The U.S. Food and Drug Administration (FDA) has approved Alhemo (concizumab-mtci) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A with factor…
In a significant advancement for von Willebrand Disease (VWD) research and treatment, an international team of experts, including patients, clinicians, and researchers, has developed the first-ever core outcome set (COS) for VWD clinical trials. The…
This analysis evaluated relationships between pain limitations and 1) family history of IBD, social determinants, depression/anxiety scores and 2) use of marijuana, opioids and other medications, anxiety, and depression. The majority of CVR…
Gene therapy preferences and informed decision-making: Results from a NHF CVR survey (External Auth)
The aim of this study was to evaluate preferences and variables which influence decision-making related to gene therapy and other novel haemophilia therapies. Haemophilia Treatment Centers and patient advocacy groups must be able to educate persons…
The aim of this analysis was to describe the relationship between self-reported mental health diagnosis, history of mental health treatment, participation in physical activity, chronic pain, and social support. Persons with inheritable bleeding…
Clinicians at US Hemophilia Treatment Centers completed semi-structured interviews about their experience with shared decision-making and provided feedback on a clinician shared decision-making tool prototype. These data highlight the need for…
As gene therapies for hemophilia are incorporated into clinical practice, shared decision-making (SDM) is recommended for implementation. The data in this study highlights the utility of a SDM tool for hemophilia gene therapy and key information…
Glanzmann Thrombasthenia (GT) is a rare inherited platelet disorder caused by a qualitative or quantitative defect of the glycoprotein IIb/IIIa complex. The bleeding phenotype, psychosocial impact, and patient perspectives in GT remain inadequately…
Current pain treatment practices have not adequately addressed pain in people with bleeding disorders. Strong evidence of a relationship between pain attitudes and pain outcomes was presented. No other studies report on pain attitudes in people with…
Disparities in healthcare and health outcomes within minoritized and marginalized populations have been extensively documented in the literature. However, despite growing evidence that race/ethnicity may have a negative impact, few studies in BDs…