6 results match your criteria: "Dills Family Foundation Center for Research at BCDI[Affiliation]"
The Roadmap to Integrate Diversity, Equity, and Inclusion in Hematology Clinical Trials: An American Society of Hematology Initiative.
Blood Adv
December 2024
Novo Nordisk A/S, Soeborg, Denmark.
Clinical trial design for classical hematologic diseases is difficult because samples sizes are often small and not representative of the disease population. ASH initiated a Roadmap project to identify barriers and make progress to integrate diversity, equity, and inclusion into trial design and conduct. Focus groups of international experts from across the clinical trial ecosystem were conducted.
View Article and Find Full Text PDFImproved prevention of bleeding episodes with emicizumab in 3 patients with concomitant hemophilia A and von Willebrand disease.
Blood Coagul Fibrinolysis
October 2024
Bleeding & Clotting Disorders Institute, Dills Family Foundation Center for Research at BCDI.
Article Synopsis
- Hemophilia A (HA) is characterized by frequent bleeding episodes, often requiring prophylactic factor VIII (FVIII) replacement to manage, especially when combined with von Willebrand disease (VWD), which is present in around 1% of the population.
- Co-existing HA and mild VWD can complicate effective treatment, and while traditional FVIII/VWF concentrates are used, some patients struggle with adherence due to the intravenous administration method.
- A new alternative, emicizumab, a non-factor subcutaneous therapy, has shown promise in reducing bleeding rates for patients with severe HA and VWD when traditional prophylaxis was insufficient, suggesting it could be a valuable option for better management.
Hereditary antithrombin deficiency pilot project registry from the American Thrombosis and Hemostasis Network.
J Thromb Haemost
November 2024
Department of Pediatric Hematology & Oncology, Yale University School of Medicine, New Haven, Connecticut, USA; National Bleeding Disorders Foundation, New York, New York, USA.
Background: Patients with hereditary antithrombin deficiency (HAD) have an increased risk of venous thromboembolism (VTE). The American Thrombosis and Hemostasis Network (ATHN) 12: HAD Pilot Project established a registry to collect data on patients with HAD.
Objectives: To inform current practice and serve as a platform to design a multicenter global registry for patients with HAD.
Costs and impact of disease in adults with sickle cell disease: a pilot study.
Blood Adv
July 2024
Leonard D. Schaeffer Center for Health Policy & Economics, University of Southern California, Los Angeles, CA.
We assessed the feasibility to estimate illness burden in adults with SCD, investigated factors associated with health-related quality of life (HRQoL), and estimated societal burden. We recruited 32 participants and collected data on fatigue, HRQoL, and work productivity and activity impairment via patient survey. Health care utilization was abstracted for the 12 months before enrollment using medical chart review.
View Article and Find Full Text PDFPractical challenges associated with efanesoctocog alfa (ALTUVIIIO) prophylaxis in a 19-month-old male with severe hemophilia A.
Pediatr Blood Cancer
August 2024
Bleeding & Clotting Disorders Institute, Dills Family Foundation Center for Research at BCDI, Peoria, Illinois, USA.
Retrospective chart review of GI bleeding in people with von Willebrand disease.
Haemophilia
July 2024
Department of Medicine, Mary M. Gooley Hemophilia Center, Rochester, New York, USA.
Introduction: Gastrointestinal (GI) bleeding events (BEs) in von Willebrand disease (VWD) are difficult to diagnose and often recurrent. Limited data from clinical trials has led to lack of consensus on treatment options.
Aim: Describe current treatments and outcomes for GI BEs in people with VWD.