Therapeutic Plasma Exchange for Fibrinogen-Associated Hyperviscosity: Results of the COPLEX Randomized Controlled Trial.

Background: Therapeutic plasma exchange (TPE) is the primary intervention for treating symptomatic hyperviscosity from hypergammaglobulinemia, yet its efficacy for treating hyperviscosity related to hyperfibrinogenemia is unclear.

Objective: Define the safety and efficacy of TPE for critically ill COVID-19 patients with elevated blood viscosity from hyperfibrinogenemia.

Method: A prospective, randomized controlled trial in critically ill COVID-19 patients at a single US healthcare system.

Assessment of joint health in females with haemophilia: The carriers ultrasound project (CUP) study.

Introduction: The needs of haemophilia carriers (HC) have been historically overlooked. It is now recognised that HC manifests bleeding symptoms, including haemarthrosis. The natural history of joint health in HC is not yet defined.

Differences and similarities in patient-reported outcomes among men and women with haemophilia.

Introduction: Both men and women can be diagnosed with haemophilia and the experience with haemophilia may be different between men and women.

Aim: This study aimed to compare patient-reported outcomes in men versus women with haemophilia.

Methods: This cross-sectional study is a post-hoc analysis of data collected as part of the Haemophilia-related Distress Questionnaire validation study.

Exploring Older Adults' Perceptions of Using Digital Health Platforms for Self-Managing Musculoskeletal Health Conditions: Focus Group Study.

Background: Digital technologies can assist and optimize health care processes. This is increasingly the case in the musculoskeletal health domain, where digital platforms can be used to support the self-management of musculoskeletal conditions, as well as access to services. However, given a large proportion of the population with musculoskeletal conditions are older adults (aged ≥60 years), it is important to consider the acceptability of such platforms within this demographic.

Race and ethnicity reporting and representation in hemophilia clinical trials.

Racial and ethnic representativeness in clinical trials is crucial to mitigate disparities in outcomes; however, diversity among hemophilia trials is unknown. The aim of this study is to examine the reporting and representation of race and ethnicity in trials of people with hemophilia (PwH). In this cross-sectional study, the ClinicalTrials.

Race and ethnicity and the success of immune tolerance induction among people with severe haemophilia A in the United States.

Introduction: Immune tolerance induction (ITI) is the only treatment to eradicate inhibitors in people with severe haemophilia A with inhibitors. Since the risk of inhibitor development is greater among Black and Hispanic persons, it has been hypothesized that race and ethnicity may influence ITI success. Limited studies have evaluated this hypothesis.

Race, ethnicity, and immune tolerance induction in hemophilia A in the United States.

Background: In racially diverse communities, treatment of chronic diseases can vary across racial and ethnic groups.

Objectives: To examine healthcare disparities in hemophilia care in the United States by evaluating receipt of immune tolerance induction (ITI) among different racial and ethnic groups.

Methods: In this cross-sectional study, people with severe hemophilia A with an inhibitor who entered the Center for Disease Control and Prevention Community Counts registry between 2013 and 2017, were aged ≥5 years at study entry, and had a history of an inhibitor ( = 614) were included.

Defining the impact of immune tolerance induction on clinically relevant outcomes in a US cohort of severe hemophilia A.

Although the near-term benefit of immune tolerance induction (ITI) for the treatment of people with severe hemophilia A with inhibitor is apparent, the magnitude of the longer-term impact of ITI on clinical outcomes remains undefined. We examined the association between receiving ITI and the success of ITI on clinical outcomes including (1) clinical events, (2) health care use, (3) quality of life/function, (4) socioeconomic status, and (5) death, using the Community Counts (CC) registry of US Hemophilia Treatment Centers between 2013 and 2017. Multivariate logistic regression, negative binomial, and Poisson models were used.

Racial and ethnic differences in reported haemophilia death rates in the United States.

Introduction: People with haemophilia's life expectancies have improved over time. Whether progress has been experienced equitably is unknown.

Aim: To examine recorded haemophilia death (rHD) rates according to race and ethnicity in the United States (US).

Corrigendum to 'Efficacy of emicizumab is maintained throughout dosing intervals for bleed prophylaxis' [Research and Practice in Thrombosis and Haemostasis, Volume 7, Issue 2, February 2023, 100077].

[This corrects the article DOI: 10.1016/j.rpth.

Cost-effectiveness of prophylactic emicizumab versus prophylactic recombinant factor VIII in patients with moderate or mild hemophilia A without inhibitors in the United States.

Prophylactic emicizumab is cost-ineffective in adults with moderate or mild hemophilia A without inhibitors at current pricing. The price of prophylactic emicizumab would need to decrease by >35% to become cost-effective in this patient population.

Racial and Ethnic Differences in Distress, Depression, and Quality of Life in people with hemophilia.

Hemophilia-related distress (HRD) has been shown to be higher among those with lower educational attainment, but potential racial/ethnic differences have not been previously described. Thus, we examined HRD according to race/ethnicity. This cross-sectional study was a planned secondary analysis of the hemophilia-related distress questionnaire (HRDq) validation study data.

Efficacy of emicizumab is maintained throughout dosing intervals for bleed prophylaxis.

Background: Across the HAVEN clinical trial program, the efficacy of emicizumab has been demonstrated in children, adolescents, and adults with hemophilia A, with or without factor VIII inhibitors. After the 4-week loading dose period, emicizumab concentrations are expected to remain at levels that provide bleed protection throughout the entire dosing interval, regardless of the chosen maintenance dosing regimen, ie, weekly, every 2 weeks, or every 4 weeks.

Objectives: The objective of this study was to examine the timing of treated bleeds within the dosing intervals for emicizumab administered during the HAVEN 1 to 4 studies.

Cross-sectional study evaluating the association of haemophilia-related distress and clinically relevant outcomes.

Introduction: In chronic diseases, disease-related distress can impact disease outcomes. Distress and haemophilia-related distress has been demonstrated in people with haemophilia (PwH). The association of haemophilia-related distress on disease outcomes among PwH is unknown.

Manipulating chromatin architecture in C. elegans.

Background: Nucleosome-mediated chromatin compaction has a direct effect on the accessibility of trans-acting activators and repressors to DNA targets and serves as a primary regulatory agent of genetic expression. Understanding the nature and dynamics of chromatin is fundamental to elucidating the mechanisms and factors that epigenetically regulate gene expression. Previous work has shown that there are three types of canonical sequences that strongly regulate nucleosome positioning and thus chromatin accessibility: putative nucleosome-positioning elements, putative nucleosome-repelling sequences, and homopolymeric runs of A/T.

Untreated bleeds in people with hemophilia A in a noninterventional study and intrapatient comparison after initiating emicizumab in HAVEN 1-3.

Background: Bleeding in people with hemophilia A can be life threatening, and intra-articular bleeds can result in joint damage. Most clinical studies focus on treated bleeds, while bleeds not treated with coagulation factor(s) (untreated bleeds) are underreported.

Objectives: We assessed the incidence of untreated bleeds during a noninterventional study (NIS) wherein people with hemophilia A, with or without factor VIII (FVIII) inhibitors, were managed according to standard practice.

Surgical outcomes in people with hemophilia A taking emicizumab prophylaxis: experience from the HAVEN 1-4 studies.

Many people with hemophilia A (PwHA) undergo surgery in their lifetime, often because of complications of their disease. Emicizumab is the first bispecific monoclonal antibody prophylactic therapy for PwHA, and its efficacy and safety have been previously demonstrated; however, there is a need to build an evidence base on the management of PwHA on emicizumab undergoing surgery. Data from the HAVEN 1-4 phase 3 clinical trials were pooled to provide a summary of all minor and major surgeries in PwHA with or without factor VIII (FVIII) inhibitors who were receiving emicizumab prophylaxis.

Effect of emicizumab prophylaxis on bone and joint health markers in people with haemophilia A without factor VIII inhibitors in the HAVEN 3 study.

Introduction: Emicizumab prophylaxis significantly reduces bleeding events; however, the associated impact on bone/joint health is unknown.

Aim: To explore the effect of emicizumab prophylaxis on bone/joint health in people with haemophilia A (PwHA) without FVIII inhibitors enrolled in HAVEN 3 (NCT02847637).

Methods: Haemophilia joint health scores (HJHS; v2.

Comparing direct oral anticoagulants and vitamin K antagonist use in morbidly obese patients with venous thromboembolism: A single center retrospective cohort study.

: Limited data exists on the safety and efficacy of direct-acting oral anticoagulants (DOAC) use in morbidly obese patients with venous thromboembolism (VTE). Given the benefits of DOAC use over vitamin K antagonists (VKAs), in terms of monitoring requirements, and dietary and drug interactions, it is important to evaluate whether this is consistent in the higher risk for VTE recurrence morbidly obese group body mass index (BMI ≥ 40 kg/m). : This retrospective, single-center cohort study included patients with a BMI of at least 40 kg/m who were admitted to Emory University Hospital from 1 January 2012 to 31 May 2020 with acute VTE, and subsequently initiated on anticoagulation treatment with either DOAC or VKA (warfarin).

Quality of life in a large multinational haemophilia B cohort (The B-Natural study) - Unmet needs remain.

Introduction: The B-Natural study is a multicentre, multinational, observational study of haemophilia B (HB) designed to increase understanding of clinical manifestations, treatment and quality of life (QoL).

Aim: To characterise and compare QoL in HB across disease severity groups and individuals with inhibitors to identify gaps in treatment.

Methods: A total of 224 individuals from 107 families were enrolled from a total of 24 centres in North America (n = 16), Europe (n = 7) and Asia (n = 1).

Shortening the Haemophilia Activities List (HAL) from 42 items to 18 items.

Introduction: The Haemophilia Activities List (HAL) was developed to measure activities and participation in persons with haemophilia (PWH). Shortening the questionnaire may facilitate use of the HAL.

Aim: The aim of this study was to determine which items of the HAL are redundant, to construct a shorter version of the HAL, and to determine the construct validity of the HAL .

Thrombocytopenia Induced by Polysulfone Dialysis Membranes.

BACKGROUND Biocompatible hemodialysis membranes have greatly advanced the treatment of renal failure. Synthetic polysulfone dialysis membranes are considered to be very biocompatible because of their low propensity to activate complement. However, these membranes can reduce platelet count through platelet activation, although the mechanism of this activation is unknown.