Joint reaction and simulated muscle forces during squatting and walking in persons with hemophilia.

Background: Persons with hemophilia experience joint bleeding that can lead to debilitating arthropathy, most commonly seen in ankles, knees, and elbows. Arthropathy can hinder participation in daily and athletic activities. We explored how hemophilic arthropathy impacts movement patterns in walking and bilateral squatting tasks in persons with hemophilia compared to healthy controls.

Concizumab improves clot formation in hemophilia A under flow.

Background: Inhibition of tissue factor pathway inhibitor (TFPI) is an emerging therapeutic strategy for treatment of hemophilia. Concizumab is a monoclonal antibody that binds TFPI and blocks its inhibition of factor (F)Xa thereby extending the initiation of coagulation and compensating for lack of FVIII or FIX.

Objectives: The objective of this in vitro study was to evaluate how concizumab affects clot formation in hemophilia A under flow.

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science.

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 and comprehensive Hemophilia Treatment Center care model, to better serve all people with inherited BDs (PWIBD), and increasing equitable access to optimal health emerged as top priorities.

Research Design And Methods: NHF, with the American Thrombosis and Hemostasis Network (ATHN), convened multidisciplinary expert working groups (WG) to distill priority research initiatives from consultation findings.

Hemostasis in the Pregnant Woman, the Placenta, the Fetus, and the Newborn Infant.

The hemostasis system is composed of procoagulant, anticoagulant, and fibrinolytic proteins that interact with endothelial and blood cells and with each other in a complex system of checks and balances to maintain blood flow while preventing both hemorrhage and thrombosis. Pregnancy is a unique physiological state in which biological alterations predispose both mother and fetus to both bleeding and clotting. The placenta is a vascular interface for maternal and fetal blood exchange which predisposes the mother to hemorrhage.

Long-term prophylaxis: what are our options and how to define success?

Currently, we are at an enviable place in hemophilia treatment. Although full prophylaxis with standard half-life recombinant or plasma-derived factor concentrates has been definitively shown to be inadequate for full protection against bleeding and arthropathy, a number of novel therapies with improved hemostatic enhancement are clinically available or in promising clinical trials. In order to compare outcomes among a number of very efficacious therapies, it is necessary to have sensitive tools employed in long-term follow-up for several years for participants with no or minimal joint disease.

Emicizumab initiation and bleeding outcomes in people with hemophilia A with and without inhibitors: A single-center report.

Background: Emicizumab, a bispecific antibody factor VIII mimetic, is approved for prophylaxis in hemophilia, and has different risks and side effects compared to factor VIII products.

Objective: To better understand the early impact of emicizumab on our patients at the University of Colorado Hemophilia and Thrombosis Center (UCHTC), we evaluated adverse reactions, factor prophylaxis overlap, and bleeding rates after starting emicizumab through a quality improvement project.

Patients/methods: A retrospective chart review and structured phone interview were conducted from June to September 2019 for all patients who had started emicizumab at the UCHTC.

Antithrombin deficiency: A pediatric disorder.

Introduction: Hereditary antithrombin (AT) deficiency is an autosomal dominant thrombophilic disorder. Guidelines do not support routine testing of children based on personal or familial thrombosis.

Aim: To investigate clinical, genetic and laboratory profiles of AT deficient children and their affected family members.

Validation of Outcome Instruments for Pediatric Postthrombotic Syndrome: Introducing the Peds-VEINES-QOL, a New Health-Related Quality of Life Instrument.

Background:  There is need for validated outcome measures for postthrombotic syndrome (PTS) following pediatric venous thromboembolism (VTE), with a focus on quality of life (QoL).

Aims:  This article assesses reliability and validity of two PTS and two QoL scales for children following lower extremity VTE.

Methods:  Pediatric patients following lower extremity VTE were recruited from three thrombosis clinics.

Outcome measures in Haemophilia: Beyond ABR (Annualized Bleeding Rate).

Options for management of haemophilia are increasing rapidly with completely novel therapeutic approaches that cannot be compared using traditional factor assays. In addition, as prophylaxis regimens have improved, bleeding rates have decreased, and consequently, it is difficult to show an impact of novel therapies on rates of spontaneous bleeding. There is currently an urgent need for a panel of outcome measures to compare therapies that are dissimilar in many essential ways.

Disseminated Intravascular Coagulation Is an Independent Predictor of Adverse Outcomes in Children in the Emergency Department with Suspected Sepsis.

Objective: To evaluate the impact of early disseminated intravascular coagulation (DIC) on illness severity in children using a database of emergency department ED encounters for children with suspected sepsis, in view of similar associations in adults.

Study Design: Laboratory and clinical data were extracted from a registry of emergency department encounters of children with suspected sepsis between April 1, 2012, and June 26, 2017. International Society of Thrombosis and Hemostasis DIC scores were calculated from laboratory values obtained within 24 hours of emergency department admission.

Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study.

The Joint Outcome Study (JOS), a randomized controlled trial, demonstrated that children with severe hemophilia A (HA) initiating prophylactic factor VIII (FVIII) prior to age 2.5 years had reduced joint damage at age 6 years compared with those treated with episodic FVIII for bleeding. The Joint Outcome Continuation Study (JOS-C) evaluated early vs delayed prophylaxis effects on long-term joint health, following JOS participants to age 18 years in an observational, partially retrospective study.

Factor VIII prophylaxis effects outweigh other hemostasis contributors in predicting severe haemophilia A joint outcomes.

Introduction: The Joint Outcome Study (JOS) demonstrated that previously untreated children with severe haemophilia A treated with prophylactic factor VIII (FVIII) concentrate had superior joint outcomes at age 6 years compared to those children treated episodically for bleeding. However, variation in joint outcome within each treatment arm was not well explained.

Aim: In this study, we sought to better understand variation in joint outcomes at age 6 years in participants of the JOS.