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CME: Transthyretin amyloidosis (ATTR) and its complications: Optimizing management strategies


AN ACCREDITED UP-TO-DATE VERSION OF THIS TOPIC CAN BE FOUND AT: Updates in the management of polyneuropathy of hereditary transthyretin amyloidosis (ATTR): Treating the condition head-on

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 is passed to offspring through autosomal dominant inheritance, whereas the wild type variant occurs independently of genetic mutations. 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

This activity is free of charge.

Release Date: June 13, 2020 -- Expiration Date: June 13, 2022

Faculty: Robert Chilton, MD


Introduction, disclosures

Introduction content: Cursory refresher and review of ATTR

Epidemiology and etiology of ATTR: U.S. & world statistics, demographics, genetics

  • ATTR symptomology and diagnosis
  • Pathophysiology: Protein malformation precipitated by TTR destabilization
  • Organ involvement and complications: cardiologic, neurologic, musculoskeletal
  • Diagnostic tests
  • Tissue biopsy
  • Bone radiotracer scintigraphy
  • Nerve conduction studies
  • Autonomic function testing
  • Serum studies
  • Imaging
  • Staging
  • Differential diagnoses
  • Underdiagnosis and barriers to diagnosis and strategies for addressing them

Treatment in patients with TRR amyloidosis

  • Pharmacotherapy: Patisiran, inotersen, symptomatic management
  • Drugs in development: ALN-TTRsc02. and AKCEA-TTR-LRx
  • Management of cardiomyopathy and polyneuropathy: Roles of diflunisal, tafamidis, beta blockers, diuretics
  • Emerging strategies and trends in recent years
  • Promoting active patient involvement in the care plan
  • Best practice: Putting it all together
  • 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 epidemiology of ATTR, including its subtypes.
  • Compare and contrast invasive and non-invasive methods in the diagnosis of ATTR’s complications.
  • Describe challenges in the management of ATTR, including under-diagnosis and challenges patients face in understanding the diagnosis and treatment of ATTR.
  • Describe available and emerging therapies for ATTR and design an appropriate therapeutic plan including active patient involvement.


ACCME Activity #0


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: Robert Chilton, MD, Professor of Medicine, Associate Program Director for Interventional Cardiology, Director Catheterization Lab/Clinical Proteomics, University of Texas, has no relevant financial disclosures.

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

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


  • 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

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

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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