Inhibitor Incidence in Severe Hemophilia B
Inhibitor incidence was found to be 10.2% in previously untreated patients with severe hemophilia B after 500 exposure days.
Bleeding Disorders
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Female Patients With Bleeding Disorders Included in the American Thrombosis and Hemostasis Network Dataset
The most common bleeding disorder in this cohort was von Willebrand disease, which comprised 62.9% of diagnoses.
Lack of Standardized Treatment for Pregnant Women With Type 3 von Willebrand Disease
Researchers conducted a systematic review to assess outcomes and complications in pregnant women with type 3 von Willebrand disease.
Are High-Throughput Sequencing Tests Useful for Diagnosing Bleeding, Thrombotic, and Platelet Disorders?
Researchers evaluated the diagnostic utility of the ThromboGenomics high-throughput sequencing test for bleeding, thrombotic, and platelet disorders.
Adolescents With Hemophilia May Struggle With Low Self-Esteem
Patients with hemophilia had lower median score on the Offer Self-Image Questionnaire compared with a control group.
Incidence of Bleeding Disorders in Adolescents Seeking Emergency Care for Heavy Menstrual Bleeding
The majority of patients admitted to the hospital after seeking emergency care for heavy menstrual bleeding did not have an underlying bleeding disorder.
BLA Submitted for Gene Therapy to Treat Hemophilia A
The US FDA has received a BLA for valoctocogene roxaparvovec (BMN 270) for the treatment of hemophilia A in adults.
Expert Q&A: The Current State of Gene Therapy for Hemophilia
Although gene therapy shows promise in curing hemophilia, questions about its efficacy, optimal administration, and outcomes remain.
Two Easily Measured Biomarkers Could Predict Hemorrhage in Thrombocytopenia
C-reactive protein and urine albumin/creatinine ratio were associated with increased risk of bleeding in patients with thrombocytopenia due to hematologic malignancy.
Emicizumab Yields Clinically Relevant Improvement in Joint Health in Hemophilia A
Patients with hemophilia A reported a median improvement of 2.25 points in hemophilia joint health score during the course of the study.
Factor XI Deficiency Linked To Increased Risk for Postpartum Hemorrhage With Cesarean Delivery
Risk for postpartum hemorrhage after cesarean delivery was nearly 2-fold greater in patients with mild factor XI deficiency compared with the control cohort.
Simoctocog Alfa Safe, Effective for Managing Inhibitors in Hemophilia A
Researchers evaluated whether simoctocog alfa, a fourth-generation recombinant factor VIII product, is safe and effective in patients with severe hemophilia A.
Inhibitors Do Not Cause Early Mortality in Nonsevere Hemophilia A
Median age at inhibitor development was 13 years, and the inhibitor-related mortality rate was 2.44 per 1000 person-years.
Severe Postpartum Hemorrhage as Evidence of an Inherited Bleeding Disorder
Of 69 patients referred for hemostatic examination due to severe postpartum hemorrhage, 16 (23%) were diagnosed with a mild bleeding disorder.
Establishment of ROTEM Sigma Parameters to Evaluate Coagulopathic Bleeding
Researchers calculated the optimal values for parameters of the ROTEM sigma-based algorithm for treating coagulopathic bleeding.
Updated Guidance on Managing Bleeding and Surgery on Antiplatelet Therapy
Antiplatelet therapy is used to manage cardiovascular events but can lead to increased risk for bleeding events both mild and severe.
Many Patients Lack Knowledge of Potential Interactions Between Apixaban, Over-the-Counter Products
Researchers found that many patients have limited knowledge concerning potential serious interactions between dietary supplements and apixaban.
Resting vs Activated Platelets: Why Knowing the Difference Makes Financial Sense
Transfusing activated platelets can increase the number of transfusions required and thus lead to additional costs compared with transfusing resting platelets.
Lower Transfusion Threshold for Preterm Neonates With Thrombocytopenia Reduces Risk for Death, Major Bleeds
Patients who were transfused at a lower platelet count threshold experienced a 7% decrease in absolute risk for death or major bleeding.
Acquired von Willebrand Syndrome in Pediatric Patients With Chronic Myeloid Leukemia
Researchers assessed whether high platelet counts in pediatric patients with chronic myeloid leukemia resulted in thrombosis or bleeding.
Children With Type 3 von Willebrand Disease More Likely To Report Bleeds, Need for Mobility Assistance
Male patients with von Willebrand disease were more likely to be diagnosed earlier and to report bleeds compared with female patients.
Bleeding Disorders Are Prevalent in Adolescents With Heavy Menstrual Bleeding
Patients with anovulatory bleeding experienced a significantly longer time to bleeding disorder diagnosis compared with patients with ovulatory bleeding.
FDA Approves Earlier Use of Nplate in Immune Thrombocytopenia
The FDA has approved the supplemental Biologics License Application for Nplate (romiplostim; Amgen) to expand treatment to newly diagnosed adult patients with immune thrombocytopenia.
FDA Approves Wilate for Hemophilia A in Adult and Adolescent Patients
The Food and Drug Administration (FDA) has approved Wilate (von Willebrand Factor/Coagulation Factor VIII Complex [Human]; Octapharma) for routine prophylaxis to reduce the frequency of bleeding episodes and on-demand treatment and control of bleeding episodes in patients with hemophilia A. The approval was based on data from the prospective, open-label, multicenter phase 3 trial that…
Advances in the Diagnosis of Type 1 von Willebrand Disease: Genetic Testing
Despite the increasing availability of gene sequencing, several barriers prevent it from having utility as a tool for diagnosis of type 1 von Willebrand disease.
Initial Laboratory Testing Sufficient for Diagnosing von Willebrand Disease in Many Pediatric Patients
For a cutoff von Willebrand factor antigen value of 100 IU/dL above which repeat testing was not necessary, negative predictive value was 96.6%.
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.
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.
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