Initial Hemophilia B Treatments Should Be Done in Hospitals, Researchers Suggest
The initial treatment in patients newly diagnosed with hemophilia B should definitely be done in hospital according to a study conducted by scientists in Turkey.
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Rare New Case of Pseudotumor in non-Hemophiliac Patient Reported
Researchers at the Mayo Clinic Arizona have reported a third rare case of hemophilic pseudotumor (HP) identified in a non-hemophiliac patient. Findings from the investigation, “Hemophilic pseudotumor in a non-hemophilic patient treated with a hybrid procedure of preoperative embolization of the feeding arteries followed by surgical resection—A…
Hemophilia Review Highlights Improved Patient-reported Outcome Measures
In an article titled “Recognizing the need for personalization of hemophilia patient-reported outcomes in the prophylaxis era” published in the scientific journal Haemophilia, researchers reviewed two new tools that are used to measure patient reported outcome measures.
Type of Chromatin Seen to Stop ‘Jumping Genes’ from Doing Harm, as in Hemophilia
New research suggests that the main developmental role of heterochromatin, a form of dense DNA found in chromosomes, is to suppress virus-like DNA elements known as “jumping genes” from replicating and attaching themselves across a person’s genome, potentially destroying important genes and causing a variety of diseases, including hemophilia and cancer. The study, by University of North Carolina…
Sangamo’s Genome Editing Therapy for Hemophilia B Named ‘Orphan Drug’ by FDA
Sangamo BioSciences announced that its zinc finger nuclease (ZFN)-mediated genome editing product, SB-FIX, has received orphan drug designation from the U.S. Food and Drug Administration (FDA), speeding its development as a potential treatment for hemophilia B. Sangamo plans to initiate a Phase 1/2 clinical trial, SB-FIX-1501, in adults with this disease later…
Hemophilia Care May Be Improved by Implementation of New WFH Form
The World Federation of Hemophilia (WFH) has created a universal case report form for the collection of core data on hemophilic patients in a standardized format that will include relevant diagnosis, therapies, and outcome. Previous WFH forms provided only an overview of the number of patients and access to treatment.
Brain Hemorrhage in Boy with Hemophilia Treated with Transfusions
Researchers in India recently reported the case of child with hemophilia and traumatic intracerebellar hemorrhage, who was successfully treated with transfusions of plasma. They recommend a conservative approach as a first line of treatment, before more invasive alternatives such as surgery. The case report, “Intracerebellar haemorrage in a haemophilia child,” was published in…
New Recombinant Factor IX Products for Hemophilia B May ‘Revolutionize’ Treatment
Patient treatment for hemophilia B has come a long way since the introduction of factor IX (FIX) concentrates. In a recent commentary published in the journal Blood and titled “A new era for hemophilia B treatment,” researchers from Oregon Health and Science University and Bloodworks Northwest highlighted recent advances in…
Researchers Question Health-Related Quality of Life for Youth, Young Men with Hemophilia
Researchers at several Canadian institutions investigated the tendencies of health-related quality of life (HRQoL) among teenagers and young adults with hemophilia A (HA) and found that young adults demonstrated more joint disease and worse HRQoL when physical function and pain were considered. The research paper, “Generic and disease-specific quality of…
Preventive Hemophilia Treatment Still Faces Obstacles, but Future Looks Promising
Prophylactic hemophilia treatment can save patients with severe forms of the disease from the long-term consequences of joint damage and other complications. A report by Dr. Suchitra S. Acharya, MD, of Hofstra Northwell School of Medicine in New York, gives a detailed overview of the current state and future perspectives…