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Hot Topics of the Day are picked by experts to capture the latest information and publications on public health genomics and precision health for various diseases and health topics. Sources include published scientific literature, reviews, blogs and popular press articles.

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117 hot topic(s) found with the query "Hemophilia "

Towards Personalized Treatment in Hemophilia: The Role of Genetic Factors in Iron and Heme Control to Identify Patients at Risk for Haemophilic Arthropathy
LFD van Vulpen et al, JPM, January 26, 2024 (Posted: Jan 29, 2024 8AM)

From the abstract: "The treatment landscape for hemophilia is changing rapidly, creating opportunities for personalized treatment. As major morbidity is still caused by hemophilic arthropathy, understanding the factors affecting joint damage and joint damage progression might lead to more individualized treatment regimens. We investigated the association of HFE mutations or HMOX1 polymorphisms affecting iron/heme handling with radiographic joint damage in 252 hemophilia patients (severe and moderate). "

How do people inherit hemophilia?
Medical News Today, September 2023 (Posted: Sep 29, 2023 11AM)

From the website: "Hemophilia is a bleeding disorder that can affect the blood’s ability to clot properly. In most cases, people inherit the gene variations for hemophilia in an X-linked recessive inheritance pattern. Hemophilia is a condition that affects the blood’s ability to coagulate, or clot. It occurs when people have low amounts of a certain type of protein in the blood known as clotting factors. Clotting factors are responsible for normal blood clotting. "

Hemostasis - A Balancing Act.
H Marijke van den Berg et al. N Engl J Med 2023 8 (9) 853-856 (Posted: Aug 31, 2023 7AM)

From the paper: "It is reassuring that this first-in-class drug is effective in treating hemophilia A or B with inhibitors. Given its reported efficacy in both types of hemophilia without inhibitors, concizumab is evolving as an attractive therapeutic for all patients with hemophilia. In the bigger picture, these new treatment options need to be weighed against the possibility of cure of hemophilia through gene therapy: two adeno-associated virus (AAV) vector–based products have been approved for clinical use, and more are in the pipeline."

Phase 3 Trial of Concizumab in Hemophilia with Inhibitors.
Tadashi Matsushita et al. N Engl J Med 2023 8 (9) 783-794 (Posted: Aug 31, 2023 7AM)

From the abstract: "Concizumab is an anti–tissue factor pathway inhibitor monoclonal antibody designed to achieve hemostasis in all hemophilia types, with subcutaneous administration. We conducted the explorer7 trial to assess the safety and efficacy of concizumab in patients with hemophilia A or B with inhibitors. Among patients with hemophilia A or B with inhibitors, the annualized bleeding rate was lower with concizumab prophylaxis than with no prophylaxis."

Researchers uncover mysteries behind immune response to hemophilia A treatment
Medical XPress, August 2023 (Posted: Aug 18, 2023 11AM)

In a recent study published in Blood, researchers from Indiana University School of Medicine shed new light on the underlying causes of immune response to factor VIII, providing valuable insights that could lead to more effective and safer hemophilia A treatments in the future. The scientists discovered that T cells, which play a vital role in the immune system and help trigger factor VIII inhibitor formation, rely on a specific set of other cells to move coagulation factor VIII to the regions of the spleen where T cells reside.

Community Counts: Understanding Hemophilia and Other Bleeding Disorders through Public Health Surveillance
CDC webinar, August 24, 2023 Brand (Posted: Jul 11, 2023 8AM)

Over the past 20 years, CDC has conducted public health surveillance to better understand the characteristics and complications that impact the bleeding disorders population. Community Counts is a public health surveillance program that gathers and shares information about common health issues, medical complications, and causes of death that affect people with bleeding disorders cared for in U.S. Hemophilia Treatment Centers.

The FDA just approved another gene therapy. Here’s what to know about them
SA Yonah, Washington Post, July 1, 2023 (Posted: Jul 01, 2023 3PM)

The Food and Drug Administration approved a treatment that uses gene therapy to treat severe hemophilia A, a rare and sometimes fatal blood disorder. The new drug, Roctavian, could save people with the severe form of the disease from a lifetime of frequent injections. The drug’s maker expects about 2,500 of the estimated 6,500 Americans with severe hemophilia A to be eligible to receive the drug with its initial approval.

Efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase 3 trial.
Guy Young et al. Lancet 2023 4 (10386) 1427-1437 (Posted: May 01, 2023 10AM)

Subcutaneous fitusiran prophylaxis resulted in statistically significant reductions in annualised bleeding rate in participants with haemophilia A or haemophilia B with inhibitors, with two-thirds of participants having zero bleeds. Fitusiran prophylaxis might show haemostatic efficacy in participants with haemophilia A or haemophilia B with inhibitors; therefore, the therapeutic might have the potential to improve the management of people with haemophilia.

Hemophilia A Gene Therapy - Some Answers, More Questions.
Lindsey A George et al. The New England journal of medicine 2023 2 (8) 761-763 (Posted: Feb 23, 2023 9AM)

Within a growing therapeutic armamentarium, adeno-associated virus (AAV)–mediated gene transfer of factors VIII and IX has been in clinical development for two decades. Thus far, the development of a gene therapy for hemophilia has been an iterative process, with future successes predicated on the investigation of unexpected observations from clinical trials to then improve the next generation of vectors.

Efanesoctocog alfa for hemophilia A: results from a phase 1 repeat-dose study.
Toshko Lissitchkov et al. Blood advances 2021 11 (4) 1089-1094 (Posted: Jan 26, 2023 7AM)

We conducted a phase 3 study involving patients 12 years of age or older with severe hemophilia A. In patients with severe hemophilia A, once-weekly efanesoctocog alfa provided superior bleeding prevention to prestudy prophylaxis, normal to near-normal factor VIII activity, and improvements in physical health, pain, and joint health.

Sources of Innovation in Gene Therapies - Approaches to Achieving Affordable Prices.
Kerstin N Vokinger et al. The New England journal of medicine 2023 1 (Posted: Jan 26, 2023 7AM)

Gene therapies are a fast-growing area of innovation and potentially hold great promise for clinical care. Most recently, a new gene therapy, etranacogene dezaparvovec (Hemgenix), was approved by the Food and Drug Administration (FDA) for use in adults with hemophilia B (factor IX deficiency); the cost of this one-time treatment has been estimated at $3.5 million. Since 2017, a total of 11 such treatments have been approved by the FDA.

Current challenges in hemophilia genetics and how they can be overcome
JM Johnson, Nat Cardiovasc Research, October 2022 (Posted: Nov 04, 2022 11AM)

Genomic sequencing in hemophilia is a high-yield test and clinically useful for diagnosis, assessing the risk of developing neutralizing antibodies (‘inhibitors’) against the affected coagulation factor, pregnancy and neonatal management, and family counseling. New genomic technologies can detect several types of DNA change with high sensitivity. Systematic collection of genotype–phenotype data is important to better understand the genetics of hemophilia.

Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A
S Fong et al, Nature Medicine, April 11, 2022 (Posted: Apr 11, 2022 2PM)

Factor VIII gene transfer with a single intravenous infusion of valoctocogene roxaparvovec (AAV5-hFVIII-SQ) has demonstrated clinical benefits lasting 5 years to date in people with severe hemophilia A. Molecular mechanisms underlying sustained AAV5-hFVIII-SQ-derived FVIII expression have not been studied in humans.

Precision Public Health in Action: Enhancing models to predict risk of adverse treatment outcomes in people with hemophilia
CJ Bean et al, CDC Blog Post, March 29, 2022 Brand (Posted: Mar 30, 2022 7AM)

CDC investigators have developed a genetic inhibitor risk prediction tool hat is based on information about the genetic variant causing a person’s hemophilia, genetic variation in the HLA region and in immune response genes. However, this tool has thus far been evaluated in a relatively small number of people with hemophilia. In this newly funded project, investigators will evaluate the performance of the existing inhibitor risk prediction tool in a diverse patient population. Previous studies of the prediction tool were conducted in populations of non-Hispanic, White persons with hemophilia.

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
MC Ozelo et al, NEJM, March 17, 2022 (Posted: Mar 17, 2022 9AM)

We conducted an open-label, single-group, multicenter, phase 3 study to evaluate the efficacy and safety of valoctocogene roxaparvovec in men with severe hemophilia A, defined as a factor VIII level of 1 IU per deciliter or lower. Participants who were at least 18 years of age and did not have preexisting anti-AAV5 antibodies or a history of development of factor VIII inhibitors. In patients with severe hemophilia A, valoctocogene roxaparvovec treatment provided endogenous factor VIII production and significantly reduced bleeding and factor VIII concentrate use relative to factor VIII prophylaxis.

Prepare the Way for Hemophilia A Gene Therapy
CD Thornburg, NEJM, March 17, 2022 (Posted: Mar 17, 2022 9AM)

Valoctocogene roxaparvovec has been granted Regenerative Medicine Advanced Therapy and Breakthrough Therapy designations from the Food and Drug Administration. If approved, this first-generation gene therapy would offer a new choice for care that could be truly transformative and liberating for eligible men with hemophilia.

Disease of the Week: Hemophilia
CDC, March 2022 Brand (Posted: Mar 02, 2022 8AM)

Hemophilia is an inherited bleeding disorder, which means it can run in families. Blood does not clot properly in people with hemophilia. Blood contains proteins called clotting factors that are needed to stop bleeding. People with hemophilia have low amounts of either factor VIII (factor 8) or factor IX (factor 9) in their blood. A lower amount of factor in the blood typically leads to more severe hemophilia. The lower the amount of the factor, the more likely it is that bleeding will occur. Hemophilia occurs in about 1 of every 5,000 male births from all racial and ethnic groups. Females can also have hemophilia, but this is much more rare.

Precision Public Health in Action: New CDC Pilot Projects Integrating Human Genomics into Public Health Surveillance and Applied Research
M Clyne et al, CDC Blog Post, February 14, 2022 Brand (Posted: Feb 15, 2022 7AM)

Six CDC projects were selected for funding in 2022 and 2023. They cover a wide range of topics, including: Assessing the impact of genetics in the control of two infectious diseases (Tuberculosis and Ebola), enhancing the reporting of gene/genome sequencing in newborn screening programs, examining the role of medications and genetics in the National Birth Defects Prevention Study (NBDPS), establishing population-based, ethnicity-specific allele frequencies for pharmacogenomic traits of public health importance using the National Health and Nutrition Examination Survey (NHANES), and enhancing the evaluation of genetic risk prediction models for inhibitor development among people with hemophilia in different populations.

Gene therapy for hemophilia A
K O'Leary, Nature Medicine, November 30, 2021 (Posted: Nov 30, 2021 9AM)

Hemophilia A is caused by gene mutations that lead to absence or dysfunction of clotting factor VIII, which manifests as frequent, spontaneous hemorrhage. In a phase 1/2 trial, an investigational gene therapy brought about sustained expression of clotting factor VII and a dramatic reduction in the bleeding rate.

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
LA George et al, NEJM, November 18, 2021 (Posted: Nov 18, 2021 5AM)

The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose. In this phase 1–2 trial, we infused an investigational adeno-associated viral (AAV) vector (SPK-8011) for hepatocyte expression of factor VIII in 18 men with hemophilia A. Four dose cohorts were enrolled. Sustained factor VIII expression in 16 of 18 participants who received SPK-8011 permitted discontinuation of prophylaxis and a reduction in bleeding episodes. No major safety concerns were reported.

Real Stories from People Living with Hemophilia – Carrie
CDC, November 2021 Brand (Posted: Nov 14, 2021 6AM)

Carrie is no stranger to hemophilia. Her father has severe hemophilia B (factor IX [9] deficiency) and she grew up watching her father experience joint bleeds and receive treatment at the Hemophilia Treatment Center (HTC) at his local hospital and at home. “It was challenging, but as a child you don’t know any different. [Hemophilia] is your normal. This is what your dad does. I had a father who never was able to run and play with us, he was never able to kneel down and give us a bath when we were kids.”

What is a ‘serious’ genetic condition? The perceptions of people living with genetic conditions
FK Boardman et al, EJHG, September 27, 2021 (Posted: Sep 28, 2021 6AM)

This study explores the concept of seriousness through the perspectives of people with a range of ‘clinically serious’ conditions (fragile X conditions, spinal muscular atrophy, cystic fibrosis, hemophilia, thalassemia). Attitudes towards suffering, quality of life (QoL) and selective pregnancy termination were elucidated from 45 in-depth qualitative interviews and 469 postal/online surveys. The majority of participants reported good health/wellbeing, and the capacity for good QoL, despite experiencing suffering with their condition.

Hemophilia
E Berntorp et al, Nature Dis Primers., June 24, 2021 (Posted: Jun 24, 2021 7AM)

Haemophilia A and B are rare congenital, recessive X-linked disorders caused by lack or deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease depends on the reduction of levels of FVIII or FIX, which are determined by the type of the causative mutation in the genes encoding the factors (F8 and F9, respectively). The hallmark clinical characteristic, especially in untreated severe forms, is bleeding.

Data from CDC’s hemophilia surveillance programs can help prevent or reduce hemophilia-related health problems
CDC, March 2021 (Posted: Mar 17, 2021 10AM)

For more than 20 years, the Centers for Disease Control and Prevention (CDC) has successfully developed, implemented, and maintained a surveillance (monitoring) program for hemophilia to advance our understanding of the condition and the needs of the hemophilia community.

Evaluation of CDC's Hemophilia Surveillance Program - Universal Data Collection (1998-2011) and Community Counts (2011-2019), United States.
Schieve Laura A et al. Morbidity and mortality weekly report. Surveillance summaries (Washington, D.C. : 2002) 2020 Sep (5) 1-18 (Posted: Sep 11, 2020 7AM)

CDC has successfully developed, implemented, and maintained a surveillance system for hemophilia. The program can serve as an example of how to conduct surveillance for a complex chronic disease by involving stakeholders, improving and building new infrastructure, expanding data collection, providing testing guidance, establishing a registry with specimen collection.

BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A.
Konkle Barbara A et al. The New England journal of medicine 2020 Sep (11) 1018-1027 (Posted: Sep 10, 2020 7AM)

In a small, early-phase study involving men with severe hemophilia A, a single intravenous injection of BIVV001 resulted in high sustained factor VIII activity levels, with a half-life that was up to four times the half-life associated with recombinant factor VIII, an increase that could signal a new class of factor VIII replacement therapy.

Investigational Hemophilia A Gene Therapy Shows Sustained Benefit
J Abbasi, JAMA, February 11, 2020 (Posted: Feb 12, 2020 8AM)

An experimental gene therapy for hemophilia A remained effective up to 3 years after a single infusion, researchers recently reported in the New England Journal of Medicine. Valoctocogene roxaparvovec encodes factor VIII, the blood-clotting protein that’s missing or low in people with hemophilia A, the most common form of the disease.

Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries.
Iorio Alfonso et al. Annals of internal medicine 2019 Sep (Posted: Nov 02, 2019 7AM)

The prevalence of hemophilia is higher than previously estimated. Patients with hemophilia still have a life expectancy disadvantage. Establishing prevalence at birth is a milestone toward assessing years of life lost, years of life with disability, and burden of disease.

Off to College Toolkit
Hemophilia Federation of America, 2019 (Posted: Aug 16, 2019 8AM)

In this toolkit, you’ll find resources about preparing and making the transition from high school to campus life. This toolkit has resources that are specific to living with a bleeding disorder and others that are more general, but are applicable if you or your child will need accommodations to be a successful student.

The national blueprint for future basic and translational research to understand factor VIII immunogenicity: NHLBI State of the Science Workshop on factor VIII inhibitors.
Meeks Shannon L et al. Haemophilia : the official journal of the World Federation of Hemophilia 2019 Jul 25(4) 595-602 (Posted: Jul 24, 2019 10AM)

Inhibitor formation against coagulation factor VIII (FVIII) is an unresolved serious problem in replacement therapy for the X-linked bleeding disorder hemophilia A. Although FVIII inhibitors have been extensively studied, much of the basic mechanism of this immune response remains to be uncovered.

Experimental gene therapy for hemophilia shows early promise
D Garde, Stat News, July 5, 2019 (Posted: Jul 07, 2019 0PM)

The Community Counts Data Visualization Tool
CDC, 2019 Brand (Posted: Jul 05, 2019 10AM)

The Community Counts data visualization tool displays de-identified data on patients with bleeding disorders who are enrolled in Community Counts in a new interactive, visual format. This tool can be used by clinicians, patients, and policymakers to learn more about the burden of bleeding disorders in the United States.

New Opportunities and Challenges in Hemophilia Care: 2019 Update
CDC Public Health Seminar, June 6, 2019 Brand (Posted: Jun 04, 2019 10AM)

How to discuss gene therapy for haemophilia? A patient and physician perspective.
Miesbach Wolfgang et al. Haemophilia : the official journal of the World Federation of Hemophilia 2019 May (Posted: May 29, 2019 8AM)

Emergency Preparedness Info for People with Blood Disorders
CDC, 2019 Brand (Posted: May 09, 2019 2PM)

Women with bleeding disorders.
Hermans C et al. Haemophilia : the official journal of the World Federation of Hemophilia 2018 May 29-36 (Posted: Mar 25, 2019 11AM)

Women can have hemophilia, too
CDC, 2019 Brand (Posted: Mar 18, 2019 5PM)

Real Stories from People Living with Hemophilia
CDC, 2019 Brand (Posted: Mar 08, 2019 1PM)

Prepare Your Health: Personal Health Preparedness for People with Bleeding Disorders
CDC A-Z Index ?Ú Public Health Matters Blog, March 4, 2019 Brand (Posted: Mar 05, 2019 9AM)

The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world.
Pierce Glenn F et al. Haemophilia : the official journal of the World Federation of Hemophilia 2019 Jan (Posted: Jan 09, 2019 9AM)

Thousands of sickle cell and hemophilia patients are waiting for gene therapies. Can manufacturers keep up?
E Mullin, Stat News, November 5, 2018 (Posted: Nov 05, 2018 11AM)

Mimicking Factor VIII to Manage the Factor VIII-Deficient State.
Ragni Margaret V et al. The New England journal of medicine 2018 Aug (9) 880-882 (Posted: Aug 30, 2018 8AM)

Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors.
Mahlangu Johnny et al. The New England journal of medicine 2018 Aug (9) 811-822 (Posted: Aug 30, 2018 8AM)

The Patients Who Don't Want to Be Cured A hemophiliac says his genetic disorder is part of his identity, and therapies like CRISPR threaten to erase it.
S Zhang, The Atlantic, August 29, 2018 (Posted: Aug 30, 2018 8AM)

They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. Experimental gene therapies have yielded promising results in early trials. But the drugs have left some patients wary, worried that success will not last.
G Kolata, New York Times, AUgust 13, 2018 (Posted: Aug 14, 2018 11AM)

Gene therapy in hemophilia A: a cost-effectiveness analysis.
Machin Nicoletta et al. Blood advances 2018 Jul 2(14) 1792-1798 (Posted: Aug 01, 2018 10AM)

Genetic Risk Factors and Inhibitor Development in Hemophilia: What Is Known and Searching for the Unknown.
Margaglione Maurizio et al. Seminars in thrombosis and hemostasis 2018 Jun (Posted: Jun 27, 2018 9AM)

Community Counts
Explore health issues, medical complications, and causes of death that affect people with bleeding disorders who receive care at U.S. Hemophilia Treatment Centers Brand (Posted: Jun 14, 2018 2PM)

Basics About Hemophilia
Brand (Posted: Jun 12, 2018 1PM)

FDA plans to speed path to approval for some gene therapies, starting with hemophilia
I. Swetlitz, StatNews, May 22, 2018 (Posted: May 23, 2018 8AM)

Hemophilia groups warned of 5 deaths among patients taking Roche’s big new blockbuster Hemlibra
J Carroll, Endpoints News, Mar 28, 2018 (Posted: Mar 28, 2018 8AM)

What is an inhibitor?
All people with hemophilia and VWD type 3 are at risk for developing an inhibitor ? an antibody ? to treatment used to stop or to prevent a bleeding episode: Anthony's story. Brand (Posted: Mar 20, 2018 2PM)

Hemophilia Drug With Big Price Tag May Be Economical In the Long Run
S Collins, Feb 2018 (Posted: Mar 12, 2018 3PM)

Chris Bombardier: Reaching New Heights
He is the first person with hemophilia to summit Mount Everest, Hemaware, Feb 2018 (Posted: Feb 27, 2018 1PM)

Gene therapies for hemophilia hit the mark in clinical trials.
Pickar Adrian K et al. Nature medicine 2018 Feb (2) 121-122 (Posted: Feb 14, 2018 10AM)

Hemophilia Gene Therapies Show Promise
J Abbasi, JAMA, Feb 13, 2018 (Posted: Feb 13, 2018 3PM)

Factor VIII Inhibitors: Generating a National Blueprint for Future Research
NHLBI Conference, May 15-18, 2018 Brand (Posted: Feb 09, 2018 2PM)

Hemophilia gene therapy comes of age.
George Lindsey A et al. Blood advances 2017 Dec (26) 2591-2599 (Posted: Jan 13, 2018 10AM)

Gene Therapy for Severe Hemophilia A
Karen Sokal-Gutierrez, NEJM Resident360, Dec 2017 (Posted: Jan 02, 2018 9AM)

Closing In on Treatment for Hemophilia B.
Porteus Matthew et al. The New England journal of medicine 2017 Dec (23) 2274-2275 (Posted: Dec 07, 2017 8AM)

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
George Lindsey A et al. The New England journal of medicine 2017 Dec (23) 2215-2227 (Posted: Dec 07, 2017 8AM)

Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy
KJ Passi, NEJM, August 31, 2017 (Posted: Aug 30, 2017 6PM)

Emicizumab Prophylaxis in Hemophilia A with Inhibitors
J Oldenburg et al, NEJM, August 31, 2017 (Posted: Aug 30, 2017 6PM)

The role of recombinant activated factor VII in the haematological management of elective orthopaedic surgery in haemophilia A patients with inhibitors.
Castaman Giancarlo et al. Blood transfusion = Trasfusione del sangue 2017 May 1-9 (Posted: Aug 22, 2017 9AM)

Sports participation recommendations for patients with bleeding disorders.
Howell Cailly et al. Translational pediatrics 2017 Jul (3) 174-180 (Posted: Aug 22, 2017 9AM)

Hemophilia Treatment Center (HTC) Directory
Brand (Posted: Aug 22, 2017 9AM)

Emicizumab Prophylaxis in Hemophilia A with Inhibitors.
Oldenburg Johannes et al. The New England journal of medicine 2017 Jul (Posted: Jul 17, 2017 1PM)

Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy.
Pasi K John et al. The New England journal of medicine 2017 Jul (Posted: Jul 11, 2017 11AM)

Making Strides Toward Preventing Inhibitors in Bleeding Disorders
Brand (Posted: Apr 17, 2017 9AM)

Recent advances in hemophilia B therapy.
Horava Sarena D et al. Drug delivery and translational research 2017 Feb (Posted: Apr 17, 2017 9AM)

Gene Therapy for Hemophilia.
Nienhuis Arthur W et al. Molecular therapy : the journal of the American Society of Gene Therapy 2017 Apr (Posted: Apr 17, 2017 9AM)

Molecular diagnosis of von Willebrand disease.
Baronciani L et al. Haemophilia : the official journal of the World Federation of Hemophilia 2017 Feb (Posted: Mar 13, 2017 6PM)

Emergency department utilization by haemophilia patients in United States.
Zakieh A et al. Haemophilia : the official journal of the World Federation of Hemophilia 2017 Feb (Posted: Mar 13, 2017 5PM)

FDA Gives Breakthrough Designation to Hemophilia Gene Therapy
National Hemophilia Foundation, January 30, 2017 (Posted: Feb 01, 2017 5PM)

Hemophilia Therapy — Navigating Speed Bumps on the Innovation Highway
D DiMichele, NEJM, May 25, 2016 (Posted: May 26, 2016 0AM)

A First in Hemophilia B Therapy
R Voelker, JAMA, April 19, 2016 (Posted: Apr 22, 2016 11AM)

Whole-exome sequencing to identify genetic risk variants underlying inhibitor development in severe hemophilia A patients
MM Gorski et al, Blood, March, 2016 (Posted: Apr 20, 2016 3PM)

Personalizing the royal treatment for hemophilia
ML Hulbert, Science Trans Med, April 20, 2016 (Posted: Apr 20, 2016 3PM)

World Hemophilia Day 2016
Brand (Posted: Apr 15, 2016 1PM)

The past and future of haemophilia: diagnosis, treatments, and its complications.
Peyvandi Flora et al. Lancet (London, England) 2016 Feb (Posted: Mar 16, 2016 5PM)

Key Findings: Study of Hemophilia Care Outcomes over 50 Year Span Reveals Progress, Ongoing Health Challenges
Brand (Posted: Mar 16, 2016 5PM)

Von Willebrand disease in the emergency department.
Livesey K et al. Haemophilia : the official journal of the World Federation of Hemophilia 2015 Nov (Posted: Dec 01, 2015 1PM)

Recommendations on Standardized Testing and Surveillance for Inhibitors in Patients with Hemophilia A and B
Hemophilia Foundation, October 2015 (Posted: Nov 22, 2015 2PM)

Health-related quality of life assessment in haemophilia patients on prophylaxis therapy: a systematic review of results from prospective clinical trials.
Oladapo A O et al. Haemophilia : the official journal of the World Federation of Hemophilia 2015 Sep (5) e344-58 (Posted: Nov 22, 2015 8AM)

New products for the treatment of haemophilia.
Laffan Mike et al. British journal of haematology 2015 Oct (Posted: Nov 22, 2015 8AM)

New and improved AAVenues: current status of hemophilia B gene therapy.
Brimble Mark A et al. Expert opinion on biological therapy 2015 Nov 1-14 (Posted: Nov 22, 2015 8AM)

New Recommendation on Inhibitors In Light of CDC Findings
Hemophila Foundation, November 10, 2015 (Posted: Nov 22, 2015 8AM)

Treatment of Hemophilia A and B and von Willebrand disease: A Systematic Review
et al. Stockholm:Swedish Council on Health Technology Assessment (SBU) 2011 May (Posted: Aug 28, 2015 0PM)

Hemophilia And Von Willebrand Disease In Children: Emergency Department Evaluation And Management.
Schwartz Kevin R et al. Emerg Med Pract 2015 Sep (9) 1-24 (Posted: Aug 28, 2015 0PM)

New approaches to gene and cell therapy for hemophilia.
Ohmori T et al. J. Thromb. Haemost. 2015 Jun S133-42 (Posted: Jul 30, 2015 8AM)

A systematic review of definitions and reporting of bleeding outcome measures in haemophilia.
Chai-Adisaksopha C et al. Haemophilia 2015 Jul 14. (Posted: Jul 30, 2015 8AM)

Targeting Antithrombin to Treat Hemophilia.
Ragni Margaret V et al. N. Engl. J. Med. 2015 Jul 23. (4) 389-91 (Posted: Jul 30, 2015 8AM)

Pain Toolkit
From the Hemophilia Foundation (Posted: May 26, 2015 0PM)

Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens.
Oldenburg Johannes et al. Blood 2015 Mar 26. (13) 2038-2044 (Posted: Apr 14, 2015 9AM)

The current status of prophylactic replacement therapy in children and adults with haemophilia.
Ljung Rolf et al. Br. J. Haematol. 2015 Mar 29. (Posted: Apr 14, 2015 9AM)

A new recombinant factor VIII: from genetics to clinical use.
Santagostino Elena et al. Drug Des Devel Ther 2014 2507-15 (Posted: Mar 02, 2015 9AM)

Gene therapy for hemophilia.
Rogers Geoffrey L et al. Front Biosci (Landmark Ed) 2015 556-603 (Posted: Mar 02, 2015 9AM)

Non-genetic risk factors and their influence on the management of patients in the clinic.
Álvarez Teresa et al. Eur. J. Haematol. 2015 Feb 2-6 (Posted: Mar 02, 2015 9AM)

Genetic risk factors for inhibitors in haemophilia A.
Bardi Edit et al. Eur. J. Haematol. 2015 Feb 7-10 (Posted: Mar 02, 2015 9AM)

Strategies for inhibitor treatment and costs in the short and long term: a critical evaluation of recent clinical studies.
Baghaipour Mohammad Reza et al. Eur. J. Haematol. 2015 Feb 30-7 (Posted: Mar 02, 2015 9AM)

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Disclaimer: Articles listed in Hot Topics of the Day are selected by Public Health Genomics Branch to provide current awareness of the scientific literature and news. Inclusion in the update does not necessarily represent the views of the Centers for Disease Control and Prevention nor does it imply endorsement of the article's methods or findings. CDC and DHHS assume no responsibility for the factual accuracy of the items presented. The selection, omission, or content of items does not imply any endorsement or other position taken by CDC or DHHS. Opinion, findings and conclusions expressed by the original authors of items included in the Clips, or persons quoted therein, are strictly their own and are in no way meant to represent the opinion or views of CDC or DHHS. References to publications, news sources, and non-CDC Websites are provided solely for informational purposes and do not imply endorsement by CDC or DHHS.
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