CME: Present and emerging strategies in the medical management of Hunter syndrome
Activity Description / Statement of Need:
Mucopolysaccharidoses (MPS) are a group of genetic diseases characterized by a deficiency of lysosomal enzymes responsible for the hydrolysis of glycosaminoglycans (GAGs), whose manifold biological roles contribute to a variety of clinical manifestations in patients presenting with MPS. MPS has seven different subcategories, of which Hunter syndrome, an X-linked recessive genetic disease, is MPS II. The syndrome is characterized by a deficiency in iduronate 2-sulfatase, which results in relatively high levels of the GAGs heparan and dermatan sulfate, leading to physical signs similar to MPS I, with the addition of aggressive behavior and developmental delay. The major complications of Hunter syndrome have to do with its widespread and varied symptoms. The nature of this disease is that multiple organ systems are impacted simultaneously, considerably decreasing patient quality of life. The most common symptoms are facial disfigurements, hepatosplenomegaly, and skeletal joint stiffness, but presentation can fluctuate widely between patients.
This activity identifies the critical components of the management process and offer solutions to close gaps in diagnosis and care, with the ultimate goals of improvement of Hunter syndrome management, treatment adherence, and health and cost outcomes.
Target Audience:
Pediatricians, neurologists, endocrinologists, and primary care physicians; physician assistants, nurse practitioners, and pharmacists; and any other HCPs with an interest in or who may clinically encounter patients with Hunter syndrome.
This program is supported by an educational grant from Takeda.
This activity is free of charge.
Release Date: March 24, 2022 -- Expiration Date: March 24, 2024
Faculty: Natalia Gomez-Ospina, MD, PhD
Agenda
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Faculty introduction |
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Introductory content: review of Hunter syndrome · Epidemiology: statistics (global prevalence) · MPS risk and causes o Pre-natal testing · Pathophysiology, clinical presentation, disease course, and complications · Diagnosis and the impact of late diagnosis or misdiagnosis o Urine GAG analysis o Enzyme test · Patient case(s) |
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Treatment · Enzyme replacement therapy, including clinical data · Physical therapy: role in patient care · Emerging treatment options o Gene therapy o Enzyme replacement therapy · Potential barriers to treatment and solutions, including patient access programs · Patient case(s) |
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Summary, conclusions, and best practice recap |
Learning Objectives
By the end of the session the participant will be able to:
- Describe the risks and consequences of mis- and underdiagnosis of Hunter syndrome
- Recall strategies to improve recognition and diagnosis of Hunter syndrome
- Discuss present approaches to treating Hunter syndrome
- Formulate a care plan in the treatment of a patient with Hunter syndrome
Accreditation
ACCME Activity #202281720
This activity has been planned and implemented in accordance with the Essential Areas and Policies of the Accreditation Council for Continuing Medical Education through ScientiaCME. ScientiaCME is accredited by the 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.
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 th
e 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's (ABP) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABP MOC credit.
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 (0.1 CEUs) 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 # 0574-0000-22-018-H01-P. This is an Knowledge (K)-type activity.
Nurse Practitioners (NPs): The American Academy of Nurse Practitioners accepts AMA PRA Category 1 Credit(s)™ from organizations accredited by the ACCME. ScientiaCME will provide NPs who successfully complete each activity with a certificate of participation indicating that the activity was designated for AMA PRA Category 1 Credit(s)™.
Physician Assistants: The American Academy of Physician Assistants accepts AMA PRA Category 1 Credit(s)™ from organizations accredited by the ACCME.
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: Natalia Gomez‐Ospina, MD, PhD, Assistant Professor, Department of Pediatrics, Division of Medical Genetics, and Division of Stem Cell and Regenerative Medicine, Stanford University, has received financial compensation from Graphite Bio and Codexis for consulting, research and speaker. She is also an employee of ImmuneAI.
Faculty WILL discuss off-label use of a commercial product.
Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, CEO of ScientiaCME, has no relevant financial disclosures.
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 Takeda.
Instructions
- Read the learning objectives above
- Take the Pre-Test (optional). Completion of the pre-test will help us evaluate the knowedge 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.
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
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.
Additional Courses That Are Related To This Activity
Treatment strategies in Hunter Syndrome
Child and adolescent Crohn’s disease: Updates in medical and nutritional strategies