Hepatocellular Cancer Screening and Gene Therapy Preparedness the Focus of New Recommendations

Hepatocellular Cancer Screening and Gene Therapy Preparedness the Focus of New Recommendations

Two new documents have been issued by the NBDF’s Medical and Scientific Advisory Council (MASAC), which periodically writes recommendations and advisories on treatment, research, and other general health concerns of the inheritable bleeding disorders community.

Study Results Encourage Discussion of Therapeutic Options for VWD Patients with Menorrhagia

Study Results Encourage Discussion of Therapeutic Options for VWD Patients with Menorrhagia

While the majority of women with von Willebrand disease (VWD) will experience heavy menstrual bleeding (HMB) often associated negative impacts on overall health and quality of life, available data relevant to effective treatment is limited.

Small Study Looks at Use of Epidural Analgesia During Labor in VWD Type 1

Small Study Looks at Use of Epidural Analgesia During Labor in VWD Type 1

A research abstract presented at the recent Thrombosis & Haemostasis Summit of North America 2020 Virtual Conference suggested that epidural analgesia may be a safe option for pregnant women with von Willebrand disease (VWD) type 1, provided clotting factors are maintained at certain levels.

Live Medical Preconference Symposium at NHF’s BDC to Tackle Rare Bleeding & Platelet Disorders

Live Medical Preconference Symposium at NHF’s BDC to Tackle Rare Bleeding & Platelet Disorders
Our distinguished faculty for this year's syposium will be reviewing the treatments currently available to manage bleeding in these patients and share significant advances being made to improve genetic diagnosis, including valuable insights on how to interpret rare platelet gene panels.

CDC Calls for Stakeholder Comments on Pain, Pain Management and Opioid Use

CDC Calls for Stakeholder Comments on Pain, Pain Management and Opioid Use
The CDC is interested in hearing from patients with acute or chronic pain, patients’ family members and/or caregivers, and health care providers who care for patients with pain or conditions that can complicate pain management such as opioid use disorder or overdose.  

Healthcare Provider Webinars on Females with Bleeding Disorders Recertified

Healthcare Provider Webinars on Females with Bleeding Disorders Recertified
These educational activities are designed specifically for healthcare providers who may be new to this field or who function outside the hemophilia treatment center network, including primary care physicians, obstetrician/gynecologists, nurse/nurse practitioners, dentists, oral surgeons and other allied professionals.

PT Study Focuses on Comprehensive Survey of Physical Activities Risks for 101 Activities

PT Study Focuses on Comprehensive Survey of Physical Activities Risks for 101 Activities
A veteran group of HTC physical therapists conduct comprehensive review of the ranges and risk drivers associated with 101 activities, including specific bleeding risks in six joints and three injury types, bruising, head injury, and muscle bleeding.

Current and Emerging Therapies the Focus of New Medscape Activity

Current and Emerging Therapies the Focus of New Medscape Activity
Medscape recently launched a new education opportunity for healthcare providers. “Hemophilia 2018: Current and Emerging Therapies” is a discussion focused on the latest advances in the management of hemophilia, including the evolving role of extended half-life factor products, emerging data on small molecule approaches and gene therapy. The activity, which centers on a video recording of a live presentation at the 2017 American Society of Hematology Annual Meeting, is intended for hematologists, pediatricians, and other healthcare professionals who treat patients with hemophilia.

FDA Approves Once-Weekly Subcutaneous Therapy for Hemophilia A Patients with Inhibitors

FDA Approves Once-Weekly Subcutaneous Therapy for Hemophilia A Patients with Inhibitors
The U.S. Food and Drug Administration (FDA) has approved HEMLIBRA® (emicizumab) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors.

New Medscape Activity Looks at Integrating Extended Half Life Products into Clinical Practice

New Medscape Activity Looks at Integrating Extended Half Life Products into Clinical Practice
“Optimizing Transition to Extended Half-Life Factor Replacement Products,” is the latest educational opportunity to become available from Medscape. It is intended for hematologists, pediatricians, nurses, and other healthcare professionals who manage patients with hemophilia.

FDA Expands Labeling for Epclusa® to Include Co-Infected Patients with HCV/HIV and Warning for HBV Reactivation

FDA Expands Labeling for Epclusa® to Include Co-Infected Patients with HCV/HIV and Warning for HBV Reactivation
The U.S. Food and Drug Administration (FDA) has approved expanded labeling for Epclusa®, a combination therapy for adults with chronic hepatitis C viral (HCV) infection.

“Hands On” Physicians Workshop Will Tackle Hemophilia and Inhibitors

“Hands On” Physicians Workshop Will Tackle Hemophilia and Inhibitors
The Nationwide Children’s Hospital Pediatric Hematology Division along with its affiliated institution, The Ohio State University College of Medicine, are jointly hosting an upcoming educational training on inhibitors. The Center for Advanced Training in Hemophilia and Inhibitor Management (CATHIM) workshop will focus on the clinical care of patients with hemophilia and inhibitors.

Partners Module Offers a Primer for Nurses Interested in Clinical Research

Partners Module Offers a Primer for Nurses Interested in Clinical Research
The Partners in Bleeding Disorders Education Program recently relaunched an educational activity focused on clinical research at hemophilia treatment centers (HTCs) entitled “The Basics of Research at Hemophilia Treatment Centers.” The purpose of the module is to enable learners who care for patients with bleeding disorders to participate in research, interpret, and apply specialty protocols and outcome findings effectively.

BloodCenter of Wisconsin Announces New Test for VWD

BloodCenter of Wisconsin Announces New Test for VWD
The BloodCenter of Wisconsin (BCW) recently announced the launch of a new and more sensitive test for von Willebrand disease (VWD), a genetic disorder characterized by either a qualitative or quantitative flaw in von Willebrand factor (VWF). Milwaukee-based BCW is a not-for-profit organization that specializes in blood services, organ, tissue and marrow donation, diagnostic testing, medical services and research.

New Medscape Activity Looks at Cutting Edge Concepts in Hemophilia Care

New Medscape Activity Looks at Cutting Edge Concepts in Hemophilia Care
“Hemophilia Updates: Incorporating New Concepts Into Practice” is the latest educational opportunity to become available from Medscape. This activity is intended for hematologists, pediatricians, nurses, and other healthcare providers who manage patients with hemophilia. It is part of the online series, Clinical Advances in Hemophilia: Management for Life. The purpose of the program is to increase the knowledge, skills and competence of clinicians in providing individualized management of hemophilia.

NHF - McMaster University Clinical Practice Guideline: Care Models for the Management of Hemophilia is Available for Review and Comment

NHF - McMaster University Clinical Practice Guideline: Care Models for the Management of Hemophilia is Available for Review and Comment
NHF is partnering with McMaster University, an international leader on clinical practice guidelines, to develop evidence-based guidelines for hemophilia care.

NHF Announces Judith Graham Pool Postdoctoral Research Fellowship Recipients

NHF Announces Judith Graham Pool Postdoctoral Research Fellowship Recipients
The National Hemophilia Foundation (NHF) is pleased to announce that Wei Cheng, PhD , of Washington University in St. Louis, and Colin Kretz, PhD, of the University of Michigan have been selected as individual award recipients of NHF’s Judith Graham Pool Postdoctoral Research Fellowship.

New Website Contains Latest HCV Treatment and Management Recommendations

New Website Contains Latest HCV Treatment and Management Recommendations
The American Association for the Study of Liver Diseases and the Infectious Diseases Society of America (IDSA), in collaboration with the International Antiviral Society-USA, have developed a website for the rapid dissemination of evidence-based, expert-developed recommendations for hepatitis C virus (HCV) management. 

Gene Therapy Research Using Platelet Cells Could Benefit Inhibitor Patients

Gene Therapy Research Using Platelet Cells Could Benefit Inhibitor Patients
Investigators at the University of North Carolina (UNC) School of Medicine and Medical College of Wisconsin (MCW) developed a gene therapy approach that uses platelets, avoiding the kind of antibody response (inhibitors) that can neutralize an infused factor therapy.

FDA Approves Simeprevir, New HCV Oral Therapy; Approval Pending for Sofosbuvir

FDA Approves Simeprevir, New HCV Oral Therapy; Approval Pending for Sofosbuvir
In November, the US Food and Drug Administration (FDA) approved simeprevir, a new oral drug, to treat the hepatitis C virus (HCV). Olysio™, manufactured by Johnson & Johnson, is a protease inhibitor that halts the progression of HCV, preventing it from reproducing. 

NHF Announces the 2013 NHF/Novo Nordisk Career Development Award Recipient

NHF Announces the 2013 NHF/Novo Nordisk Career Development Award Recipient
The National Hemophilia Foundation (NHF) is pleased to announce Annette von Drygalski, MD, PharmD, Assistant Clinical Professor of Medicine, University of California at San Diego, as the recipient of the NHF/Novo Nordisk 2013 Career Development Award (CDA).

NHF Announces 2013 NHF-Baxter Clinical Fellows, New Institutions Join Program

NHF Announces 2013 NHF-Baxter Clinical Fellows, New Institutions Join Program
The National Hemophilia Foundation (NHF) is pleased to announce this year's NHF-Baxter Clinical Fellowship award recipients: Tyler Buckner, MD, of the University North Carolina at Chapel Hill, and Christopher Ng, MD, of the University of Colorado Denver. 

New Blood Center in Philadelphia

New Blood Center in Philadelphia
In January, Penn Medicine announced its union with the Children's Hospital of Philadelphia (CHOP) to establish the Penn-CHOP Blood Center for Patient Care and Discovery, which is being positioned as that region's first dedicated center for the treatment and research of blood diseases.

International Study Finds Similar Inhibitor Risk for FVIII Products

International Study Finds Similar Inhibitor Risk for FVIII Products
Results of a recent international study published in The New England Journal of Medicine (NEJM) suggest that plasma-derived factor VIII (pdFVIII) products and recombinant factor VIII (rFVIII) products used to treat hemophilia A are associated with similar rates of inhibitor development. 

Medical College of Wisconsin Receives NIH Grant for VWD Studies

Medical College of Wisconsin Receives NIH Grant for VWD Studies
In March, the Medical College of Wisconsin (MCW) received a five-year, $10 million National Heart, Lung, and Blood Institute Program Project Grant. This grant will allow researchers to continue genetic studies on patients with von Willebrand disease (VWD), the most common hereditary bleeding disorder estimated to occur in 1% to 2% of the population.

Preliminary Recommendations for Protease Inhibitors in Co-infected Patients

Preliminary Recommendations for Protease Inhibitors in Co-infected Patients
In December 2011, a group of Maryland physicians published provisional treatment guidelines  for the use of protease inhibitors in patients co-infected with human immunodeficiency virus (HIV) and hepatitis C virus (HCV) in the online version of the journal Clinical Infectious Diseases.

TSA Launches Helpline for Fliers with Medical Conditions and Disabilities

TSA Launches Helpline for Fliers with Medical Conditions and Disabilities
On December 22, 2011, the Transportation Security Administration (TSA) announced the launch of TSA Cares, a toll-free, helpline number for fliers with disabilities and medical conditions. Travelers can call ahead about screening policies, procedures and what to expect at the security checkpoint.

International Study Finds Misdiagnosis in Rarest VWD Type

International Study Finds Misdiagnosis in Rarest VWD Type
The findings of an international study on the rate of misdiagnosis among patients with the rarest type of von Willebrand disease (VWD) were published in February 2011 in the Journal of Thrombosis and Haemostasis. The lead investigator of the study was Maha Othman, MD, MSc, PhD, Department of Anatomy and Cell Biology, Queen's University in Ontario, Canada.

Wall Street Journal Article Stresses Importance of Medical IDs

Wall Street Journal Article Stresses Importance of Medical IDs
“The Jewelry Prescription: Medical Bracelets Go High Tech,” an article in the Wall Street Journal on August 31, 2010, highlighted the importance of carrying or wearing a form of medical identification (ID), particularly for individuals with complex and/or chronic medical conditions and/or food or drug allergies. It featured recent advances in personal medical identification.

London Researchers Study Menorrhagia in Girls with Bleeding Disorders

London Researchers Study Menorrhagia in Girls with Bleeding Disorders
In a study published in the August Journal of Pediatric and Adolescent Gynecology, researchers from the Royal Free Hospital (RFH) in London reviewed the management and treatment outcomes in adolescents with inherited bleeding disorders and menorrhagia (excessive menstrual bleeding). 

CHOP Researchers Study Factors V and VIII Activation

CHOP Researchers Study Factors V and VIII Activation
In July, investigators from The Children’s Hospital of Philadelphia (CHOP) published a review of their research into the molecular processes that ensure factors V (FV) and VIII (FVIII) become “activated” to carry out their role as coagulation proteins. These and other proteins function in the “clotting cascade,” an intricate series of chemical and molecular reactions between clotting factors that lead to clot formation.

Baxter Issues Drug Warning for FEIBA Products

Baxter Issues Drug Warning for FEIBA Products
Baxter Healthcare Corporation made a public announcement in August that it is updating certain sections of the prescribing information for FEIBA VH® and FEIBA NF®after consistent reports of thrombosis (excessive clots forming in blood vessels) and thromboembolic events associated with the use of the products, particularly in high doses or in at-risk patients. 

Research Update: DNA Affects VWD Testing in African Americans

Research Update: DNA Affects VWD Testing in African Americans
Researchers from the Medical College of Wisconsin (MCW) in Milwaukee and several other institutions published a study in the July issue of Blood, the journal of the American Society of Hematology, suggesting a DNA-based explanation for discrepancies found in certain lab tests that help diagnose von Willebrand disease (VWD) in African Americans.

Results of Obesity Review in Mississippi's Hemophilia Population

Results of Obesity Review in Mississippi's Hemophilia Population
When it comes to obesity in adults, Mississippi ranks first in the nation—with prevalence rates of more than 33%. To determine the prevalence among the state’s hemophilia patients, researchers at the University of Mississippi Medical Center (UMMC) studied potential obesity risk factors in this population, including race/ethnicity and hemophilia severity.

Swedish Study Assesses Osteoporosis Prevention in People with Severe Hemophilia

Swedish Study Assesses Osteoporosis Prevention in People with Severe Hemophilia
In May researchers in Sweden published the results of a study analyzing whether physical activity, accompanied by a long-term prophylactic (preventive) treatment regimen, is a significant determining factor in the prevention of osteoporosis in patients with severe hemophilia.

Mayo Clinic Study Shows Women with Bleeding Disorders' Satisfaction with Global Endometrial Ablation

Mayo Clinic Study Shows Women with Bleeding Disorders' Satisfaction with Global Endometrial Ablation
In an April 2010 study, researchers at the Mayo Clinic examined health-related quality of life (HRQoL) and satisfaction of women with bleeding disorders who had undergone global endometrial ablation (GEA) for excessive menstrual bleeding.

Mount Sinai Hematologist and Researcher Receives Lifetime Achievement Award

Mount Sinai Hematologist and Researcher Receives Lifetime Achievement Award
The Hemophilia and Thrombosis Research Society (HTRS) bestowed its Lifetime Achievement Award on long-time researcher and clinician Louis M. Aledort, MD. He received the award at the HTRS and North American Specialized Coagulation Laboratory Association Scientific Symposium on Friday, April 16, 2010, in Chicago, IL.

Mayo Clinic Examines Circumcision-Induced Bleeding

Mayo Clinic Examines Circumcision-Induced Bleeding
Researchers at the Mayo Clinic (MC) in Rochester, MN, published a retrospective study in March of circumcision in patients with bleeding disorders. The purpose of the study was to examine the outcomes of circumcisions performed on patients who were evaluated at MC to determine the extent of related complications and create guidelines for clinical management.

BloodCenter of Wisconsin Establishes New Institute

BloodCenter of Wisconsin Establishes New Institute
The BloodCenter of Wisconsin in Milwaukee has created a new Medical Sciences Institute (MSI). It is expected to increase the BloodCenter’s revenue by more than $1 million annually. The institute plans to hire seven clinical researchers to complete its staffing. The BloodCenter also encompasses the Blood Research Institute, which was created in 1991 to conduct benign hematology research. Jacquelyn Fredrick, the BloodCenter’s president and chief executive officer, said MSI will advance “discoveries made in the laboratory to the patients’ bedside.”