The Impact of Emicizumab Prophylaxis on Hospitalizations and Emergency Department Visits Among Hemophilia A Patients Is Age Related.

Introduction: Hemophilia A (HA) therapy poses a significant healthcare burden. In recent years, emicizumab has been approved for prophylaxis. The current study explores the impact of transitioning to emicizumab on emergency department (ED) visits and hospitalizations in pediatric and adult HA patients.

Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review.

Damoctocog alfa pegol (BAY 94-9027, Jivi), is a site-specifically PEGylated, extended half-life recombinant factor VIII (FVIII) that is approved in several European and non-European countries for on-demand treatment and prophylaxis of bleeding in previously treated patients aged ≥ 12 years with hemophilia A. Reliable measurements can be obtained using most one-stage and chromogenic FVIII assays over a wide concentration range. The efficacy, safety and pharmacokinetics (PK) of damoctocog alfa pegol have been studied extensively in the PROTECT VIII clinical trials, and its long-term safety and effectiveness profile is continuing to build through observational and interventional real-world studies.

Management of surgery in persons with hemophilia A receiving emicizumab prophylaxis: data from a national hemophilia treatment center.

Background: Persons with hemophilia A may require surgical procedures. Real-world data on invasive procedures in persons with hemophilia A receiving emicizumab prophylaxis are limited.

Objectives: To evaluate the safety of invasive procedures in persons with hemophilia A receiving emicizumab prophylaxis and their outcomes in a longitudinally followed cohort.

Four Decades of Carrier Detection and Prenatal Diagnosis in Hemophilia A: Historical Overview, State of the Art and Future Directions.

Hemophilia A (HA), a rare recessive X-linked bleeding disorder, is caused by either deficiency or dysfunction of coagulation factor VIII (FVIII) resulting from deleterious mutations in the F8 gene encoding FVIII. Over the last 4 decades, the methods aimed at determining the HA carrier status in female relatives of HA patients have evolved from phenotypic studies based on coagulation tests providing merely probabilistic results, via genetic linkage studies based on polymorphic markers providing more accurate results, to next generation sequencing studies enabling highly precise identification of the causative F8 mutation. In parallel, the options for prenatal diagnosis of HA have progressed from examination of FVIII levels in fetal blood samples at weeks 20-22 of pregnancy to genetic analysis of fetal DNA extracted from chorionic villus tissue at weeks 11-14 of pregnancy.

Efficacy and safety of damoctocog alfa pegol prophylaxis in patients ⩾40 years with severe haemophilia A and comorbidities: analysis from the PROTECT VIII study.

Background: Advances in treatment have enabled patients with haemophilia A to live longer and therefore may be subjected to comorbidities associated with ageing, in addition to disease-associated morbidities. There have been few reports to date on efficacy and safety of treatment specifically in patients with severe haemophilia A and comorbidities.

Objective: To explore the efficacy and safety of damoctocog alfa pegol prophylaxis in patients with severe haemophilia A aged ⩾40 years with comorbidities of interest.

Non-typhoidal soft-tissue infection after gender affirming subcutaneous mastectomy case report.

We present a case of a 32-year-old transgender male who underwent chest masculinization, complicated by purulent soft tissue infection of bilateral chest incisions. Cultures tested positive for non-typhoidal , methicillin-resistant , and . Herein, we discuss multiple factors contributing to the complexity of treating this patient's clinical course.

Altered abundances of human immunoglobulin M and immunoglobulin G subclasses in Alzheimer's disease frontal cortex.

The immune system has been described to play a role in the development of Alzheimer's disease (AD), but the distribution of immunoglobulins and their subclasses in brain tissue has not been explored. In this study, examination of pathologically diagnosed frontal cortex gray matter revealed significantly higher levels of IgM and IgG in late-stage AD (Braak and Braak stages V and VI) compared to age-matched controls. While levels of IgG2 and IgG4 constant region fragments were higher in late-stage AD, concentration of native-state IgG4 with free Fc regions was increased in AD III and VI.

Molecular Mechanisms of Skewed X-Chromosome Inactivation in Female Hemophilia Patients-Lessons from Wide Genome Analyses.

Introduction: Hemophilia A (HA) is an X-linked bleeding disorder caused by factor VIII (FVIII) deficiency or dysfunction due to F8 gene mutations. HA carriers are usually asymptomatic because their FVIII levels correspond to approximately half of the concentration found in healthy individuals. However, in rare cases, a carrier may exhibit symptoms of moderate to severe HA primarily due to skewed inactivation of her non-hemophilic X chromosome.

Women with Hemophilia: Case Series of Reproductive Choices and Review of Literature.

 Very little is known regarding reproductive choices, pregnancy, and delivery of women with moderate to severe hemophilia. Our aim was to describe our experience with three hemophiliac women and their journey to achieve motherhood.  Medical charts of women with moderate to severe hemophilia A treated at our center were evaluated.

Confirmed long-term safety and efficacy of prophylactic treatment with BAY 94-9027 in severe haemophilia A: final results of the PROTECT VIII extension study.

Introduction: The phase 2/3 PROTECT VIII main study demonstrated efficacy and safety of BAY 94-9027 (damoctocog alfa pegol; Jivi ), a B-domain-deleted recombinant factor VIII (FVIII), site-specifically PEGylated to extend its half-life.

Aim: To report the final efficacy and safety data for BAY 94-9027 from the PROTECT VIII extension.

Methods: Previously treated males aged 12-65 years with severe haemophilia A (FVIII <1%) who completed the multicentre, open-label PROTECT VIII main study were eligible for the extension.

The potential role of emicizumab prophylaxis in severe von Willebrand disease.

Background: Severe von Willebrand disease (VWD) may be associated with chronic joint damage and may require prophylactic therapy. Emicizumab is a humanized bispecific antibody, which mimics the function of coagulation factor VIII (FVIII), and it has been approved for prophylaxis in hemophilia A.

Methods: This is the first study assessing the potential future role of emicizumab as an alternative prophylactic treatment in patients with severe VWD, based upon a thrombin generation (TG) ex vivo analysis.

A Call to Arms: Emergency Hand and Upper-Extremity Operations During the COVID-19 Pandemic.

Purpose: Limited data exist regarding volumetric trends and management of upper-extremity emergencies during periods of social restriction and duress, such as the coronavirus disease 2019 pandemic. We sought to study the effect of shelter-in-place orders on emergent operative upper-extremity surgery.

Methods: All patients undergoing emergent and time-sensitive operations to the finger(s), hand, wrist, and forearm were tracked over an equal number of days before and after shelter-in-place orders at 2 geographically distinct Level I trauma centers.

Dorsal migration of lumbar disc fragments causing cauda equina syndromes: A three case series and literature review.

Background: Dorsal migration of an intervertebral lumbar disc fragment is exceedingly rare and may result in spinal cord or cauda equina compression. Radiologically, these lesions may be misdiagnosed as extradural masses or epidural hematomas.

Case Description: We present three cases involving dorsal migration of sequestered lumbar disc fragments resulting in cauda equina syndromes.

mutations reduce T-synthase activity in advanced Alzheimer's disease.

Introduction: Mutations in brain tissues that cumulate with age may contribute to Alzheimer's disease (AD). Abnormal glycoprotein and Tn antigen expression have been demonstrated in AD. We identified / mutations in AD and age-matched normals without AD.

Comparing Physician and Patient Perspectives on Prophylactic Treatment with BAY 94-9027 for Severe Haemophilia A: A Post Hoc Analysis.

Introduction: BAY 94-9027 is a newly developed extended half-life product to treat haemophilia, allowing for fewer injections than with standard products. This post hoc analysis aimed to compare physicians' and patients' opinions on BAY 94-9027 prophylaxis, and explore how qualitative interview data is aligned with the data from the Haemophilia-specific Quality of Life questionnaire for Adults (Haemo-QoL-A).

Methods: Exploratory qualitative interviews were conducted with physicians and patients by phone upon the exit of patients from the PROTECT VIII extension phase following a semi-directed guide.

BAY 81-8973 demonstrated efficacy, safety and joint status improvement in patients with severe haemophilia A in the LEOPOLD I extension for ≤2 years.

Objectives: BAY 81-8973 (Kovaltry ), a full-length, unmodified, recombinant human factor VIII, provided excellent bleeding control for patients with haemophilia A in the pivotal 1-year LEOPOLD I trial. The LEOPOLD I extension evaluated long-term efficacy and safety of BAY 81-8973 prophylaxis.

Methods: After completing LEOPOLD I, patients continued receiving 20-50 IU/kg BAY 81-8973 two- or three-times weekly in the extension.

BAY 94-9027 prophylaxis is efficacious and well tolerated for up to >5 years with extended dosing intervals: PROTECT VIII extension interim results.

Introduction: BAY 94-9027 is an extended-half-life, site-specifically PEGylated, B-domain-deleted recombinant factor VIII (FVIII). The PROTECT VIII main study demonstrated efficacy of bleed control using extended-interval prophylaxis with BAY 94-9027 for 36 weeks.

Aim: To report long-term efficacy and safety of prophylaxis with BAY 94-9027 in a descriptive analysis of the ongoing PROTECT VIII extension with a total treatment time of up to >5 years.

Safety and efficacy of BAY 94-9027, an extended-half-life factor VIII, during surgery in patients with severe hemophilia A: Results of the PROTECT VIII clinical trial.

Introduction: Ensuring hemostasis during invasive procedures is a challenge in patients with severe hemophilia A. This analysis evaluated efficacy and safety of BAY 94-9027, an extended-half-life recombinant factor VIII (FVIII), in the surgical setting.

Materials And Methods: Patients participating in an open-label BAY 94-9027 clinical trial who underwent major surgery were included in the analysis.

Age and Number of Surgeries Increase Risk for Complications in Polytrauma Patients with Operative Maxillofacial Fractures.

Background: Poly-trauma patients often sustain complex head/neck injuries requiring prolonged hospitalizations and multiple operations. Few studies have evaluated the associated injury patterns and risk factors for poor clinical outcomes.

Methods: Consecutive poly-trauma patients with operative maxillofacial fractures treated at a level 1 trauma medical center between 1995 and 2013 were evaluated.

Endoscopic sleeve gastroplasty versus laparoscopic sleeve gastrectomy: a case-matched study.

Background And Aims: Endoscopic sleeve gastroplasty (ESG) reduces the gastric lumen to a size comparable with that of laparoscopic sleeve gastrectomy (LSG). However, there is a paucity of research comparing outcomes between the 2 procedures. Our study compared the 6-month weight loss outcomes and adverse events of ESG with LSG in a case-matched cohort.

Nonoperative management of complicated hiatal hernia after transhiatal esophagectomy- case report.

Introduction: Hiatal hernia is rare after transhiatal esophagectomy occurring in less than 2% of patients [5]. Due to the rare incidence of hiatal hernias after transhiatal esophagectomy overlooking this differential in a symptomatic patient can be problematic. Patients can presents with recurrent pneumonia, complaints of reflux, and in the case presented small bowel obstructions.