Bleeding Disorders
Conference Coverage
Two scoring systems helpful in diagnosing heparin-induced thrombocytopenia
SAN DIEGO – Both the 4Ts Score or the HIT Expert Probability (HEP) Score performed well in a real-world study.
Conference Coverage
Researchers seek better understanding of von Willebrand disease
SAN DIEGO – Researchers are exploring the causes of VWD and ways to tailor treatments.
Conference Coverage
Why is gene therapy for hemophilia taking so long?
SAN DIEGO – The short answer is that gene therapy vectors are complicated.
Conference Coverage
Most patients off transfusions after gene therapy for thalassemia
SALT LAKE CITY – As the vector copy number goes up, so does the likelihood of stopping transfusions.
News
Deaths in patients on emicizumab
Two hemophilia organizations have notified the public of 5 deaths in adult patients receiving emicizumab (Hemlibra). All 5 deaths—occurring in...
Clinical Review
Von Willebrand Disease: Approach to Diagnosis and Management
von Willebrand disease (VWD) is an inherited bleeding disorder caused by deficient or defective plasma von Willebrand factor (VWF). VWF is an...
News
Product approved for hemophilia patients in Japan
Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved the bispecific factor IXa- and factor X-directed antibody emicizumab (Hemlibra...
Conference Coverage
Bleeding episodes more common in boys with VWD
SAN DIEGO – Bleeding episodes and need for treatment were more frequent among boys with types 1 and 2 von Willebrand disease.
Feature
Of ‘miracles’ and money: Why hemophilia drugs are so expensive
The U.S. hemophilia drug market, which serves about 20,000 patients, is worth $4.6 billion a year.
Feature
Hemophilia A treatment gains approval in Europe
Emicizumab is now approved in Europe and the United States for routine prophylaxis of bleeding episodes in people with factor VIII inhibitors.
Feature
States strive to curb costs for a crucial – but exorbitant – hemophilia treatment
“There aren’t a lot of options available to Medicaid programs in terms of controlling costs, because we don’t set the initial costs”