Monitoring and Treatment of Paroxysmal Nocturnal Hemoglobinuria in Patients with Aplastic Anemia in Asia: An Expert Consensus.

Paroxysmal nocturnal hemoglobinuria (PNH) clones can be identified in a significant proportion of patients with aplastic anemia (AA). Screening for PNH clones at the time of an AA diagnosis is recommended by national and international guidelines. In this report, an expert panel of physicians discusses current best practices and provides recommendations for managing PNH in patients with AA in the Asia-Pacific region.

The Impact of Genetic Mutations on the Efficacy of Immunotherapies in Lung Cancer.

Lung cancer is the leading cause of cancer-related mortality worldwide, primarily driven by genetic mutations. The most common genetic alterations implicated in lung cancer include mutations in , , , , , , , , , , , and . Targeted therapies have been developed to inhibit cancer growth by focusing on these specific genetic mutations.

Alternate Sampling Matrices for Therapeutic Drug Monitoring of Immunosuppressants.

Background: Immunosuppressant (IS) therapeutic drug monitoring (TDM) relies on measuring mostly pharmacologically inactive erythrocyte-bound and/or plasma protein-bound drug levels. Variations in hematocrit and plasma protein levels complicate interpretation of blood calcineurin inhibitor (CNI) and inhibitors of the molecular target of rapamycin (mTORi) concentrations. Variable binding of mycophenolic acid (MPA) to albumin similarly complicates its TDM in plasma.

Mucosal Spread of Sinonasal Adenocarcinoma into the Nasolacrimal Duct Without Bony Erosion of the Nasolacrimal Canal: A Case Report.

Sinonasal malignancy is a rare but recognized cause for nasolacrimal obstruction leading to epiphora. While direct mucosal spread through the nasolacrimal duct can occur in benign sinonasal tumors such as inverting papilloma, the same phenomenon has not been described in malignant lesions. The authors present a case of a low-grade nonintestinal type sinonasal adenocarcinoma centered on the inferior meatus of the sinonasal cavity, showing mucosal invasion into the nasolacrimal duct with bony expansion but no erosion of the nasolacrimal canal.

Alignment of Palliative Care Service Structure and Standards of Care for Adolescents and Young Adults with Cancer: An International Survey of Clinical Practice.

Access to timely, age-appropriate palliative care services and end-of-life communication are two standards of care for adolescents and young adults (AYAs) living with cancer where cure is uncertain or unlikely. Health professionals' capacity to facilitate these standards is critical. This study aimed to understand AYA oncology health professionals' experienced practices in, and barriers to, delivering these standards of care across palliative care and end-of-life communication in Australia, New Zealand, and the United Kingdom (UK).

Clinical and Metabolic Characteristics and Follow-Up Results of Adrenal Incidentalomas: A 10-Year Experience.

Objective: In this retrospective study, we aimed to investigate the clinical, hormonal, and radiological characteristics of patients with adrenal incidentalomas (AIs), to assess the prevalence of metabolic syndrome components in patients with AIs, and to determine whether changes in tumor size or hormonal activity occur during long-term follow-up in patients with AIs.

Methods:  We retrospectively analyzed data from 384 patients diagnosed with AI between 2010 and 2020. Data regarding radiological, hormonal, and metabolic investigations in diagnosis and also during follow-ups were collected.

Changes in peak oxygen consumption in Fabry disease and associations with cardiomyopathy severity.

Background: Fabry disease (FD) causes multiorgan sphingolipid accumulation, with cardiac involvement responsible for the largest burden of morbidity and mortality. Exercise intolerance in FD is prevalent, yet the mechanisms of this are poorly understood. The aim of this study was to assess exercise intolerance in FD and identify whether this correlates with the phase of cardiomyopathy.

Sonographic Sling Position and the Outcome of the Tension-Free Vaginal Tape-Obturator in Asian Chinese.

Introduction And Hypothesis: The objective was to assess the sonographic tension-free vaginal tape-obturator (TVT-O) position and the outcome in Asian Chinese women.

Methods: A prospective cohort study of 254 patients who underwent TVT-O surgery between 2013 and 2022. The sonographic position of the TVT-O was recorded and correlated with the outcomes, including the subjective cure rates, Incontinence Impact Questionnaire 7 (IIQ-7), and retention of urine.

Enhancing wellbeing in medical practice: Exploring interventions and effectiveness for improving the work lives of resident (junior) doctors: A systematic review and narrative synthesis.

Introduction: Globally, resident doctors face challenges like long work hours, critical decision-making stress, and exposure to death and distress, prompting concern for their wellbeing. This study addresses the need for interventions to improve their working conditions, vital for enhancing quality of life, patient care and retaining a skilled workforce.

Methods: Following PRISMA guidelines, a systematic literature review until 3 January 2024 explored interventions for resident Ddoctors pre- and post-COVID-19.

Assessment of the BODE Index and Its Association With Inflammatory Mediators in Chronic Obstructive Pulmonary Disease (COPD) Patients.

Background Chronic obstructive pulmonary disease (COPD) is a respiratory condition impacting daily activities of susceptible individuals and increasing the risk of respiratory infections and cardiovascular disease. Body Mass Index, Airflow Obstruction, Dyspnea, and Exercise Capacity (BODE) index is applied clinically to measure the survival of COPD patients. Inflammatory mediators, including cytokines and chemokines, significantly contribute to the COPD pathology.

Cecal volvulus in Marfan Syndrome.

Gastrointestinal pathology in adult patients with Marfan Syndrome is rarely reported in literature. Nevertheless, it could be life threatening when it occurs. In our paper, we are presenting the first reported case of caecal volvulus in an adult patient with Marfan Syndrome, our findings and management.

Safe and well-tolerated long-term parenteral nutrition regimen: Omega-3-fatty-acid-enriched medium chained/ long chained triglycerides emulsion.

Background And Aims: A limited number of randomized controlled trials (RCTs) have examined the use of lipid emulsions (LEs) of different compositions in home parenteral nutrition (HPN), and there are very few data on the long-term use of omega-3 (n-3) polyunsaturated fatty acids (PUFAs). The study's objective was to assess safety and tolerability of an n-3 PUFA-enriched LE in adult patients suffering from chronic intestinal failure (CIF) requiring long-term HPN.

Methods: In this prospective, randomized, controlled, double-blind, multicentre, international clinical trial, which was conducted at eleven sites, adult patients in need of HPN including lipids received either the investigational product, an n-3 PUFA-enriched medium/long-chain triglyceride (MCT/LCT) LE, or the reference product, a standard MCT/LCT LE, for an average duration of eight weeks.

Atherectomy Techniques: Rotablation, Orbital and Laser.

Coronary artery disease remains the leading cause of morbidity and mortality worldwide despite advancements in percutaneous coronary intervention (PCI). With an increasing ageing population, there is a significant challenge in addressing severe calcification in atherosclerotic plaque during angioplasty. This review article focuses on atherectomy strategies such as rotational atherectomy (RA), orbital atherectomy (OA) and excimer laser coronary angioplasty (ELCA) aimed at modifying calcified lesions and improving PCI outcomes.

The enigmatic role of SIRT2 in the cardiovascular system: Deciphering its protective and detrimental actions to unlock new avenues for therapeutic intervention.

Cardiovascular diseases (CVDs) are leading causes of mortality throughout the world, and hence, there is a critical need to elucidate their molecular mechanisms. The Sirtuin (SIRT) family of NAD+-dependent enzymes has recently been shown to play a critical role in cardiovascular health and disease, and several SIRT isoforms, especially SIRT1 and SIRT3, have been amply investigated. However, the precise function of SIRT2 is only partially explored.

UKHCDO gene therapy taskforce: Guidance for implementation of haemophilia gene therapy into routine clinical practice for adults.

Introduction: 2022 was a landmark year with two adeno-associated viral vectors (AAVs) receiving conditional marketing authorization from EMA for the treatment of persons with severe haemophilia A and severe to moderately severe haemophilia B and a third in 2024. Gene therapy is a transformative, irreversible treatment with long-lasting effects, necessitating development of new clinical pathways to ensure optimal outcomes.

Aim: To develop a consensus framework and service specification for delivery of AAV gene therapy for haemophilia in adults within the UK using the hub-and-spoke model proposed by the European Association of Haemophilia and Allied Disorders and the European Haemophilia Consortium.