CME: Updates in care and improving the healthcare experience of patients with mucopolysaccharidosis I (MPS I)
ACCREDITATION EXPIRED: July 24, 2024
Activity Description / Statement of Need:
In this online, self-learning activity:
Mucopolysaccharidoses (MPS) are a group of genetic diseases characterized by a deficiency of lysosomal enzymes responsible for the hydrolysis of glycosaminoglycans (GAGs), resulting in a variety of clinical manifestations in patients presenting with MPS. MPS has several subcategories, and MPS type I (MPS I) arises from the inheritance of an alteration in the IDUA gene, which encodes for alpha-L-iduronidase. Affecting an estimated one in 100,000 live births, MPS I is categorized as either attenuated MPS I (also known as Scheie or Hurler-Scheie syndromes) or severe MPS I with cognitive impairment (also known as Hurler syndrome).
Progressive in nature, MPS I is associated with multi-organ complications and sequelae. Patients exhibit a spectrum of clinical presentations, including facial deformities, organomegaly, cognitive impairments, upper airway obstructions, skeletal deformities, and cardiomyopathy. The burden of MPS I is considerable, with reports of caregivers contributing 51 hours per week on average to help patients perform daily activities of living. Quality of life for patients and their caregivers is significantly reduced with MPS I, affecting the social, emotional, and financial well-being of a family. It is reported that parents fear for their child’s delayed language acquisition, ability to fit in amongst peers and the society, fear of the expense for the necessary care, and fear for the death of a child from obstructive sleep apnea.
This learning activity has been designed to bring HCPs’ knowledge of the strategies for treatment and management of MPS I up to date and to improve their competence and performance in treating it.
Target Audience:
The following HCPs: Pediatricians, neurologists, endocrinologists, genetic disease specialists; physician assistants, nurse practitioners, and pharmacists in the aforementioned areas of specialty; and any other HCPs with an interest in or who may clinically encounter patients with MPS I.
Commercial Support Disclosure: This program is supported by an educational grant from Sanofi.
This activity is free of charge.
Release Date: July 24, 2022 -- Expiration Date: July 24, 2024
Faculty: Paul Orchard, MD
Agenda
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Faculty introductions, disclosures |
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MPS I introduction section
o Prevalence of the disease: Statistics
· Multi-organ pathogenesis · Disease manifestations · Early disease-detection
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MPS I treatment, healthcare burden, and barriers to care · HSCT o Timing o Optimizing outcomes o Posttransplant care · Enzyme replacement therapy o Treatment recommendations o Dosing, scheduling, and frequency o Safety and efficacy
o Reduced patient and caregiver quality of life o Need for ongoing treatment o Patient and caregiver education
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Conclusions, and best practice recap |
Learning Objectives
By the end of the session the participant will be able to:
- Describe the importance of earlier treatment and diagnosis of MPS I in patients.
- Summarize present and emerging treatment options for MPS I.
- Formulate a medical plan to treat a patient with MPS I.
- List present barriers to care in the treatment of patients with MPS I.
Accreditation
ACCME Activity #202326570
ACCREDITATION FOR THIS COURSE HAS EXPIRED. YOU MAY VIEW THE PROGRAM, BUT CME / CE IS NO LONGER AVAILABLE AND NO CERTIFICATE WILL BE ISSUED.
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.
Faculty Disclosure: Paul Orchard, MD, has received financial compensation for consulting for Orchard Therapeutics, Neurogene, and Avrobio and has had clinical trials and research supported by Genzyme/Sanofi, Bluebirdbio, and Immusoft.
Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, President of ScientiaCME, has no relevant financial disclosures.
Faculty will 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 Sanofi.
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.
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.
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