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.
Bleeding Disorders
BLEEDING DISORDERS
LATEST ARTICLES
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.
Using Diluent Buffer May Lead to Overestimation of Low Factor VIII Levels
Accurate measurement of low factor VIII levels is necessary for proper monitoring of factor VIII replacement therapy.
Clot Waveform Analysis: A Novel Method to Classify Fibrinogen Disorders
Clot waveform analysis of the Clauss fibrinogen assay may allow classification of fibrinogen disorders without measuring fibrinogen antigen levels.
FDA Approval Expands Access to Factor Xa Inhibitor Antidote Andexxa
Andexxa was initially granted accelerated approval in May 2018 for patients treated with rivaroxaban or apixaban, when reversal of anticoagulation was needed due to life-threatening or uncontrolled bleeding.
FDA Approves Updated Labeling for Hemophilia A Treatment Novoeight
The indication has been changed from “control and prevention of bleeding episodes” to “on-demand treatment and control of bleeding episodes” in adults and children with hemophilia A.
Amicar Tablet Generic Gets FDA Approval
Aminocaprioc Acid Tablets are useful in enhancing hemostasis when fibrolysis contributes to bleeding.
Advances in Treatment and Prevention of Hemophilia A: Takeaways From the 2018 ASH Annual Meeting
Research presented at the 2018 ASH Annual Meeting provided insights into the role of factor VIII as a therapeutic agent for severe hemophilia A.
Andexanet Alfa and the Role of Reversal Agents for Factor Xa Inhibitors
Andexanet alfa is the first anticoagulant reversal agent to receive global US FDA approval for patients with life-threatening or uncontrolled bleeding.
Should Infants Be Treated With von Willebrand Factor Concentrate After Complex Cardiac Surgery?
Researchers comment on the implications of treating newborns and infants with von Willebrand factor (VWF) concentrate.
Clinical Diagnosis of Factor V Inhibitors: A Single Institution’s Experience
A review of the management and outcomes for patients with autoimmune Factor V (FV) inhibitors.
Factor VIII, Factor IX Levels May Have Predictive Value in Identifying Severity of Hemophilia
Residual plasma levels of clotting factor XIII or factor IX accounted for around 70% of bleeding phenotype in patients with clinically severe hemophilia.
FDA Approves Expanded Indication for Hemlibra in Hemophilia A
The expanded approval was based on data from two Phase 3 clinical trials: HAVEN 3 and HAVEN 4.
Jivi With Step-Wise Dosing Approved for Management of Hemophilia A
The approval was supported by data from the phase 2/3 international, open-label PROTECT VIII trial (N=126) that evaluated previously treated patients aged ≥12 years with severe hemophilia A.
Want to read more?
Please login or register first to view this content.
Next post in Bleeding Disorders
