Advances in Therapeutic Management of PNH

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Series Description:

This educational series will assist clinicians in improving the care of patients with paroxysmal nocturnal hemoglobinuria (PNH) and other complement mediated hematologic diseases. After this series, clinicians will be aware of the pathophysiologic mechanisms of PNH, the application and limitations of currently available therapies, and the impact of PNH and its treatments on patients’ clinical outcomes including quality of life. Lastly, this educational series will present the latest data on emerging treatment options, and how these treatments may impact clinical care.

Episode Description:

Module 1: This module reviews the three complement mediated pathways (classic, lectin, and alternative) and how impairment in the complement pathway contributes to the pathophysiologic issues in PNH.

Module 2: This module reviews the prevalence of PNH, sign and symptoms associated with this disease, and clinical indications for testing and diagnosis.

Module 3: This module highlights the current emerging therapies for PNH and the impact it has on patients and clinical care.

Learning Objectives:

  1. Explain how impairment in the complement pathway contributes to the pathophysiologic issues in PNH
  2. Identify how current and emerging therapies target the complement pathway in patients with PNH
  3. Outline safety and efficacy of new and emerging therapies for PNH
  4. Associate potential clinical impact of emerging therapies in addressing limitations of current treatments in patients with PNH

Moderators and/or Speakers, Institutions, and Credentials:

Module 1:
Caroline Piatek, MD
Associate Professor
Jane Anne Nohl Division of Hematology
Keck School of Medicine
University of Southern California
Los Angeles, CA

Module 2:
David Araten, MD
Assistant Professor
Department of Medicine
NYU Grossman School of Medicine
New York, NY

Module 3:
Gloria Gerber, MD
Assistant Professor
Division of
Johns Hopkins University
Baltimore, MD

Target Audience: hematologists, hematologists/oncologists, nurse practitioners, physician associates, and other members of the health care team who are involved in the care of patients with paroxysmal nocturnal hemoglobinuria (PNH)


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