Clinical Practice and Hemophilia Gene Therapy the Focus of New Accredited Activity

Clinical Practice and Hemophilia Gene Therapy the Focus of New Accredited Activity
This activity is intended for learners to enhance their ability to recognize the key differences in gene therapies for hemophilia A compared with hemophilia B, and to leverage evolving data about gene therapies for hemophilia to better care for patients.

Enduring Education Focuses on Hemophilia Non-Factor Replacement Therapy

Enduring Education Focuses on Hemophilia Non-Factor Replacement Therapy
"Exploring Non-Factor Replacement Therapies for Hemophilia Care: Achieving Positive Outcomes," is intended for U.S.-based hematologist/oncologists, pediatricians, primary care physicians, emergency medicine physicians, nurses, pharmacists, and other members of the comprehensive hemophilia care team

Study Provides New Data on Neurodevelopment in Younger Hemophilia Population

Study Provides New Data on Neurodevelopment in Younger Hemophilia Population
Study investigators assessed the impacts of hemophilia type and severity, intracerebral hemorrhage, hemophilia treatment type, socioeconomic status, and neurodevelopmental disorders or symptoms on cognitive, adaptive, and social-emotional functioning compared with the general population.

First Sickle Cell Patient to Receive Gene Therapy Featured in NYT

First Sickle Cell Patient to Receive Gene Therapy Featured in NYT
Sickle cell disease is linked to an abnormality in the hemoglobin protein, which normally helps carry oxygen throughout the body via red blood cells. This causes red blood cells, normally disc shaped and flexible enough to travel smoothly in the body, to become rigid and misshapen to resemble a “C” or sickle.

Intellia Therapeutics Withdraws from Hemophilia B Gene Editing Program, Leaving Regeneron in Charge

Intellia Therapeutics Withdraws from Hemophilia B Gene Editing Program, Leaving Regeneron in Charge
In a significant development within the gene editing arena, Intellia Therapeutics has announced its decision to opt out of a hemophilia B program, relinquishing control of CRISPR/Cas9-based gene editing work on factor IX treatments to its partner, Regeneron.

FDA Grants Expanded Approval to Wilate® for Prophylaxis in All Types of VWD

FDA Grants Expanded Approval to Wilate® for Prophylaxis in All Types of VWD

Octapharma’s human plasma-derived von Willebrand Factor (VWF)/Factor VIII replacement product Wilate® has been granted expanded approval for prophylaxis by the U.S. Food and Drug Administration (FDA) to include all types of von Willebrand disease (VWD).

Hepatocellular Cancer Screening and Gene Therapy Preparedness the Focus of New Recommendations

Hepatocellular Cancer Screening and Gene Therapy Preparedness the Focus of New Recommendations

Two new documents have been issued by the NBDF’s Medical and Scientific Advisory Council (MASAC), which periodically writes recommendations and advisories on treatment, research, and other general health concerns of the inheritable bleeding disorders community.

Study Results Encourage Discussion of Therapeutic Options for VWD Patients with Menorrhagia

Study Results Encourage Discussion of Therapeutic Options for VWD Patients with Menorrhagia

While the majority of women with von Willebrand disease (VWD) will experience heavy menstrual bleeding (HMB) often associated negative impacts on overall health and quality of life, available data relevant to effective treatment is limited.

The VWD Guidelines and Me: Points of Shared Decision-Making

The new VWD guidelines were published in 2020 and heavily integrate the patient voice. But how can you use these in your conversations with healthcare professionals? This session will take a deeper dive into the specific recommendations, where patients and healthcare professionals need to decide together on the best plan of care. You will learn essential skills in shared decision-making to advocate for the care you need.

How Do YOU Navigate?

Learning through the lived experiences of others is helpful in navigating the landscape of bleeding disorder care. In this workshop, participants will be given an opportunity to learn through investigating scenarios building on the previously delivered material but also through discussions of the issues they are facing in their own everyday life. By the end, participants will receive practical tips and will feel connected and empowered.

Choose to Take Control

Being a good consumer is more than just access to products and care, it is also about knowing the do’s and don’ts that impact your daily life with a bleeding disorder. It’s about understanding your disease, choosing a care team, clearly communicating your needs and accepting responsibility for making the decisions that ensure your safety and wellbeing. During this interactive presentation, we will discuss tips, tricks and tools available to help you become a better self-advocate and an overall good consumer.

Pregunte a los expertos

¿Tiene una pregunta candente que hacerle a nuestro panel de expertos? Esta sesión ofrecerá a los participantes la oportunidad de interactuar, participar y hacer preguntas que inviten a la reflexión a un panel de proveedores de atención médica interdisciplinarios en tiempo real. Esta discusión incluirá Miguel Escobar, MD; Kelli Fraga, PT, DPT; Sabrina Farina, LMSW

Women Bleed Too

Women and girls with bleeding disorders often deal with particular issues and taboos related to diagnosis, symptoms, access to care. In this session, an expert who works to improve the quality of life for women and girls with bleeding disorders will discuss essential information on the challenges of getting a diagnosis and accessing care and treatment. In addition, an affected individual will share their experience as a woman with a bleeding disorder.

What Everyone Who Treats with Factor Needs To Know About Inhibitors

If you treat with clotting factor, you may be at risk of developing an inhibitor. This workshop is for people who do not have an inhibitor but want to learn more about risk. We will explain the basics of inhibitors, how they develop, and how and why you should have a conversation with your healthcare provider about screening and monitoring for an inhibitor.

Basics: VWD

This session will review the signs and symptoms of von Willebrand disease, discuss genetics and inheritance patterns, and define and compare the three types of VWD

OUCH! Tackling Pain Together

NHF’s Medical and Scientific Advisory Council (MASAC) recently approved new treatment guidelines for providers who administer opioids to treat pain in people with bleeding disorders. Come learn about the new guidelines from expert providers and consumers and what they might mean to you.

Just Do It... or Don't

Given relatively limited literature specifically on return to physical activity after injury for people with bleeding disorders, many physical therapists may lack competence in developing tailored recovery plans and providing prudent guidance to patients and their families. We will consider and apply recommended guidelines for return to activity and sport, critically appraise existing return to activity evaluation tools and applications, and compare and contrast return-to-work evaluation tools and applications for people with bleeding disorders.

Hemo 201: What to Do When You Have No Clue

Hemophilia is a uniquely challenging and sometimes perplexing condition for physical therapists to manage and treat. Physical therapists who see patients across the lifespan may manage conditions that range from athletic injuries to frailty. Clinicians often rely on reviewing limited evidence, consulting colleagues, and ultimately critical thinking and best clinical judgment to develop a management plan. This session will outline a framework for different scenarios seen in the clinic and look at six broad management schemes.

Pain Management: Tool Box Not a Pill Box

The session will discuss pain related to bleeding disorders in the developmental stages and how infants, children and adolescents with bleeding episodes experience, express and cope with pain. It will review the latest research on management and assessment of pain in children and provide guidance on pain education for families and caregivers. Treatment and intervention strategies for physical therapists will be reviewed and followed by a group discussion.

The Advantage of Prophylaxis for Adults with Joint Issues

It is a reality familiar to most adults with hemophilia of a certain age:  repeated joint bleeds which subsequently causes joint deterioration, affecting everyday quality of life. But the story does not have to end there. Learn how prophylactic therapy can be used to halt the momentum of degenerative joint damage, decrease pain and help you regain mobility.

Transcription of "The Advantage of Prophylaxis for Adults with Joint Issues"

Marijuana: Breaking it Down, the Good, the Bad, and the Consumption

*ASWB

Moderators:

Kathaleen Schnur, LSW
Social Worker, Hemophilia Center of Western Pennsylvania

Alfredo A. Narvaez, Jr., MSW, LMSW
Social Worker, Louisiana Comprehensive Hemophilia Center
Tulane University Medical Center

Speaker:

Khalid Namoos
Medical Fellow, UCLA David Geffen School of Medicine

By the end of this session, the participant should be better able to:

Ethics: Blurred Boundaries

*ASWB

Moderators:

Kathaleen Schnur, LSW
Social Worker, Hemophilia Center of Western Pennsylvania

Lucy Ramirez, MSW, LCSW
Clinical Social Worker, Rush Hemophilia & Thrombophilia Center

Speaker:

Alana Brunner, LCPC, NCC, CCMHC
Mental Health Therapist
St. Luke’s Psychiatric Wellness

By the end of this session, the participant should be better able to:

Cannabis Applications & Implications

*ANCC

Speaker:

Khalid Namoos
Medical Fellow, UCLA David Geffen School of Medicine

By the end of this session, the participant should be better able to:

  • Compare cannabis formulations and discuss the evidence for their potential use
  • Discuss the evidence for clinical indications for cannabis use
  • Describe the pharmacokinetics and physiological effects of cannabis 

Mild Phenotypes: The Struggle is Real

*ANCC  

Moderator:

Jennifer Donkin, RN, DNP, CPNP, CFNP
Nurse Practitioner
Children's Hospital of Los Angeles

Speakers:

Sharon L. Littig, RN
Clinical Coordinator
Cardeza Foundation Hemophilia Center at Jefferson University

Penni J. Smith, MPA, BSN, RN-BC
Hemophilia/Hematology RN Coordinator
Utah Center for Bleeding and Clotting Disorders
Primary Children’s Hospital  

Disorders of Fibrinogen

*ANCC

Moderator:

Kimberly Hurdstrom, RN, BSN
Hemophilia & Thrombosis Center, University of Colorado School of Medicine
University of Colorado Denver

Speaker:

Marilyn Manco-Johnson, MD
Director, Hemophilia & Thrombosis Center
Professor, Pediatrics Hematology-Oncology
University of Colorado School of Medicine
University of Colorado Denver

By the end of this session, the participant should be better able to:

Forging a New Path – Gene Therapy Readiness and HTC Implementation

*CME/ACPE

Moderator/Speaker:

Steven W. Pipe, MD, Faculty Chair
Professor of Pediatrics and Pathology
Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases,
Pediatric Medical Director, Hemophilia and Coagulation Disorders Program,
Director, Special Coagulation Laboratory,
University of Michigan
Ann Arbor, Michigan

Speakers:

Challenging Cases

*ANCC

Moderators:

Sharon L. Littig, RN
Clinical Coordinator
Cardeza Foundation Hemophilia Center at Jefferson University

Jessica Pindilli, BSN, RN
University of Wisconsin Hospital & Clinics
UWHC Comprehensive Program for Bleeding Disorders

Speakers:

Maria Tovar-Herrera, RN-BC, BSN
RN Coordinator
Rush Hemophilia and Thrombophilia Center

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