Project GYM: Physical Fitness Improves Wellbeing in Young Men With Hemophilia
Researchers investigated the safety and feasibility of Project GYM, a personal trainer-led physical fitness program for young men with hemophilia.
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Use of a Smart Medical Internet of Things System on Quality of Life in Patients With Hemophilia
Researchers sought to determine whether home nursing based on smart medical Internet of Things systems could improve the quality of life of patients with hemophilia.
Long-Term Prophylaxis Cost-Effective for Von Willebrand Disease
Researchers sought to determine whether long-term prophylaxis against bleeds in patients with von Willebrand disease would be cost effective.
Dental Caries and Other Aspects of Oral Health That Impact Patients With Hemophilia
Researchers sought to determine patterns of oral health in patients with hemophilia.
Plasma-Derived VWF Effective for Young Children With Severe Von Willebrand Disease
Researchers sought to determine whether plasma-derived VWF would be effective in treating hemorrhage in young children with severe VWD.
Trials Included in Many Coagulation Factor Package Inserts Have Significant Biases
Researchers sought to determine whether biases were present in clinical trials included in the package inserts of coagulation factors used for the treatment of hemophilia.
New Study Evaluating BPA Use With Emicizumab Among Patients With Hemophilia A
Researchers are seeking to evaluate outcomes after initiation of emicizumab and concomitant BPAs in patients with hemophilia A and FVIII inhibitors.
Extension Study Shows Safety, Efficacy of rVIII-SingleChain for Previously Treated Hemophilia A
Researchers sought to determine whether rVIII-SingleChain therapy would be safe and effective as a long-term prophylaxis option for previously treated patients with hemophilia A.
PRO Tool, GOAL-Hēm, Now More Patient-Centric for Patients With Hemophilia
Researchers sought to revise the GOAL-Hēm patient-reported outcomes tool to be more patient-centric for patients with hemophilia.
Efanesoctocog Alfa Appears Effective in Severe Hemophilia A
Researchers sought to determine whether efanesoctocog alfa may yield high sustained FVIII activity and improve bleed protection in patients with severe hemophilia A.
Valoctocogene Roxaparvovec Evaluated in Severe Hemophilia A
In open-label study, substantial increase seen in factor VIII activity at weeks 49 to 52 of follow-up.
Gene Therapy Gets Fast Track Status for Hemophilia A
In preclinical studies, ASC618 has been shown to have at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with native human FVIII bioengineered constructs.
Reduced Cardiovascular Morbidity Seen in Patients With Hemophilia
Researchers sought to determine the incidence of fatal and nonfatal CVD during 5 years of follow up in adults with hemophilia.
Millennials With Hemophilia: Attitudes and Expectations From Treatment
Researchers sought to determine the attitudes and expectations of patients with hemophilia belonging to Generation Y regarding their treatment.
Perspectives on Participation in Sports in Patients With Hemophilia
Researchers sought to provide perspectives and recommendations for engaging patients with hemophilia in physical activity.
Durable Response Observed With Gene Therapy for Hemophilia A
The phase 3 GENEr8-1 study evaluated valoctocogene roxaparvovec in adults with severe hemophilia and residual FVIII levels less than or equal to 1 IU/dL.
Social Participation, Self-Perception, and Social Support in Boys With Hemophilia
Researchers sought to evaluate relationships between self-perception and social participation in boys with hemophilia.
Researchers Developed a Parenting Booklet on Physical Activity Restrictions and Recommendations in Pediatric Hemophilia
Researchers sought to develop a booklet for parents of children with hemophilia to help them manage physical activity restrictions based on the self-determination theory.
Care Delivery Model for Patients With Severe Hemophilia A in Tehran, Iran
Researchers sought to develop a care delivery model for patients with severe hemophilia A to improve on care and the use of resources.
Immune Tolerance Induction Therapy With Recombinant Antihemophilic Factor Seems Effective in Hemophilia A
Researchers sought to determine whether ITI therapy with ADVATE® would be safe and effective in patients with hemophilia A and neutralizing antibodies to coagulation factor VIII.
Experts Fail To Reach Consensus on Treatment of Hemophilia A
An expert panel sought to determine how to treat some patients with severe hemophilia A.
Magnetic Resonance Imaging of Joints Over Time in Boys With Severe Hemophilia A
Researchers sought to determine whether MRI would be useful in evaluating joint changes in boys with severe hemophilia.
Dronedarone Plus DOACs Increase Risk for Bleeding Events in AF
A study finds that dronedarone increased the risk for major bleeding events in patients with atrial fibrillation also taking a DOAC.
Strenuous Physical Activity Increases Bleed Risk in Patients With Hemophilia A
Researchers sought to determine whether patients with hemophilia A would be at higher risk of bleeding events when participating in high-collision physical activity.
Health Outcomes With Extended Vs Standard Half-Life Factor Products for Patients With Hemophilia
Researchers sought to determine whether switching to EHL factor concentrates would have an impact on health-related quality of life for patients with hemophilia.
Patient Care Provided by Hemophilia Treatment Centers in the United States
Researchers sought to summarize hemophilia treatment centers in the United States, how they function, and the benefits of this care approach.
A Case of Peripartum Hemophilia A in a Patient With Sickle Cell Trait
A new report described the presentation, diagnosis, and management of a patient with sickle cell trait who developed peripartum hemophilia A.
Comparison of Approaches for Detection of Type 1 von Willebrand Disease
Researchers sought to determine whether the HemosIL VWF activity assay would be an effective method for identifying VWD.
Mobile Health Technology in the Management of Hemophilia
Researchers sought to determine whether mobile health technology may play a helpful role in the management of hemophilia.
Positive Impact Seen With Neurodynamics Nerve Flossing on Femoral Neuropathy in Patients With Hemophilia
Researchers sought to determine whether the neurodynamics nerve flossing technique would be beneficial in the treatment of femoral neuropathy in children with hemophilia.
MarzAA Safe and Efficacious as Prophylactic Treatment in Patients With Severe Hemophilia With Inhibitors
Researchers sought to determine whether a novel variant of activated human factor VII Marzeptacog alfa (activated) would be effective as prophylactic treatment in patients with severe congenital hemophilia with inhibitors.
Bleeding Patterns of Infants and Toddlers With von Willebrand Disease
Researchers sought to define bleeding patterns and other characteristics in infants and toddlers with von Willebrand disease.
Recombinant FVIIIFc May Be Cost Effective for Treating Severe Hemophilia A
Researchers sought to determine whether rFVIII Fc-fusion protein would be a cost-effective treatment option for patients with severe hemophilia A.
In Silico Reduction in Antithrombin Level in Patients With Hemophilia A
Researchers sought to determine whether antithrombin would have the potential to restore thrombin generation in patients with hemophilia A.
Standardized Ultrasound Guidelines Needed for Patients With Hemophilia, According to Expert Panel
Researchers sought to determine whether clearer guidelines are needed for utilizing ultrasounds to assess musculoskeletal outcomes in patients with hemophilia.
Aerobic, Resistance, Combined Training Improves Lipid Profiles and Quality of Life in Overweight Patients With Hemophilia A
Researchers sought to determine whether combined training would improve lipid profiles and quality of life in patients with hemophilia A who were overweight.
Continuing Prophylaxis into Young Adulthood Improves Outcomes for Patients With Hemophilia
People with hemophilia tend to have less adherence to prophylaxis as they transition to adulthood but continuing the therapy can improve their joint health.
Plasma-Derived Von Willebrand Factor/FVIII Safe, Effective in Patients Younger Than 12 With Hemophilia A
Researchers sought to determine whether plasma-derived von Willebrand factor/factor VIII would be safe and effective in patients with hemophilia A younger than 12 years.
Joint Health Deteriorated in Patients With Hemophilia A During COVID-19 Lockdown
Researchers sought to determine the impact of the COVID-19 lockdown on patients with hemophilia.
Patients With Hemophilia Can Safely Perform Knee Extensions
Researchers sought to determine whether patients with hemophilia could safely perform moderate intensity training without adverse effects.
Emicizumab May Improve Outcomes for Acquired Hemophilia A
Researchers sought to determine whether emicizumab would help improve outcomes for patients with acquired hemophilia A.
Phase 3 Results Confirm Emicizumab Is Safe for Hemophilia A
Researchers sought to confirm whether emicizumab is safe for treating patients with hemophilia A with factor VIII inhibitors.
Recombinant von Willebrand Factor Prophylaxis Effective at Preventing Bleeding in Patients With von Willebrand Disease
Researchers sought to determine whether recombinant VWF would be effective in reducing spontaneous bleeding for patients with severe VWD previously treated in an on-demand manner.
Monoclonal Antibody Marstacimab Appears to Be Safe and Effective in Patients With Severe Hemophilia
Researchers sought to determine whether marstacimab would be effective in treating patients with severe hemophilia.
Gene Therapy Reduces Bleed Rates Among Patients With Hemophilia A
Researchers sought to determine whether valoctocogene roxaparvovec would reduce the risk of bleeding and rate of factor VIII infusion in patients with hemophilia A.
FDA Issues Safety Alert Regarding Use of Hydroxyethyl Starch Products
There are currently 4 FDA-approved hydroxyethyl starch products on the market.
Updates on the Etiology, Diagnosis, Management of von Willebrand Syndrome and Hemophilia A
Acquired hemophilia A (AHA) and acquired von Willebrand syndrome (aVWS) are rare, life-threatening bleeding disorders that occur in patients with no bleeding history and no family history of a bleeding disorder.
First-in-Human Results Show Promise With BT200, a New Anti-von Willebrand Factor Agent
Researchers sought to determine whether BT200, a new anti-von Willebrand factor, would have efficacy in patients with von Willebrand disease.
Novel Tool Developed to Measure the Impact of Hemophilia on Families
Researchers have developed and validated a novel tool, the Hemophilia Family Impact Tool (H-FIT) to measure the impact of hemophilia on families.
More Bleeding Events After PCI in Women With Baseline Risk Factors
The researchers explored the association of sex with outcomes among patients treated with ticagrelor monotherapy vs ticagrelor plus aspirin.
ABO-Incompatible Platelet Transfusion May Lead to Worse Clinical Outcomes in ICH
Researchers sought to determine whether transfusing ABO-incompatible platelets would lead to worse clinical outcomes and slower platelet recovery in patients with ICH.
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