Large Study Compares Serum Thrombopoietin Levels in Pediatric Patients With Acquired Aplastic Anemia, Immune Thrombocytopenia
A large, single-center analysis of pediatric patients with either acquired aplastic anemia (aAA) or immune thrombocytopenia (ITP) has compared the serum thrombopoietin (TPO) levels in these ...
Etranacogene Dezaparvovec Bests Extended Half-Life Factor IX Products in Hemophilia B Prophylaxis
For patients with severe or moderately severe hemophilia B, a single dose of etranacogene dezaparvovec gene therapy reduced bleeding rates and eliminated routine infusions associated with ...
Emicizumab Effective as a Hemophilia A Prophylaxis in Infants
The bispecific monoclonal antibody emicizumab was an effective prophylactic treatment for infants with severe hemophilia A without factor VIII (FVIII) inhibitors, according to data from ...
Age Linked to Desmopressin Response in von Willebrand Disease
Response to desmopressin in patients with von Willebrand disease (VWD) and low von Willebrand factor (VWF) level improves as they get older, according to findings presented at ...
Health Disparities Persist in Hemophilia Life Expectancy
Between 1999 and 2020, recorded hemophilia death (rHD) rates in the U.S. improved in males across all racial and ethnic groups ...
Ultrasound May Be Useful to Assess Subclinical Synovial Inflammation in Hemophilia
A study published in Haemophilia highlights the potential utility of ultrasound screenings to assess preclinical joint damage in ...
Valoctocogene Roxaparvovec May Provide Lower Bleeding Rates Than Emicizumab in Severe Hemophilia A
In patients with severe hemophilia A, prophylactic treatment with valoctocogene roxaparvovec gene therapy provided a lower annualized bleeding rate ...
Switching to Different FVIII Product Not Associated with Anti-FVIII Inhibitor Development
Individuals with hemophilia A who switched to a different factor VIII (FVIII) product did not develop anti-FVIII inhibitors nor did their immune profile change ...
Fitusiran Significantly Reduces Bleeding Events, Improves HRQoL in Hemophilia A, B
Once-monthly prophylaxis with the investigational small interference RNA therapeutic fitusiran was associated with a significant reduction in bleeding events when compared with clotting factor concentrate ...
STASEY Study Confirms Safety of Emicizumab Prophylaxis in Hemophilia A With FVIII Inhibitors
The safety profile of emicizumab prophylaxis was confirmed in a large, international population of patients with hemophilia A and factor VIII (FVIII) inhibitors ...
