Characterization of Men with Hemophilia B and Factors Associated with Treatment Practices, Participating in the Community Counts Registry from 2014 to 2018.

Hemophilia B is an inherited, X-linked, bleeding disorder caused by a mutation of the clotting factor 9 (FIX) gene. The mutation reduces the amount of FIX protein and results in spontaneous and trauma-related bleeding episodes. In 1994, approximately 2,800 men with hemophilia B (MWHB) were treated at hemophilia treatment centers (HTCs) in the United States (US). To date, studies examining health outcomes for MWHB in the US have not been compared across disease severities. Treatment of MWHB has become more complex with changes in prophylaxis practices in the US and the introduction of novel treatment products. Observational studies that describe health outcomes among MWHB and current treatment practices are important to inform future clinical practices. These cross-sectional analyses used data from MWHB enrolled in the Community Counts surveillance Registry (Registry) from 2014 to 2018. The first paper compared the sample of MWHB in the Registry to the population of MWHB who received treatment in HTCs and described the demographic, clinical factors, and health outcomes across disease severities. From 2014-2018, the population of MWHB who received care in HTCs included 4,816 MWHB, of which 2091 participated in the Registry. The second paper examined demographic, clinical factors, and health outcomes associated with treatment regimen, prophylaxis versus episodic; and used a marginal model. The final model included ethnicity, health insurance, history of a joint bleed, and interactions between severity by chronic pain as well as age by history of central venous access device utilization. The third paper examined demographic, clinical factors, and health outcomes associated with treatment product type utilization, standard half-life products versus extended half-life products, among MWHB on continuous prophylaxis; and used a marginal model. The final model included disease severity, enrollment year, HTC region, and percent of missed treatment dose. The second and third paper demonstrated that patient-level treatment outcomes were clustered by the HTCs where they received care. Future studies should examine the treatment dosage and frequency of administration of treatment products for MWHB on prophylaxis and replicate these studies for hemophilia A to determine if the factors associated with treatment are similar for all men with hemophilia

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 HALshort. Methods: A secondary analysis was performed on pooled data of two published studies using the HAL (seven domains, 42 items, optimum score: 100) in adults with haemophilia A/B. Data were divided into a derivation (62%) and a validation set (38%). Redundant items were identified by evaluation of: floor and ceiling effects, proportions of missing and ‘not applicable’ responses, inter-item correlations, component loadings in an exploratory factor analysis, internal consistency, and item-total correlations. Correlations with the SF-36 and EQ-5D-5L were used to determine construct validity of the HALshort. Results: Data on 680 PWH were evaluated. In the derivation dataset (n = 420), median age was 30 years (range 18–80), 43% had severe haemophilia and 61% received prophylaxis. Median (IQR) HAL sum score was 65.0 (55.7–88.8). The stepwise procedure resulted in a HALshort of 18 items with a median sum score of 63.3 (54.4–86.7). Construct validity was similar for the HAL and HALshort in the validation dataset (n = 260). Conclusion: This clinimetric study resulted in a >50% shortening of the HAL. The 18-item HALshort reduces patient burden and is expected to capture the information on activities and participation. The HALshort needs further validation

Community compensation in the context of Carbon Capture and Storage: Current debates and practices

Societal opposition has the potential to slow down the implementation of Carbon Capture and Storage (CCS). One of the difficulties is that the perceived benefits associated with a CCS facility for local communities tend to be low compared to its perceived burdens. As is the case for other low carbon technologies, community compensation (or community benefits) has been suggested as a way to restore this perceived imbalance. A diverse literature has looked into the role of community compensation across various land uses and research fields. Synthesis is limited, while at the same time, the provision of community compensation in practice is moving from an ad hoc to a more institutionalized approach. Therefore, it is important to take stock of the literature. This paper provides a review of the community compensation literature in the form of four debates, drawing together environmental social science research on different low carbon technologies (e.g. CCS, renewable energy). In addition, current practices in community compensation for four European countries are discussed. The two parts of this paper are brought together in a set of lessons for the provision of community compensation for future CCS projects; in turn, suggestions for further research are made to address remaining knowledge gaps

A genome-wide association study of resistance to HIV infection in highly exposed uninfected individuals with hemophilia A

Human genetic variation contributes to differences in susceptibility to HIV-1 infection. To search for novel host resistance factors, we performed a genome-wide association study (GWAS) in hemophilia patients highly exposed to potentially contaminated factor VIII infusions. Individuals with hemophilia A and a documented history of factor VIII infusions before the introduction of viral inactivation procedures (1979-1984) were recruited from 36 hemophilia treatment centers (HTCs), and their genome-wide genetic variants were compared with those from matched HIV-infected individuals. Homozygous carriers of known CCR5 resistance mutations were excluded. Single nucleotide polymorphisms (SNPs) and inferred copy number variants (CNVs) were tested using logistic regression. In addition, we performed a pathway enrichment analysis, a heritability analysis, and a search for epistatic interactions with CCR5 Δ32 heterozygosity. A total of 560 HIV-uninfected cases were recruited: 36 (6.4%) were homozygous for CCR5 Δ32 or m303. After quality control and SNP imputation, we tested 1 081 435 SNPs and 3686 CNVs for association with HIV-1 serostatus in 431 cases and 765 HIV-infected controls. No SNP or CNV reached genome-wide significance. The additional analyses did not reveal any strong genetic effect. Highly exposed, yet uninfected hemophiliacs form an ideal study group to investigate host resistance factors. Using a genome-wide approach, we did not detect any significant associations between SNPs and HIV-1 susceptibility, indicating that common genetic variants of major effect are unlikely to explain the observed resistance phenotype in this populatio

Correction to: Cluster identification, selection, and description in Cluster randomized crossover trials: the PREP-IT trials

An amendment to this paper has been published and can be accessed via the original article

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

Abstract Introduction: 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/m2). Materials and methods: This retrospective, single‐center cohort study included patients with a BMI of at least 40 kg/m2 who were admitted to Emory University Hospital from 1st January 2012 to 31st May 2020 with acute VTE, and subsequently initiated on anticoagulation treatment with either DOAC or VKA (warfarin). Univariate and bivariate analyses were used to evaluate differences in demographics by treatment type and BMI. Multivariate Cox proportional hazard regression was used to assess the risk of VTE recurrence by type of treatment among morbidly obese patient subgroup. Results: There were 247 (11.8%) morbidly obese (≥ 40 kg/m2) patients who were more likely than non‐obese patients to be younger, female, and of non‐white race. Thirty percent of the study population (n=74) had a BMI >50 kg/m2. T ime‐to‐event analysis confirmed that the hazard of experiencing a recurrent thrombosis was not statistically significantly different among morbidly obese patients treated with a DOAC compared with VKA (hazard ratio [HR]: 0.28, confidence interval [CI] 0.07‐1.11, p = 0.07). Conclusions: This study aligns with previous literature and confirms that morbidly obese patients receiving DOAC or VKA have similar risks of recurrent VTE

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