In this online, self-learning activity:
Gaucher disease (GD) is characterized by a deficiency of the lysosomal enzyme glucocerebrosidase, resulting in the accumulation of sphingolipids throughout the body but most manifesting prominently in the bones. GD is subcategorized based on clinical features: type 1 GD is the non-neuronopathic form and affects mainly the inner organs, while types 2 and 3 are the acute and sub-acute neuropathic forms, whose pathology manifests predominantly within central nervous system. GD has an estimated prevalence of 0.70 to 1.75 per 100 000 in the general population, it affects individuals of Ashkenazi Jewish heritage in significantly higher numbers. One of the first of GD’s complications is the chronic anemia and a persistent bleeding risk. Another is the hepatosplenomegaly, which may be a part of the initial clinical presentation, as may the anatomical abnormalities of bone deformities and stunted growth.
This learning activity has been designed to bring healthcare professionals’ knowledge of the strategies for treatment and management of GD up to date and to improve their competence and performance in treating it.
The following healthcare professionals: Pediatricians, neurologists, endocrinologists, and primary care physicians; physician assistants and nurse practitioners in those areas of specialty; pharmacists who practice in specialty pharmacies that treat patients with rare diseases; and any other healthcare professionals with an interest in or who may clinically encounter patients with GD.
Commercial Support Disclosure: This program is supported by an educational grant from Takeda.
This activity is free of charge.
Release Date: April 06, 2022 -- Expiration Date: April 06, 2024
Faculty: Neal J. Weinreb, MD, FACP
Faculty introduction, disclosures |
Primer and updates on GD’s epidemiology, pathophysiology, presentation, complications, and diagnosis · Epidemiology, pathophysiology, risk factors · Complications: Hematologic, bone · Classification and clinical features of the disease by subtype · Diagnosis and risk of misdiagnosis |
Treatment and barriers to care · Therapeutic goals based on organ systems affected · ERT: Indications, modifications during therapy (dose changes or interruptions), efficacy based on the evidence in neuronopathic and non- neuronopathic disease, practical considerations · SRT: Place in therapy, efficacy and clinical trial evidence, practical considerations · Investigational glucosylceramide synthase inhibitors · Patient case(s) |
Summary, conclusions, and best practice recap |
By the end of the session the participant will be able to:
ACCME Activity #202280751
This activity has been planned and implemented in accordance with the accreditation requirements 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 1.25 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 1.25 MOC points in the American Board of Internal Medicine's (ABIM) 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 ABIM MOC credit.
ABIM MOC Credit Type: Medical Knowledge
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 1.25 contact hours (0.125 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 #0574-0000-22-025-H01-P. This is an Application (A)-type activity.
Pharmacists: You 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.
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.
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: Neal Weinreb, MD, FACP, Voluntary Associate Professor of Human Genetics and Medicine (Hematology) University of Miami Miller School of Medicine, Miami, FL, discloses that he serves on Medical/Scientific Advisory Board Member and/or Consultant for Genzyme-Sanofi, Takeda, Shire, Aurobio, Aeglea, and Pfizer for which he has received financial compensation.
Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, President of ScientiaCME, has no relevant financial disclosures.
Faculty will not discuss off-label use 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 Takeda.
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Present and emerging strategies in the medical management of Hunter syndrome