52 results match your criteria: "Hemophilia and Thrombosis Treatment Center[Affiliation]"
Development of the World Federation of Hemophilia Shared Decision-Making Tool.
Haemophilia
November 2024
The World Federation of Hemophilia, Montreal, Quebec, Canada.
Introduction: The use of shared decision-making (SDM) in clinical settings is becoming more prevalent. The evolving and increasingly complex treatment landscape of haemophilia management has augmented the need and desire for SDM between patients and their healthcare team. SDM tools have been used in other chronic conditions and can be an effective form of education for patients and clinicians.
View Article and Find Full Text PDFHow clinicians and persons with hemophilia may approach shared decision-making.
Expert Rev Hematol
June 2024
World Federation of Hemophilia, Montréal, Québec, Canada.
International Prophylaxis Study Group (IPSG) haemophilia joint MRI scale version 2.0.
Haemophilia
May 2024
Department of Diagnostic Imaging, Department of Medical Imaging, Research Institute, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
The benefits of gene therapy in people with haemophilia.
J Viral Hepat
April 2024
Hemophilia and Thrombosis Treatment Center, Rady Children's Hospital San Diego, San Diego, California, USA.
Haemophilia is an inherited bleeding disorder which causes significant morbidity and mortality, especially in the severe form. Prophylaxis with factor replacement has high efficacy in reducing bleeding but is limited by the need for frequent intravenous infusion and fluctuations in haemostasis between doses. Additional prophylaxis therapies are being developed which may overcome some of the current treatment barriers.
View Article and Find Full Text PDFGene Therapy in Hemophilia A: Achievements, Challenges, and Perspectives.
Semin Thromb Hemost
February 2025
Rady Children's Hospital San Diego, Hemophilia and Thrombosis Treatment Center, San Diego, California.
Strides in advancements of care of persons with hemophilia include development of long-acting factor replacement therapies, novel substitution and hemostatic rebalancing agents, and most recently approved gene therapy. Several decades of preclinical and clinical trials have led to development of adeno-associated viral (AAV) vector-mediated gene transfer for endogenous production of factor VIII (FVIII) in hemophilia A (HA). Only one gene therapy product for HA (valoctocogene roxaparvovec) has been approved by regulatory authorities.
View Article and Find Full Text PDFCorrection: Huang et al. Systemic Anticoagulation and Inpatient Outcomes of Pancreatic Cancer: Real-World Evidence from U.S. Nationwide Inpatient Sample. 2023, , 1985.
Cancers (Basel)
March 2024
Institute of Medicine, Chung Shan Medical University, Taichung 402, Taiwan.
In the original publication [...
View Article and Find Full Text PDFEffects of SGLT2 inhibitors on clinical cancer survival in patients with type 2 diabetes.
Diabetes Metab
January 2024
Graduate Institute of Business Administration, College of Management, Fu Jen Catholic University, Taipei, Taiwan; Artificial Intelligence Development Center, Fu Jen Catholic University, Taipei, Taiwan; Big Data Center, Lo-Hsu Medical Foundation, Lotung Poh-Ai Hospital, Yilan, Taiwan; Department of Food Nutrition and Health Biotechnology, College of Medical and Health Science, Asia University, Taichung, Taiwan; Division of Radiation Oncology, Lo-Hsu Medical Foundation, Lotung Poh-Ai Hospital, Yilan, Taiwan; Department of Healthcare Administration, College of Medical and Health Science, Asia University, Taichung, Taiwan; Centers for Regional Anesthesia and Pain Medicine, Wan Fang Hospital, Taipei Medical University, Taipei, Taiwan; Department of Management, College of Management, Fo Guang University, Yilan, Taiwan. Electronic address:
Purpose: According to the preclinical data, sodium-glucose cotransporter 2 (SGLT2) inhibitors (SGLT2is) may exert anticancer effects. Here, we clarified the cancer-specific mortality (primary outcome) and all-cause mortality (secondary outcome) of SGLT2is and their dose-dependency in patients with cancer undergoing standard curative treatments.
Methods: We analyzed data from patients with type 2 diabetes mellitus (T2DM) diagnosed with cancer between January 1, 2016, and December 31, 2018, enrolled from the Taiwan Cancer Registry database.
Efficacy, safety and cost of emicizumab prophylaxis in haemophilia A patients with inhibitors: A nationwide observational study in Taiwan.
Haemophilia
November 2023
Center for Rare Disease and Hemophilia, Department of Pediatrics, Taichung Veterans General Hospital, Taichung, Taiwan.
Introduction: Emicizumab mimicking the cofactor function of activated factor VIII (FVIII) restores haemostasis.
Methods: This nationwide observational study aimed to retrospectively investigate efficacy, safety, and cost in 1 year before and up to 3 years after emicizumab prophylaxis for haemophilia A (HA) patients with FVIII inhibitors.
Results And Discussion: A total of 39 severe HA patients with a median age of 23.
Evaluation of collagen turnover biomarkers as an objective measure for efficacy of treatment with rurioctocog alfa pegol in patients with hemophilia A: a secondary analysis of a randomized controlled trial.
J Thromb Haemost
January 2024
Nordic Bioscience, Copenhagen, Denmark.
Background: Patients with hemophilia who have recurrent hemarthroses develop hemophilic arthropathy (HA). Regular prophylaxis with factor (F) VIII (FVIII) can reduce HA, but there is a need for objective outcome measures to evaluate treatment efficacy.
Objectives: Evaluate and assess collagen turnover biomarkers in patients with hemophilia A to determine the efficacy of rurioctocog alfa pegol treatment and understand their potential as tools for guiding treatment decisions and monitoring outcomes.
Evaluating Gene Therapy as a Potential Paradigm Shift in Treating Severe Hemophilia.
BioDrugs
September 2023
Division of Hematology/Oncology, Department of Medicine, University of California, San Diego, La Jolla, CA, USA.
Hemophilia is characterized by a deficiency in coagulation factors VIII or IX. The general standard of care for severe hemophilia is frequent intravenous recombinant or plasma-derived factor replacement to prevent bleeding. While this treatment is effective in preventing bleeding, frequent infusions are burdensome for patients.
View Article and Find Full Text PDFDevelopment of a haemophilia A gene therapy shared decision-making tool for clinicians.
Haemophilia
September 2023
Hemophilia and Thrombosis Treatment Center, Rady Children's Hospital San Diego, San Diego, California, USA.
Introduction: As gene therapies are incorporated into clinical practice, shared decision-making (SDM) is recommended for implementation.
Aim: To inform development of a clinician SDM tool for haemophilia A gene therapy.
Methods: Clinicians at US Hemophilia Treatment Centers completed semi-structured interviews about their experience with SDM and provided feedback on a clinician SDM tool prototype.
Gene Therapy for Hemophilia A: A Mixed Methods Study of Patient Preferences and Shared Decision-Making.
Patient Prefer Adherence
April 2023
Hemophilia and Thrombosis Treatment Center, Rady Children's Hospital San Diego, San Diego, CA, USA.
Purpose: As gene therapies for hemophilia are incorporated into clinical practice, shared decision-making (SDM) is recommended for implementation. SDM tools may facilitate informed decision-making for gene therapy and other novel therapies.
Objective: To inform the development of SDM tools for hemophilia gene therapy.
Systemic Anticoagulation and Inpatient Outcomes of Pancreatic Cancer: Real-World Evidence from U.S. Nationwide Inpatient Sample.
Cancers (Basel)
March 2023
Institute of Medicine, Chung Shan Medical University, Taichung 402, Taiwan.
Pancreatic cancer can induce a hypercoagulable state which may lead to clinically apparent thrombosis. However, the effect of anticoagulants remains ambiguous. This study aimed to investigate the potential effect of long-term systemic anticoagulant usage on hospitalization outcomes of patients with pancreatic cancer.
View Article and Find Full Text PDFDirect oral anticoagulants in pediatric venous thromboembolism: Experience in specialized pediatric hemostasis centers in the United States.
Res Pract Thromb Haemost
January 2023
Department of Pediatrics, Division of Pediatric Hematology, Oncology and Marrow & Blood Cell Transplantation, Children's Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY, USA.
Article Synopsis
- Pediatric hematologists in the US have been using direct oral anticoagulants (DOACs) off-label for treating venous thromboembolism (VTE) in kids and teens before official FDA approval in 2021.
- The ATHN 15 study analyzed the use of DOACs from 2015 to 2021 across 15 specialized pediatric centers, focusing on their safety and effectiveness in patients aged 0 to 21 years.
- Results indicated that rivaroxaban was the most commonly prescribed DOAC, with a bleeding complication rate of 13.8% and a recurrence rate of 4%, showing that DOACs have an adequate safety and effectiveness profile for young patients.
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
N Engl J Med
February 2023
From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).
Article Synopsis
- The study investigates the effects of gene therapy (etranacogene dezaparvovec) for treating moderate-to-severe hemophilia B, comparing it with traditional factor IX replacement therapy.
- A phase 3 open-label trial involved 54 men who received a single infusion of AAV5 vector after a lead-in period of factor IX prophylaxis, with the goal of reducing annualized bleeding rates.
- Results showed a significant decrease in bleeding rates from 4.19 to 1.51 and an increase in factor IX activity, demonstrating that gene therapy is not only noninferior but also superior to standard treatment.
Novel Approach to Improve the Identification of the Bleeding Phenotype in Noonan Syndrome and Related RASopathies.
J Pediatr
June 2023
Rady Children's Institute of Genomic Medicine, San Diego, CA. Electronic address:
Objectives: To characterize the bleeding phenotype in Noonan syndrome (NS), to test the utility of following national guidelines in detecting this phenotype, to evaluate thromboelastography (TEG) as a diagnostic tool, and to evaluate the cohort for genotype-phenotype correlations.
Study Design: Participants with a clinical diagnosis of NS or related RASopathies were enrolled in a cohort study. Study procedures included clinical bleeding assessment, coagulation testing per guidelines, and hematology consultation.
De novo antiphospholipid antibody syndrome in two infants.
Pediatr Blood Cancer
April 2023
Department of Pediatrics, Weill Cornell Medicine, New York, New York, USA.
Gene therapy preferences and informed decision-making: Results from a National Hemophilia Foundation Community Voices in research survey.
Haemophilia
January 2023
Hemophilia and Thrombosis Treatment Center, Rady Children's, Hospital San Diego, San Diego, California, USA.
Introduction: To inform education and treatment discussions, it is important to understand how persons with haemophilia prefer to learn about and discuss new therapies and to identify variables that influence decision-making.
Aim: The aim of this study was to evaluate preferences and variables which influence decision-making related to gene therapy and other novel haemophilia therapies.
Methods: An online survey was sent to men with severe haemophilia enrolled in the National Hemophilia Foundation Community Voices in Research online platform for patient-powered research.
Five Challenging Cases of Hereditary Antithrombin Deficiency Characterized by Thrombosis or Complicated Pregnancy.
J Blood Med
October 2022
Bleeding & Clotting Disorders Institute, University of Illinois College of Medicine - Peoria, Peoria, IL, USA.
Hereditary antithrombin deficiency (ATD) is a rare autosomal dominant condition (estimated prevalence 1:500-1:5000). Most ATD patients have AT activity levels 40-60% of normal. We present treatments for venous thromboembolism (VTE) in five cases of hereditary ATD.
View Article and Find Full Text PDFEffect of sleep disorders on the risks of cancers and site-specific cancers.
Sleep Med
December 2022
Graduate Institute of Business Administration, College of Management, Fu Jen Catholic University, Taipei, Taiwan; Artificial Intelligence Development Center, Fu Jen Catholic University, Taipei, Taiwan; Department of Food Nutrition and Health Biotechnology, College of Medical and Health Science, Asia University, Taichung, Taiwan; Big Data Center, Lo-Hsu Medical Foundation, Lotung Poh-Ai Hospital, Yilan, Taiwan; Division of Radiation Oncology, Lo-Hsu Medical Foundation, Lotung Poh-Ai Hospital, Yilan, Taiwan; Department of Healthcare Administration, College of Medical and Health Science, Asia University, Taichung, Taiwan; Centers for Regional Anesthesia and Pain Medicine, Wan Fang Hospital, Taipei Medical University, Taipei, Taiwan. Electronic address:
Purpose: Whether preexisting sleep disorder is an independent risk factor for cancer remains unclear. Therefore, we performed this propensity score-matched population-based cohort study to compare the incidence rate ratios (IRRs) of specific cancers between patients with and without sleep disorders.
Patients And Methods: Patients were categorized into two groups on the basis of the presence or absence of sleep disorders and matched at a 1:1 ratio.
Sarcopenia Is an Independent Risk Factor for Severe Diabetic Nephropathy in Type 2 Diabetes: A Long-Term Follow-Up Propensity Score-Matched Diabetes Cohort Study.
J Clin Med
May 2022
Graduate Institute of Business Administration, College of Management, Fu Jen Catholic University, Taipei 242, Taiwan.
Background: Diabetic nephropathy is a common cause of chronic kidney disease (CKD) and end-stage kidney disease (ESKD) worldwide and results in tremendous wastage of medical resources. Determining the indicators of diabetic nephropathy, such as sarcopenia, and implementing early interventions to prevent disease progression is crucial. Purpose: The effect of sarcopenia on the risk of severe diabetic nephropathy in patients with type 2 diabetes (T2DM) remains unclear.
View Article and Find Full Text PDFPrepare the Way for Hemophilia A Gene Therapy.
N Engl J Med
March 2022
From the Hemophilia and Thrombosis Treatment Center, Rady Children's Hospital San Diego, San Diego, and the Department of Pediatrics, University of California San Diego Health Sciences, La Jolla.
Case Report: Vaccine-Induced Immune Thrombotic Thrombocytopenia in a Pancreatic Cancer Patient After Vaccination With Messenger RNA-1273.
Front Med (Lausanne)
November 2021
Division of Hematology and Oncology, Department of Internal Medicine, Hemophilia and Thrombosis Treatment Center, Chang Gung Memorial Hospital at Keelung, Keelung, Taiwan.
Vaccination plays an important role during the COVID-19 pandemic. Vaccine-induced thrombotic thrombocytopenia (VITT) is a major adverse effect that could be lethal. For cancer patients, cancer-related thromboembolism is another lethal complication.
View Article and Find Full Text PDFCharacteristics, complications, and sites of bleeding among infants and toddlers less than 2 years of age with VWD.
Blood Adv
April 2021
Division of Blood Disorders, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA.
Article Synopsis
- * A significant 68% of cases were identified due to family history, with an average diagnosis age of 7 months; type 2 VWD was diagnosed earlier than types 1 or 3.
- * Bleeding events were common, occurring in 70% of the patients, especially from oral mucosa and circumcision, highlighting the importance of family history and early diagnosis in treatment.
Transition of care for pediatric and adult patients with venous thromboembolism: A National Quality Improvement Project from the American Thrombosis and Hemostasis Network (ATHN).
Thromb Res
April 2021
Wayne State University, Children's Hospital of Michigan, Detroit, MI, United States of America.
Background: Transition of care (TOC) for management of anticoagulation from inpatient to outpatient setting for patients with acute venous thromboembolism (VTE) poses serious safety concerns. We implemented a national quality improvement educational initiative to address this issue.
Methods: Pediatric and adult patients admitted for their first VTE were prospectively enrolled at 16 centers from January 2016 to December 2018.