Archive
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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Archived Hot Topics of the Day By Date
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)
How do people inherit hemophilia?
Medical News Today, September 2023
(Posted: Sep 29, 2023 11AM)
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)
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)
Researchers uncover mysteries behind immune response to hemophilia A treatment
Medical XPress, August 2023
(Posted: Aug 18, 2023 11AM)
Community Counts: Understanding Hemophilia and Other Bleeding Disorders through Public Health Surveillance
CDC webinar, August 24, 2023
(Posted: Jul 11, 2023 8AM)
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)
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)
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)
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)
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)
Current challenges in hemophilia genetics and how they can be overcome
JM Johnson, Nat Cardiovasc Research, October 2022
(Posted: Nov 04, 2022 11AM)
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)
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
(Posted: Mar 30, 2022 7AM)
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
MC Ozelo et al, NEJM, March 17, 2022
(Posted: Mar 17, 2022 9AM)
Prepare the Way for Hemophilia A Gene Therapy
CD Thornburg, NEJM, March 17, 2022
(Posted: Mar 17, 2022 9AM)
Disease of the Week: Hemophilia
CDC, March 2022
(Posted: Mar 02, 2022 8AM)
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
(Posted: Feb 15, 2022 7AM)
Gene therapy for hemophilia A
K O'Leary, Nature Medicine, November 30, 2021
(Posted: Nov 30, 2021 9AM)
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
LA George et al, NEJM, November 18, 2021
(Posted: Nov 18, 2021 5AM)
Real Stories from People Living with Hemophilia – Carrie
CDC, November 2021
(Posted: Nov 14, 2021 6AM)
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)
Hemophilia
E Berntorp et al, Nature Dis Primers., June 24, 2021
(Posted: Jun 24, 2021 7AM)
Data from CDC’s hemophilia surveillance programs can help prevent or reduce hemophilia-related health problems
CDC, March 2021
(Posted: Mar 17, 2021 10AM)
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)
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)
Investigational Hemophilia A Gene Therapy Shows Sustained Benefit
J Abbasi, JAMA, February 11, 2020
(Posted: Feb 12, 2020 8AM)
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)
Off to College Toolkit
Hemophilia Federation of America, 2019
(Posted: Aug 16, 2019 8AM)
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)
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
(Posted: Jul 05, 2019 10AM)
New Opportunities and Challenges in Hemophilia Care: 2019 Update
CDC Public Health Seminar, June 6, 2019
(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
(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
(Posted: Mar 18, 2019 5PM)
Real Stories from People Living with Hemophilia
CDC, 2019
(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
(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
(Posted: Jun 14, 2018 2PM)
Basics About Hemophilia
(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 Roches 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.
(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
(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
(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
(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
(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
(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)
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.
- Page last reviewed:Feb 1, 2024
- Page last updated:Apr 15, 2024
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