A systematic review of outcome measures evaluating treatment efficacy in vulval lichen sclerosus and evaluation of patients' priorities.

Vulval lichen sclerosus (VLS) is an inflammatory skin disease characterised by itching, apareunia, loss of vulval architecture and scarring. Heterogeneity in outcome reporting precludes comparison between treatments. This study aimed to systematically review outcome measures used to evaluate the efficacy of VLS treatments and present patients' treatment priorities.

A protocol for the development of core outcome sets for effectiveness trials and clinical audits in renal cell cancer (R-COS).

Background: There is inconsistency in outcomes collected in renal cell cancer (RCC) intervention effectiveness studies and variability in their definitions. This makes critical summaries of the evidence base difficult and sub-optimally informative for clinical practice guidelines and decision-making by patients and healthcare professionals. A solution is to develop a core outcome set (COS), an agreed minimum set of outcomes to be reported in all trials in a clinical area.

Postpartum peripheral neuropathy due to vitamin B12 deficiency, potentially precipitated by nitrous oxide use.

Neurological dysfunction which would concern the anaesthetist on labour ward is typically associated with neuraxial blocks. However, an appreciation of other causes is crucial. We present a case of peripheral neuropathy secondary to vitamin B12 deficiency, which highlights the importance of a thorough neurological examination, alongside an understanding of neurological pathophysiology.

Role of circulating exosomal biomarkers and their diagnostic accuracy in pancreatic cancer.

Background And Aim: New biomarkers have the potential to facilitate early diagnosis of pancreatic cancer (PC). Circulating exosomes are cell-derived protein complexes containing RNA that can be used as indicators of cancer development. The aim of this review is to evaluate the current literature involving PC patient groups for highly accurate exosomal biomarkers.

Acute lung injury after balloon pulmonary angioplasty results in a similar haemodynamic response and possible clinical advantage at follow-up.

Acute lung injury (ALI) is a common but poorly defined and understood complication of balloon pulmonary angioplasty (BPA) for chronic thromboembolic pulmonary hypertension (CTEPH). Little data are available on the medium term clinical outcomes of BPA complicated by ALI. We analyzed per-procedure data from 282 procedures in 109 patients and per-patient data from 85 patients.

Hyperelastic, shape-memorable, and ultra-cell-adhesive degradable polycaprolactone-polyurethane copolymer for tissue regeneration.

Novel polycaprolactone-based polyurethane (PCL-PU) copolymers with hyperelasticity, shape-memory, and ultra-cell-adhesion properties are reported as clinically applicable tissue-regenerative biomaterials. New isosorbide derivatives (propoxylated or ethoxylated ones) were developed to improve mechanical properties by enhanced reactivity in copolymer synthesis compared to the original isosorbide. Optimized PCL-PU with propoxylated isosorbide exhibited notable mechanical performance (50 MPa tensile strength and 1150% elongation with hyperelasticity under cyclic load).

Managing surgery in hemophilia with recombinant factor VIII Fc and factor IX Fc: Data on safety and effectiveness from phase 3 pivotal studies.

Background: Surgical procedures impose hemostatic risk to people with hemophilia, which may be minimized by optimal factor (F) replacement therapy.

Methods: This analysis evaluates the efficacy and safety of extended half-life factor replacement recombinant FVIII and FIX Fc fusion proteins (rFVIIIFc and rFIXFc) during surgery in phase 3 pivotal (A-LONG/Kids A-LONG and B-LONG/Kids B-LONG) and extension (ASPIRE and B-YOND) studies. Dosing regimens were determined by investigators.

Illustrated State-of-the-Art Capsules of the ISTH 2022 Congress.

The ISTH London 2022 Congress is the first held (mostly) face-to-face again since the COVID-19 pandemic took the world by surprise in 2020. For 2 years we met virtually, but this year's in-person format will allow the ever-so-important and quintessential creativity and networking to flow again. What a pleasure and joy to be able to see everyone! Importantly, all conference proceedings are also streamed (and available recorded) online for those unable to travel on this occasion.

An alginate-based encapsulation system for delivery of therapeutic cells to the CNS.

Treatment options for neurodegenerative conditions such as Parkinson's disease have included the delivery of cells which release dopamine or neurotrophic factors to the brain. Here, we report the development of a novel approach for protecting cells after implantation into the central nervous system (CNS), by developing dual-layer alginate beads that encapsulate therapeutic cells and release an immunomodulatory compound in a sustained manner. An optimal alginate formulation was selected with a view to providing a sustained physical barrier between engrafted cells and host tissue, enabling exchange of small molecules while blocking components of the host immune response.

The Hemophilia Joint Health Score version 2.1 Validation in Adult Patients Study: A multicenter international study.

Background: The Hemophilia Joint Health Score (HJHS) was developed and validated to detect arthropathy in children. Additional evidence is required to show validity in adults. We studied the convergent and discriminant construct validity of the HJHS version 2.

The lived experience of women with a bleeding disorder: A systematic review.

Background: Research studies have described the morbidity associated with inherited bleeding disorders such as hemophilia and von Willebrand disease in women, but their effect on daily living has long been underrecognized. This systematic review sought to document the lived experience of women with a bleeding disorder by assessing research findings on quality of health care, socioeconomic factors, and mental health.

Methods: A systematic search was carried out in Web of Science, the Cumulative Index to Nursing and Allied Health Literature, and PubMed on July 31, 2020.

Musculoskeletal ultrasound in hemophilia: Results and recommendations from a global survey and consensus meeting.

Introduction: For persons with hemophilia, optimization of joint outcomes is an important unmet need. The aim of this initiative was to determine use of ultrasound in evaluating arthropathy in persons with hemophilia, and to move toward consensus among hemophilia care providers regarding the preferred ultrasound protocols for global adaptation.

Methods: A global survey of hemophilia treatment centers was conducted that focused on understanding how and why ultrasound was being used and endeavored to move toward consensus definitions of both point-of-care musculoskeletal ultrasound (POC-MSKUS) and full diagnostic ultrasound, terminology to describe structures being assessed by ultrasound, and how these assessments should be interpreted.

Ethnicity and COVID-19 cardiovascular complications: a multi-center UK cohort.

Background: Recent reports suggest an association between ethnicity and COVID-19 mortality. In the present multi-center study, we aimed to assess the differences underlying this association, and ascertain whether ethnicity also mediates other aspects of COVID-19 like cardiovascular complications.

Methods: Data were collected from a mixed-ethnicity UK cohort of 613 patients admitted and diagnosed COVID-19 positive, across six hospitals in London during the second half of March 2020: 292 were White Caucasian ethnicity, 203 were Asian and 118 were of Afro-Caribbean ethnicity.

Impaired platelet-dependent thrombin generation associated with thrombocytopenia is improved by prothrombin complex concentrates in vitro.

Background: Impaired thrombin generation (TG) in patients with acquired coagulopathy, is due to low coagulation factors and thrombocytopenia. The latter is typically treated with platelet transfusions and the former with plasma and occasionally with prothrombin complex concentrates (PCCs). We hypothesized that manipulating the concentrations of coagulation factors might result in restoration of platelet-dependent TG over and above that of simple replacement therapy.

Global coagulation assays in hemophilia A: A comparison to conventional assays.

Background: Global assays measure the interactions of coagulants, anticoagulants, and platelets on thrombin generation and may reflect the comprehensive coagulation potential in patients with hemophilia better than conventional assays.

Objectives: The objectives of the current study were to investigate the value of global assays for measuring and monitoring the coagulation potential of patients with hemophilia A (HA).

Patients/methods: Rotational thromboelastometry, thrombin generation assay (TGA), and activated partial thromboplastin time (APTT) clot waveform analysis were investigated in a cohort of patients with severe, moderate, and mild HA and compared with conventional assays.

Profound vitamin D deficiency in four siblings with Imerslund-Grasbeck syndrome with homozygous CUBN mutation.

Imerslund-Grasbeck syndrome (IGS, OMIM 261100) is a rare autosomal recessive disease characterized by vitamin B12 malabsorption resulting in megaloblastic anemia and asymptomatic proteinuria. IGS is caused by bi-allelic mutations in either CUBN or AMN that respectively encode the cubilin and amnionless subunits of the cobalamin-intrinsic factor receptor. We report four siblings (three boys, one girl) of non-consanguineous parents of Jewish background, aged 10 months to 12 years, with homozygous CUBN frameshift c.

Fixed doses of N8-GP prophylaxis maintain moderate-to-mild factor VIII levels in the majority of patients with severe hemophilia A.

Background: N8-GP is an extended half-life recombinant factor VIII developed for prophylaxis and treatment of bleeds in patients with hemophilia A.

Objective: To assess pharmacokinetic (PK) characteristics of N8-GP in previously treated patients with severe hemophilia A, model the time spent at hemophilia thresholds of ≥1 and ≤5 IU/dL (moderate) or >5 IU/dL (mild) FVIII levels during N8-GP prophylaxis, and investigate the relationship between N8-GP half-life and von Willebrand factor (vWF).

Methods: PK assessments were obtained from patients with severe hemophilia A (FVIII < 1 IU/dL) participating in 4 clinical trials: pathfinder 1 (20-60 years); pathfinder 2 (12-17 and ≥18 years); pathfinder 5 (0-11 years), and pathfinder 7 (25-71 years).

Meta-analysis of laparoscopic transcystic transcholedochal common bile duct exploration for choledocholithiasis.

Background: It is not clear whether laparoscopic transcystic exploration (LTCE) laparoscopic choledochotomy (LCD) is superior in the management of choledocholithiasis. In this meta-analysis, the success of LTCE LCD was evaluated.

Methods: Cochrane Central Register of Controlled Trials, Web of Science, Trip, PubMed, Ovid and Embase databases were searched systematically for relevant literature up to May 2017.

Conventional and conservative management of placenta accreta is two ends of a single continuum: A report of three cases and literature review.

Placenta accreta (PA) is a critical condition that represents a significant source of morbidity and mortality observed in women with multiple prior cesarean sections. Precise prenatal identification of affected pregnancies permits optimal obstetric management. Antenatal diagnosis leads to less blood loss and a requirement for blood transfusion than women diagnose during cesarean section.

Application of the modified Zajicek criteria to diagnose probable spinal cord neurosarcoidosis.

Neurosarcoidosis represents a significant diagnostic challenge, as clinical features overlap with other neuroinflammatory conditions, and biopsy of affected neuronal tissue is often high risk or not feasible. Here we highlight application of the modified Zajicek criteria to diagnose probable spinal neurosarcoidosis in the absence of histology from affected neuronal tissue.