Novel Fusion Protein to Sustain Factor VIII Activity in Hemophilia A
BIVV001, a novel fusion protein, has longer half-life than recombinant factor VIII products and may be a safe therapy for hemophilia A.
Bleeding Disorders
BLEEDING DISORDERS
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Low Bone Mass Common in Patients With Hemophilia
Investigators assessed whether there was a link between low bone mineral density, 25(OH)D concentrations, and bone turnover markers in hemophilia.
Label Mix-Up Prompts Recall of IV Amiodarone, Tranexamic Acid
Inadvertent administration may result in serious adverse events.
Effects of ABO Blood Group on von Willebrand Factor Biology, Primary Hemostasis
ABO blood group has been associated with cardiovascular disease and thrombotic risk; however, influences on hemostasis are still not well understood.
Understanding the Association Between COVID-19, Thromboembolism, and Therapeutic Anticoagulation
In expanded research, investigators studied the effects of therapeutic and prophylactic anticoagulation treatment in hospitalized patients with COVID-19.
Von Willebrand Factor Level Associated With Long-Term Outcomes in Patients After Coronary Stenting
Researchers studied the effect of certain biomarkers on long-term adverse cardiovascular outcomes in patients with coronary morbidities undergoing PCI.
PwHA: Annualized Bleeding Rates High With Current Treatment Standards
There is a need for novel, effective treatments for pediatric patients with hemophilia A with FVIII inhibitors to reduce annualized bleeding rates.
Risk Factors for Early Post-Surgical Wound Complications in Hemophilia
Investigators studied the frequency and risk factors linked to wound healing complications after elective orthopedic surgery in patients with hemophilia.
Prevalence of Endotheliopathy in COVID-19–Associated Coagulopathy
A team of investigators sought to determine whether endotheliopathy is involved in COVID-19–associated coagulopathy pathogenesis.
Assessing Prevalence and Treatment of Cold Agglutinin Disease in Warmer and Colder Climates
Investigators aimed to improve the understanding of the epidemiologic, clinical, and laboratory findings of cold agglutinin disease.
Characterization of NBEAL2 Genetic Variants in Gray Platelet Syndrome
In addition to well-described platelet defects in gray platelet syndrome, immune defects, such as abnormal cells also exist.
Deep Vein Thrombosis Detection in Patients With Hemophilia A Undergoing Total Knee Arthroplasty
A prospective, single-center study aimed to determine the prevalence of DVT after TKA among patients with hemophilia A and found that contrast-enhanced CT led to better detection compared with ultrasound.
Fetal Precautions Can Affect Mode of Delivery in Congenital Bleeding Disorders
In about 10% of pregnancies in which the mother has a congenital bleeding disorder, recommended fetal precautionary measures may influence the mode of delivery.
The Utility of Disease Registries to Assess Inhibitor Development in Hemophilia A
Researchers assessed whether hemophilia A disease registry could be used instead of clinical studies to determine orphan drug safety in pediatric patients.
Importance of Bleeding Phenotype, Diagnostic Trends in Congenital Platelet Defects
There was a median bleeding score of 21 among patients with Glanzmann thrombasthenia, 9 in Bernard-Soulier syndrome, 10 in dense granule deficiency, 7 in ADP pathway defect, 8 in TxA2 pathway defect, 6 in isolated thrombocytopenia, and 8 in complex abnormalities.
Increasing Factor IX Levels May Be Independent of Puberty in Children With Hemophilia B Leyden
Genetic mutations associated with the heterogeneity of hemophilia B Leyden may also be linked to mechanisms that increase factor IX levels with age.
VTE-BLEED Score May Not Accurately Differentiate Patients With VTE for Bleeding Risk
VTE-BLEED prognostic scores did not accurately identify patients with VTE who were at high risk of bleeding events.
von Willebrand Disease: Recent Developments in Diagnosis, Treatments
New developments in lab assays have allowed for more specialized assessments of VWD activity, leading to better diagnoses and various treatment options.
von Willebrand Disease False Levels, Misdiagnosis Still an Issue With Off-Site Testing
The percentage of discordance in onsite and off-site laboratory testing of von Willebrand disease remains high.
Successful Management of Severe COVID-19 in Otherwise Healthy Pediatric Patient
Sustained improvement was noted after the administration of tocilizumab, an interleukin-6 antagonist.
United States Hemophilia Occurrence Rate Higher Than Previously Measured
The exact number of hemophilia cases are unknown and estimates of the size and characteristics of the US hemophilia population are needed for healthcare planning and resource needs assessment.
Genetic Mosaicism and Transmission of Hemophilia
Approximately 30% of patients with hemophilia are considered sporadic cases, causing a challenge when determining carrier status and disease transmission risk to offspring.
Bleeding Risk Assessed With Concomitant Direct Oral Anticoagulants and Verapamil or Diltiazem
Concomitant use of dabigatran with verapamil or diltiazem was found to be associated with an increased risk of bleeding in patients with normal kidney function, according to the findings of a recently published retrospective cohort study.
Characterizing Healthcare Needs, Utilization in Female Patients With Hemophilia A
Identifying hemophilia A in women and girls with factor VIII deficiency with bleeding events using healthcare claims still remains a challenge.
Patterns of Type 2 von Willebrand Disease in Patients in Iran
Researchers assessed 136 patients with VWD in Iran over 13 years to characterize clinical and molecular patterns associated with the disease.
FDA Approves Sevenfact for Hemophilia A or B With Inhibitors
The FDA has approved Sevenfact (coagulation factor VIIa [recombinant]-jncw; LFB Biotechnologies) for the treatment and control of bleeding episodes occurring in patients aged ≥12 years with hemophilia A or B with inhibitors.
Caution Urged for Genetic Screening in Children Suspected of Having Inherited Bleeding Disorders
The authors stated, however, that genetic screening should be used with caution in children.
Age May Affect Factor VIII Levels in Nonsevere Hemophilia A
Neither factor VIII clotting activity nor factor VIII antigens were significantly different for any blood group in patients with nonsevere hemophilia A.
Exposure to Opioids in Pediatric and Adult Patients With Hemophilia
Researchers found that exposure to opioids in patients with hemophilia may be underreported in the American Thrombosis and Hemostasis Network dataset.
Von Willebrand Factor Plasma Levels and Risk for Major Adverse Cardiovascular Events
Patients who experienced major adverse cardiovascular events reported higher von Willebrand factor plasma levels compared with patients in the control cohort.
Health and Psychological Well-Being in Patients With Hemophilia
Patients with hemophilia were found to experience worse general health but higher levels of psychological well-being compared with individuals in the control arm.
Esperoct Now Available for Treatment of Hemophilia A
Novo Nordisk announced the launch of Esperoct® (antihemophilic factor [recombinant], glycopegylated-exei) for the treatment of adult and pediatric patients with hemophilia A for routine prophylaxis.
Coagulation Disorders and Bleeding Risks With Cefoperazone-Sulbactam
Of patients who received cefoperazone-sulbactam, 9.2% reported a coagulation disorder and 15.7% reported a decrease in platelet count.
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.
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.
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