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.
Treatment strategies for children with hemophilia who have inhibitors should involve eradicating the inhibitor and managing bleeding.
Only factor VIII trough level and prophylactic treatment showed associations with joint health in univariate analysis.
Platelet aggregation and platelet activation were both independently associated with bleeding risk.
Currently, molecular testing occurs at the tail end of the diagnostic workflow for inherited bleeding disorders.
For both type 1 and type 2 von Willebrand disease, higher BMI was associated with increased VWF:Ag and FVIII:C levels.
Variation in disease phenotype and clinical circumstances makes it difficult to create a standardized treatment approach to von Willebrand disease.
Patients’ responses to surveys assessing quality of life indicated impairment in both adults and adolescents.
A bleeding assessment tool yielded higher scores for patients with suspected or confirmed platelet function disorders.
Researchers analyzed the clinical features of factor XIII deficiency in neonates and used the data to develop a diagnostic algorithm.
For all anticoagulants examined, patients with cancer had higher bleeding incidence rates than patients without cancer.
There was no difference in mortality rate between patients taking various direct-acting oral anticoagulants (DOACs), though bleeding locations varied for each DOAC.
High shear stress in blood vessels due to cardiovascular disease may be associated with the development of acquired von Willebrand syndrome.
Assay values were not strongly correlated with von Willebrand factor ristocetin cofactor or antigen activity.
Because most studies on heavy menstrual bleeding have been conducted in adults, treatment guidelines for adolescents are lacking.
Patients with cyanotic congenital heart diseases were found to have higher levels of hematocrit, hemoglobin, and reticulocytes.
The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to PB2452 (PhaseBio), an investigational reversal agent for ticagrelor.
Researchers identified 11 novel mutations that may play a pathogenic role in hereditary factor X deficiency.
Neonates with hemophilia who were delivered either vaginally or by Cesarean section had similar rates of intracranial bleeding.
Researchers reviewed 33 studies published over a span of 20 years to identify risk factors and symptoms of intracranial hemorrhage in patients with hemophilia.
Weekly prophylaxis with glycopegylated recombinant factor VIII was well tolerated and had a good safety profile for low-bleeding patients with hemophilia A.
Current guidelines do not adequately inform management of patients with inherited bleeding disorders undergoing routine dental procedures.
Simplifying factor VIII reconstitution may increase treatment adherence and satisfaction in patients with hemophilia A.
Unnecessarily high platelet count thresholds may expose patients undergoing lumbar or epidural procedures to excessive transfusion-related risks.
Gene therapy with adeno-associated viral viruses could provide an efficacious, safe, and cost-effective treatment option for patients with hemophilia.
Researchers conducted a retrospective chart review to assess the bleeding risk associated with gastrointestinal endoscopy.
Researchers assessed potential risk factors for major bleeding in patients without cardiovascular disease to develop sex-specific prediction models.
Researchers assessed whether prophylactic treatment with von Willebrand factor/factor VIII concentrates reduced bleeding risk.
Population-based screening of newborns is crucial to identifying severe combined immunodeficiency before the onset of potentially fatal infection.
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.
Underlying lymphoproliferative syndromes, male gender, and older age may be predictive of relapse in patients with acquired hemophilia A.
Researchers assessed safety outcomes in patients with hemophilia A without inhibitors.
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.
Researchers assessed the influence of adopting population pharmacokinetics to inform prophylaxis for patients with hemophilia A.
Acute major bleeding decreased by 92% in patients who were treated with andexanet alfa.
Using rotational thromboelastometry may allow assessment of bleeding risks in patients with clinically relevant von Willebrand disease.
Researchers conducted the first real world study to compare the safety and effectiveness of apixaban and rivaroxaban in patients with VTE.
Changes in clinical management of trauma hemorrhage have significantly reduced mortality rates, but variation in treatment strategies still remains.
The need for tranexamic acid to be administered intravenously creates a barrier to rapid treatment of patients with fibrinolysis.
Many unique challenges surround the diagnosis and treatment of inherited mild bleeding disorders.
The interactions between von Willebrand factors and its cleaving protease ADAMTS-13 recently emerged as risk factors for pediatric thromboembolism.
Propensity for bleeding was previously thought to relate to greater fibrinolytic activity, but this may not be the case.