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rFVIIIFc (efraloctocog alfa, Eloctate ) is an extended half-life (EHL) factor VIII licensed for use in patients with hemophilia A for prophylaxis and treatment of bleeding and surgical episodes. Pharmacokinetic studies in adults have shown a mean 1.5-fold increase in half-life compared to full-length factor VIII. When compared to adults, the half-life is decreased by 8% in adolescents between 12 and 17 years, by 18% in children 6 to <12 years, and by 33% in children between the ages of 2 and <6 years. There is a considerable interindividual variation in the prolongation of the half-life particularly in children and across the age groups, the range extending from no increase to a 2.5-fold increase. In addition to age, von willebrand factor (VWF) antigen level has demonstrated a significant impact on rFVIIIFc half-life, with higher VWF levels associated with greater prolongation of half-life. The pivotal and pediatric clinical trials have demonstrated the efficacy and safety of rFVIIIFc for use in regular prophylaxis and in management of bleeds and surgery. In these studies, just under half the participants showed a zero annualized bleed rate (ABR), and the median ABR (1.6 in the pivotal study for the individualized prophylaxis arm) showed a further decrease in the extension study. On average, the patients required fewer infusions (reduced by at least a third), and the mean weekly consumption seems to be in keeping with standard recombinant factor VIII. EHL rFVIIIFc has made decreased infusion frequency a possibility. However, the interindividual variability in dose and infusion frequency highlights the need for a personalized approach based on individual patient's half-life and/or response to treatment.

By inactivating the tissue factor pathway inhibitor, concizumab can lead to sufficient thrombin generation for clotting. In this trial, the median annualized bleeding rate was 9.8 with no prophylaxis and 0 with concizumab prophylaxis.

ObjectiveRisk assessment for venous thromboembolism (VTE) and thromboprophylaxis in those with risk factors is established in adult practice. Evidence to support efficacy and safety of this approach in adolescents is lacking. We aimed to describe thrombotic risk factors and to determine the proportion of potentially preventable events in a retrospective cohort study of adolescents with VTE.Design, setting and patientsData were collected between 2008 and 2014 from eight tertiary UK centres. Qualifying events were radiologically confirmed VTE in subjects aged 12–17 years. Central venous line-related upper venous system events were excluded.Results76 cases were identified, 41 males, median age 15 years. Frequent risk factors were: reduced mobility, 45%; thrombophilia, 24%; malignancy, 20%; surgery, 18%; combined oral contraceptive pill, 12%; congenital venous anomaly, 5%. 28 (37%) had no significant underlying diagnosis and no provoking event/hospitalisation, presenting as outpatients with VTE which was considered ‘unpreventable’. Of 48 where there had been opportunity for risk assessment, chemical thromboprophylaxis was not indicated in 26 and was contraindicated in 8. 14/76 (18%) had an indication to consider thromboprophylaxis and no contraindication. Of these, four had cerebral palsy, five malignancy and two inflammatory bowel disease. All had reduced mobility with recent surgery in eight. Four received chemical thromboprophylaxis prior to presentation.ConclusionsAmong a cohort of adolescents with VTE, a small proportion (13%) had an indication to consider chemical thromboprophylaxis but did not receive it. VTE risk assessment and prevention should focus on adolescents with immobility or surgery, particularly in those with malignancy.

Modern haemophilia care based on good diagnosis and effective prophylaxis has allowed boys born with haemophilia to grow up leading essentially normal lives. Nevertheless, there remain challenges notably those posed by inhibitors and patient expectations. There is now a significant cohort of men in their 30s, 40s and 50s who have been looked after extremely well but many of whom now have significant ankle arthropathy because they played football, even when advised not to do so at a time when prophylaxis was limited or started late. The imminent era of longer-acting clotting factors and gene therapy will also impact on future patient expectations. Meeting and managing these challenges will be much enhanced by the development and maintenance of good relationships between the patient and the haemophilia team.

Individuals with haemophilia are usually excluded from patrolling police roles as these often necessitate confrontation. We describe an individual with moderate to severe haemophilia B who has been recruited by the Metropolitan Police Service as a Special Constable with the support of his haematologist.

Introduction: Severe factor X deficiency is a rare serious bleeding disorder historically treated with fresh frozen plasma (FFP) and more recently with prothrombin complex concentrate (PCC) which contains activated factors II, VII, IX and X. The infusion volume of PCC is smaller than FFP, but there is a risk of thromboembolic complications given the presence of activated forms of vitamin K-dependent factor concentrates when treating an isolated coagulation factor deficiency. Methods: We describe the case of a nine-year-old girl of consanguineous origin with co-existent congenital merosin deficient muscular dystrophy and severe factor X deficiency treated with twice-weekly PCC prophylaxis via an indwelling central venous access device (CVAD). Infusion occlusion of her fifth CVAD occurred 24-months post-insertion; thrombus within the right subclavian and brachiocephalic veins was seen on radiological imaging. She started peripheral treatment with BPL Factor X concentrate as infusion volumes were smaller and given her immobility further thrombotic risk was predicted to be reduced. A sixth CVAD was inserted seven months later and BPL Factor X prophylaxis was continued. Results:BPL Factor X concentrate was effective in maintaining trough levels of 13IU/ml 72-hours post-dose, with no intercurrent bleeding episodes or further problems in terms of occlusion of her portacath. Further radiological screening has not been undertaken. Conclusion: BPL Factor X has been shown to be a safe and effective alternative to PCC for treatment of severe factor X deficiency in this case.

  In an improbable setting Mosher finds irresistible stories, and after applying himself \"to the seat of the chair,\" he finally finds his writer's voice in which to tell those tales to a widening audience: \"Disappearances,\" \"Where the Rivers Flow North,\" \"A Stranger in the Kingdom,\" are just a few of the titles in his growing body of work about \"Kingdom County.\"