Long-Term Efficacy and Safety of Canakinumab in Patients With Tumor Necrosis Factor Receptor-Associated Periodic Syndrome: Results From a Phase III Trial.

Objective: We aimed at assessing efficacy, safety, and tolerability of canakinumab in patients with tumor necrosis factor receptor-associated periodic syndrome (TRAPS) during a 72-week long-term, open-label extension of the CLUSTER study.

Methods: Patients received open-label canakinumab 150 or 300 mg, either every 4 weeks (q4w) or every 8 weeks, with up-titration permitted after on-treatment flares (maximum dose: 300 mg q4w). Efficacy assessments included physician global assessment of disease activity, number of flares, and serum C-reactive protein (CRP) and serum amyloid A protein (SAA) levels.

Portomesenteric Vein Thrombosis in Patients Undergoing Sleeve Gastrectomy: an Updated Systematic Review and Meta-Analysis of 101,914 Patients.

Introduction: Portomesenteric vein thrombosis (PMVT) is a rare but potentially fatal complication of sleeve gastrectomy (SG). The rising prevalence of SG has led to a surge in the occurrence of PMVT, while the associated risk factors have not been fully elucidated. This study aims to determine the incidence and risk factors of PMVT in patients undergoing SG.

Hepatitis B cure: How and when.

Background: First- and second- generation new treatments are being evaluated to provide a cure for hepatitis B. The life cycle of HBV includes several well- categorized steps that are targets for new treatments. A cure remains a major challenge even if it is measured by HBsAg seroclearance alone.

Evaluating the real-life effect of MP-AzeFlu on asthma outcomes in patients with allergic rhinitis and asthma in UK primary care.

Background: MP-AzeFlu (Dymista®; spray of azelastine/fluticasone propionate) is the most effective allergic rhinitis (AR) treatment available. Its effect on asthma outcomes in patients with AR and asthma is unknown.

Methods: This pre-post historical cohort study, using the Optimum Patient Care Research Database, included patients aged ≥12 years, from UK general practice with active asthma (defined as a recorded diagnosis, with ≥1 prescription for reliever or controller inhaler) in the year before or at the initiation date.

Scoring system to facilitate diagnosis of Gaucher disease.

Background: Gaucher disease (GD) manifests heterogeneously and other conditions are often misdiagnosed in its place, leading to diagnostic delays. The Gaucher Earlier Diagnosis Consensus (GED-C) initiative proposed a point-scoring system (PSS) based on the signs and covariables that are most indicative of GD to help clinicians identify which individuals to test for GD.

Aims: To validate the PSS retrospectively in a test population including patients with GD and other conditions with overlapping manifestations.

ARIA masterclass 2018: From guidelines to real-life implementation.

Over the past 20 years, ARIA (Allergic Rhinitis and its Impact on Asthma) has developed various guidelines for the treatment of allergic rhinitis (AR) and asthma multimorbidity. Over time, the ARIA initiative has evolved to ensure the highest level of bestpractices adoption in real life settings. It has evolved towards Integrated Care Pathways (ICPs) using mobile technology, and has now entered a new phase in which change management is key to provide an active and healthy life to all AR patients.

Screening and management of viral hepatitis and hepatocellular carcinoma in Mongolia: results from a survey of Mongolian physicians from all major provinces of Mongolia.

Background: According to Globocan, Mongolia has the highest worldwide hepatocellular carcinoma (HCC) incidence (78.1/100 000, 3.5× higher than China).

A systematic review of transcatheter aortic valve implantation via carotid artery access.

Background: The carotid artery is a novel access route for transcatheter aortic valve implantation (TAVI). This may represent a viable alternative in patients unsuitable for TAVI via traditional transfemoral access, up to 20%, as well as other access routes such as subclavian, transapical and aortic. This systematic review summarises the current evidence for its safety and feasibility.

A phenotype-genotype correlation of ADAMTS13 mutations in congenital thrombotic thrombocytopenic purpura patients treated in the United Kingdom.

Background: ADAMTS13 mutations play a role in thrombotic thrombocytopenic purpura (TTP) pathogenesis.

Objectives: To establish a phenotype-genotype correlation in a cohort of congenital TTP patients.

Patients/methods: Clinical history and ADAMTS13 activity, antigen and anti-ADAMTS13 antibody assays were used to diagnose congenital TTP, and DNA sequencing and in vitro expression were performed to identify the functional effects of the ADAMTS13 mutations responsible.

Therapeutic goals in the treatment of Fabry disease.

Purpose: Fabry disease is a progressive multiorgan, multisystem disorder that is caused by a deficiency in the lysosomal enzyme α-galactosidase A. Serious renal, cardiac, and cerebrovascular involvement are responsible for much of the morbidity and premature mortality associated with Fabry disease, and neuropathic pain, gastrointestinal problems, and hypohidrosis negatively affect quality of life of patients with Fabry disease. Fabry disease is X-linked, but women are often symptomatic and may be as severely affected as men.

Enhanced propensity of T lymphocytes in patients with systemic lupus erythematosus to apoptosis in the presence of tumour necrosis factor alpha.

Objective: To determine the effect of inflammation through exposure to tumour necrosis factor (TNF)alpha on T lymphocytes in patients with systemic lupus erythematosus (SLE).

Methods: We studied the effect of TNFalpha on T-lymphocyte apoptosis in patients with SLE, rheumatoid arthritis (RA), and in healthy controls. Apoptosis of CD4 and CD8 T lymphocytes and naive and memory subpopulations was determined by flow cytometry using 7-amino-actinomycin D (7AAD) and propidium iodide (PI).

Characterisation of tumoral markers correlated with ErbB2 (HER2/Neu) overexpression and metastasis in breast cancer.

The receptor tyrosine kinase ErbB2 (HER2/neu) is overexpressed in ˜30% of breast cancers and is associated with poor prognosis and an increased likelihood of metastasis. Clinical treatments such as trastuzumab are effective in less than 35% of women diagnosed as ErbB2-positive, highlighting the necessity of searching for novel targets and alternative therapies. Herein, a proteomic screening strategy combining quantitative-based gel electrophoresis and MS was used to compare the protein expression of 48 normal human breast and tumour tissues differing in ErbB2 expression and lymph node status.

High response rate to donor lymphocyte infusion after allogeneic stem cell transplantation for indolent non-Hodgkin lymphoma.

The role of donor lymphocyte infusion (DLI) in the management of lymphoid malignancies after allogeneic stem cell transplantation (SCT) has not been clearly characterized. There is emerging evidence pointing to the effectiveness of this approach, particularly in patients with low-grade disease, although to date this has been reported only in small numbers of patients, and thus the utility of this treatment remains uncertain. A total of 28 patients with low-grade lymphoid malignancies previously treated with allogeneic SCT received a total of 68 infusions of donor lymphocytes.

Prevalence of the ADAMTS-13 missense mutation R1060W in late onset adult thrombotic thrombocytopenic purpura.

Background: Thrombotic thrombocytopenic purpura (TTP) is most commonly associated with deficiency or inhibition of von Willebrand factor-cleaving protease (ADAMTS-13) activity. ADAMTS-13 mutations and polymorphisms have been reported in childhood congenital TTP, but their significance in adult onset TTP remains unclear.

Objectives: We sought to identify common ADAMTS-13 mutations in adults with late onset TTP and to investigate whether they may predispose acute clinical episodes of the disorder in adulthood.

Access to expensive drugs in the NHS: myths and realities for cancer patients.

Setting: Patient support organisation (Myeloma UK) and Hospital Myeloma Clinic.

Type Of Study: Case review of two patients, one whose treatment is funded by a private insurance company in the UK, and the other who is funded by the National Health Service (NHS) and questionnaire survey of 51 haematologists from Myeloma UK database.

Results: The treatment options available for private patients with myeloma in the UK are broader and more in line with national and international practice than are options available to UK NHS patients.

The Objective Structured Clinical Exam - practical aspects.

Assessment of clinical skills in addition to factual knowledge has become part of the examination system in both undergraduate and postgraduate examinations. There is an increasing interest in the concept of competence and how it can be measured, particularly for the process of revalidation of practitioners. The Objective Structured Clinical Examination (OSCE) has been introduced for the assessment of clinical skills into postgraduate anaesthetic examinations in the United Kingdom.