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CME: Congenital Thrombotic Thrombocytopenic Purpura (cTTP) (Upshaw-Schulman syndrome): Therapeutic Updates, Best Practices, and Emerging Therapies


Activity Description / Statement of Need:

In this online, self-learning activity:

Thrombotic thrombocytopenic purpura (TTP) is a rare blood disorder that impacts three in a million adults per year, with congenital or hereditary TTP (cTTP, also known as Upshaw-Schulman syndrome) accounting for a third of the overall incidence. The incidence of TTP rises with increasing age and the mortality rate of untreated TTP may be as high as 90%.

In recent years molecular mechanisms contributing to TTP have been identified: patients diagnosed with TTP have larger von Willebrand factor molecules (vWF) and a defective protease enzyme of A Disintegrinlike And Metalloprotease with ThromboSpondin type 1 motif 13 (ADAMTS13), which cleaves larger vWF molecules and inhibits platelet adhesion. In congenital or hereditary TTP (cTTP, also known as Upshaw-Schulman syndrome), the gene that codes of ADAMTS13 is defective and cannot properly produce the enzyme, whereas in acquired TTP, antibody production leads to downstream enzymatic deactivation.

Target Audience:

The following healthcare professionals: hematologists; physician assistants, nurse practitioners, nurses, and pharmacists who practice in hematology; and any other healthcare professionals with an interest in or who clinically encounter patients with cTTP.

This program is supported by an educational grant from Shire.

Release Date: October 02, 2018 -- Expiration Date: October 02, 2020

Faculty: Ilene Weitz, MD


Cursory refresher and review on congenital (cTTP) epidemiology and diagnosis

  • Epidemiology: statistics on prevalence
  • Pathogenesis and etiology: the genetics of cTTP [Learning Objective #1]
  • Symptomology and clinical presentation
  • cTTP: often in newborns, pregnant patients
  • Thrombosis, thrombocytopenia, purpura
  • Neurological, cardiac, renal complications
  • ADAMTS13 gene deficiency
  • Diagnostic criteria [Learning Objective #2]
    • Thrombocytopenia
    • Microangiopathic hemolytic anemia
    • Blood film test
  • Patient case(s)

Treatment of cTTP [Learning Objectives #3-5]

  • Treatment overview and goals of therapy for cTTP
  • cTTP: current practice guidelines and standard care options
  • Acute episodes [Learning Objective #3]
    • Plasma infusion for ADAMTS13 repletion, goal platelet count ≥ 150 x 103/mcL, usually within 1-2 days
    • Lack of immediate response suggestive of acquired TTP
    • Factor VIII for patients intolerant of plasma infusion
    • Emerging therapy and the evidence so far behind it: recombinant ADAMTS13 [Learning Objectives #4]
    • Routine monitoring: microvascular thromboses [Learning Objective #3]
  • Consequences of failure to achieve normal platelet counts
  • Special populations (e.g., pregnancy)
  • Role of family genetic testing
  • Barriers to care [Learning Objective #5]
  • Patient case(s) [Learning Objectives #3 & 5]

Summary, conclusions, and best practice recap

Learning Objectives

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

  • Describe the pathophysiology of cTTP such that it might inform treatment mechanisms.
  • Identify diagnostic criteria and differential diagnoses for cTTP
  • Describe current treatment standards for the management of cTTP and apply them to patient cases
  • Describe new and emerging therapies for the treatment of cTTP and the existing literature support its use in practice
  • Evaluate the likelihood cTTP in a patient case and develop a treatment plan



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: Ilene C. Weitz, M.D. Professor, University of Southern California, Los Angeles, CA has no relevent financial disclosures

Disclosures of Educational Planners: Charles Turck, PharmD has no relevent financial disclosures.

Commercial Support Disclosure: This program is supported by an educational grant from Shire.


  • 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.