Bleeding Disorders

New Tool for Reconstituting Factor VIII for Hemophilia A Prophylaxis

By

Sonali Gattani

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

By

Vicki Moore, PhD

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

By

Caleb Rans, PharmD

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

By

Megan Garlapow, PhD

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

By

Sonali Gattani

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

By

Sonali Gattani

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

By

Caleb Rans, PharmD

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

By

Da Hee Han, PharmD

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

By

John Schieszer, MA

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

By

Sonali Gattani

Researchers assessed safety outcomes in patients with hemophilia A without inhibitors.

Assessing Children With Bleeding Symptoms: Coagulation Disorder or Abuse?

By

Lauren Dembeck, PhD

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

By

Lauren Dembeck, PhD

Researchers assessed the influence of adopting population pharmacokinetics to inform prophylaxis for patients with hemophilia A.

Andexanet Alfa Efficacious in Reducing Antifactor Xa Activity

By

Sonali Gattani

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

By

Megan Garlapow, PhD

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

By

Sonali Gattani

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

By

Lauren Dembeck, PhD

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

By

John Schieszer, MA

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

By

Caleb Rans, PharmD

Many unique challenges surround the diagnosis and treatment of inherited mild bleeding disorders.

Researchers Review Coagulation Risk Factors in Neonates and Children

By

Megan Garlapow, PhD

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

By

Megan Garlapow, PhD

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

By

Megan Garlapow, PhD

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

By

Sonali Gattani

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

By

Da Hee Han, PharmD

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

By

Diana Ernst, RPh

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

By

Diana Ernst, RPh

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

By

Bryant Furlow

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

By

Caleb Rans, PharmD

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?

By

Roxanne Nelson, RN, BSN

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

By

Lauren Dembeck, PhD

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

By

Megan Garlapow, PhD

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

By

Diana Ernst, RPh

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

By

Da Hee Han, PharmD

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.