EAHAD 2023: Treatments Show Promise for Hemophilia A and B
At EAHAD 2023, researchers reported positive results from trials of patients with hemophilia A and B, including those who are difficult to treat.
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Gene Therapy Improved Health-Related QoL Among Men With Severe Hemophilia A
Researchers sought to determine whether gene therapy with valoctocogene roxaparvovec would improve health-related QoL for men with severe hemophilia A.
Survey Reveals Disparities in Management of CALR-Mutated Essential Thrombocythemia
Researchers sought to determine the management of CALR-mutated essential thrombocythemia that has progressed for patients in Australia.
High Adherence, Excellent Outcomes Seen With EHL Factor VIII and IX Concentrates in Hemophilia
Researchers sought to determine whether prophylactic EHL factor VIII or IX concentrates would improve adherence rates and outcomes in patients with hemophilia.
Treatment Adherence Lower in the Transition to Adulthood in Severe Hemophilia
Researchers sought to determine treatment adherence in people with severe hemophilia.
Researchers Identify Characteristics Predicting PGT or PND Use Among Hemophilia Carriers
Researchers sought to determine characteristics that predict the use of diagnostic PGT or PND for hemophilia carriers.
Simoctocog Alfa Appears Effective in Patients With Previously Untreated Severe Hemophilia A
Researchers sought to determine whether simoctocog alfa would be safe and effective for bleeding prophylaxis patients with severe hemophilia A.
Personalized Prophylactic Approach May Be Beneficial in Hemophilia A
Researchers sought to determine whether rurioctocog alfa pegol prophylaxis would be effective for reducing ABRs in patients with hemophilia A.
Multifaceted Barriers Prevent Physical Activity in Patients With Hemophilia
Researchers sought to determine factors that prevent patients with hemophilia from engaging in physical activity.
Emicizumab Improves Coagulation Potential in Plasma From Infants and Toddlers With Hemophilia A
Researchers sought to determine the coagulation potential of emicizumab in plasmas obtained from infants and toddlers with severe hemophilia A.
Cost-Ineffectiveness Seen With Prophylactic Emicizumab in Some Patients With Hemophilia A
Researchers sought to determine the cost-effectiveness of prophylactic emicizumab in patients with moderate or mild hemophilia A without inhibitors in the United States.
Tranexamic Acid Is Not Inferior to Recombinant VWF in Alleviating Heavy Menstrual Bleeding in Patients With VWD
Researchers sought to compare tranexamic acid with VWF for alleviating heavy menstrual bleeding in patients with mild-to-moderate VWD.
Efanesoctocog Alfa Appears to Prevent Inhibitor Development, Reduce Bleeding in Children With Severe Hemophilia A
Researchers sought to determine whether efanesoctocog alfa would be an effective treatment for children with severe hemophilia A.
Fidanacogene Elaparvovec Gene-Based Therapy Shows Promise in Hemophilia B
Researchers sought to determine whether gene therapy with fidanacogene elaparvovec would reduce bleeding risk in male patients with hemophilia B.
Improvement in Joint Health Observed With Gene Therapy in Hemophilia A
Researchers sought to determine whether gene therapy with dirloctocogene samoparvovec would improve joint health for patients with hemophilia A.
Valoctocogene Roxaparvovec Shows Hemostatic Efficacy in Patients With Severe Hemophilia A Without Inhibitors
Researchers sought to determine whether valoctocogene roxaparvovec would yield hemostatic efficacy in patients with severe hemophilia A without inhibitors.
High Rate of Sustained Response Seen After TPO-RA Discontinuation in Immune Thrombocytopenia
Researchers sought to determine whether responses would be sustained after TPO-RA discontinuation among patients with persistent or chronic immune thrombocytopenia.
FDA Greenlights Gene Therapy Roctavian for Severe Hemophilia A
Roctavian is the first gene therapy approved for severe hemophilia A.
Fidanacogene Elaparvovec, a Gene Therapy for Hemophilia B, Under Review
A regulatory decision is expected in the second quarter of 2024.
Orelabrutinib Shows Promise for Immune Thrombocytopenia in Phase 2 Study
Researchers sought to determine whether orelabrutinib would be an effective treatment for patients with immune thrombocytopenia.
Fitusiran Shows Promise as Prophylactic Treatment in Hemophilia A and B
Researchers sought to determine whether fitusiran would be helpful for reducing bleeding risk in patients with hemophilia A or B.
Withholding Transfusions Before Central Venous Catheter Placement Linked to Worse Outcomes in Severe Thrombocytopenia
Researchers sought to determine whether withholding prophylactic platelet transfusions before central venous catheter placement would improve bleeding outcomes in patients with severe thrombocytopenia.
Economic Burden Associated With Having Hemophilia With Inhibitors
Researchers sought to determine the disease-related economic burden for patients who have hemophilia with inhibitors.
Recombinant Factor FVIII Fc Fusion Protein Linked to Rapid Tolerization in Severe Hemophilia A With Inhibitors
Researchers sought to determine whether there is a link between recombinant factor VIII Fc fusion protein and time to tolerization in patients with severe hemophilia A .
Fitusiran Prophylaxis Linked to Improved Bleeding Rates for Severe Hemophilia A or B Without Inhibitors
Researchers sought to determine whether a lower bleeding rate would be achieved with prophylaxis with fitusiran for hemophilia A or B.
Blueprint Developed for Research Priorities of Ultra-Rare Bleeding Disorders
Researchers sought to develop recommendations for future research opportunities to address the priorities of patients with ultra-rare bleeding disorders.
Many Hemophilia Study Participants Reported Decreased Physical Activity Levels During COVID-19 Lockdown
Researchers sought to determine whether there were changes in participant-reported physical activity among people with moderate to severe hemophilia during the COVID-19 lockdown.
On-Demand Treatment With B-Domain-Deleted Recombinant Factor VIII Effective in Hemophilia A
Researchers sought to determine whether TQG202 would lead to effective bleeding control when used on-demand for patients with moderate-to-severe hemophilia A.
Rondaptivon Pegol Shows Favorability Tolerability and Safety in Hemophilia A
Researchers sought to determine whether rondaptivon pegol would be an effective treatment to increase endogenous FVIII levels in patients with hemophilia A.
Results With N8-GP Within Range for Previously Untreated Pediatric Patients With Hemophilia A
Researchers sought to determine whether inhibitor incidence with N8-GP prophylaxis would be within range for patients with previously untreated hemophilia A.
Study Shows Positive Long-Term Efficacy and Safety With Romiplostim for Immune Thrombocytopenia
Researchers sought to determine whether romiplostim would be effective in treating pediatric patients with immune thrombocytopenia.
FDA Extends Review Period for Valoctocogene Roxaparvovec for Hemophilia A
Valoctocogene roxaparvovec is an investigational adeno-associated virus (AAV) gene therapy.
Gene Therapy Etranacogene Dezaparvovec Shows Favorable Safety for Patients With Hemophilia B
Researchers sought to determine whether etranacogene dezaparvovec would be safe for treating patients with hemophilia B.
Altuviiio Approved as Once-Weekly Factor VIII Therapy for Hemophilia A
Altuviiio temporarily replaces the missing coagulation factor VIII needed for effective hemostasis.
Study Finds Many Mothers of Children With Hemophilia Experience Guilt
Researchers sought to assess experiences of guilt and coping strategies in mothers of children with hemophilia.
Emicizumab Appears Safe, Effective in Patients With Mild or Moderate Hemophilia A
Researchers sought to determine whether emicizumab would be effective in patients with non-severe hemophilia A without inhibitors.
Efanesoctocog Alfa Prophylaxis Highly Effective in Hemophilia A
Researchers sought to determine whether efanesoctocog alfa would be effective as bleeding prophylaxis for patients with severe hemophilia A.
Women With Immune Thrombocytopenia Show No Increased Risk of Severe Bleeding During Pregnancy
Researchers sought to determine whether women with ITP have an increased risk of severe bleeding during pregnancy.
Researchers Develop Reliable Predictor for Perioperative Diagnosis of Acquired VWS in Infants With Congenital Heart Disease
Researchers sought to investigate diagnostic tools of acquired VWS compared with multimer analysis as diagnostic criterion standard.
Recent Data Shows Prophylaxis Is Initiated at a Younger Age for Patients With Severe Hemophilia
Researchers sought to determine whether there were any changes in prophylactic treatment practices among previously untreated patients with hemophilia.
A1 Domain Required for Assembly of Von Willebrand Factor Tubules With in Vivo Helical Parameters
Researchers sought to determine the role of the A1 domain of the VWF glycoprotein in VWF tubule assembly.
Problem Joints Are Associated With Worse Quality of Life and Work-Productivity Loss in Adults With Hemophilia
Researchers sought to assess the impact of problem joints in patients with hemophilia.
Haplotype Mismatch Unrelated to Anti-Factor VIII Antibody Binding in Hemophilia A
Researchers sought to determine whether a mismatch between F8 gene haplotypes impacted antibody binding to factor VIII in patients with hemophilia A.
Researchers Address Kinesiophobia in Patients With Hemophilia
Researchers sought to evaluate effects of kinesiophobia among patients with hemophilia.
Long-Term Risk of ITP Higher in Patients With Persistent, Isolated Mild Thrombocytopenia
Researchers sought to assess the risk of progression to ITP or hematologic neoplasia in adults with persistent, isolated mild thrombocytopenia.
Emicizumab Is Safe and Effective in Infants With Severe Hemophilia A
Researchers sought to determine whether emicizumab would be effective in treating infants with severe hemophilia A without factor VIII inhibitors.
Factor VIII Concentrate With an Extended Half-Life for Patients With Hemophilia A
Researchers sought to determine whether using factor VIII concentrate with an extended half-life would improve outcomes in patients with hemophilia A.
First Gene Therapy for Hemophilia B Gets FDA Approval
The approval was based on data from the HOPE-B trial that included 54 male participants with severe or moderately severe hemophilia B.
Combination Regimen Highly Effective for Acquired Hemophilia A
Researchers sought to determine whether CyDRi would be a safe and effective treatment for patients with acquired hemophilia A.
Assay Developed for the Measurement of Anti-Factor VIII Antibodies in Patients With Hemophilia A
Researchers sought to develop an assay for measuring neutralizing inhibitors and non-neutralizing antibodies in patients with hemophilia A.
Physical Activity and Fitness Appears Lower Among Patients With Moderate to Severe Hemophilia
Researchers sought to determine the rates of physical activity and fitness in patients with moderate to severe hemophilia.
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