A5040182946- Hemophilia Treatment and Research
- Blood Coagulation and Thrombosis Mechanisms
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Venous Thromboembolism Diagnosis and Management
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Chronic Myeloid Leukemia Treatments
- Platelet Disorders and Treatments
- Cancer-related gene regulation
- Adolescent and Pediatric Healthcare
- Childhood Cancer Survivors' Quality of Life
- Orthopaedic implants and arthroplasty
- Case Reports on Hematomas
- Lymphoma Diagnosis and Treatment
- Hemostasis and retained surgical items
- Palliative Care and End-of-Life Issues
- Health Systems, Economic Evaluations, Quality of Life
- Hearing, Cochlea, Tinnitus, Genetics
- Acute Lymphoblastic Leukemia research
- Healthcare cost, quality, practices
- Trauma and Emergency Care Studies
- Total Knee Arthroplasty Outcomes
- Autopsy Techniques and Outcomes
- Diagnosis and Treatment of Venous Diseases
- Bone and Joint Diseases
- Viral-associated cancers and disorders
University of Colorado Denver
2015-2024
University of Colorado Cancer Center
2024
BioMarin (United States)
2023
University of Colorado Anschutz Medical Campus
2017-2023
University of Colorado Health
2021
Children's Hospital Colorado
2017-2019
University of North Carolina at Chapel Hill
2006-2015
Palmetto Hematology Oncology
2014
St. Jude Children's Research Hospital
1999
Creighton University
1999
The NCCN Guidelines for Cancer-Associated Venous Thromboembolic Disease provide strategies the prevention, diagnosis, and treatment of venous thromboembolism (VTE) in adult patients with cancer. VTE is a common life-threatening condition cancer, its management often requires multidisciplinary efforts. panel comprised specialists spanning various fields, including cardiology, hematology, medical oncology, internal medicine, interventional radiology, pharmacology. content featured this issue...
Haemophilia is characterized by frequent haemarthrosis, leading to acute/chronic joint pain.To assess self-reported prevalence, description and management of pain in adult males with mild-to-severe haemophilia history pain/bleeding.Participants completed a survey five patient-reported outcome instruments assessing pain, functional impairment health-related quality life (HRQoL).Of 381 participants enrolled, median age was 34 years; 77% had A, 71% severe disease 65% were overweight/obese. Many...
Background Children with cancer experience multiple symptoms due to their disease and as a result of treatment. The purpose this study was demonstrate the feasibility potential utility using latent profile analysis (LPA), type cluster analysis, in children identify groups patients who similar levels symptom severity impairment physical function. Procedure We analyzed patient‐reported functional data previously collected Pediatric Patient Reported Outcomes Measurement Information System...
Estimates of the size and characteristics US haemophilia population are needed for healthcare planning resource needs assessment. A network comprehensive treatment centres (HTCs) located throughout United States receives federal support diagnosis management other rare bleeding disorders.Estimate incidence prevalence among males using HTC network.During period 2012-2018, de-identified surveillance data were collected on all who visited an that included year birth, gender, race, Hispanic...
Abstract Introduction People with inherited and long‐term conditions such as haemophilia have been shown to adapt their levels of disability, often reporting better quality life (QoL) than expected from the general population (the disability paradox). Aim To investigate paradox in people United States by examining preference differences health state valuations versus population. Methods We conducted a discrete choice experiment including duration capture states based on patient‐reported...
Background The National Hemophilia Foundation (NHF) conducted extensive all-stakeholder inherited bleeding disorder (BD) community consultations to inform a blueprint for future research. Sustaining and expanding the specialized comprehensive Treatment Center care model, better serve all people with BDs (PWIBD), increasing equitable access optimal health emerged as top priorities.Research Design Methods NHF, American Thrombosis Hemostasis Network (ATHN), convened multidisciplinary expert...
Abstract Introduction Pain, functional impairment, anxiety, and depression associated with joint disease may affect health‐related quality of life (HRQoL) in people hemophilia. Objective To report detailed patient‐reported outcomes (PRO) assessments related to HRQoL participants the Functional Impairment, Quality Life (P‐FiQ) study. Methods Pain were assessed via PRO instruments US adult males hemophilia A or B a history pain bleeding. included EQ‐5D‐5L visual analog scale, Brief Inventory...
Abstract Introduction For people with haemophilia B (PwHB), bleeding may occur despite prophylaxis, negatively affecting health‐related quality of life (HRQoL). The pivotal phase 3 HOPE‐B trial investigating the adeno‐associated virus gene transfer product, etranacogene dezaparvovec (EDZ), demonstrated sustained factor IX (FIX) activity and bleed protection in PwHB baseline FIX levels ≤2%. Aim Assess how EDZ affects HRQoL participants. Methods was evaluated using generic disease‐specific...
Plain Language SummaryWhat is the study about?Hemophilia A a genetic bleeding disorder that causes for long periods because of lower than normal levels factor VIII (FVIII), protein important blood clotting. FVIII replacement therapy typical approach to manage bleeds, but new and improved therapies are becoming widely available. Prophylaxis with medication emicizumab gene valoctocogene roxaparvovec two such novel treatments approved use in adults severe hemophilia without inhibitors.Two...
The α9 acetylcholine receptor (α9 AChR) is specifically expressed in hair cells of the inner ear and believed to be involved synaptic transmission between efferent nerves cells. Using a recently developed method, we modified bacterial artificial chromosome containing mouse AChR gene with reporter encoding green fluorescent protein (GFP) generate transgenic mice. GFP expression mice recapitulated known temporal spatial AChR. However, observed previously unidentified dynamic changes cochlear...
Standardized and disease-specific patient-reported outcome (PRO) instruments assessing pain, functional impairment health-related quality of life (HRQoL) in people with haemophilia (PWH) have been used studies, but infrequently comprehensive care settings for individual assessment or treatment planning.To assess the impact pain on HRQoL PWH.P-FiQ enrolled 381 adult PWH a history joint pain/bleeding included 5 PROs clinical evaluation (Hemophilia Joint Health Score v2.1 [HJHS]).Median age was...
Abstract Introduction Females may have haemophilia with the same factor VIII (FVIII) or IX (FIX) levels as affected males. Characterization of females would be useful for health care planning to meet their unique needs. Federally‐funded treatment centres (HTCs) in United States contribute data on all individuals bleeding disorders receiving Population Profile (HTC PP) component Community Counts Public Health Surveillance Bleeding Disorders project. Aims To estimate number at HTCs and compare...
Emicizumab, a bispecific antibody factor VIII mimetic, is approved for prophylaxis in hemophilia, and has different risks side effects compared to products.
Health-related quality of life (HRQoL) is impaired in patients with hemophilia; however, the impact mild/moderate hemophilia B and affected women not well characterized.To evaluate factors that affect HRQoL adults caregivers children.US adult children completed distinct ~1-hour online surveys including patient-reported outcome instruments.In total, 299 150 participated. Adults moderate reported poorer health status (median EQ-5D-5L index score, 0.63) than those mild (0.73) or severe (0.74)...
Summary Perioperative clotting factor replacement is administered to reverse the inherent haemostatic defect in persons with haemophilia (PWH), potentially increasing their risk for developing venous thromboembolism (VTE) postoperatively. It was our objective determine prevalence of VTE PWH undergoing total hip or knee arthroplasty (THA, TKA). Patients A B who underwent THA TKA were enrolled this prospective, multicentre observational cohort study. Lower extremity duplex ultrasound performed...
Hemophilia B is a rare congenital blood disorder characterized by factor IX deficiency. Clinical profiles of hemophilia range from mild to severe forms the disease. The objective this study was characterize economic burden associated with differing clinical US health system perspective. Using IBM MarketScan database (June 2011-February 2019), claims-based algorithm developed identify 4 distinct (mild, moderate, moderate-severe, and severe) in adult males based on frequency hemorrhage events...
Gene therapy has shown promise in clinical trials for patients with haemophilia, but patient preference studies have focused on factor replacement treatments.We conducted a discrete choice experiment (DCE) to investigate the relative importance and differential preferences provide gene attributes.We surveyed male adults haemophilia United States recruited from panels including National Hemophilia Foundation Community Voices Research platform using an online survey over 4 months 2020/21....
Abstract Introduction Levels of pain and dysfunction appear to differ among people with hemophilia despite similar levels joint disease. Objective To determine patient characteristics that influence function independent status. Methods US adults completed a survey included information on clinical characteristics, demographics, patient‐reported outcome instruments assessing (Brief Pain Inventory v2 Short Form [ BPI ]), functional impairment (Hemophilia Activities List HAL health status ( EQ...
The Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B-HERO-S) initiative was launched in an effort to address specific gaps the understanding of psychosocial impact mild-moderate-severe hemophilia B. original (HERO) qualitative study evaluated needs people with A or multiple countries; however, a majority participants had more common moderate-severe A. B-HERO-S designed collaboration community evaluate adults caregivers children B, including affected women girls...
Background: Hemophilia is marked by frequent joint bleeding, resulting in pain and functional impairment. Objective: This study aimed to assess the reliability of five patient-reported outcome (PRO) instruments people with hemophilia (PWH) a non-bleeding state. Methods: Adult male PWH any severity inhibitor status, history or completed PRO (EQ-5D-5L, Brief Pain Inventory v2 [BPI], International Physical Activity Questionnaire [IPAQ], Short Form 36 Health Survey [SF-36v2], Activities List...