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CME: Hereditary transthyretin amyloidosis (ATTR) treatment strategies: best practices and emerging therapies

Activity Description / Statement of Need:

In this online, self-learning activity:

Transthyretin amyloidosis (ATTR) is a progressive, multisystem, life-threatening disorder characterized by the extracellular deposition of misfolded, insoluble amyloid fibrils. As TTR is a protein present in human serum, and its role is to transport thyroxine and retinol-binding proteins, it is vital for behavior, cognition, nerve regeneration, and axonal growth. TTR itself is innately amyloidogenic even without the presence of genetic mutations. The familial variant of the disease (hATTR) is ATTR that is passed to offspring through autosomal dominant inheritance, whereas the wild type variant (wtATTR) refers to ATTR that occurs independently of genetic mutations. hATTR may present as late as mid-adulthood, but its symptoms usually start between the ages of 2 and 10 years. Left untreated, the average life expectancy of ATTR is 3 to 15 years from symptom onset.

 

 

Target Audience:

The following HCPs: neurologists, cardiologists, and hematologists; physician assistants, nurse practitioners, nurses, and pharmacists in the aforementioned areas of specialty; and any other HCPs with an interest in or who may clinically encounter patients with ATTR.


Commercial Support Disclosure: This program is supported by educational grants from Akcea Therapeutics and Alnyam

This activity is free of charge.


Release Date: March 28, 2019 -- Expiration Date: March 28, 2021

Faculty: Michelle Kaku, MD -- Deepa Gopal, MD, MS

Agenda

Introduction content: Cursory refresher and review of hATRR, including contrast wtATTR

  • Epidemiology and etiology of hATRR
    • Statistics
    • Age
    • Genetics
    • Symptomology and diagnosis of TRR amyloidosis
      • Pathophysiology
      • Onset and classification
      • Signs and symptoms
      • Differential diagnoses
      • Diagnostic tests
      • Progression of disease
      • Complications

Treatment in patients with TRR amyloidosis

  • Pharmacotherapy: patisiran, symptomatic management
  • Drugs in development
  • Emerging strategies and trends in recent years
  • Best practice: putting it all together
  • Patient case(s)

Learning Objectives

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

  • Describe the pathophysiology of ATTR such that it might inform treatment mechanisms
  • Describe available therapies used for treatment of ATTR and explain current literature supporting use of those therapies
  • Design and implement an appropriate therapeutic plan for treatment of ATTR
  • Describe future therapies currently being investigated for the treatment of ATTR

Accreditation

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 1.0 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.

PhysiciansFor maintenance of certification (MOC) credit, 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 credit for this activity. Please note: not all activities on this site provide MOC credit. If this activity does not specify that it provides MOC credit in this section, then it does NOT provide MOC credit.

Pharmacists

ScientiaCME is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmaceutical education. This program is approved for 1.0 hours (0.1 CEUs) of continuing education.  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 # 0574-0000-19-008-H01-P.  ACPE Accreditation effective 4/10/2019, expires 4/10/2021. This is a Knowledge (K)-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.

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 Disclosure: Michelle Kaku, MD, Assistant Professor of Neurology, Boston University, has no relevant financial disclosures.

Deepa Gopal, MD, MS, Assistant Professor of Cardiovascular Medicine, Boston University, has no relevant financial disclosures.

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

Commercial Support Disclosure:

 This program is supported by educational grants from Akcea Therapeutics and Alnyam

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.
  • Take the post-test
  • Complete the activity evaluation and CME registration. A CE certificate will be emailed to you immediately.

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.


Additional Courses That Are Related To This Activity

Treatment strategies in Fabry disease

Fabry disease: Updates from recent research findings

Gaucher disease: Updates from recent research findings

Hereditary transthyretin amyloidosis (ATTR) treatment strategies: best practices and emerging therapies