Hawaii Chapter - NHF
Phone 808-284-7417

NEWS


If you want to be in the know about what’s going on at our organization, you’ve come to the right place!

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


BioMarin recently shared a community update on their Hemophilia A Clinical Development Program for valoctocogene roxaparvovec, the company’s investigational gene therapy.

WASHINGTON, D.C.— December 31, 2021 — In response to additional rulemaking for the Centers for Medicare & Medicaid Services’ (CMS)

FOR IMMEDIATE RELEASE

Media & Community Inquiries

Brett Spitale, VP of Advancement

Dec. 31, 2021 – New York, NY – NHF is deeply saddened to announce that former NHF CEO and community member, Val Bias, passed away suddenly on Thursday, Dec. 30.

Val and I first got to know one another 30 years ago, when we were opposing one another in an election for president of the National Hemophilia Foundation (US). I was fighting for HIV/AIDS advocacy from within, from the Board, and Val was part of a more activist group that was impatient the NHF wasn’t doing enough, fast enough. He and I came together, realized our goals were the same, and also recognized we were so much stronger together.

Results of a recently published study suggest that intense physical activities, particularly those that include a high collision-risk, can increase bleeding risk in individuals with severe or moderately severe hemophilia A. Investigators also found that increasing the time between the administration of factor (FVIII) and physical activity also heightened bleeding risk.


Sigilon Therapeutics recently announced a “strategic reprioritization,” as the company shifts focus to their non-hemophilia, investigational therapies, including those for MPS-1 and diabetes. This closely follows recent setbacks associated with their phase 1/2 clinical study of SIG-001 in individuals with severe or moderately severe hemophilia.

Interested in working in the hematology field, but don’t think that medical school is right for you? Consider an allied health field, like genetic counseling or physical therapy. Recently, NHF sat down with Mitch Starkman, a physical therapist, and Stefanie Dugan, a genetic counselor, to discuss their career paths and professional experiences. Read these Q&As and watch their respective videos to learn more! 

 

The onset of childbirth and the postpartum period are times when women with von Willebrand disease (VWD) are at an increased risk for excessive bleeding, exposing them to further, and in some instances, serious complications. While there exist therapies with VWD-specific indications, it is not uncommon for these patients to still experience excessive bleeding while receiving treatment. These scenarios are challenging as there is sparce clinical data and a subsequent lack of clear guidance on the optimal management of bleeding in these particular settings.

uniQure and CSL Behring recently announced that etranacogene dezaparvovec, an investigational hemophilia B gene therapy currently in clinical trials, has achieved a primary endpoint of “non-inferiority” in annualized bleeding rate (ABR) 18-months post administration. The non-inferiority component of a clinical trial measures whether the investigational therapy is not worse/less efficacious than a currently available product. In this case, etranacogene dezaparvovec is being compared to baseline factor IX prophylactic replacement therapy as part of the phase III HOPE-B pivotal trial.

PRESS RELEASE





FOR IMMEDIATE RELEASE

MEDIA CONTACT:

Sigilon Therapeutics today reported a new development in the company’s phase 1/2 clinical study of SIG-001 in individuals with severe or moderately severe hemophilia. This follows the events of this past summer when the U.S. Food and Drug Administration (FDA) placed a clinical hold on the trial due to an unwanted inhibitor response in one of the three patients participating in the study.

Historically, data on infants and toddlers (ITs) with von Willebrand disease (VWD), particularly relevant to bleeding patterns, has been lacking. To address this absence of data, a team of researchers from the U.S. Hemophilia Treatment Center Network (USHTCN) and the Centers for Disease Control and Prevention conducted retrospective analysis of HTC patients with VWD who are less than two years of age. The data were obtained through the USHTCN. The results were published in the journal Blood Advances.

Spark Therapeutics announced updated data from a phase 1/2 clinical trial of SPK-8011

For more than 40 years, the hemophilia treatment center (HTC), the U.S. HTC Network (USHTCN), and its model of integrated, patient-centered care has been essential in helping people with hemophilia (PWH) achieve optimal health outcomes.

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NHF Statement In Response to New Michigan Law:

Media Contact:

Ilana Ostrin

212 328 3769


iostrin@hemophilia.org

The Indiana Hemophilia & Thrombosis Center and Partners in Bleeding Disorders Education Program have announced a new education program designed for physicians who practice at federally funded hemophilia treatment centers (HTCs) in the U.S. The Partners Physician Academy (PPA) will focus specifically on supporting and accelerating third-and-fourth year fellows and junior physicians for up to two years post-fellowship.

NHF is very pleased to announce that the Kevin Child Scholarship (KCS) application deadline has been set for August 2, 2021! The scholarship is named in tribute to Kevin Child, who died of AIDS in 1989, shortly before graduating from college. It is awarded annually to a student with hemophilia who is about to enter or currently enrolled in a program of higher education.  

Shared decision making (SDM), the concept by which patients and healthcare providers work closely together to arrive at personalized treatment-related decisions, can have significant positive impacts for individuals with hemophilia.

A new paper published in The Journal of Haemophilia Practice advances the concept of shared decision making (SDM) between the hemophilia patient and their healthcare provider (HCP).

A new article, “Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia,” was published in the Journal of Clinical Medicine (JCM).



In this review, the authors discuss some of the primary considerations relevant to investigational gene therapies that employ adeno-associated viral (AAV) vectors, with particular focus on immunogenicity and genotoxicity – the former denotes the ability of a foreign substance to trigger an immune response, while the latter refers to a substances ability to damage genetic material.

FOR IMMEDIATE RELEASE

Media Contact

Ilana Ostrin

iostrin@hemophilia.org

212-328-3769

FOR PUBLIC COMMENT - JUNE 3, 2021

BioMarin recently reported key findings from preclinical studies of their investigational, gene therapy Roctavian (valoctocogene roxaparvovec), which was developed for the treatment of adults with severe hemophilia A. The findings were presented at the American Society of Gene & Cell Therapy (ASGCT) Virtual Meeting, which was held May 11-14, 2021.

The National Hemophilia Foundation (NHF) today, in conjunction with the American Kidney Fund (AKF), Arthritis Foundation, and American Autoimmune Related Diseases Association (AARDA), released findings from a new national online survey of patients and caregivers that illustrates the alarming challenges t

Important research using data from the My Life, Our Future (MLOF) Research Repository Phase One has been published in Frontiers in Medicine.

“Scholarship season” is now in full swing, time for an appointment reminder!

uniQure recently announced that their commercialization and license agreement with CSL Behring  for their investigational hemophilia B gene therapy was finally closed on May 5, 2021 – the agreement had been pending the completion of a series of antitrust reviews in the U.S., Australia, and the U.K.

Findings from a recently published study in the Journal of Anesthesia suggest that pregnant patients with hemophilia whose factor VIII or IX levels drop below 50% at the time of receiving neuraxial anesthesia are more likely to experience postpartum complications.

Hemophilia Treatment Centers (HTCs) across the United States are participating in the 2021 National HTC Patient Satisfaction Survey. If you, or someone you care for, had contact with the HTC in 2020, they want to hear from you! You can take the survey online at www.htcsurvey.com, or fill out the paper survey you received in the mail. The survey closes on June 30, 2021.

NHF's Wednesday Webinars for May 2021 will focus on Women's Health Month,  featuring topics  of interest to both patients and healthcare providers who are interested in issues faced by women with inheritable blood disorders.

May Webinars

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

May 5, 2021

VWD Guidelines: Diagnoses

Speaker: Robert Sidonio, MD

 

May 12, 2021

uniQure recently announced that the U.S. Food and Drug Administration (FDA) has removed a clinical hold on their hemophilia B gene therapy program. The FDA had determined that uniQure satisfactorily addressed all issues raised by the agency that were related to a single patient diagnosed with hepatocellular carcinoma (HCC), a common form of liver cancer.

Kevin Mills, PhD has joined the National Hemophilia Foundation as chief scientific officer.

A graduate of University of Colorado Boulder, MIT, and Harvard Medical School, Dr. Mills will be responsible for overseeing NHF’s research strategy, including developing and expanding the foundation’s research capabilities, and setting research goals.

Dear NHF Family,

Like everyone else, I was closely following the trial and eventual verdict in the case of Derek Chauvin for the murder of George Floyd, and I know that many of you were anxiously awaiting as well. Mr. Floyd’s death was senseless and an ugly act of violence.

It was my hope that the jury would evaluate the facts and render a a fair verdict, and in turn, pave the way for greater accountability at all levels of our society, including law enforcement.

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.

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.

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Hawaii Chapter - NHF
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Phone 808-284-7417

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