Accreditation/Credit Designation

Physicians' Education Resource®, LLC (PER®), is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education (CME) for physicians.

Physicians' Education Resource®, LLC, designates this enduring material for a maximum of 1.5 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Acknowledgment of Commercial Support

This activity is supported by educational grants from Ablynx, a Sanofi Company; Apellis Pharmaceuticals; Dova Pharmaceuticals, Inc; Rigel Pharmaceuticals, Inc; and Sanofi Genzyme.

Community Practice Connections™: “D” is for Diagnosis: Solving the Mystery of Rare Hematologic Disorders

Release Date: February 28, 2019
Expiration Date: February 28, 2020
Media: Internet - based

Activity Overview

Hematologists and primary care physicians play a pivotal role in the diagnosis of disorders and management of patients who present with hematologic symptoms of unknown origin. This program is designed to raise your awareness of the rare hematologic disorders Gaucher disease, paroxysmal nocturnal hemoglobinuria, thrombotic thrombocytopenic purpura, and immune thrombocytopenia. Through a review of the diagnostic and treatment paradigms for each disease, you will improve your ability to provide accurate and early diagnosis and appropriate therapy for your patients with these conditions. This activity will also highlight emerging options for treating patients with these rare blood disorders, clinical data, and expert interviews to help place emerging data into the context of clinical practice. The faculty share their best practices and review new and emerging strategies, and place novel data in the context of existing diagnostic and therapeutic algorithms.

Acknowledgement of Commercial Support

This activity is supported by educational grants from Ablynx, a Sanofi Company; Apellis Pharmaceuticals; Dova Pharmaceuticals, Inc; Rigel Pharmaceuticals, Inc; and Sanofi Genzyme.

Instructions for This Activity and Receiving Credit

  • You will need to log in to participate in the activity.
  • Each presentation may contain an interactive question(s). You may move forward through the presentation; however, you may not go back to change answers or review audio files/content until you finish the presentation.
  • At the end of the activity, educational content/audio files will be available for your reference.
  • In order to receive a CME certificate, you must complete the activity.
  • Complete the Posttest and pass with a score of 70% or higher, complete the Evaluation, and then click on “Request for Credit.” You may immediately download a CME certificate upon completion of these steps.

Target Audience

This activity is intended for medical oncologists, hematologists, and other oncology healthcare professionals interested in understanding the current treatment guidelines and diagnostic strategies for patients who present with hematologic symptoms of unknown origin. Fellows, researchers, nurses, nurse practitioners, physician assistants, and other healthcare professionals are also invited to participate.

Learning Objectives

Upon successful completion of this activity, you should be better prepared to:

  1. Illustrate the importance of rigorous differential diagnoses for patients with rare hematologic disorders
  2. Detail the pathophysiology of multiple rare diseases with hematologic implications and how underlying biology informs treatment decisions
  3. Describe established evidence concerning treatment strategies to care for patients with thrombocytopenia or thrombosis resulting from rare blood disorders
  4. Incorporate methods to proactively mitigate the impact of disease- and treatment-related adverse events resulting from treating rare blood disorders

Faculty, Staff, and Planners' Disclosures


Pramod K. Mistry
Pramod K. Mistry, MD, PhD, FRCP, FAASLD
Professor of Medicine and Pediatrics
Professor of Cellular & Molecular Physiology
Department of Internal Medicine (Digestive Diseases)
Yale University School of Medicine
New Haven, CT

Disclosures: Grant/Research Support: Sanofi Genzyme; Consultant: Sanofi Genzyme; Other: Sanofi Genzyme

Nichola Cooper MA, MBBS, MD, FRCP
Senior Lecturer and Honorary Consultant Haematologist
Hammersmith Hospital
Imperial College London
London, United Kingdom

Disclosures: Grant/Research Support: Novartis sponsorship for educational preceptorship; Consultant: Advisory board for Novartis and Amgen; Other: Speaking at educational events, Novartis, and Amgen.

Peter Hillmen
Peter Hillmen, MB ChB, PhD
Professor of Experimental Haematology
Leeds Institute of Cancer & Pathology
Honorary Consultant Haematologist
Leeds Teaching Hospitals NHS Trust
Leeds, United Kingdom

Disclosures: Grant/Research Support: Apellis Pharmaceuticals; Other: Chair of PNH Global Registry and International Ad Board for Alexion.

Marie Scully
Marie Scully, MD
Clinical Lead, Haemopathology and Blood Transfusion
Department of Haematology
University College London Hospitals NHS Trust
London, United Kingdom

Disclosures: Grant/Research Support: Baxalta, Shire; Speaker’s Bureau: Ablynx, Alexion, Shire.

Neal Weinreb
Neal Weinreb, MD, FACP
Regional Coordinator and Chair
International Collaborative Gaucher Group
UHealth Sylvester Cancer Center
University of Miami
Coral Springs, FL

Disclosures: Consultant: Genzyme, a Sanofi Company; Shire HGT; Pfizer Corporation; Other: Medical advisory boards, Genzyme and Pfizer.

The staff of PER® have no relevant financial relationships with commercial interests to disclose.

Disclosure Policy and Resolution of Conflicts of Interest (COI)

As a sponsor accredited by the ACCME, it is the policy of PER® to ensure fair balance, independence, objectivity, and scientific rigor in all of its CME activities. In compliance with ACCME guidelines, PER® requires everyone who is in a position to control the content of a CME activity to disclose all relevant financial relationships with commercial interests. The ACCME defines “relevant financial relationships” as financial relationships in any amount occurring within the past 12 months that creates a COI.

Additionally, PER® is required by ACCME to resolve all COI. PER® has identified and resolved all COI prior to the start of this activity by using a multistep process.

Off-Label Disclosure and Disclaimer

This CME activity may or may not discuss investigational, unapproved, or off-label use of drugs. Participants are advised to consult prescribing information for any products discussed. The information provided in this CME activity is for continuing medical and nursing education purposes only, and is not meant to substitute for the independent clinical judgment of a physician relative to diagnostic, treatment, or management options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER® or any of the companies that provided commercial support for this activity.

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