Management of Bleed Events in the Phase 2 Study of Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia A and B with and Without Inhibitors
Background: Hemophilia is an inherited bleeding disorder caused by an impairment in the body’s ability to accomplish the natural clotting process. People with hemophilia experience bleeds because there is an inadequate amount of…
Centralized Inhibitor Testing in the United States: Laboratory Methods Used for the Community Counts Registry for Bleeding Disorders Surveillance
Assessing the Availability and Use of Resources to Support Youth with Hemophilia, their Families and Care Teams during Transition to Adult Health Care
CHESS – Improving research and advocacy through an improved understanding of the economic and social burden of hemophilia
Real-World Pharmacy Dispensation and Expenditures Associated with Standard and Extended Half-Life Recombinant Factor VIII Products in Hemophilia A
Objectives: Contemporary real-world data on units dispensed and expenditures associated with use of standard half-life (SHL) and extended half-life (EHL) factor VIII replacement products in U.S. patients with hemophilia A are…
Physician practice patterns in the US show significant variation in how PK parameters are currently used to personalize care for US hemophilia A patients
Introduction & Objective: Standard approaches to prophylaxis may be further improved by taking into account individuals’ pharmacokinetic (PK) profile, thereby increasing the likelihood of therapeutic success [Valentino 2012,…
Sports/Recreational Activity-Specific Range and Drivers of Risk in People With Hemophilia: Results of the Activity-Intensity-Risk (AIR) Survey and Consensus Meeting of US Physical Therapists
Objective: Limited evidence supports activity-associated bleeding risk assessment for people with hemophilia (PWH), and consumer materials generically describe types of risk and a single activity risk score based on input from a…
Real-World Specialty Pharmacy Dispensation and Expenditures Associated with Prophylactic Regimens Using Standard and Extended Half-Life Recombinant Factor IX Products in Severe Hemophilia B
Impact of targeted education on obesity in children with hemophilia-a single HTC quality project
Mild-Severe Hemophilia B Impacts Relationships for US Adults and Children With Hemophilia B and Their Families: Results From the Bridging Hemophilia B Experiences, Results, and Opportunities Into Solutions (B-HERO-S) Study
Objective: The B-HERO-S study evaluated the impact of mild-severe hemophilia B on the lives of affected adults and children. Here we assess the impact of hemophilia B on relationships. Methods: US adults…