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The National Heart, Lung, and Blood Institute (NHLBI) is currently seeking public feedback that will inform future research the agency will conduct on inhibitors to factor VIII (FVIII) in patients with hemophilia A. To this end, the NHLBI has released a Request for Information (RFI) to solicit perspectives and comments from the bleeding disorders community on potential strategies, scientific opportunities and priorities, and the requisite infrastructure to optimize inhibitor research.

As individuals with hemophilia live longer and reach life expectancy rates comparable to the general population, their healthcare providers will continue to encounter clinical challenges inherent in treating and managing aging patients, including cardiovascular disease (CVD). There are several established risk factors associated with CVD such as hypertension, overweight, obesity and an abnormal lipid profile.

Medscape has launched a new 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.

Bayer receives FDA approval for hemophilia A treatment

Bayer received FDA approval for new extended half-life hemophilia A product for patients 12 yrs and older.

The new vial size will reduce the reconstitution time that was needed to prepare multiple vials for a similar dose.

The therapy is for people with severe and moderately severe hemophilia B.

Catalyst Updates on Phase 2/3 Trial of Subcutaneous Therapy

Catalyst Biosciences recently announced updated positive interim data from a phase 2/3, multinational clinical study of marzeptacog alfa (MarzAA), a subcutaneously administered factor VIIa therapy. MarzAA is being developed by Catalyst for the prophylactic treatment of individuals with hemophilia A or B with inhibitors.

NHF 70th: Pursuing discovery of the next generation of therapies

An interview with one of the recipients of NHF's Innovative Investigator Awards

NHF 70th: Pursuing discovery of the next generation of therapies

An interview with one of the recipients of NHF's Innovative Investigator Awards

Sangamo Therapeutics, Inc. recently announced positive preliminary data from the Phase 1/2 clinical trial designed to evaluate SB-525, the company’s gene therapy program for people with hemophilia A, or factor VIII (FVIII) deficiency. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer for the development and commercialization of hemophilia A gene therapy programs.

Spark Reports Progress on Hemophilia A Gene Therapy Candidate

The therapy is administered via a one-time intravenous infusion, which is designed to elicit the production of therapeutic levels of factor VIII.

Court Rules on Shire/Roche Preliminary Injunction

The preliminary injunction was part of an ongoing patent lawsuit.

New Medscape Activity Features the Science Behind Gene Therapy

The activity will examine the technology behind the emerging approaches to gene therapy in hemophilia.

Shire is discontinuing the manufacture and distribution of BEBULIN®, the company’s factor IX human plasma-derived prothrombin complex concentrate product. BEBULIN® is indicated for the prevention and control of bleeding episodes in adult patients with hemophilia B. The National Hemophilia Foundation has been notified by Shire that this decision was based on the reduced demand and is not connected with any new safety or efficacy findings in relation to the therapy.

Healthcare providers interested in broadening their knowledge of the science and clinical management of inhibitors are enthusiastically invited to participate in NHF’s webinar on “Current Research and Future Approaches to Eradicating Inhibitors in Hemophilia.” This CME and CNE-accredited activity reviews the basic science behind the development of inhibitor antibodies to treatment for hemophilia, the associated management complications and current barriers to research.

A new patient financial assistance program has become available for individuals with hemophilia. The Assistance Fund (AF) is a charitable foundation that was created to provide eligible patients with chronic diseases help with paying medication copayments, coinsurance, deductibles, insurance premiums and incidental medical expenses.

As “the dog days” of August approach, Autumn may still seem a long way off. Though with a quick glance of the calendar, we see that colleges begin their class terms earlier and earlier and Fall semester is right around the corner! Whether going away or staying closer to home, being a first-year college student can be a time fraught with great excitement, anticipation and perhaps some trepidation about this next big step. HANDI, NHF’s information resource center, would like to highlight several resources aimed at helping new college students in this transition.

NHF Comments on Trump Administration Drug Pricing Blueprint

NHF submitted a letter on the "American Patients First" drug pricing blueprint.

HTRS

The HTRS MRA provides financial support for qualified fellows or junior attending/junior faculty pursuing clinical, translational, or basic science research in hemostasis and/or thrombosis under the guidance of an experienced mentor.

Study Shows Impact of Missing HTC Clinic Visits

New study from Emory University School of Medicine focused on causes and ramifications of missing clinic visits in patients with bleeding disorders.

The rate of attendance to scheduled clinic visits have been linked to health outcomes in several chronic disease groups such as diabetes, hypertension, HIV and congenital heart disease. Higher clinic attendance rates are generally associated with better outcomes in these patients. However, the potential effects of non-attendance (NA) for individuals with bleeding disorders is not well understood.

Pfizer Initiates First Stage of Phase 3 Hemophilia B Gene Therapy Trial

The first stage will collect data that will be eventually used to evaluate the efficacy and safety of the investigational hemophilia B therapy.

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.