Public Health Webinar Series on Blood Disorders

November 7, 2024

The thalassemias are among the most common genetic disorders in the world; yet they exhibit a broad spectrum of phenotypes. Thalassemias can be subdivided based on the hemoglobin chain affected (alpha versus beta) and genotype, which roughly corresponds to severity level (major, minor, and intermedia). The thalassemia phenotypes can also be categorized into those that are transfusion-dependent (mostly thalassemia major) and those that are not (mostly thalassemia intermedia). Individuals born with severe forms require clinical management that may include chronic transfusion therapy and iron chelation. Early diagnosis of thalassemia results in optimal outcomes.

Thalassemia is typically managed by a hematologist; however, patients often present to pediatricians with symptoms, which lead to a diagnosis of thalassemia. In addition, it is important that pediatricians and hematologists discuss the management of a pediatric patient’s thalassemia and any impact that their condition may have upon other aspects of patient care that fall under the pediatrician’s purview.

In this webinar, Alexis Thompson, MD, MPH, will discuss the diagnosis and clinical management of thalassemia, with an emphasis on information that is most relevant for pediatricians. She will provide information on how to accurately diagnose thalassemia, as well as the pathophysiology and management most commonly associated with severe forms of thalassemia.

Presenter:
Alexis A. Thompson, MD, MPH
Chief of the Division of Hematology, Children’s Hospital of Philadelphia
Elias Schwartz, MD
Endowed Chair in Hematology, Children’s Hospital of Philadelphia

Moderator:
Jeanne Boudreaux, MD
Director, Comprehensive Thalassemia Center,
Children’s Healthcare of Atlanta

October 1, 2024

This roundtable is dedicated to women’s health and thrombosis, offering invaluable insights into preventing blood clots during contraception, pregnancy, and the postpartum period.

In this webinar, Naomi Tepper, M.D., Medical Officer at the CDC’s Division of Reproductive Health, discusses the latest contraceptive guidelines, followed by an expert panel discussion featuring global leaders in thrombosis research.

June 20, 2024

Venous thromboembolism (VTE), a significant health concern in the United States, impacts up to 900,000 people each year and is responsible for about 100,000 deaths annually. VTE, which includes conditions such as deep vein thrombosis and pulmonary embolism, presents a significant public health challenge due to its prevalence and potential for serious complications. There are various VTE treatment strategies a physician may consider, based on the patient’s condition, that include the use of anticoagulants, thrombolytics, filters, or thrombectomy. Mechanical thrombectomy is a type of minimally invasive procedure in which a radiologist uses specialized equipment to remove a clot from a patient’s vein.

In this webinar, Drs. Gonsalves and Knox will discuss the evolving role of mechanical thrombectomy in VTE management. They will explore the impact of VTE on affected populations, with a particular focus on how recent advancements in mechanical thrombectomy are reshaping VTE treatment strategies. They will then provide insights into the latest research, technological breakthroughs, and clinical practices in this field. Lastly, they will discuss the challenges and prospects of integrating these innovative treatments into various healthcare environments.

Presenter:
Carin Gonsalves, MD
Professor of Radiology
Co-Director, Division of Interventional Radiology, Thomas Jefferson University
Co-Founder and Co-Director, Pulmonary Embolism Response Team, Jefferson Health

Presenter:
Michael Knox, MD
Interventional Radiologist, Co-Chair, Pulmonary Embolism Response Team, Corewell Health

Moderator:
Alejandra Gutierrez Bernal, MD
Assistant Professor, Cardiovascular Critical Care Cardiology Lead, Pulmonary Embolism Response Team, University of Minnesota

May 16, 2024

Hemophilia A and B are rare inherited bleeding disorders caused by deficiencies in coagulation factor VIII (FVIII) or factor IX (FIX). Nearly all people with hemophilia A or B have an identifiable DNA change in the F8 or F9 genes, respectively. The inheritance of hemophilia has historically been described as a “recessive” X-linked pattern that only affects males. However, females who have one or more hemophilia-causing genotypes can also have bleeding and hemophilia. The misperception of hemophilia as a male-only disorder has led to clinical definitions and descriptions of disease informed by the male experience and under-recognition of the healthcare needs of females with hemophilia.

There is growing evidence that hemophilia genotype-positive females can commonly experience excessive bleeding, delays in diagnosis, undertreatment, and poor quality of life. Different than in males, coagulation factor levels do not correlate well with bleeding, and some genotype-positive females experience excessive bleeding even with factor levels within the normal reference range. Genetic testing, factor levels, and bleeding symptom assessment are all important in making a diagnosis of hemophilia, symptomatic carrier, or asymptomatic carrier in females. Females also commonly experience heavy menstrual bleeding and are at high risk for postpartum hemorrhage. However, the causes of excessive reproductive tract bleeding are not well understood.

In this webinar, Dr. Johnsen will discuss various considerations for females who have hemophilia genotypes, including inheritance patterns, factor levels, bleeding manifestations, and nomenclature for diagnosis of hemophilia. She will also describe the Hemophilia A Research Program (HARP), a new study seeking to better understand the hemophilia bleeding risk in pregnancy and identify the causes of inhibitor formation in early childhood.

Presenter:
Jill Johnsen, MD
Associate Professor, Division of Hematology and Oncology, University of Washington
Faculty, Institute for Stem Cell and Regenerative Medicine, University of Washington
Co-Lead, Hemophilia A Cohort Research Program (HARP)

Moderator:
Roshni Kulkarni, MD
Director Emerita, MSU Center for Bleeding and Clotting Disorders
Professor Emerita, Pediatric Hematology/Oncology, Department of Pediatrics and Human Development, Michigan State University

September 28, 2023

Venous thromboembolism (VTE) is a major cause of morbidity and mortality in the United States, affecting up to 900,000 people and accounting for up to 100,000 deaths annually. The focus of care for patients once diagnosed with blood clots is to prevent recurrent VTE, major bleeding, and death. While these are highly relevant clinical outcomes, they do not capture patient-centered outcomes such as quality of life (QoL). Importantly, decreased QoL is prevalent after VTE, occurring in up to half of patients.

Post-VTE syndrome occurs despite adequate anticoagulation therapy, ranging from mild to severe symptoms, and can have a major impact on QoL. Moreover, post-VTE syndrome has been associated with higher risk of depressive disorders, unemployment, and social isolation. In this webinar, Rachel Rosovsky, MD, and Jeffrey Kline, MD, present data and discuss the importance of understanding psychosocial factors that can affect patients’ diagnoses and long-term recovery from VTE.

Presenter:
Rachel P. Rosovsky, MD, MPH
Director, Thrombosis Research
Division of Hematology
Massachusetts General Hospital
Associate Professor of Medicine
Harvard Medical School
Boston, Massachusetts

Presenter:
Jeffrey A. Kline, MD
Associate Chair for Research
Brooks Bock Endowed Professor
Wayne State University School of Medicine Department of Emergency Medicine
Detroit, Michigan

August 24, 2023

Over the past 20 years, the Centers for Disease Control and Prevention has conducted public health surveillance to better understand the characteristics and complications that impact the bleeding disorders population. Community Counts is a public health surveillance program that gathers and shares information about common health issues, medical complications, and causes of death that affect people with bleeding disorders cared for in U.S. Hemophilia Treatment Centers.

In this presentation, Drs. Dupervil and Byams provide an overview of Community Counts, its components, and its role in monitoring important health outcomes. They will present key findings on the occurrence of bleeding disorders, and information collected to date on population demographics, clinical characteristics, and other health indicators.

Brandi Dupervil, DHSc, MPH
Epidemiologist Epidemiology and Surveillance Branch
Division of Blood Disorders
National Center on Birth Defects and Developmental Disabilities
Centers for Disease Control and Prevention

Vanessa R. Byams, DrPH, MPH
Lead Health Scientist
Epidemiology and Surveillance Branch
Division of Blood Disorders, National Center on Birth Defects and Developmental Disabilities
Centers for Disease Control and Prevention

Skye Peltier, MPH, PA-C
Clinical Consultant
American Thrombosis and Hemostasis Network Clinical Co-Director
Children’s Minnesota
Center for Bleeding and Clotting Disorders

March 23, 2023

In this webinar, Drs. Goldenberg and Sochet discuss key aspects of venous thromboembolism (VTE) in children, including incidence, risk factors, and outcomes, as well as critical knowledge gaps in optimal treatment and prevention. They will then summarize the design and findings of two recently published investigator-initiated clinical trials of VTE treatment (Kids-DOTT phase 3 multinational trial on duration of anticoagulation in patients younger than 21 years old with provoked VTE) and prevention (COVAC-TP, U.S. national phase 2 on pharmacological thromboprophylaxis in children hospitalized for COVID-19-related illness, including multisystem inflammatory syndrome in children). Lastly, they will outline key questions for future research.

Presenter:
Neil A. Goldenberg, MD, PhD
Professor, Global Principal Investigator, Kids-DOTT Trial
Associate Dean for Research, Johns Hopkins All Children’s Hospital
Director, Johns Hopkins All Children’s Institute for Clinical & Translational Research, Johns Hopkins All Children’s Hospital

Presenter:
Anthony A. Sochet, MD, MHS
National Principal Investigator, COVAC-TP Trial
Assistant Professor of Anesthesiology and Critical Care Medicine Johns Hopkins University School of Medicine
Associate Director, Pediatric Critical Care Medicine Fellowship Program
Johns Hopkins All Children’s Hospital

Moderator:
Julie Jaffray, MD
Pediatric Hematologist/Oncologist Rady Children’s Hospital San Diego

June 22, 2023

Although relatively new in terms of clinical application, several gene therapy-based treatments have, in recent years, received approval from the Food and Drug Administration (FDA) and begun to be used in real world settings in the United States. In addition, clinical trials using either gene transfer or genome editing continue to show promise, with the potential to impact treatment for patients with a wide range of hereditary disorders in the future. This webinar will showcase thalassemia as a case example in emerging approaches in gene therapy.

In this presentation, Drs. Rivella and Sheth will review the status of the FDA-approved gene therapy process for thalassemia, discussing the science behind it, the results of the clinical trials associated with it, and the clinical implications as gene therapy begins to be applied in a real-world setting. In addition, they will explore future clinical trials involving potential alternative gene therapy treatments.

Presenter:
Stefano Rivella, PhD
Professor of Pediatrics, Children’s Hospital of Philadelphia
Scientific Director of The Sickle Cell and Red Cell Disorders Curative Therapy Center, Children’s Hospital of Philadelphia

Presenter:
Sujit Sheth, MD
Chief, Division of Pediatric Hematology/Oncology
Professor of Clinical Pediatrics, Weill Cornell Medicine

Moderator:
Muin J. Khoury MD, PhD
Director, Office of Genomics and Precision Public Health
National Center on Birth Defects and Developmental Disabilities
Centers for Disease Control and Prevention

February 23, 2023

Through advances in treatment, people with hemophilia are enjoying longer lives. Aging can bring new health concerns such as declining physical and cognitive function, bone loss, and cardiovascular disease. Aging with hemophilia presents unique challenges that require attention by hemophilia providers and treatment centers to successfully support people with hemophilia as they transition through later stages of life.

In this presentation, Dr. Kempton will discuss comorbidities that are either unique to or more common in people with hemophilia, as well as common challenges of aging that may require special attention in the hemophilia population.

Presenter:
Christine L. Kempton, MD, MSc
Professor, Department of Hematology and Medical Oncology & Department of Pediatrics, Emory University School of Medicine
Director, Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory
Vice Chair for Faculty Development, Department of Hematology & Medical Oncology, Emory University School of Medicine

Presenter:
“Bobby” Duc Tran, MD, MSc
Assistant Professor, Department of Hematology & Medical Oncology
Hemophilia of Georgia Center for Bleeding & Clotting Disorders, Emory University School of Medicine

March 24, 2022

In this webinar, Drs. Wendelboe and Ortel describe how novel methods, such as natural language processing, might be incorporated into surveillance systems for venous thromboembolism. This work is part of a larger collaborative project between the U.S. Centers for Disease Control and Prevention, the University of Oklahoma, and Duke University.

Presenter:
Aaron M. Wendelboe, PhD
Edward E. and Helen T. Bartlett Chair in Public Health
Professor, Department of Biostatistics and Epidemiology
University of Oklahoma Health Sciences Center

Presenter:
Thomas Lee Ortel, MD, PhD
Chief, Division of Hematology
Professor of Medicine and Pathology
Member of the Duke Cancer Institute
Duke University School of Medicine

Moderator:
Karon Abe, PhD
Captain, US Public Health Service
Chief, Epidemiology & Surveillance Branch
Division of Blood Disorders
National Center on Birth Defects and Developmental Disabilities