Education Spotlight Sessions
The following information is preliminary and subject to change.
The Education Spotlight Sessions are intended to provide a more in-depth review on a scientific topic. Each 90-minute session will be presented once in a small-venue format on either Sunday or Monday and will include ample time for audience questions and participation.
These sessions are restricted to medical and research professionals only; no businesspersons or media will be admitted.
Hemophilia: A Disease in Transition
Hemophilia patient care has made significant advances during the past 50 years. In the current era, bleeding prevention and maintaining an active, healthy lifestyle are the main goals of care. Factor replacement therapy is effective, although this approach comes at a price of frequent venipuncture and inhibitor development, particularly for those with hemophilia A.
Subclinical bleeding remains a concern fueling the debate over the optimal trough level and best marker of adequate therapy. Personalization of therapy is now easier with tools to support routine pharmacokinetic analysis and musculoskeletal ultrasound. Newer agents that impact hemostasis without the need for frequent venipuncture are now available and more are making their way through the trial process towards market authorization and wider introduction. During this phase, the biggest risk is the inherent failure of small clinical trials to detect rare serious adverse events. Some severe but not unexpected adverse thrombotic events have already come to notice with the factor VIII mimetic antibody and the antithrombin lowering siRNA therapies. Other studies rebalancing hemostasis through inhibition of the tissue factor pathway inhibitor and activated protein C are also underway. The post-market authorization surveillance stage of these studies will be important to establish the long-term landscape of hemophilia care in 2019 and beyond. Although better tools will exist to manage patients in 2019, gene therapy is likely to receive market authorization by 2024. Where gene therapy will best fit in this landscape is yet to be determined.
Dr. Christine Kempton will discuss the current treatment of hemophilia, including recent advances such as emicizumab and extended half-life products.
Dr. Edward Tuddenham will discuss the future care of hemophilia, including the possible role of gene therapy for treatment of hemophilia A and B with and without inhibitors.
David Lillicrap, MD
Kingston, ON, Canada
Christine L Kempton, MD, MSc
Hemophilia Care in 2019
Edward Tuddenham, MDback to top
UCL Cancer Institute
London, United Kingdom
Hemophilia Care in 2024
Managing Acute and Chronic Pain in Patients With Sickle Cell Disease
While sickle cell disease (SCD) has a vast array of clinical complications, the most common presentation of SCD is pain. Over the past two decades, the complexity of providing optimal pain management for SCD has changed. There is now an older population that requires transition and care in an adult health care setting. Additionally, the understanding and management of chronic pain is changing, particularly in light of the current dialogue on the use of chronic opiates. This session will focus on the complexities of providing optimal acute and chronic pain management to patients with SCD.
Dr. Paula Tanabe will discuss evidence-based treatment of vaso-occlusive episode (VOE), including a review of recommendations from the National Heart, Lung, and Blood Institute. As per National Institutes of Health guidelines, VOEs should have prompt assessment and intervention within 60 minutes. Given the complexities of the health system, successful implementation of these recommendations may sometimes require novel approaches. In this session, Dr. Tanabe will discuss specific and effective site strategies to achieve these goals. Furthermore, she will discuss novel non-opiate approaches to acute pain management, including selection inhibition, lidocaine, and ketamine.
Dr. Wally Smith will review the historical view of the epidemiology and mechanisms of pain in SCD and how it has influenced treatment approaches. He will review current definitions and understandings of acute and chronic SCD pain, the evolution to chronic pain, and suggest how those understandings should change our treatment approach. Lastly, Dr. Smith will review and discuss the paucity of evidence on balancing benefits and harms when using opioids to treat chronic SCD pain.
Payal C Desai, MD
The Ohio State University
Paula Tanabe, PhD, MSN, MPH, RN, FAEN, FAAN
Current Best Practices in the Management of Acute Pain in Sickle Cell Disease
Wally R. Smith, MDback to top
Virginia Commonwealth University
Current Best Practices in the Management of Chronic Pain in Sickle Cell Disease
Population Risks of Developing Myeloid Malignancies
The seminal work that first led to the understanding of the risk of myeloid malignancies at the population level came from studies on Japanese atomic bomb survivors that showed increased risk from radiation exposure. This risk remains elevated even 40 to 60 years after radiation exposure. In the past decade, other leukemogenic exposures have garnered attention because of their potential impact on large populations. The association of several cancers, including leukemias, to Agent Orange has raised concerns regarding the risk in 2.8 million U.S. military personnel exposed to this defoliant in Vietnam. Advancements in cancer therapies have improved survival, resulting in a growing population of cancer survivors in the U.S., estimated to reach 18 million by 2022. Between 10 and 20 percent of these survivors will develop myeloid malignancies attributed to prior exposure to chemotherapy and radiation. Approximately 1.5 to 2 percent of incident cancers could be related to exposure to radiation from CT scans, with myeloid malignancies among the top three cancers. This risk is not subtle considering that the number of CT scans performed annually in the U.S. now exceeds 70 million. Therefore, characterizing population risks of myeloid malignancies has public health implications and may provide data that can help with treatment decisions in clinical situations that are not well covered by randomized clinical trials. This spotlight session will provide a framework on how to integrate and use the knowledge gained from population studies in individual patient care.
Dr. Yasushi Miyazaki will summarize known environmental risk factors and will discuss, in detail, myeloid malignancies related to Rd exposure, showing cytogenetic and molecular characteristics. There are several environmental factors known as risk factors for myeloid malignancies, such as benzene (Bz) and ionizing radiation (IRd). Some others are also suggested to increase the risk of myeloid neoplasms, but epidemiological evidence is not as firm as Bz or IRd.
Dr. Sudipto Mukherjee will discuss the risk of myeloid malignancies in three different population cohorts with distinct leukemogenic exposures: long-term cancer survivors who received prior chemotherapy and radiation therapy, U.S. military personnel exposed to Agent Orange, and in those undergoing diagnostic CT scans.
Sudipto Mukherjee, MD, PhD, MPH
Yasushi Miyazaki, MD, PhD
Atomic Bomb Disease Institute of Nagasaki University
Environmental Exposures and Myeloid Malignancy Risk
Sudipto Mukherjee, MD, PhD, MPHback to top
From Fat Man to Agent Orange and Beyond: When Myeloid Malignancies Strike
The Story Is the Story: Using Narrative Medicine to Cure Disease
In her essay "Moving on," the physician-writer Abigail Zuger, MD, reflects on her career of seeing patients as she considers retirement. Dr. Zuger writes, "When I close the door to my airless little clinic room for the last time, I will be closing hundreds of charts in the middle and walking away before the stories end. In all of medicine, is there anything more difficult to do than that?" This spotlight session will feature three extraordinarily skilled and prolific physician-writers and their take on the intersection of storytelling, writing, and medicine.
Dr. Danielle Ofri will probe why doctors should care about narrative. Why do medical journals publish stories and poems in their otherwise statistically significant pages? Dr. Ofri will discuss why skilled clinicians must be able to interpret their patients’ metaphors, otherwise clinicians risk missing out on the many layers of meaning, not to mention the diagnosis.
Dr. Jay Baruch will discuss how the best medicine doesn't work on the wrong story; but finding traction in patients' stories can be hard. Thinking like a writer of stories can provide a different set of tools for understanding and responding to the profound stories that patients tell.
Dr. Ilana Yurkiewicz will discuss her career as a writer and as a hematology and oncology fellow. She will also discuss how writing for public audiences from the inside can be a powerful tool for advocacy and change.
Mikkael A. Sekeres
Cleveland Clinic Taussig Cancer Center
Danielle Ofri, MD, PhD
New York University
New York, NY
Why Should I Care About the Narrative?
How the Patient Narrative Informs Treatment Decisions
Ilana Yurkiewicz, MD
back to top
Trainee Perspective: The Power of Voice