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.
The percentage of discordance in onsite and off-site laboratory testing of von Willebrand disease remains high.
Sustained improvement was noted after the administration of tocilizumab, an interleukin-6 antagonist.
Approximately 30% of patients with hemophilia are considered sporadic cases, causing a challenge when determining carrier status and disease transmission risk to offspring.
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.
Identifying hemophilia A in women and girls with factor VIII deficiency with bleeding events using healthcare claims still remains a challenge.
Researchers assessed 136 patients with VWD in Iran over 13 years to characterize clinical and molecular patterns associated with the disease.
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.
The authors stated, however, that genetic screening should be used with caution in children.
Neither factor VIII clotting activity nor factor VIII antigens were significantly different for any blood group in patients with nonsevere hemophilia A.
Researchers found that exposure to opioids in patients with hemophilia may be underreported in the American Thrombosis and Hemostasis Network dataset.
Patients who experienced major adverse cardiovascular events reported higher von Willebrand factor plasma levels compared with patients in the control cohort.
Patients with hemophilia were found to experience worse general health but higher levels of psychological well-being compared with individuals in the control arm.
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.
Of patients who received cefoperazone-sulbactam, 9.2% reported a coagulation disorder and 15.7% reported a decrease in platelet count.
Inhibitor incidence was found to be 10.2% in previously untreated patients with severe hemophilia B after 500 exposure days.
The most common bleeding disorder in this cohort was von Willebrand disease, which comprised 62.9% of diagnoses.
Researchers conducted a systematic review to assess outcomes and complications in pregnant women with type 3 von Willebrand disease.
Researchers evaluated the diagnostic utility of the ThromboGenomics high-throughput sequencing test for bleeding, thrombotic, and platelet disorders.
Patients with hemophilia had lower median score on the Offer Self-Image Questionnaire compared with a control group.
The majority of patients admitted to the hospital after seeking emergency care for heavy menstrual bleeding did not have an underlying bleeding disorder.
The US FDA has received a BLA for valoctocogene roxaparvovec (BMN 270) for the treatment of hemophilia A in adults.
Although gene therapy shows promise in curing hemophilia, questions about its efficacy, optimal administration, and outcomes remain.
C-reactive protein and urine albumin/creatinine ratio were associated with increased risk of bleeding in patients with thrombocytopenia due to hematologic malignancy.
Patients with hemophilia A reported a median improvement of 2.25 points in hemophilia joint health score during the course of the study.
Risk for postpartum hemorrhage after cesarean delivery was nearly 2-fold greater in patients with mild factor XI deficiency compared with the control cohort.
Researchers evaluated whether simoctocog alfa, a fourth-generation recombinant factor VIII product, is safe and effective in patients with severe hemophilia A.
Median age at inhibitor development was 13 years, and the inhibitor-related mortality rate was 2.44 per 1000 person-years.
Of 69 patients referred for hemostatic examination due to severe postpartum hemorrhage, 16 (23%) were diagnosed with a mild bleeding disorder.
Researchers calculated the optimal values for parameters of the ROTEM sigma-based algorithm for treating coagulopathic bleeding.
Antiplatelet therapy is used to manage cardiovascular events but can lead to increased risk for bleeding events both mild and severe.
Researchers found that many patients have limited knowledge concerning potential serious interactions between dietary supplements and apixaban.
Transfusing activated platelets can increase the number of transfusions required and thus lead to additional costs compared with transfusing resting platelets.
Patients who were transfused at a lower platelet count threshold experienced a 7% decrease in absolute risk for death or major bleeding.
Researchers assessed whether high platelet counts in pediatric patients with chronic myeloid leukemia resulted in thrombosis or bleeding.
Male patients with von Willebrand disease were more likely to be diagnosed earlier and to report bleeds compared with female patients.
Patients with anovulatory bleeding experienced a significantly longer time to bleeding disorder diagnosis compared with patients with ovulatory bleeding.
The FDA has approved the supplemental Biologics License Application for Nplate (romiplostim; Amgen) to expand treatment to newly diagnosed adult patients with immune thrombocytopenia.
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…
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.
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%.
Patients treated with bypassing therapy experienced significantly higher medical and outpatient pharmacy costs.
Researchers have found that the estimated number of men with hemophilia is higher than known diagnoses, prompting a call for improved diagnosis approaches.
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…
Researchers conducted a meta-analysis of 4 studies examining prophylaxis with factor VIII or emicizumab for hemophilia A without inhibitors.
Self-reported adherence to prophylaxis did not correlate with objective measures of treatment adherence.
Researchers highlighted the current status of diagnostic strategies and laboratory screening tests for von Willebrand disease.
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.
Researchers conducted a retrospective analysis of records from the National Inpatient Sample to identify risk factors for gastrointestinal bleeding.
In patients with hemophilia A who developed inhibitors, 99.3% of inhibitors developed within 75 days of exposure to factor VIII.
Researchers evaluated whether certain brands of recombinant Factor VIII products were associated with increased risk of developing inhibitors.