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If you want to be in the know about what’s happening in the bleeding disorders community, you’ve come to the right place!

Be sure to check back regularly to get our latest news updates.


NHF's Wednesday Webinars for April 2021 will feature an array of speakers and topics of interest to both healthcare providers and patients and families with blood disorders alike.

April Webinars
All webinars are from 2:00pm-3:00pm ET.

April 7th, 2021
What is the Burden of Problem Joints with Hemophilia?

Speaker: Paul McLaughlin

April 14th, 2021
WFH Humanitarian Aid Program

Speaker: Assad Haffar

The American Rescue Plan (ARP), signed into law on March 11, 2021, provides new opportunities to get health insurance coverage, and/or to reduce the cost of your premiums. Review your coverage now to make sure you are maximizing your benefits under the new law.

 

uniQure recently announced the results of a comprehensive investigation into a case of hepatocellular carcinoma (HCC) diagnosed in one patient participating in the phase III HOPE-B pivotal trial of etranacogene dezaparvovec, the company’s investigational gene therapy candidate for individuals with severe and moderately severe hemophilia B.

uniQure recently announced the results of a comprehensive investigation into a case of hepatocellular carcinoma (HCC) diagnosed in one patient participating in the phase III HOPE-B pivotal trial of etranacogene dezaparvovec, the company’s investigational gene therapy candidate for individuals with severe and moderately severe hemophilia B.

To the members of the inheritable blood disorders community,

Let me begin with a word for the thousands of members of our community – patients, providers, partners, family, and friends – who are also members of the Asian and Pacific Islander community:

We would not be the National Hemophilia Foundation without you.

As part of a patient education webinar series known as ITP INSIGHTSSM, the Platelet Disorder Support Association (PDSA) recently recorded a session on the new COVID-19 vaccines and immune thrombocytopenic purpura (ITP), a disorder characterized by low platelet levels.

The National Hemophilia Foundation (NHF) announced that Keri L. Norris, PhD, MPH, MCHES, has joined NHF as  vice president of health equity, diversity, and inclusion. Dr. Norris has more than 20 years of experience in health equity, public health, and social justice, with a particular emphasis on health disparities. Her role will be to create a health equity framework to integrate into NHF programs and services and develop culturally and linguistically appropriate programs and services to address disparities in outcomes within our community.

The National Hemophilia Foundation (NHF) announce plans for a NHF State of the Science Research Summit to bring together the inherited bleeding disorders community for a virtual conference to identify and accelerate research progress in the areas of greatest need for affected individuals and their families. The event is scheduled to be held virtually September 12-15, 2021.

In Genentech’s ongoing efforts to transparently communicate with the hemophilia community, we are sharing two updates to the Hemlibra label that were requested by the FDA. These changes, which were accepted and implemented on March 10, 2021, impact the Warnings and Precautions (sections 5.1 and 5.2) and Clinical Pharmacology (section 12.3) sections of the label.​ Importantly, the overall benefit/risk profile of Hemlibra remains positive.

The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) issued three new documents, all of which were adopted by NHF’s Board of Directors on March 4, 2021.

The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) issued three new documents, all of which were adopted by NHF’s Board of Directors on March 4, 2021.

The National Hemophilia Foundation (NHF) is refocusing its mission statement to reflect a renewed emphasis on research and an expanded scope to include inheritable blood disorders.

The newly adopted mission statement reads:

The mission of the National Hemophilia Foundation is dedicated to finding cures for inheritable blood disorders and to addressing and preventing the complications of these disorders through research, education, and advocacy, enabling people and families to thrive.

The National Hemophilia Foundation is now accepting nominations for its Awards of Excellence. These awards honor members of the bleeding disorders community who have made outstanding contributions to improving the lives of people with bleeding disorders through treatment and care, advocacy, and raising awareness.

The Children’s National Hospital and Takeda recently announced the creation of a new, first-of-its-kind program to help standardize and streamline the process of diagnosis and care for individuals with rare diseases.

NHF's Wednesday Webinars for March 2021 will feature an array of speakers and topics of interest to both healthcare providers and patients and families with blood disorders alike.

March Webinars
All webinars are from 2:00pm-3:00pm ET.

 

March 3, 2021

Cellular Therapies for Hemophilia 

Speakers: Amy Shapiro, MD, Indiana Hemophilia & Thrombosis Center, Inc. and Deya Corzo, MD, Sigilon Therapeutics

 

The U.S. Food and Drug Administration (FDA) has partially rescinded a pair of supplemental Biologics License Applications (BLAs) the agency had granted, in error, for two recombinant factor IX products, BeneFIX® (Pfizer) and IXINITY (Aptevo). The supplemental BLAs in question were both approved by the FDA in 2020 and represented additional indications that ultimately ran against exclusivity rights of another product.   

Background:

Fitusiran is an RNA drug that can prevent antithrombin protein from being made in the liver. Antithrombin is an important brake for the coagulation system. Insufficient amounts of circulating antithrombin in normal adults lead to thrombosis. In persons with hemophilia (PWH) A or B, who don’t have sufficient clotting, lowering antithrombin with fitusiran has been shown to increase coagulation and protect PWH from bleeding. It is presently in Phase 3 clinical trials.

Octapharma recently announced the final results from the NuProtect study, which evaluated the immunogenicity of Nuwiq® in previously untreated patients (PUPs) with severe hemophilia A.Nuwiq® is a recombinant factor VIII therapy, is indicated for on-demand treatment and control of bleeding episodes and routine prophylaxis to reduce the frequency of bleeding episodes and perioperative m

Spark Therapeutics announced preliminary data from part one of their ongoing phase 1/2 open-label, non-randomized, dose-finding study of SPK-8016, the company’s investigational gene therapy for hemophilia A.

Come the Spring of 2021 patients and caregivers will be presented with a new opportunity to provide valuable feedback on the care they receive at hemophilia treatment centers (HTC) across the United States.

NHF's Wednesday Webinars for February 2021 will feature an array of speakers and topics of interest to both healthcare providers and patients and families with blood disorders alike.

February Webinars
All webinars are from 2:00pm-3:00pm ET.

On January 28th, 2021, President Biden signed an executive order aimed at stabilizing and strengthening the Affordable Care Act (ACA) and Medicaid. The President specifically stated that this order does not change any law but rather rolls back actions taken by the Trump administration over the last four years.

BioMarin recently announced positive updates from its ongoing phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. The trial currently includes 134 participants, all of whom have received a single dose of the therapy and have been observed for at least one year post treatment.

CSL Behring has announced a voluntary pharmacy level product recall of one batch of Mononine Coagulation Factor IX (Human), a plasma-derived product used to treat hemophilia B. CSL Behring’s notification states, “Patients can continue to use product they may have. Although the potential for safety risk to patients is considered low, it cannot be fully excluded.”

The affected batch is identified as: P100125501

The new joint clinical practice guidelines on the diagnosis and management of von Willebrand disease, developed by the American Society of Hematology (ASH), the International Society of Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) now have snapshot guides available in English, Spanish, and French.

The snapshots provide a high-level summary of both the diagnosis and management of VWD.

 

The new joint clinical practice guidelines on the diagnosis and management of von Willebrand disease, developed by the American Society of Hematology (ASH), the International Society of Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) now have snapshot guides available in English, Spanish, and French.

The snapshots provide a high-level summary of both the diagnosis and management of VWD.

 

“From Principles to Practice: Preparing for the Advent of Gene Therapy” represents the newest educational activity in Medscape’s series “Clinical Advances in Gene Therapy for Hemophilia.” Presented through a collaboration between Medscape and the National Hemophilia Foundation, the series includes various discussions relevant to the latest data on gene therapy as well as the practicalities associated with using gene therapy for hemophilia.

“From Principles to Practice: Preparing for the Advent of Gene Therapy” represents the newest educational activity in Medscape’s series “Clinical Advances in Gene Therapy for Hemophilia.” Presented through a collaboration between Medscape and the National Hemophilia Foundation, the series includes various discussions relevant to the latest data on gene therapy as well as the practicalities associated with using gene therapy for hemophilia.

Organizations Collaborate to Develop International State-of-the-Art Guidelines on the Diagnosis and Management of von Willebrand Disease
bmarshall
Jan 11, 2021

Jan 11, 2021

 The American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), National Hemophilia Foundation (NHF), and World Federation of Hemophilia (WFH) have developed joint clinical practice guidelines on the diagnosis and management of von Willebrand disease (VWD), the world’s most common inherited bleeding disorder. The guidelines were published today in Blood Advances.

VWD affects approximately 1% of the world’s population, and it is the most common bleeding disorder. Although VWD occurs among men and women equally, women are more likely to notice the symptoms because of heavy or abnormal bleeding during their menstrual periods and after childbirth. This inherited condition results in the decreased production, absence, or abnormal function of the clotting protein von Willebrand factor.

VWD can cause unusual bleeding from small wounds or minor procedures, frequent nosebleeds, bruising, bleeding in joints, and heavy menstrual periods and post-partum bleeding in women. Symptoms may vary from patient to patient or in a single patient over the course of his or her life. Primary care providers, pediatricians, obstetricians, and gynecologists who observe unusual bleeding often refer their patients to a hematologist for further testing and management. Many individuals with mild symptoms do not receive a diagnosis right away and live for many years with untreated bleeding or do not realize they have VWD until they experience a severe bleed that could have been prevented.

“While VWD is a common bleeding disorder, it is also complex, presenting challenges in the timely diagnosis and appropriate management of bleeding for patients,” said 2021 ASH President Martin S. Tallman, MD, of Memorial Sloan Kettering Cancer Center.  “Because diagnosis is not straightforward and symptoms range in severity, there is a need for trustworthy guidelines to help improve the quality of care for patients. There are no better partners for ASH in this effort than ISTH, NHF, and WFH, whose collective expertise in VWD from the scientific and patient perspectives around the world enhanced this effort.”

Notably, the guidelines make key statements on thresholds of laboratory testing to classify VWD to be more inclusive of patients who experience bleeding, but whose blood work does not meet currently accepted thresholds for diagnosis. In addition, the guidelines for the first time recommend that VWD patients who suffer from frequent, severe bleeding that decreases quality of life should receive routine VWD prophylaxis – an injectable concentrate of clotting protein – several times a week. Together, the guidelines on diagnosis and management include a combined 19 recommendations.

The guidelines were developed by two expert panels made up of 32 individuals, including U.S.-based and international hematologists, individuals living with VWD, and scientists with expertise in evidence synthesis and appraisal and guideline development methodology. Clinical questions were developed by the panels and prioritized, and an international survey was completed to identify the most important clinical questions. A systematic review of available evidence was conducted by the University of Kansas Medical Center and the panel referred to this evidence to make recommendations. This process shed light on the lack of strong evidence on which to base recommendations, and therefore the report calls for more research.

“These guidelines are an excellent example of collaboration across multiple societies ensuring that the guidelines develop represent the most up to date and relevant advice about diagnosis and treatment of this common, but often misunderstood bleeding disorder,” said ISTH Past President Claire McLintock, MD. “Working as part of the incredible team that developed the guidelines in diagnosis of VWD was hugely rewarding and inspiring.”

People with VWD made up approximately one quarter of each guideline panel’s composition. As full voting members they contributed their expertise to every aspect of the rigorous guideline development process, from the prioritization of clinical questions, through the evaluation of the available evidence, to the consideration of factors such as stakeholder values and preferences, equity, and feasibility. Their involvement, actively supported by the panel chairs and collaborating organizations, was critical to ensuring the development of recommendations with the greatest pertinence and in which the community will have great confidence. Collaboration with WFH and NHF was essential to engaging the broader VWD patient community.

With their extensive established functional networks with healthcare professionals in centers for comprehensive care of bleeding disorders and patient organizations throughout the US and around the world, the four collaborating organizations, ASH, ISTH, NHF, and WFH will all play important roles in the creation and dissemination of resources based on the guidelines, and their implementation in individual care and broader advocacy efforts.

“These guidelines are an extremely important step in our quest to address the difficulties individuals go through to obtain an accurate, timely diagnosis, and appropriate treatment.  The challenge ahead will be for us to educate both those living with VWD and healthcare professionals on the guideline recommendations,” said Leonard Valentino, MD, President and CEO of NHF. “We were excited to work with ASH, ISTH, and WFH to create these and introduce them to the community.”

“In 2017 we gathered members of the global VWD community for the first WFH International Symposium on VWD. Delegates from countries around the world told us that the number one tool they needed in order to provide comprehensive care to people with VWD was clinical practice guidelines,” explained WFH President Cesar Garrido. “The WFH is very proud to deliver these guidelines today, with our partners from ASH, ISTH, and NHF, to the global community. In addition to offering guidance for clinicians and individuals with VWD as they partner in personalized care decisions, they constitute the foundation for important advocacy initiatives to improve access to the recommended diagnostic techniques and treatment options in all countries.”

The publication of the guideline will be accompanied by tools and educational resources to help patients, hematologists and other health care providers understand and implement the recommendations.

Related links about the new VWD guidelines:

•    VWD guidelines in Blood Advances

•    ASH resources

•    ISTH resources

•    NHF resources

•    WFH resources

About the American Society of Hematology

The American Society of Hematology (www.hematology.org) is the world’s largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 60 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. ASH publishes Blood (www.bloodjournal.org), the most cited peer-reviewed publication in the field, and Blood Advances (www.bloodadvances.org), an online, peer-reviewed open access journal. For more information about ASH’s portfolio of clinical practice guidelines, visit www.hematology.org/guidelines.

About the International Society of Thrombosis and Haemostasis

Founded in 1969, the ISTH is the leading worldwide not-for-profit organization dedicated to advancing the understanding, prevention, diagnosis and treatment of thrombotic and bleeding disorders. ISTH is an international professional membership organization with more than 7,700 clinicians, researchers and educators working together to improve the lives of patients in more than 110 countries around the world. Among its highly regarded activities and initiatives are education and standardization programs, research activities, meetings and congresses, peer-reviewed publications, expert committees and World Thrombosis Day on 13 October. Visit ISTH online at www.isth.org.


About the National Hemophilia Foundation

NHF (the National Hemophilia Foundation) is a 501(c)(3) nonprofit organization dedicated to finding better treatments and cures for inheritable bleeding disorders and to preventing the complications of these disorders through education, advocacy and research. NHF’s programs and initiatives are made possible through the generosity of individuals, corporations and foundations, as well as through a cooperative agreement with the Centers for Disease Control and Prevention (CDC). Additional information on hemophilia and NHF can be found at www.hemophilia.org.

About the World Federation of Hemophilia

For over 50 years, the World Federation of Hemophilia (WFH), an international not-for-profit organization, has worked to improve the lives of people with hemophilia and other inherited bleeding disorders. Our vision of Treatment for All is that one day, all people with a bleeding disorder will enjoy a more certain future filled with promise, no matter where they live. For decades, we have been working around the world to improve diagnosis, treatment and ensure patients have sustainable access to care. We have a responsibility to deliver life-changing programs and services. Established in 1963, the WFH is a global network of patient organizations in 147 countries and has official recognition from the World Health Organization. Visit WFH online at www.wfh.org.

 

 

Organizations Develop Guidelines on the Diagnosis and Management of VWD
bmarshall
Jan 11, 2021

Jan 11, 2021

 The American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), National Hemophilia Foundation (NHF), and World Federation of Hemophilia (WFH) have developed joint clinical practice guidelines on the diagnosis and management of von Willebrand disease (VWD), the world’s most common inherited bleeding disorder. The guidelines were published today in Blood Advances.

VWD affects approximately 1% of the world’s population, and it is the most common bleeding disorder. Although VWD occurs among men and women equally, women are more likely to notice the symptoms because of heavy or abnormal bleeding during their menstrual periods and after childbirth. This inherited condition results in the decreased production, absence, or abnormal function of the clotting protein von Willebrand factor.

VWD can cause unusual bleeding from small wounds or minor procedures, frequent nosebleeds, bruising, bleeding in joints, and heavy menstrual periods and post-partum bleeding in women. Symptoms may vary from patient to patient or in a single patient over the course of his or her life. Primary care providers, pediatricians, obstetricians, and gynecologists who observe unusual bleeding often refer their patients to a hematologist for further testing and management. Many individuals with mild symptoms do not receive a diagnosis right away and live for many years with untreated bleeding or do not realize they have VWD until they experience a severe bleed that could have been prevented.

“While VWD is a common bleeding disorder, it is also complex, presenting challenges in the timely diagnosis and appropriate management of bleeding for patients,” said 2021 ASH President Martin S. Tallman, MD, of Memorial Sloan Kettering Cancer Center.  “Because diagnosis is not straightforward and symptoms range in severity, there is a need for trustworthy guidelines to help improve the quality of care for patients. There are no better partners for ASH in this effort than ISTH, NHF, and WFH, whose collective expertise in VWD from the scientific and patient perspectives around the world enhanced this effort.”

Notably, the guidelines make key statements on thresholds of laboratory testing to classify VWD to be more inclusive of patients who experience bleeding, but whose blood work does not meet currently accepted thresholds for diagnosis. In addition, the guidelines for the first time recommend that VWD patients who suffer from frequent, severe bleeding that decreases quality of life should receive routine VWD prophylaxis – an injectable concentrate of clotting protein – several times a week. Together, the guidelines on diagnosis and management include a combined 19 recommendations.

The guidelines were developed by two expert panels made up of 32 individuals, including U.S.-based and international hematologists, individuals living with VWD, and scientists with expertise in evidence synthesis and appraisal and guideline development methodology. Clinical questions were developed by the panels and prioritized, and an international survey was completed to identify the most important clinical questions. A systematic review of available evidence was conducted by the University of Kansas Medical Center and the panel referred to this evidence to make recommendations. This process shed light on the lack of strong evidence on which to base recommendations, and therefore the report calls for more research.

“These guidelines are an excellent example of collaboration across multiple societies ensuring that the guidelines develop represent the most up to date and relevant advice about diagnosis and treatment of this common, but often misunderstood bleeding disorder,” said ISTH Past President Claire McLintock, MD. “Working as part of the incredible team that developed the guidelines in diagnosis of VWD was hugely rewarding and inspiring.”

People with VWD made up approximately one quarter of each guideline panel’s composition. As full voting members they contributed their expertise to every aspect of the rigorous guideline development process, from the prioritization of clinical questions, through the evaluation of the available evidence, to the consideration of factors such as stakeholder values and preferences, equity, and feasibility. Their involvement, actively supported by the panel chairs and collaborating organizations, was critical to ensuring the development of recommendations with the greatest pertinence and in which the community will have great confidence. Collaboration with WFH and NHF was essential to engaging the broader VWD patient community.

With their extensive established functional networks with healthcare professionals in centers for comprehensive care of bleeding disorders and patient organizations throughout the US and around the world, the four collaborating organizations, ASH, ISTH, NHF, and WFH will all play important roles in the creation and dissemination of resources based on the guidelines, and their implementation in individual care and broader advocacy efforts.

“These guidelines are an extremely important step in our quest to address the difficulties individuals go through to obtain an accurate, timely diagnosis, and appropriate treatment.  The challenge ahead will be for us to educate both those living with VWD and healthcare professionals on the guideline recommendations,” said Leonard Valentino, MD, President and CEO of NHF. “We were excited to work with ASH, ISTH, and WFH to create these and introduce them to the community.”

“In 2017 we gathered members of the global VWD community for the first WFH International Symposium on VWD. Delegates from countries around the world told us that the number one tool they needed in order to provide comprehensive care to people with VWD was clinical practice guidelines,” explained WFH President Cesar Garrido. “The WFH is very proud to deliver these guidelines today, with our partners from ASH, ISTH, and NHF, to the global community. In addition to offering guidance for clinicians and individuals with VWD as they partner in personalized care decisions, they constitute the foundation for important advocacy initiatives to improve access to the recommended diagnostic techniques and treatment options in all countries.”

The publication of the guideline will be accompanied by tools and educational resources to help patients, hematologists and other health care providers understand and implement the recommendations.

Related links about the new VWD guidelines:

•    VWD guidelines in Blood Advances

•    ASH resources

•    ISTH resources

•    NHF resources

•    WFH resources

About the American Society of Hematology

The American Society of Hematology (www.hematology.org) is the world’s largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 60 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. ASH publishes Blood (www.bloodjournal.org), the most cited peer-reviewed publication in the field, and Blood Advances (www.bloodadvances.org), an online, peer-reviewed open access journal. For more information about ASH’s portfolio of clinical practice guidelines, visit www.hematology.org/guidelines.

About the International Society of Thrombosis and Haemostasis

Founded in 1969, the ISTH is the leading worldwide not-for-profit organization dedicated to advancing the understanding, prevention, diagnosis and treatment of thrombotic and bleeding disorders. ISTH is an international professional membership organization with more than 7,700 clinicians, researchers and educators working together to improve the lives of patients in more than 110 countries around the world. Among its highly regarded activities and initiatives are education and standardization programs, research activities, meetings and congresses, peer-reviewed publications, expert committees and World Thrombosis Day on 13 October. Visit ISTH online at www.isth.org.


About the National Hemophilia Foundation

NHF (the National Hemophilia Foundation) is a 501(c)(3) nonprofit organization dedicated to finding better treatments and cures for inheritable bleeding disorders and to preventing the complications of these disorders through education, advocacy and research. NHF’s programs and initiatives are made possible through the generosity of individuals, corporations and foundations, as well as through a cooperative agreement with the Centers for Disease Control and Prevention (CDC). Additional information on hemophilia and NHF can be found at www.hemophilia.org.

About the World Federation of Hemophilia

For over 50 years, the World Federation of Hemophilia (WFH), an international not-for-profit organization, has worked to improve the lives of people with hemophilia and other inherited bleeding disorders. Our vision of Treatment for All is that one day, all people with a bleeding disorder will enjoy a more certain future filled with promise, no matter where they live. For decades, we have been working around the world to improve diagnosis, treatment and ensure patients have sustainable access to care. We have a responsibility to deliver life-changing programs and services. Established in 1963, the WFH is a global network of patient organizations in 147 countries and has official recognition from the World Health Organization. Visit WFH online at www.wfh.org.

 

 

New Research Fellowship Available from NHF
bmarshall
Jan 11, 2021

Jan 11, 2021

The National Hemophilia Foundation has created a new research fellowship that seeks to create greater diversity in the field of benign hematology with the Jeanne M. Lusher Diversity Fellowship (JML). The fellowship, which hopes to foster career-long interest in inheritable bleeding and blood disorders, is open to Black, Indigenous, and people of color (BIPOC) who are enrolled in a doctoral program in a biomedical field relevant to blood disorders. The goal of the fellowship is to identify clinicians from under-represented communities and genders early in their career and establish an interest in inheritable bleeding and blood disorders that will sustain them throughout their careers.

The fellowship honors the distinguished legacy of Dr. Jeanne M. Lusher, a pioneering clinician and researcher who died in 2016. Dr. Lusher’s tremendous impact on the bleeding disorders community began in 1961 when she helped to diagnose a young girl with a rare instance of hemophilia. As a researcher, she focused on inhibitor development in factor VIII patients. Along with a colleague, she was the first to identify inhibitors as antibodies to factor VIII in 1966. She authored more than 270 peer-reviewed papers, nine books and more than 60 book chapters. Dr. Lusher served as chair of NHF’s Medical and Scientific Advisory Council (MASAC) from 1994-2000.

“Dr. Lusher was a tremendous mentor to young hematologists who wanted to work with patients with bleeding disorders,” said Michelle Wiktop, DNP, NHF’s head of research.  “We hope this new fellowship will eventually create a research community that  reflects the diversity of the patient community we serve.”

More information on the JML Fellowship, including how to apply, can be found here.

The National Hemophilia Foundation has created a new research fellowship that seeks to create greater diversity in the field of benign hematology with the Jeanne M. Lusher Diversity Fellowship (JML). The fellowship, which hopes to foster career-long interest in inheritable bleeding and blood disorders, is open to Black, Indigenous, and people of color (BIPOC) who are enrolled in a doctoral program in a biomedical field relevant to blood disorders.

The National Hemophilia Foundation has created a new research fellowship that seeks to create greater diversity in the field of benign hematology with the Jeanne M. Lusher Diversity Fellowship (JML). The fellowship, which hopes to foster career-long interest in inheritable bleeding and blood disorders, is open to Black, Indigenous, and people of color (BIPOC) who are enrolled in a doctoral program in a biomedical field relevant to blood disorders.

 The American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), National Hemophilia Foundation (NHF), and World Federation of Hemophilia (WFH) have developed joint clinical practice guidelines on the diagnosis and management of von Willebrand disease (VWD), the world’s most common inherited bleeding disorder.

 The American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), National Hemophilia Foundation (NHF), and World Federation of Hemophilia (WFH) have developed joint clinical practice guidelines on the diagnosis and management of von Willebrand disease (VWD), the world’s most common inherited bleeding disorder.

Looking Ahead to 2021
bmarshall
Jan 8, 2021

Jan 8, 2021

As we look ahead into the new year, and the changes it will bring, I’ve been reflecting on the importance of the events of 2020 and what we have learned. This past year, tumultuous and strange as it was, has taught us all some lessons that will guide me, NHF, and the bleeding disorders community in the years ahead.

The first lesson is how important it is to be in regular communication with you. The past year has been one where clear and unambiguous communication of facts has never been more relevant. At the outset of the coronavirus pandemic, we instituted a series of webinars that sought to answer your questions, help you find resources, and increase your understanding of how this virus was affecting our community. In 2021, we will continue this series each Wednesday, bringing you not only the latest information we have about COVID-19, but also information on new vaccines and treatments for coronavirus, new developments in the diagnosis and treatment of bleeding disorders, research into women with bleeding disorders, our approach to overcoming health inequities in the bleeding disorders community and much more.

The second lesson is that it is ever more critical that we listen to you, our community members. We are in an era of transformational discovery in bleeding disorders treatment and care. Gene therapy and nonfactor therapies will change our understanding of what it means to live with a bleeding disorder. But to fully understand it, we must make sure that you—the people who live with a bleeding disorder and your family members—are involved every step of the way. In 2021, we will put a major emphasis on understanding the gaps in care you experience and working with you and our entire community to close those gaps through research. We are currently assembling working groups to discuss, debate and plan the direction of research into bleeding disorders. In September 2021, we will be holding a State of the Science Summit that will bring researchers, healthcare providers, pharmaceutical and biotech companies, and you, together to prioritize and finalize plans for future research. Your voice and active participation along the journey are key to its success. The voice of the community must be at the center of bleeding disorders research. Therefore, I invite each of you to consider participating and contributing to this important work. Look for future news about how you can get involved in surveys, discussion groups and the summit.

The third and most important lesson 2020 provided me is that it is imperative that we expand our reach within the bleeding disorders community. This pandemic, and who bore the greatest burden, has made clear the appalling inequity in access to healthcare in our country – and the care of people with bleeding disorders is no exception. We must guarantee that people of color, people from underserved areas, those who don’t have financial resources to pay for medications and services, and women with bleeding disorders have the same access to treatment and services, are included in research, are welcomed and represented in our chapters, and most important, that their concerns and needs are heard and acted upon.

All these lessons have provided the imperative that NHF must widen our community. In over 35 years as a hematologist, I treated people with hemophilia and von Willebrand disease, but I also treated people with other blood disorders who didn’t have the array of treatment options, research opportunities, clinical services and support that many of you have access to. There are people with rare bleeding disorders, people with sickle cell disease, platelet disorders and Glanzmann’s thrombasthenia, thrombocytopenia, thalassemia, venous thrombosis and other inherited blood disorders who are treated at hemophilia treatment centers yet are not part of our community. In 2021, we will be reaching out to advocacy organizations along with people with these disorders, healthcare providers, and researchers to understand how we can work together to improve the lives of not just people with bleeding disorders, but people with blood disorders. We have a vision of a world without inheritable blood disorders.  We will strive to move our mission to finding cures for inheritable blood disorders and to addressing and preventing the complications of these disorders through research, education, and advocacy, enabling people and families to thrive. Together, with open, direct and honest communication and a renewed focus on research, that includes all members of our community, we can make that mission and vision a reality. Come along with us on this journey—together we are stronger and together we have a louder voice!

Peace and prosperity,



Leonard A. Valentino, MD

A Message from NHF President and CEO Leonard A. Valentino, MD
bmarshall
Jan 8, 2021

Jan 8, 2021

Dear Community,

 

Not a big surprise that the turning of the page in the calendar did not make all our worries go away. The big stories that consumed our thoughts at the end of last year—the election, the pandemic, and racial inequity—carried over and, in the case of the election, boiled over.

 

On January 6th, many of us watched with shock what was occurring in Washington, DC. Regardless of your political beliefs, I’m sure you were as stunned as I was to see thousands of people storming the hallowed halls of our government. I’m angry, saddened, embarrassed, and appalled at the shameful violence and those who took part in this unacceptable behavior. We have also seen similar displays at state capitols throughout the country.  

 

It also made me think about the history of activism in our community. In the 1990s, after thousands of our community members were ravaged by HIV/AIDS, our community protested —angrily, righteously—but peacefully. In a time when bipartisan divisions were high, our community members worked with both Republicans and Democrats not only on the passage of the Ricky Ray Hemophilia Relief Act, but to ensure that our blood and blood products would remain safe for all Americans for generations to come. In doing so, we earned the respect of lawmakers, staff, and set a template for other patient advocacy organizations to enact change at the Federal level.

 

What we witnessed on January 6th was not advocacy. It was a degradation of the freedoms enshrined in the Bill of Rights —to speak without censure from the government, to peacefully assemble. It was an attempt by a small fraction of Americans to subvert the will of the people and our democratic process.

But Congress reassembled in the Capitol that same evening to finish its Constitutional duty to officially certify the Electoral College vote. In one day, we watched a serious threat to our democracy matched by the resilience of our lawmakers and those working to protect it.

We’ve experienced many dark moments together over the past 10 months. All of you have personally experienced disruption and loss but please know that I and the NHF team are here for you, and that you can always reach out directly to me or anyone at NHF if you are feeling stress, especially if it’s affecting your health. We are in this together and are willing to lend support where we can.

Even with all of this, I still have a positive outlook for  2021. I draw inspiration from our history of advocacy, working together to create positive change. That is the spirit that I hope our community will embrace as we prepare to work with a new Administration and Congress in 2021. I hope you will join us at Washington Days in March as we continue to work to ensure access to care for our community.

 

 We will continue to support each other as we journey through 2021.

Stay safe,

Leonard A. Valentino, MD

Dear Community,

 

Not a big surprise that the turning of the page in the calendar did not make all our worries go away. The big stories that consumed our thoughts at the end of last year—the election, the pandemic, and racial inequity—carried over and, in the case of the election, boiled over.

 

Dear Community,

 

Not a big surprise that the turning of the page in the calendar did not make all our worries go away. The big stories that consumed our thoughts at the end of last year—the election, the pandemic, and racial inequity—carried over and, in the case of the election, boiled over.

 

As we look ahead into the new year, and the changes it will bring, I’ve been reflecting on the importance of the events of 2020 and what we have learned. This past year, tumultuous and strange as it was, has taught us all some lessons that will guide me, NHF, and the bleeding disorders community in the years ahead.

As we look ahead into the new year, and the changes it will bring, I’ve been reflecting on the importance of the events of 2020 and what we have learned. This past year, tumultuous and strange as it was, has taught us all some lessons that will guide me, NHF, and the bleeding disorders community in the years ahead.

COVID-19 Vaccines and Bleeding Disorders: Frequently Asked Questions (FAQs)
jangarola
Dec 28, 2020

Dec 28, 2020

NHF recognizes that individuals with bleeding disorders may have questions and concerns relevant to the new COVID-19 vaccines, including any implications specific to their conditions. The following FAQs are therefore meant to address some of the most common questions. Please note that these answers were created for broad purposes and that affected individuals should engage closely with their healthcare provider to discuss the possibilities of vaccination, including potential contraindications (if any), and specific questions related to safety and efficacy. Given the nature of this virus, rapid developments in vaccines, and the upcoming transition in our federal government, this continues to be a very fluid situation.

Two mRNA COVID-19 vaccines – from Pfizer-BioNTech and Moderna – have already been approved and are currently being administered to people in the US and internationally. There are several other vaccines that are in development and will be considered for “Emergency Use Authorization” (EUA) from the U.S. Food and Drug Administration (FDA), including two viral vector vaccines – developed by Oxford-Astrazeneca and Johnson & Johnson.  These additional vaccines are currently in large, phase III clinical trials and yielding promising results thus far. NHF will keep the bleeding disorders community updated as new information becomes available.

 

What is an “Emergency Use Authorization” (EUA)?

An Emergency Use Authorization (EUA) is a mechanism to facilitate the availability and use of medical countermeasures, including vaccines, during public health emergencies, such as the current COVID-19 pandemic. Under an EUA, FDA may allow the use of unapproved medical products in an emergency to diagnose, treat, or prevent serious or life-threatening diseases or conditions such as infection with the virus that causes COVID-19 disease when certain statutory criteria have been met, including that there are no adequate, approved, and available alternatives.

 

These vaccines were developed very fast. Are they safe?

It is true that the Pfizer/BioNTech and Moderna vaccines have been developed very quickly. But the technology behind the vaccines is not new. They both are what are called mRNA vaccines, which function very differently than the types of vaccines used in the past. mRNA vaccines, rather than using a weak or ineffective sample of the virus to trigger antibodies  – like in a flu shot – mRNA vaccines instruct your cells to create a harmless protein (called “spike” protein) that your body recognizes as foreign, creating an immune system response. Scientific papers describing this mRNA technology, discovered by Hungarian scientist Katalin Karikó (who oversees mRNA work for BioNTech) were first published in 2005.

 

Is it safe for a person with a bleeding disorder to receive the COVID vaccine?

If you have a bleeding disorder, there are no contraindications to being vaccinated with either vaccine now available via EUA status for intramuscular administration. It should be noted that, immune tolerance therapy, treatment for hepatitis C, and HIV and other conditions including the use of immunosuppressive agents do not preclude a person from receiving either available vaccines. For patients in a clinical study, vaccination should be reported to the study investigators.

 

Would I be in a priority group to receive the vaccine with a bleeding disorder?

Since people with bleeding disorders are not at a greater risk of contracting COVID19 or developing a severe form of the disease, they are not considered a priority group. General selection rules will apply for those with a bleeding disorder. Bleeding disorder patients in the risk groups identified by age, state of health, health care or other essential worker will be vaccinated as a priority like others in the general population with the same risks. Priority groups will vary by state.

 

Is there a particular type of vaccine I should choose?

Currently there is no reason to choose a particular type of vaccine. If you are considering enrolling in a gene therapy clinical trial, you should avoid any vaccines that use a modified adeno-associated virus (AAV) since these viruses are used in gene therapy. No vaccinations currently approved or in development are using AAV viruses. However, vaccines that are using a different virus, adenovirus, are being tested and are unrelated to AAV therefore would not be a problem with enrolling in a gene therapy clinical trial in the future.

 

Will I have a bleed if I take the vaccination?

The vaccination is administered intra-muscularly but the smallest gauge needle needs should be used (25-27 gauge), if possible. Some vaccines must be administered using the accompanying needle–syringe combination, and so the use of an alternative needle may not be possible or desirable.

It would be preferable for you to infuse with a factor replacement product prior to or right after the vaccination and applying pressure for 10 minutes after the vaccination. Patients receiving emicizumab may be vaccinated by intra-muscular injection at any time without receiving an additional dose of FVIII. Patients with von Willebrand Disease or rare bleeding disorders should consult with their hematologist regarding special precautions prior to receiving the vaccination. All rare bleeding disorder patients (including those with thrombocytopenia and/or platelet function disorders) should be vaccinated. Patients on anticoagulants should have prothrombin time testing performed within 72 hours prior to injection to determine international normalized ratio (INR); if results are stable and within the therapeutic range, they can be vaccinated intramuscularly.

Following the vaccination, the area should be monitored for hematoma formation immediately for 10 minutes to reduce bleeding and swelling and by self-inspection 2-4 hours later at home to ensure that there is no delayed hematoma. Discomfort at the injection site is to be expected. Discomfort in the arm felt for 1-2 days after injection should not be alarming unless it worsens and is accompanied by swelling.

Any adverse events should be reported to the physician and any allergic reactions need to be reported immediately to your physician or you need to go to the emergency room. To read more about vaccinations in general please click on this link  for NHF’s Medical and Scientific Advisory Council’s recommendations, MASAC Document 221 – Recommendations on Administration of Vaccines to Individuals with Bleeding Disorders.

 

Should I take the vaccination if I am pregnant or breastfeeding?

Currently, there is limited data on the safety of COVID-19 vaccines for women who are pregnant or breastfeeding, although studies are planned. The Centers for Disease Control and Prevention (CDC) and the FDA have safety monitoring systems in place to capture information about vaccination during pregnancy and will closely monitor reports. Individuals who are pregnant and considering vaccination for COVID-19 are encouraged to speak with their clinicians. According to the CDC, key considerations that should inform these conversations include likely exposure to the virus that causes COVID-19; risks of COVID-19 to the pregnant patients and their fetus; and current knowledge about available vaccines including efficacy, known side effects, and the lack of available data in this population. 

 

Below are some helpful resources:

How COVID-19 Vaccines Work (CDC)

COVID-19: Mitigation, Treatment, and Vaccines (NHF Webinar)

MASAC Document 221 – Recommendations on Administration of Vaccines to Individuals with Bleeding Disorders.

Fact Sheet for Recipients and Caregivers – EUA of the Pfizer-BioNTech COVID-19 Vaccine to prevent Coronavirus Disease 2019 in Individuals 16 Years of Age and Older

Fact Sheet for Recipients and Caregivers – EUA of the Moderna COVID-19 Vaccine  to Prevent Coronavirus Disease 2019 in individuals 18 Years of Age and Older

COVID-19 Vaccination Considerations for People Who Are Pregnant (CDC)

Last revised: December 21, 2020

NHF recognizes that individuals with bleeding disorders may have questions and concerns relevant to the new COVID-19 vaccines, including any implications specific to their conditions. The following FAQs are therefore meant to address some of the most common questions. Please note that these answers were created for broad purposes and that affected individuals should engage closely with their healthcare provider to discuss the possibilities of vaccination, including potential contraindications (if any), and specific questions related to safety and efficacy.

NHF recognizes that individuals with bleeding disorders may have questions and concerns relevant to the new COVID-19 vaccines, including any implications specific to their conditions. The following FAQs are therefore meant to address some of the most common questions. Please note that these answers were created for broad purposes and that affected individuals should engage closely with their healthcare provider to discuss the possibilities of vaccination, including potential contraindications (if any), and specific questions related to safety and efficacy.

COVID-19 Vaccination Guidance from NHF, WFH, EAHAD, and EHC for the Bleeding Disorders Community
vprice
Dec 23, 2020

Dec 23, 2020

COVID-19 vaccination guidance for people with bleeding disorders

Guidance from the World Federation of Hemophilia (WFH), European Association for Haemophilia and Allied Disorders (EAHAD), European Haemophilia Consortium (EHC), and U.S. National Hemophilia Foundation (NHF)

It is important that hemophilia treatment centres, in close collaboration with patient organizations, take action to inform people with bleeding disorders about the COVID-19 vaccines and contribute to an effective vaccination program.

 

  1. People with bleeding disorders are not at greater risk of contracting COVID-19 or developing a severe form of the disease, so they are not considered a priority group for vaccination.

 

  1. The vaccine should be administered intramuscularly. The smallest gauge needle available (25-27 gauge) should be used, if possible. Some vaccines must be administered using the accompanying needle–syringe combination, and so the use of an alternative needle may not be possible or desirable. Pressure should be applied to the site for at least 10 minutes post-injection to reduce bleeding and swelling. Additionally, self-inspection/palpation of the injection area several minutes and 2-4 hours later is recommended to ensure that there is no delayed hematoma. Discomfort in the arm felt for 1-2 days after injection should not be alarming unless it worsens and is accompanied by swelling. Any adverse events (e.g., hematoma, allergic reaction) should be reported to a hemophilia treatment centre.

 

  1. Patients should contact their physician immediately or go to the nearest hospital emergency room right away if they experience an allergic reaction (fever, warmth, redness, itchy skin rash, shortness of breath, or swelling of the face or tongue) as it can be life-threatening. Patients with a history of allergic reactions to extended half-life clotting factor concentrates containing polyethylene glycol (PEG) should discuss vaccine choice with their physician because some vaccines contain PEG as an excipient.

 

  1. Many individuals with bleeding disorders may not have ready access to hemostatic therapies prior to vaccination. In these cases, make efforts to access other clotting factors if possible. Alternatively, follow the instructions above making sure the smallest possible needle is used and maintain pressure for more than 10 minutes.

 

  1. For patients with severe/moderate hemophilia, the injection should be given after a factor VIII (FVIII) or factor IX (FIX) injection. For patients with a basal FVIII or FIX level above 10%, no hemostatic precautions are required.

 

  1. Patients on emicizumab (with or without an inhibitor) can be vaccinated by intramuscular injection at any time without hemostatic precautions and without receiving a dose of FVIII.

 

  1. Patients with Type 1 or 2 Willebrand disease (VWD), depending on their baseline von Willebrand factor (VWF) ristocetin cofactor (RiCof) activity levels, should use therapies (i.e., DDAVP if available, tranexamic acid), in consultation with their hemophilia treatment centre. Patients with Type 3 VWD should be given a VWF-containing injection.

 

  1. All rare bleeding disorder patients (including those with thrombocytopenia and/or platelet function disorders) should be vaccinated. Patients on anticoagulants should have prothrombin time testing performed within 72 hours prior to injection to determine international normalized ratio (INR); if results are stable and within the therapeutic range, they can be vaccinated intramuscularly.

 

  1. There are no specific contraindications to vaccination related to complications of hemophilia and other bleeding disorders or their therapies. Immune tolerance, treatment of hepatitis C and HIV, and other conditions do not contraindicate vaccination.

 

  1. Vaccination is not contraindicated for patients on immunosuppressive agents (cortisone, other immunosuppressive drugs).

 

  1. Potential contraindications should be discussed individually with the physician because recommendations vary in different jurisdictions due to lack of data in special populations (e.g., pregnant or breastfeeding women).

 

  1. The U.K. Medicines and Healthcare Products Regulatory Agency and the U.S. Centers for Disease Control and Prevention have advised caution in using the Pfizer/BioNTech vaccine in people with a history of significant allergic reactions. Specific recommendations for people with a history of allergic/anaphylactoid reactions can be found in the advisory published by each agency.

 

  1. For patients in a clinical study, vaccination should be reported to the study investigators.

 

Information will be updated on the WFH, EAHAD, EHC, and NHF websites as needed.

COVID-19 vaccination guidance for people with bleeding disorders

COVID-19 WFH Announcements and Statements

Specific Risks of COVID-19 to the Bleeding Disorders Community

COVID-19: Practical Recommendations for People with Hemophilia

Practical guidance for the in‐hospital management of PWH published in Haemophilia

COVID-19 vaccination guidance for people with bleeding disorders

Guidance from the World Federation of Hemophilia (WFH), European Association for Haemophilia and Allied Disorders (EAHAD), European Haemophilia Consortium (EHC), and U.S. National Hemophilia Foundation (NHF)

It is important that hemophilia treatment centres, in close collaboration with patient organizations, take action to inform people with bleeding disorders about the COVID-19 vaccines and contribute to an effective vaccination program.

 

COVID-19 vaccination guidance for people with bleeding disorders

Guidance from the World Federation of Hemophilia (WFH), European Association for Haemophilia and Allied Disorders (EAHAD), European Haemophilia Consortium (EHC), and U.S. National Hemophilia Foundation (NHF)

It is important that hemophilia treatment centres, in close collaboration with patient organizations, take action to inform people with bleeding disorders about the COVID-19 vaccines and contribute to an effective vaccination program.

 

NHF Applauds Congressional Enactment of The Hemophilia SNF Access Act
vprice
Dec 22, 2020

Dec 22, 2020

The National Hemophilia Foundation is thrilled to announce that its top legislative priority in 2020, The Hemophilia SNF Access Act, was included in the end-of-year omnibus federal spending and economic relief package passed by Congress on December 21, 2020 (HR 133, The Consolidated Appropriations Act, 2021).  The bill now goes to President Trump for his signature, which is expected in the coming days.

This critical legislation will rectify a long-standing problem to improve access to skilled nursing facilities (SNFs) for Medicare beneficiaries with hemophilia and other bleeding disorders.  NHF has heard from many community members over the years about challenges accessing SNF facilities due to the way that Medicare reimburses bleeding disorders treatments. 

The legislation, introduced in January 2020 in the Senate by Senators Bob Menendez (D-NJ), Michael Enzi (R-WY) and Sheldon Whitehouse (D-RI) and in the House in February 2020 by Representatives Darin LaHood (R-IL), Brian Higgins (D-NY), Debbie Dingell (D-MI) and Gus Bilirakis (R-FL), is the culmination of years of continued efforts by NHF and other bleeding disorders organizations.

 “We have been advocating to improve access to skilled nursing facilities for Medicare beneficiaries with bleeding disorders for many years,” said Dr. Leonard A. Valentino, president and CEO of the National Hemophilia Foundation. “NHF applauds Congress for enacting this vital legislation, which will benefit the hundreds of Americans with bleeding disorders who are Medicare beneficiaries as soon as October 1, 2021.  It may seem like a small shift in policy, but it will have a huge impact on our community.”

The bill was a top talking point for the 450 volunteer advocates with bleeding disorders who participated in NHF’s annual advocacy event, Washington Days, on February 28th and many other advocates contacted their Congressional representatives in support of the bill throughout the year.  “Our thanks go not only to our lead champions and their tireless staff who pushed for the SNF change to be included in the year end bill, but also to the hundreds of bleeding disorders advocates – patients, families and hemophilia treatment center staff – who raised their voices in support of the bill this year. We could not have done it without you!” said Nathan Schaefer, NHF’s vice president of public policy.

The National Hemophilia Foundation is thrilled to announce that its top legislative priority in 2020, The Hemophilia SNF Access Act, was included in the end-of-year omnibus federal spending and economic relief package passed by Congress on December 21, 2020 (HR 133, The Consolidated Appropriations Act, 2021).  The bill now goes to President Trump for his signature, which is expected in the coming days.

The National Hemophilia Foundation is thrilled to announce that its top legislative priority in 2020, The Hemophilia SNF Access Act, was included in the end-of-year omnibus federal spending and economic relief package passed by Congress on December 21, 2020 (HR 133, The Consolidated Appropriations Act, 2021).  The bill now goes to President Trump for his signature, which is expected in the coming days.

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