Bleeding Disorders

Prevalence of Gastrointestinal Bleeding in Patients With von Willebrand Disease

Risk for gastrointestinal bleeding in adult patients with von Willebrand disease (VWD) may be 2.5 times higher compared with adults without VWD, according to results from a retrospective study published in Thrombosis Research. The study included an identification of risk factors. Prevalence of gastrointestinal bleeding was determined by analyzing National Inpatient Sample (NIS) records of…

Inhibitor Development Is Rare After 75 Days of Exposure to Factor VIII in Severe Hemophilia A

By

Sonali Gattani

In previously untreated patients with severe hemophilia A, nearly all inhibitory antibodies (inhibitors) against factor VIII develop within 75 days of exposure, according to research published in Blood. Researchers followed 1038 previously untreated pediatric patients with severe hemophilia A who received treatment with factor VIII. Median age of the cohort at first exposure was 1.1…

Risk for Factor VIII Inhibitor Development Appears Lower With Third-Generation Products

By

Lauren Dembeck, PhD

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

By

Caleb Rans, PharmD

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

By

Vicki Moore, PhD

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

By

Marielle Fares, PharmD, BCGP

Platelet aggregation and platelet activation were both independently associated with bleeding risk.

High-Throughput Sequencing May Improve Diagnosis of Inherited Bleeding Disorders

By

Roxanne Nelson, RN, BSN

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

By

Sonali Gattani

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

By

Caleb Rans, PharmD

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

By

Lauren Dembeck, PhD

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

By

Sonali Gattani

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

By

Vicki Moore, PhD

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

By

Vicki Moore, PhD

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

By

Sonali Gattani

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

By

Caleb Rans, PharmD

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

By

Vicki Moore, PhD

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

By

Lauren Dembeck, PhD

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

By

Vicki Moore, PhD

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

By

Diana Ernst, RPh

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

By

Megan Garlapow, PhD

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

By

Megan Garlapow, PhD

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

By

Lauren Dembeck, PhD

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

By

Lauren Dembeck, PhD

Once-weekly prophylactic injection of glycopegylated recombinant factor VIII (N8‐GP) for severe hemophilia A appears to be efficacious and safe, according to results from the pathfinder 2 extension phase. The study, published in Haemophilia, included patients aged 12 years or older with severe hemophilia A who had experienced up to 2 bleeds during the pathfinder 2…

Safety of Buccal Administration of Dental Anesthetic in Patients With Hemophilia

By

Sonali Gattani

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

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.