Hawaii Chapter - NBDF
Phone 808-284-7417

News

Check out the NBDF newsfeed below or CLICK HERE to see our Koko Ohana Newsletter.


A new paper published in The Lancet Haematology journal includes data from the HOPE-B clinical trial program that is investigating the hemophilia B gene therapy etranacogene dezaparvovec – the product’s commercial name is Hemgenix® (CSL Behring).

 

Keeping Your Informed 

The HANDI Resources Center Team is pleased to announce new 2024 scholarship opportunities available for the bleeding disorders community!

Government Relations Update – March 2024

Federal: NBDF’s 2024 Washington Days was held March 6-8.  NBDF is grateful to the more than 400 volunteer advocates from forty-four states who participated in the 231 meetings with Members of Congress and the state advocacy training.  This year’s priority issues are the HELP Copays Act (HR 830/S 1375), appropriations for NIH and CDC programs, and federal action addressing commercial health plans’ narrow prescription drug formularies and exclusions.

State

NBDF recently learned that Novo Nordisk now has 3,000 IU vial size of Rebinyn® back in stock. Rebinyn is the company's recombinant factor IX extended half-life therapy for the treatment of hemophilia B. It is indicated for the on-demand treatment and control of bleeding episodes and the management of bleeding around surgery. It is also indicated for routine prophylaxis to reduce the frequency of bleeding episodes.

The U.S. Food and Drug Administration (FDA) has approved a supplemental Biologics License Application for IXINITY® (Medexus), a recombinant factor replacement therapy to treat patients with hemophilia B. 

The new approval expands the product indication to include the on-demand, prophylactic, and perioperative treatment of children under 12 years of age. The previous indication was limited to adults and older children more than 12 years of age.

For Immediate Release 

CONTACT:  

Carrie Strehlau and Kyla Clark 

National Bleeding Disorders Foundation 

212-382-3773 

kclark@hemophilia.org%C2%A0" title="Email for Kyla Clark">kclark@hemophilia.org 

 

National Bleeding Disorders Foundation Celebrates Five Years of Community Voices in Research 

NBDF is pleased to announce HANDI Highlights, which is designed to connect the bleeding disorders community with timely, practical, and readily-accessible resources.

Takeda recently announced positive results from a phase 2 clinical trial of their investigational therapy (Mezagitamab), to treat patients with persistent or chronic primary immune thrombocytopenia (ITP). The therapy is developed with laboratory engineered monoclonal antibodies, which are cloned versions of human antibodies produced by the body’s immune system.

The National Bleeding Disorders Foundation (NBDF) marked its 75th Anniversary in 2023 by recognizing the contribution of various community stakeholders. As part of this campaign, a video was created to celebrate the pivotal role of nursing in comprehensive care for bleeding disorder patients. The video features interviews with experienced nurses who for decades have cared for patients at hemophilia treatment centers throughout the U.S. 

The Partners Physician Academy (PPA) is offering a new training course for early career physicians practicing in hematology and specializing in bleeding disorders care.

The training will cover the key conditions that physicians treat and manage when working within the hemophilia treatment center network, such as hemophilia, von Willebrand disease (VWD), and rare bleeding disorders.

Happy Bleeding Disorders Awareness Month! In keeping with the spirit of the month, NBDF’s HANDI Resource Center is pleased to announce the availability of a new Hemophilia Fact Sheet. This two-page PDF document was created to fill the need for a current, accessible resource containing basic information about hemophilia.

Government Relations Update – February 2024 

A new study published in the journal Blood Coagulation & Fibrinolysis, looked at the impact of hereditary factor X deficiency (HFXD) on patients and caregivers in the United States. HFXD is an ultra-rare bleeding disorder that occurs in approximately 1 in 500,000 to 1 in a million people globally.

We are pleased to announce that the Emergency Care for Patients with von Willebrand Disease treatment manual is now available in a Spanish language version. Funding for the translation, Cuidado de Emergencia a Pacientes con Enfermedad de von Willebrand, was provided by Akron Children’s Hospital.

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NBDF employees are talented, dynamic, and passionate individuals dedicated to supporting the inheritable blood and bleeding disorders community. They value diversity, accountability, service, respect, and support.

Phil Gattone, M.Ed., NBDF President and CEO 

Federal:

The U.S. Food and Drug Administration (FDA) has approved Fabhalta® (iptacopan), the first oral therapy for the treatment of adults with a rare blood disorder known as paroxysmal nocturnal hemoglobinuria (PNH). It is manufactured by Novartis.

PNH is an acquired (not inherited) condition that causes the immune system to attack and prematurely breakdown red blood cells and platelets. Left untreated, the condition can become serious and even be life threatening. While it can occur at any age, it is most often diagnosed in young adults.

Novo Nordisk recently informed NBDF that they are experiencing a temporary shortage of their product Rebinyn®, specifically in the 3,000 IU vial size. Rebinyn is a recombinant factor IX extended half-life therapy for the treatment of hemophilia B.

It is indicated for the on-demand treatment and control of bleeding episodes and the management of bleeding around surgery. It is also indicated for routine prophylaxis to reduce the frequency of bleeding episodes.

For Immediate Release

CONTACT:

Carrie L. Strehlau

National Bleeding Disorders Foundation

cstrehlau@hemophilia.org

901-230-4665

National Bleeding Disorders Foundation Names Phil Gattone, M.Ed., Next Chief Executive Officer

Gattone brings decades of experience in nonprofit community engagement, fundraising, strategy

Celebrate Black History Month with NBDF! 

This year we will share stories from the Black community within the bleeding disorders community. These individuals are truly an inspiration to the entire inheritable blood and bleeding disorders community. We hope that you take the time to read their stores and that their courage is an inspiration to you! 

In recent years, the arrival of novel hemophilia treatments that are effective and less burdensome to administer have opened up new possibilities for the consumers of these therapies. This begs the question – How might this evolving landscape be impacting treatment preferences for people with hemophilia? 

Takeda announced today that it is conducting a voluntary market withdrawal for two product lots of 650 IU VONVENDI® [von Willebrand factor] in the U.S. Takeda said that the withdrawal is being conducted out of an abundance of caution due to misprinted product labels with the incorrect expiration date.

The expiration date printed on the label on the outside of the package of the affected lots is six months after the actual expiration date (January 27, 2025).

Federal  Updates:

Pfizer has announced positive results from their global phase 3 BASIS clinical study of marstacimab, the company’s investigational, subcutaneous therapy that targets an anticoagulant protein known as tissue factor pathway inhibitor (TFPI).

Dear National Hemophilia Foundation Community, Staff and Friends,

When Saylor Behrens' biology professor told her class that hemophilia is the result of incest, she grew angry. She knew better. Her younger brother, Wyatt, has hemophilia A.

It wasn't just the false narrative being spread that made her indignant- it was the sheer lack of awareness about hemophilia that morphed her brother's bleeding disorder into a scary, incredibly rare condition that could be brushed off. 

Every day, chapters across the country work with the blood and bleeding disorders community around them. Since 2021, Jacob Murdock has served as Chapter Executive Director in Nevada. He took time out of his busy schedule to tell us why loves the Nevada Chapter.

How long has your Chapter been serving the community?

We were founded as the Hemophilia Foundation of Nevada in 1990. We became a national chapter of NHF in 2011. The chapter was founded by Renee Paper, a nurse and a pioneer in the treatment of women with bleeding disorders.

The Rare Diseases Diversity Coalition (RDDC) is currently seeking applicants for its 2023 Rare Disease Fellowship Program, which is aimed at inspiring the next generation of the medical workforce to work in the rare disease space and achieve greater health equity for people of color living with rare diseases.

The Fellowship allows rare disease organizations to collaborate with fellows to better understand the health impacts of that disease on communities of color. In addition, the Fellow will gain a greater awareness of the challenges of living with rare diseases

NHF’s Medical and Scientific Advisory Council (MASAC) recently issued four new documents, which are now available for easy access.

In July 2020 Ferring Pharmaceuticals suspended manufacturing of the nasal spray  STIMATE®, a popular product used to treat certain bleeding disorders.

Between 2013-2017, the “My Life Our Future” (MLOF) project offered eligible individuals with hemophilia free genotyping, which is historically hard to access, expensive, and not covered by insurance.

Novo Nordisk has notified the National Hemophilia Foundation that they have received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for their investigational, subcutaneous therapy concizumab.

Federal Updates:

FOR IMMEDIATE RELEASE

Ilana Ostrin

Senior Director of Public Relations and Communications

The foundation's staff is comprised of dedicated people use their talents to help the blood and bleeding disorders community. One of them is Karina Lopez, MPH! Karina joined us to talk about her progression from an internship to a full-time position as a Project Management Specialist at the organization and one of the projects she's most proud of during her time here.

Watch the interview or read the full conversation below.

Having everyone's perspectives is critical to forming solid research or a well-rounded program. But how do you get the perspectives of people who are not already be involved, whether by choice or circumstance? HEDI Program Specialist, Marissa Melton, starts a conversation on how to engage unengaged communities.

As NHF continues to celebrate it's 75th Anniversary, the organization sat down with community member Eldon Ham to learn more about his story.

For Immediate Release

Renee Peck

Pathway to Cures
rpeck@pathwaytocures.org

212-328-3790

 

New York, NY, February 27, 2023 – Pathway to Cures (P2C), a venture philanthropy fund focused on transformative treatments for inheritable blood disorders, announces the appointment of its investment committee.

Coalition Advocates for Substance Use Disorder/Behavioral Health Services for Patients With Bleeding Disorders

Sanofi recently announced that results of two clinical studies of their investigational therapy fitusiran were recently published in The Lancet and The Lancet Haematology journals. Both phase 3 trials were evaluating the efficacy and safety of fitusiran for the prophylactic treatment of adults and adolescents with hemophilia A or B. The enrolled participants, all of whom are 12 years of age and up, had been previously treated with existing factor replacement therapy or bypassing agent (BPA) prophylaxis.

Federal Updates:

Washington Days Recap: Nearly 400 volunteer advocates attended Washington Days March 8-10th and participated in 235 meetings with Members of Congress and staff.  The focus of this year was soliciting cosponsors for H.R. 830, the HELP Copays Act, and securing level funding for the federal hemophilia programs.

During legislative session, NHF's chapter network, public policy staff, and the bleeding disorders community at large has been hard at work advocating across the United States! For the month of March, here are some advocacy and policy highlights from the beginning of this year:

To end Bleeding Disorders Awareness Month and celebrate National Doctors Day, Dr. Aaron Chen shares his story of how living with thalassemia inspired his calling as a physician. Watch his interview or read the full transcript below.

April is National Minority Health Month. To learn about the experiences of the bleeding disorders community in an island state, Chloe, a member of the communications team, sat down with Kyra Calbero, Interim Executive Director of the foundation's Hawaii chapter.

Watch a video below that summarizes the conversation, or read the whole transcript.

The foundation's staff is comprised of dedicated people use their talents to help the blood and bleeding disorders community.

A new member of the team is Chloe Fellwock, a communications assistant who joined began her tenure in June 2022. A member of the communications team sat down with her to talk about working this role as her first job out of college, what she's learned, and her favorite moments with the team.

Answering Your Questions on the Proposed Change to the FDA's Blood Donation Policy

Akron Pharmaceuticals, one of several companies that manufactures and markets the product aminocaproic acid, recently filed for chapter 7 bankruptcy and subsequently closed.   

Researchers from the Children’s Healthcare of Atlanta and Emory University recently published the results of a small study investigating the subject of guilt in mothers of children with hemophilia (CWH).

March 15, 2023 – The National Hemophilia Foundation (NHF) today announced that a series of manuscripts central to advancing patient-focused research for the inherited bleeding disorders (IBD) community have been published in the journal Expert Review of Hematology. These papers describe initial recommendations from six multi-disciplinary working groups established to evaluate and address the most urgent priorities for the community, and will form the basis for NHF’s community-driven National Research Blueprint (NRB).

Resource Links

Hawaii Chapter - NBDF
75-5660 Kopiko Street, Suite C7 #294
Kailua-Kona, HI 96740
Phone 808-284-7417

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