Bleeding Disorders

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.

von Willebrand Disease False Levels, Misdiagnosis Still an Issue With Off-Site Testing

By

Rita Aghjayan

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

By

Cassandra Pardini, PharmD

Sustained improvement was noted after the administration of tocilizumab, an interleukin-6 antagonist.

Genetic Mosaicism and Transmission of Hemophilia

By

Roxanne Nelson, RN, BSN

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

By

Cassandra Pardini, PharmD

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

By

Vicki Moore, PhD

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

By

Vicki Moore, PhD

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

By

Brian Park, PharmD

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

By

John Schieszer

The authors stated, however, that genetic screening should be used with caution in children.

Age May Affect Factor VIII Levels in Nonsevere Hemophilia A

By

Lauren Dembeck, PhD

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

By

Sonali Gattani

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

By

Vicki Moore, PhD

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

By

Jonathan Goodman, MPhil

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

By

Brian Park, PharmD

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

By

Vicki Moore, PhD

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

By

Vicki Moore, PhD

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

By

Vicki Moore, PhD

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

By

Megan Garlapow, PhD

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?

By

Lauren Dembeck, PhD

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

By

Lauren Dembeck, PhD

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

By

Jonathan Goodman, MPhil

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

By

Brian Park, PharmD

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

By

Tori Rodriguez, MA, LPC

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

By

Megan Garlapow, PhD

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

By

Sonali Gattani

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

By

Sonali Gattani

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

By

Jonathan Goodman, MPhil

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

By

Jonathan Goodman, MPhil

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

By

Vicki Moore, PhD

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

By

Lauren Dembeck, PhD

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

By

Roxanne Nelson, RN, BSN

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

By

John Schieszer, MA

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

By

Bill Dubiel, CEO, LightIntegra

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

By

Megan Garlapow, PhD

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

By

Megan Garlapow, PhD

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

By

Jonathan Goodman, MPhil

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

By

Lauren Dembeck, PhD

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

By

Diana Ernst, RPh

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

By

Brian Park, PharmD

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

By

Caleb Rans, PharmD

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

By

Megan Garlapow, PhD

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

By

Vicki Moore, PhD

Patients treated with bypassing therapy experienced significantly higher medical and outpatient pharmacy costs.

Hemophilia Prevalence Globally and at Birth in Men

By

Sonali Gattani

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

By

Brian Park, PharmD

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

By

Vicki Moore, PhD

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

By

Lauren Dembeck, PhD

Self-reported adherence to prophylaxis did not correlate with objective measures of treatment adherence.

Challenges in Diagnosing and Managing von Willebrand Disease

By

Marielle Fares, PharmD

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

By

Caleb Rans, PharmD

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

By

Vicki Moore, PhD

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

By

Sonali Gattani

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

By

Lauren Dembeck, PhD

Researchers evaluated whether certain brands of recombinant Factor VIII products were associated with increased risk of developing inhibitors.