Health Care Costs With Bypassing Therapy vs Factor VIII Replacement for Hemophilia A
Patients treated with bypassing therapy experienced significantly higher medical and outpatient pharmacy costs.
Bleeding Disorders
BLEEDING DISORDERS
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Hemophilia Prevalence Globally and at Birth in Men
Researchers have found that the estimated number of men with hemophilia is higher than known diagnoses, prompting a call for improved diagnosis approaches.
Investigational Therapy for Hemophilia A Granted Orphan Drug Designation
The Food and Drug Administration (FDA) has granted Orphan Drug designation to SIG-001 (Sigilon Therapeutics) for the treatment of hemophilia A. SIG-001 utilizes Sigilon’s Shielded Living Therapeutics platform to implant engineered human cells to produce stable blood plasma levels of factor VIII. The cells are also shielded by Sigilon’s proprietary Afibromer biomaterials matrix that minimizes…
Efficacy Comparison of Emicizumab and Factor VIII Prophylaxis Treatments in Hemophilia A Without Inhibitors
Researchers conducted a meta-analysis of 4 studies examining prophylaxis with factor VIII or emicizumab for hemophilia A without inhibitors.
Discrepancies in Self-Reported and Objective Measures of Prophylaxis Adherence in Hemophilia
Self-reported adherence to prophylaxis did not correlate with objective measures of treatment adherence.
Challenges in Diagnosing and Managing von Willebrand Disease
Researchers highlighted the current status of diagnostic strategies and laboratory screening tests for von Willebrand disease.
Recommendations for Optimizing the Use of Group O Red Blood Cells in Transfusions
Group O Rh(D)-negative blood cells can be administered to patients with any ABO Rh(D) type, making them a valuable but limited resource.
Prevalence of Gastrointestinal Bleeding in Patients With von Willebrand Disease
Researchers conducted a retrospective analysis of records from the National Inpatient Sample to identify risk factors for gastrointestinal bleeding.
Inhibitor Development Is Rare After 75 Days of Exposure to Factor VIII in Severe Hemophilia A
In patients with hemophilia A who developed inhibitors, 99.3% of inhibitors developed within 75 days of exposure to factor VIII.
Risk for Factor VIII Inhibitor Development Appears Lower With Third-Generation Products
Researchers evaluated whether certain brands of recombinant Factor VIII products were associated with increased risk of developing inhibitors.
Clinical Management of Inhibitors in Children With Hemophilia
Treatment strategies for children with hemophilia who have inhibitors should involve eradicating the inhibitor and managing bleeding.
Influence of Factor VIII Prophylaxis on Joint Health in Children With Hemophilia A
Only factor VIII trough level and prophylactic treatment showed associations with joint health in univariate analysis.
Predicting Bleeding Risk in Acute Myeloid Leukemia With Thrombocytopenia With Platelet Function Tests
Platelet aggregation and platelet activation were both independently associated with bleeding risk.
High-Throughput Sequencing May Improve Diagnosis of Inherited Bleeding Disorders
Currently, molecular testing occurs at the tail end of the diagnostic workflow for inherited bleeding disorders.
Body Mass Index Associated With Increased von Willebrand Factor, Factor VIII Levels
For both type 1 and type 2 von Willebrand disease, higher BMI was associated with increased VWF:Ag and FVIII:C levels.
Evaluating Factor Replacement Therapies for von Willebrand Disease
Variation in disease phenotype and clinical circumstances makes it difficult to create a standardized treatment approach to von Willebrand disease.
Impaired Health‐Related Quality of Life in Patients With Hemophilia A With Inhibitors
Patients’ responses to surveys assessing quality of life indicated impairment in both adults and adolescents.
Diagnostic Utility of Bleeding Assessment Tool for Identifying Platelet Function Disorders
A bleeding assessment tool yielded higher scores for patients with suspected or confirmed platelet function disorders.
Detection and Management of Factor XIII A-Subunit Deficiency in Neonates
Researchers analyzed the clinical features of factor XIII deficiency in neonates and used the data to develop a diagnostic algorithm.
Bleeding Risks for Patients With Cancer on Anticoagulant Therapy
For all anticoagulants examined, patients with cancer had higher bleeding incidence rates than patients without cancer.
Treating Major Bleeding in Patients Taking Direct-Acting Oral Anticoagulants
There was no difference in mortality rate between patients taking various direct-acting oral anticoagulants (DOACs), though bleeding locations varied for each DOAC.
The Role of Cardiovascular Diseases in the Pathogenesis of Acquired von Willebrand Syndrome
High shear stress in blood vessels due to cardiovascular disease may be associated with the development of acquired von Willebrand syndrome.
Whole Blood Ristocetin-Induced Platelet Aggregometry for Determining von Willebrand Disease Severity
Assay values were not strongly correlated with von Willebrand factor ristocetin cofactor or antigen activity.
Addressing Shortcomings in Management of Heavy Menstrual Bleeding in Adolescents
Because most studies on heavy menstrual bleeding have been conducted in adults, treatment guidelines for adolescents are lacking.
Discriminating Coagulation Patterns in Cyanotic, Noncyanotic Congenital Heart Disease
Patients with cyanotic congenital heart diseases were found to have higher levels of hematocrit, hemoglobin, and reticulocytes.
Novel Reversal Agent for Ticagrelor Gets FDA’s Breakthrough Tx Designation
The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to PB2452 (PhaseBio), an investigational reversal agent for ticagrelor.
Novel Mutations Associated With Hereditary Factor X Deficiency Identified
Researchers identified 11 novel mutations that may play a pathogenic role in hereditary factor X deficiency.
Intracranial Bleeding Rates in Neonates With Hemophilia by Mode of Delivery
Neonates with hemophilia who were delivered either vaginally or by Cesarean section had similar rates of intracranial bleeding.
Management of Intracranial Hemorrhage in Patients With Hemophilia
Researchers reviewed 33 studies published over a span of 20 years to identify risk factors and symptoms of intracranial hemorrhage in patients with hemophilia.
Once-Weekly Prophylaxis for Low-Bleeding Patients With Severe Hemophilia A
Weekly prophylaxis with glycopegylated recombinant factor VIII was well tolerated and had a good safety profile for low-bleeding patients with hemophilia A.
Safety of Buccal Administration of Dental Anesthetic in Patients With Hemophilia
Current guidelines do not adequately inform management of patients with inherited bleeding disorders undergoing routine dental procedures.
New Tool for Reconstituting Factor VIII for Hemophilia A Prophylaxis
Simplifying factor VIII reconstitution may increase treatment adherence and satisfaction in patients with hemophilia A.
Optimal Platelet Transfusion Threshold Unknown in Patients Undergoing Lumbar or Epidural Procedures
Unnecessarily high platelet count thresholds may expose patients undergoing lumbar or epidural procedures to excessive transfusion-related risks.
Advances in the Treatment of Hemophilia: Clinical Gene Therapy
Gene therapy with adeno-associated viral viruses could provide an efficacious, safe, and cost-effective treatment option for patients with hemophilia.
Congenital Bleeding Disorders Not Associated With Increased Endoscopic Bleeding Risk With Proper Prophylaxis
Researchers conducted a retrospective chart review to assess the bleeding risk associated with gastrointestinal endoscopy.
Sex-Specific Models for Predicting Bleeding Risk Associated With Aspirin Use
Researchers assessed potential risk factors for major bleeding in patients without cardiovascular disease to develop sex-specific prediction models.
Efficacy of Secondary Prophylaxis in Patients With Severe Inherited von Willebrand Disease
Researchers assessed whether prophylactic treatment with von Willebrand factor/factor VIII concentrates reduced bleeding risk.
A Novel Screening Program for Severe Combined Immunodeficiency in Newborns
Population-based screening of newborns is crucial to identifying severe combined immunodeficiency before the onset of potentially fatal infection.
Esperoct Approval Provides New Treatment Option for Hemophilia A
Novo Nordisk announced that the Food and Drug Administration (FDA) has approved Esperoct (turoctocog alfa pegol, N8-GP) for the treatment of patients with hemophilia A (congenital factor VIII deficiency) for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment and control of bleeding episodes and perioperative management of bleeding.
Long-Term Outcome Profile in Patients With Acquired Hemophilia A
Underlying lymphoproliferative syndromes, male gender, and older age may be predictive of relapse in patients with acquired hemophilia A.
Episodic Treatment Compared With Prophylaxis in Patients With Hemophilia A
Researchers assessed safety outcomes in patients with hemophilia A without inhibitors.
Assessing Children With Bleeding Symptoms: Coagulation Disorder or Abuse?
Several factors need to be considered when determining whether a child’s bleeding symptoms are the result of a coagulation disorder, physical abuse, or both.
Pharmacokinetics-Based Hemophilia A Prophylaxis May Improve Clinical Outcomes
Researchers assessed the influence of adopting population pharmacokinetics to inform prophylaxis for patients with hemophilia A.
Andexanet Alfa Efficacious in Reducing Antifactor Xa Activity
Acute major bleeding decreased by 92% in patients who were treated with andexanet alfa.
Use of Rotational Thromboelastometry With Ristocetin to Identify Clinically Relevant von Willebrand Disease
Using rotational thromboelastometry may allow assessment of bleeding risks in patients with clinically relevant von Willebrand disease.
Safety Comparison of Apixaban vs Rivaroxaban for Thromboembolism Prevention
Researchers conducted the first real world study to compare the safety and effectiveness of apixaban and rivaroxaban in patients with VTE.
Updates to Transfusion Strategies for Major Hemorrhage in Trauma
Changes in clinical management of trauma hemorrhage have significantly reduced mortality rates, but variation in treatment strategies still remains.
Tranexamic Acid Concentrations Required for Fibrinolysis Inhibition
The need for tranexamic acid to be administered intravenously creates a barrier to rapid treatment of patients with fibrinolysis.
Improving the Clinical Diagnosis of Inherited Bleeding Disorders
Many unique challenges surround the diagnosis and treatment of inherited mild bleeding disorders.
Researchers Review Coagulation Risk Factors in Neonates and Children
The interactions between von Willebrand factors and its cleaving protease ADAMTS-13 recently emerged as risk factors for pediatric thromboembolism.
Self-Reported Bleeding Symptoms May Not Be Associated With Greater Fibrinolytic Activity
Propensity for bleeding was previously thought to relate to greater fibrinolytic activity, but this may not be the case.
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