Treatments and Clinical Outcomes of Bleeding Related to Pregnancy, Surgery, or Spontaneous or Traumatic Bleeds in Women and Girls With Factor VIII and IX Deficiency: Results From a Retrospective Chart Review
The Need for Comprehensive Care for Persons with Chronic Platelet Disorders
Evaluating BIVV001, a New Class of Factor VIII Replacement Therapy: A Phase 3 Study (XTEND-1) Design
Final Results of PUPs A-LONG Study: Evaluating Safety and Efficacy of rFVIIIFc in Previously Untreated Patients With Hemophilia A
Objective: PUPs A-LONG aimed to evaluate the safety, including inhibitor development, and efficacy of extended half-life (EHL) recombinant factor VIII Fc fusion protein (rFVIIIFc) in previously untreated patients (PUPs) with…
Final Results of PUPs B-LONG Study: Evaluating Safety and Efficacy of rFIXFc in Previously Untreated Patients With Hemophilia B
Objective: PUPs B-LONG aimed to evaluate the safety and efficacy of recombinant factor IX Fc fusion protein (rFIXFc) for prevention and treatment of bleeds in previously untreated patients (PUPs) with hemophilia B. …
Characteristics of persons with hemophilia A treated with emicizumab with or without factor VIII inhibitors
Objective: Emicizumab is indicated for prophylaxis in persons with hemophilia A with or without factor VIII (FVIII) inhibitors to reduce or prevent bleeds. This study provides an early view of individuals treated with emicizumab…
A systematic review of mortality statistics and causes of death in people with congenital hemophilia A (PwcHA)
Objectives: While publications have reported on mortality in PwcHA, a contemporary evidence-based understanding of mortality in congenital hemophilia A (HA) is absent. This systematic review aims to establish a benchmark of…
3 apps in 1: MyCBDR, myWAPPS and myPROBE
Non-severe hemophilia is not benign? - Insights from the PROBE Study
Objective: There are limited data on the impact of haemophilia on health status and health-related quality of life (HRQL) in people affected by non-severe haemophilia. Aim is to evaluate the health status of people living with…
Summary of thrombotic or thrombotic microangiopathy events in persons with hemophilia A taking emicizumab