemolytic anemia and painful vaso-occlusive crises (VOCs), which may lead to emergency department (ED) visits, hospitalization, and potentially fatal complications. In one US study, 45% of deaths among people with SCD were related to cardiopulmonary causes, and VOCs alone have been shown to increase the risk of death by 50%. SCD also imposes other significant health burdens on patients: it is associated with a significant reduction in quality of life, malnutrition, the development of mental health disorders like depression and anxiety, loss of work or school days, frequent antibiotic use leading to higher rates of antimicrobial resistance, acute chest syndrome, and stroke.

Target Audience:

HCPs including but not limited to: hematologists and primary care physicians; physician assistants, nurse practitioners, nurses, and pharmacists who specialize in the aforementioned areas of specialty; and any other HCPs with an interest in or who may clinically encounter patients with SCD.


Commercial Support Disclosure: This program is supported by an educational grant from Agios. 

This activity is free of charge.


Release Date: November 17, 2025 -- Expiration Date: November 17, 2027

Faculty: Saranya Veluswany, MD

Agenda

Faculty Introduction, disclosures

The burden of SCD and its complications 

  • Epidemiology of SCD
  • Hospitalization and ED visits
  • Complications
  • Health burden

Practical implementation of pharmacotherapeutic treatment options 

  • Goals of therapy
  • Disease-modifying therapy
    • Hydroxyurea
    • P selectin inhibitor
    • Anti-oxidant, protein synthesis modulating amino acid therapy
  • Iron overload and chelation therapy
  • Curative treatment: gene therapy, hematopoietic stem cell transplantation, and candidate selection
  • Overall health and chronic pain management
  • Investigational therapy
    • Erythrocyte pyruvate kinase activation
    • C5 inhibition
    • Zinc finger nuclease gene-edited cell therapy
  • Management challenges and solutions 
    • Barriers to the transition from pediatric to adult care
    • Importance of the MDT
    • Related patient education
  • Patient case(s) 

Summary, conclusions, and best practice recap

Learning Objectives

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

  • Describe the genetic basis and common genotypes of sickle cell disease in USA.
  • Describe the epidemiology of sickle cell disease.
  • Discuss the acute clinical and chronic complications associated with sickling disorders.
  • Recognize the unique challenges with access to care in this population.
  • Describe the general therapeutic approaches to sickle cell disease including new and emerging therapeutics and apply that information to a patient case.

Accreditation

ACCME Activity #203331495

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

Credit Designation: ScientiaCME designates this educational activity for a maximum of 1.0 AMA PRA Category 1 Credit(s)™ toward the AMA Physician's Recognition Award. Physicians should only claim credit commensurate with the extent of their participation in the activity.

CME-MOC_badge

ABIM MOC Recognition Statement: Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.

ABIM MOC Credit Type: Medical Knowledge

ABP MOC Recognition Statement: Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 MOC points in the American Board of Pediatrics' (ABP) Maintenance of Certification (MOC) program. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABP MOC credit.

ABP MOC Credit Type: Lifelong Learning and Self-Assessment

Physicians: For maintenance of certification (MOC) points, you must enter your board certification ID # and birth date correctly.  It is the learner's responsibility to provide this information completely and accurately at the completion of the activity. Without providing it, the learner will NOT receive MOC points for this activity. By providing this data, you acknowledge that it will be shared with ACCME and the applicable certifying board. Please note: Not all activities on this site provide MOC points. If this activity does not specify that it provides MOC points in this section, then it does NOT provide MOC points. This activity provides MOC points only for ABIM.

Pharmacists

ScientiaCME is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 1.0 contact hours  of continuing pharmacy education credit. Proof of participation will be posted to your NABP CPE profile within 4 to 6 weeks to participants who have successfully completed the post-test. Participants must participate in the entire presentation and complete the course evaluation to receive continuing pharmacy education credit. ACPE #0574-0000-25-019-H01-P. This is an Application (A)-type activity. 

PharmacistsYou must enter your NABP # and birth date correctly so that proof of participation can be posted to your NABP CPE profile. It is the learner's responsibility to provide this information completely and accurately at the completion of the activity. Without providing it, the learner will NOT receive CPE credit for this activity.

Nurses: This activity is designated for up to 1.0 AMA PRA Category 1 Credit(s)™, and the American Nurses Credentialing Center (ANCC) accepts AMA PRA Category 1 Credit(s)™ to meet the continuing education renewal requirements of nursing re-certification for APRNs and RN specialty. Some state nursing boards accept AMA PRA Category 1 Credit(s)™ for re-licensure requirements, some do not. Check your state board of nursing's CE requirements before applying credit from this course to your re-licensure.

Physician Assistants: The American Academy of Physician Assistants (AAPA) accepts AMA PRA Category 1 Credit™ assigned by organizations accredited by the ACCME as satisfying Category 1 CME for National Commission on Certification of Physician Assistants (NCCPA) national certification maintenance. This activity is designated for up to 1.0 AMA PRA Category 1 Credit(s)™.

Nurse Practitioners: The American Academy of Nurse Practitioners Certification Board (AANPCB) states that continuing education providers accredited by the ACCME may provide acceptable, accredited Advanced Practice Provider content. This activity is designated for up to 1.0 AMA PRA Category 1 Credit(s)™.


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 by ScientiaCME.

Disclosures of Faculty: Saranya Veluswany, MD, Assistant Professor of Pediatrics, Children’s Hospital Los Angeles, has received financial compensation from Novo Nordisk and Pharmacosmos for grant/research support.

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

Faculty WILL NOT discuss off-label uses of a commercial product. 

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.

Commercial Support Disclosure: This program is supported by an educational grant from Agios. 

Instructions

  • 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

PC
Windows 7 or above
Internet Explorer 8
*Adobe Acrobat Reader
MAC
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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