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


Advanced Considerations for the Recognition, Appropriate Diagnosis, and Timely Management of von Willebrand Disease and Other Bleeding Disorders among Women in Non-Hematology Health Care Settings

Take part in a survey to shape VWD Guidelines. We want to hear from people with VWD, people who care for those with VWD, or clinicians and other healthcare professionals who treat people with VWD.

A recent article appearing in the journal Haemophilia, includes updates on the My Life Our Future (MLOF) project, which was established in 2012 to offer U.S. hemophilia patients ready access to free genotyping, a type of genetic testing that is often hard to access, expensive and not covered by insurance. MLOF is a partnership between the American Thrombosis and Hemostasis Network (ATHN), the National Hemophilia Foundation (NHF), Bloodworks Northwest and Bioverativ (formerly Biogen).

Medscape has launched the latest educational activity in their “Clinical Advances in Gene Therapy for Hemophilia” series, which is presented through a collaboration between Medscape, the National Hemophilia Foundation, the European Haemophilia Consortium and the World Federation of Hemophilia. The program is supported by an independent educational grant from BioMarin.

The goal of this effort is to create and maintain state-of-the-art guidelines on the diagnosis and management of VWD.

Catalyst Biosciences, Inc. recently released an update relevant to an ongoing Phase 1/2 clinical trial for CB 2679d/ISU304, the company’s investigational therapy for the treatment of patients with severe hemophilia B/factor IX (FIX) deficiency. Catalyst develops its therapies using engineered proteases, enzymes that increase the rate of chemical reactions within cells. These proteases can target the proteins associated with certain underlying diseases and conditions, such as FIX.

Because of their lower factor levels, hemophilia patients are generally thought to be better protected from cardiovascular disease (CVD). To determine whether data would support this long-held assumption, a team of investigators enrolled patients from 19 U.S. hemophilia treatment centers (HTCs) in the “CVD in Hemophilia” study. The lead author of the study was Barbara Konkle, MD, Professor of Medicine in the Division of Hematology at the University of Washington School of Medicine in Seattle, WA.

HTC Studies Cardiovascular Disease in Hemophilia Patients

Investigators looked into whether having hemophilia protected patients against cardiovascular disease.

Update on Latest ACA Lawsuit

More information on how the current ACA lawsuit could affect the bleeding disorders community.

Grifols Announces Recall of One Lot of Profilnine. This recall is being conducted as a precautionary measure.

Genentech Announces the Launch of New Safety Website

The new web portal for patients and caregivers is intended to provide timely and accurate information on targeted serious adverse events of interest for HEMLIBRA.

This priority review is specifically for adults and children with hemophilia A without FVIII inhibitors.

Roche Holding AG announced that the U.S. Food and Drug Administration (FDA) has granted “priority review” to HEMLIBRA® a subcutaneously administered therapy currently being investigated for the treatment of patients with hemophilia A, without factor VIII (FVIII) inhibitors. The therapy, which is co-developed by Genentech, Chugai and Roche, is already FDA-approved for routine once-weekly prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with FVIII inhibitors.

CSL Behring recently announced that the US Food and Drug Administration (FDA) has approved a new 3500 IU (international unit) vial size for the company’s recombinant factor IX (rFIX) product Idelvion®. Idelvion combines or “fuses” recombinant factor IX (rFIX) with albumin, a protein found in blood plasma that helps factor IX stay active in the bloodstream longer.

uniQure presented new hemophilia B clinical trial data on May 19, 2018 at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Chicago, IL. The data showed its hemophilia B investigational gene therapy candidate AMT-060 could be successfully delivered to patients with certain pre-existing antibodies. Individuals testing positive for “anti AAV5 neutralizing antibodies” (NABs), are often excluded from trials, as they were thought to impede the delivery of some gene therapies.

Our dedicated providers and inspiring volunteers and advocates can be recognized with an Award of Excellence!

Our dedicated providers and inspiring volunteers and advocates can be recognized with an Award of Excellence!

New Study on Aging, VWF Levels and Bleeding Risk

Investigators looked to establish whether VWF levels increase as patients with VWD age, and if so, whether this can be associated with lower risk for bleeding.

NHF's Dawn Rotellini Elected to WFH Board of Directors

"It has become my passion to make a difference for people throughout the world. We all need access to treatment and care and education.”

Spark Sees Continued Progress in Hemophilia B Gene Therapy

The overall annualized bleeding rate for all 15 participants was reduced by 98% to an annual rate of 0.2 bleeds per patient.

In the Phase III HAVEN 3 study, patients who received HEMLIBRA prophylaxis every week or every two weeks experienced a 96% and 97% reduction in treated bleeds.

Novo Nordisk Announces BLA Submission for Extended Half-Life FVIII Therapy

Genentech  informed NHF of a postmarketing fatality that has occurred in an elderly patient who received treatment with emicizumab- kxwh (Hemlibra) for acquired hemophilia A.

According to a recent study, most post pubertal girls and adolescents who experience heavy menstrual bleeding are not screened for von Willebrand disease (VWD), despite years of recommendations from the American College of Obstetricians and Gynecologists. Investigators came to this conclusion after conducting a retrospective analysis of a national claims database of public and privately insured individuals from 2011-2013.  

Study Shows VWD Testing Remains Low

Most postpubertal girls and adolescents who experience heavy menstrual bleeding are not screened for von Willebrand disease (VWD), despite years of recommendations from the American College of Obstetricians and Gynecologists.

Prophylaxis study results for boys with severe hemophilia A

Researchers investigated an approach to prophylactic therapy in which patients began by receiving weekly infusions of standard half-life recombinant factor VIII and ultimately transition to “tailored frequency-escalated prophylaxis."

Honoring the Past, Embracing the Future

NHF's Red Tie Soiree paid tribute to longtime advocates for the bleeding disorders community while looking forward to the future of research and treatment.

Authors of an article published recently in the Journal of Thrombosis and Haemostasis (JTH) looked at the treatment of individuals with factor X deficiency (FX) with a human plasma-derived concentrate (pdFX). FX is one of the rarer bleeding disorders with an estimated incidence of 1 in 500,000 to 1 in a million and though they represent a very small patient population the symptoms associated with FX are no less problematic, especially if not properly treated.

Advanced Considerations for the Recognition, Appropriate Diagnosis, and Timely Management of von Willebrand Disease and Other Bleeding Disorders among Women in Non-Hematology Health Care Settings

The study looked at the treatment of individuals with factor X deficiency with a human plasma-derived concentrate.

Joan Gill, MD

Dedicated clinician and distinguished researcher passed away on May 9, 2018.

NHF 70th: Unite for Bleeding Disorders

Our Unite for Bleeding Disorders walks bring the community together in a way that honors the past while looking forward to the future.

Medicaid

CMS rejects time limit proposal in Kansas; allows work requirements in several other states.

Healthcare professionals who manage patients with hemophilia can enhance their knowledge of gene therapy for hemophilia.

Medscape has launched a new education program for providers seeking to enhance their knowledge of gene therapy for hemophilia. “Clinical Advances in Gene Therapy for Hemophilia,” is presented through a collaboration between Medscape, the National Hemophilia Foundation, the European Haemophilia Consortium and the World Federation of Hemophilia. The program is supported by an independent educational grant from BioMarin.

coreHEM Publishes Core Outcome Set for Hemophilia Clinical Trials;

coreHEM has produced the first set of guidelines recommending a specific, minimum set of outcomes to include in hemophilia gene therapy clinical trials.

Mary Lesh, RN, MS, CPNP

In recognition of National Nurses Week, Mary Lesh, RN, MS, CPNP speaks of the care HTC nurses provide to people with bleeding disorders.

NHLBI Factor VIII Inhibitor Workshop Registration Now Open

The workshop aims to solicit hemophilia community-wide input into a coordinated national blueprint for future research on factor VIII immunogenicity and factor VIII inhibitor prevention and eradication.

A study conducted at the Los Angeles-based Orthopaedic Institute for Children (OIC) suggests that enhanced education on intravenous self-infusion of factor therapy can significantly reduce prosthetic joint infections for hemophilia patients. The lack of adherence to proper self-infusion protocol, including the practice of universal precautions, can put patients at a higher risk for infections.   

Knee joint

OIC study suggests that enhanced education on intravenous self-infusion of factor therapy can significantly reduce prosthetic joint infections for hemophilia patients.

Researchers from Sweden and the U.S. recently published an article that focused on potential correlations between patients with von Willebrand disease (VWD), hospitalizations and prophylaxis. The authors conducted a retrospective study of inpatients and outpatients, both with and without VWD. Their objective was to investigate the frequency of hospital admittances and determine whether the implementation of a prophylactic treatment regimen is associated with a reduction in hospitalizations.

Doctor counseling a patient

The study investigated the frequency of hospital admittances for people with VWD to determine if the implementation of a prophylactic treatment regimen is associated with a reduction in hospitalizations.

There is no time like Spring and its milder temps, to get re-energized and become an engaged steward of your health through exercise and nutrition. The National Hemophilia Foundation (NHF)’s information resource center HANDI would like to make consumers aware of several readily accessible educational tools to get you started on living well.

HANDI hopes the following resources will help to kick start your campaign!

Shire recently announced that the U.S. Food and Drug Administration (FDA) has approved the company’s recombinant von Willebrand factor (rVWF) product VONVENDI®, for perioperative management of bleeding in adults (age 18 and older) with von Willebrand disease (VWD). The therapy had already been approved for the on-demand treatment and control of bleeding episodes in adults 18 and older who have VWD.

Emicizumab-kxwh (HEMLIBRA, Genentech) was approved for use by the US Food and Drug Administration on November 16, 2017 for individuals with hemophilia A and inhibitors.

NHF's MASAC has issued the latest revision to their most comprehensive treatment document.

The designation is for people with hemophilia A without inhibitors.

Dawn Rotellini

NHF is expanding its work with the bleeding disorders community around the world.

ICER is an independent and non-partisan research organization that evaluates the clinical and economic value of prescription drugs, medical tests, and other healthcare and healthcare delivery innovations.

Grifols, S.A. recently announced that in 2017, the company donated over 25 million international units (IU) of factor products used to treat hemophilia A and B. According to a recent press release, the donation is part of Grifols’ commitment to provide a minimum of 200 million IU of factor to the World Federation of Hemophilia (WFH) Humanitarian Aid Program over a span of eight years – Grifols began this commitment in 2014. The company expects to make an additional donation of nearly 25 million IUs in 2018.

NHF CEO Val D. Bias

National Hemophilia Foundation CEO Val D. Bias spoke to the leaders of NHF’s 52-chapter networks at the NHF 2018 Chapter Leadership Seminar in San Francisco, California.

NHF 70th the Fight to Preserve ACA Patient Protections Goes On

2018 marks NHF’s 70th year as the bleeding disorders community’s chief advocate.

Graduation hat

Applications are now open! The selected KCS recipient will receive an award of $1,000 toward their tuition costs.