A Review of Desmopressin Use in Bleeding Disorders: An Unsung Hero?

As a synthetic analogue of vasopressin, desmopressin or DDAVP has well established hemostatic properties. We present a review of DDAVP and summarize the clinical and laboratory evidence for its use in hemophilia A, von Willebrand disease (VWD), platelet function disorders, uremia, liver cirrhosis, and pregnancy, followed by illustrative examples of its broad efficacy from our local practice. In brief, DDAVP acts to release von Willebrand factor (VWF) and factor VIII from endogenously stored reserves, thereby correcting plasma deficiencies present in mild to moderately affected patients with hemophilia A and VWD.

Point-of-Care Intestinal Ultrasound Impacts Inflammatory Bowel Disease Care and Reduces the Need for Additional Investigations.

Background And Aim: Intestinal ultrasound (IUS) has emerged as a widely used point-of-care (POC) tool for guiding inflammatory bowel disease (IBD) treatment. This study aims to assess the impact of POC IUS on clinical decision-making in IBD.

Methods: We performed a retrospective review of outpatient cases utilizing POC IUS at a tertiary center between October 2021 and March 2023.

Early Infliximab Levels and Clearance Predict Outcomes After Infliximab Rescue in Acute Severe Ulcerative Colitis: Results From PREDICT-UC.

Background & Aims: The role of infliximab therapeutic drug monitoring in acute severe ulcerative colitis (ASUC) management is unknown. We aimed to identify whether infliximab therapeutic drug monitoring is associated with ASUC outcomes.

Methods: Serum and stool samples were collected from patients enrolled in the PREDICT-UC randomized controlled trial (NCT02770040), which compared intensified and standard infliximab rescue in steroid-refractory ASUC.

Enhancing treatment outcomes after gynaecological cancer (ACUMEN): a randomised controlled exercise trial protocol.

Background: Treatment for gynaecological cancer often entails challenges that negatively affect quality-of-life. While exercise has been shown to improve physical and psychosocial well-being during cancer recovery, affected women often avoid exercise due to the specific health challenges they face after treatment. The Enhancing treatment outcomes after gynaecological cancer (ACUMEN) program aims to enhance health-related quality of life in this group by promoting lifelong exercise habits.

Evaluation of the TrueBeam machine-performance-check (MPC): Collimator device check (CDC).

Purpose: To evaluate the Varian machine performance check (MPC) collimator devices check (CDC) for routine MLC and jaw testing as part of an AAPM compliant linac QA program.

Methods: CDC MLC positioning, MLC backlash, jaw positioning, and jaw parallelism were each assessed for repeatability and concordance with conventional QA. MLC and jaw positioning were also assessed for sensitivity.

Testicular Function After Immune-Checkpoint Inhibitors Treatment.

Objective: To investigate the effects of immune-checkpoint inhibitors (ICIs) on spermatogenesis and testicular endocrine function in reproductive-age men with melanoma.

Design: Prospective, mixed longitudinal and cross-sectional cohort study.

Patients: Twenty-nine men aged 19-46 years undergoing ICI therapy for melanoma at two Australian centres between 2019 and 2024.

Pretreatment and on-treatment ctDNA and tissue biomarkers predict recurrence in patients with stage IIIB-D/IV melanoma treated with adjuvant immunotherapy: CheckMate 915.

Purpose: CheckMate 915 (NCT03068455) compared adjuvant nivolumab monotherapy versus combination nivolumab+ipilimumab in patients with resected stage III/IV melanoma. This exploratory analysis was performed to identify biomarkers that correlate with benefit from adjuvant immunotherapy.

Patients And Methods: 1,844 patients received nivolumab 480 mg every 4 weeks or nivolumab 240 mg every 2 weeks with ipilimumab 1 mg/kg every 6 weeks.

Stereoelectroencephalography for Epilepsy Presurgical Assessment: A Nationwide Survey of Evolution of Practice in Australia.

Background And Objectives: Stereoelectroencephalography (SEEG) is increasingly used worldwide for epilepsy presurgical evaluation. A data-driven view of SEEG practice evolution is lacking for any world region. We aimed to perform a nationwide survey of SEEG use in Australian adult epilepsy centers.

Clinical Characteristics and Outcomes in Patients With Cryptococcaemia From a Large Population-Based Cohort.

Background: Cryptococcus bloodstream infections (BSIs) or cryptococcaemia are severe opportunistic infections with high mortality, predominantly affecting immunocompromised individuals or those with end-stage organ disease. Population-based studies examining infection trends and associations between host factors, geography, antifungal resistance, and clinical outcomes are few.

Methods: Blood cultures with growth of Cryptococcus species were retrospectively identified over a 20-year period (January 1, 2000-December 31, 2019) from a state-wide database.

Directionality of neural activity in and out of the seizure onset zone in focal epilepsy.

Epilepsy affects over 50 million people worldwide, with approximately 30% experiencing drug-resistant forms that may require surgical intervention. Accurate localisation of the epileptogenic zone (EZ) is crucial for effective treatment, but how best to use intracranial EEG data to delineate the EZ remains unclear. Previous studies have used the directionality of neural activities across the brain to investigate seizure dynamics and localise the EZ.

HIV testing in primary care in the west of Ireland: a mixed-method survey between 2013 and 2023.

Background: 2022 saw the largest number of HIV cases diagnosed in Ireland on record with a 122% increase on 2021 and a 68% increase compared to the pre-pandemic year of 2019. Late-stage diagnoses and difficulties accessing testing are more common outside of Dublin.

Aim: The aim of this survey was to assess practices and attitudes to testing in general practice in the west of Ireland.

Nivolumab and Ipilimumab Combination Treatment in Advanced Ovarian and Endometrial Clear Cell Cancers: A Nonrandomized Clinical Trial.

Importance: Gynecological clear cell cancers (CCCs) are aggressive malignant neoplasms with low response rate to chemotherapy. The treatment of patients with metastatic disease remains an area of significant unmet need.

Objective: To evaluate the efficacy of combined anti-programmed cell death 1 protein (PD-1)/cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) blockade using nivolumab and ipilimumab in advanced gynecological CCCs.

Real-World Outcomes of Pembrolizumab in Advanced Melanoma by Age and Sex: A National Population-Based Study.

Background: Immune checkpoint inhibitors are standard treatment for advanced melanoma. Pembrolizumab (programmed cell death-1 inhibitor) monotherapy is recommended as first-line treatment. However, real-world evidence on its efficacy and safety in Australia, a region with the highest melanoma incidence, remains limited.

McCune-Albright syndrome: a case of an adult with fibrous dysplasia, severe cardiopulmonary complications, acromegaly, and chronic myeloid leukemia.

McCune-Albright syndrome (MAS) is a rare mosaic disorder characterized by the classic triad of fibrous dysplasia of bone (FD), café-au-lait skin macules, and hyperfunctioning endocrinopathies. MAS is caused by a postzygotic mutation in the G-protein alpha subunit (GNAS) gene resulting in G-protein α-subunit somatic activation. There is no approved treatment for MAS.

Turning the tide on bronchiectasis in adult Aboriginal Australians: from neglect to action.

Chronic respiratory disorders are highly prevalent among adult Aboriginal and Torres Strait Islander peoples, with emerging evidence suggesting that there is a significant burden of bronchiectasis contributing to overall higher morbidity and mortality. Despite this, bronchiectasis in adult Aboriginal Australians has attracted little attention in the past few decades, and only recently is published evidence on this topic beginning to emerge. This paper highlights recent insights into the disease profiles of bronchiectasis among adult Aboriginal populations, revealing a particularly high prevalence among rural and remote residing Aboriginal people that is disproportionately higher in comparison to global diverse ethnic cohorts.

Expert Position Statement: Defining the Role of Intestinal Ultrasound in Assessing Constipation and Faecal Loading.

Background: Intestinal ultrasound has been proposed as a tool to assess non-inflammatory functional gastrointestinal disorders, including constipation and faecal loading. However, well-defined sonographic criteria for these conditions are currently lacking.

Aim: To identify and establish specific sonographic parameters that could be used to assess constipation and faecal loading with intestinal ultrasound.

Textbook Outcomes for Retroperitoneal Sarcoma Resection: A Multi-Centre Review.

For patients with retroperitoneal sarcomas (RPSs), en-bloc resection with macroscopically negative margins remains the only potentially curative treatment. Textbook outcomes (TOs) are composite measures developed to compare ideal surgical outcomes for complex oncologic resections. The aims of this study were as follows: to define TO for RPS resections; to investigate the impact of treating service and other variables on TO; and to investigate the impact of treating service on achieving a TO.

Patterns of care for vulval cancer treatment: the Queensland experience.

Objective: To evaluate patterns of care, post-operative adverse events, and recurrence rates in patients with vulval squamous cell carcinoma, undergoing sentinel node biopsy or inguino-femoral lymph node dissection.

Methods: A retrospective analysis was conducted on a cohort of 124 patients with vulval squamous cell carcinoma between 2016 and 2020 who met the study eligibility criteria. We compared the proportion of patients who underwent sentinel node biopsy versus inguino-femoral lymph node dissection, along with their rates of post-operative adverse events, recurrences, and associated socioeconomic factors.

Outcomes for high-risk defining events in follicular lymphoma following frontline immunochemotherapy.

Progression of follicular lymphoma (FL) or transformation (TFL) within 24 months of immunochemotherapy (ICT) represent high-risk defining events (HRDE) with poor overall survival (OS). We examined baseline clinical characteristics, imaging, and outcomes for patients experiencing HRDE with newly diagnosed FL requiring ICT. HRDE groups were: relapse or progression of FL within 24 months (FL24), early TFL (transformation <24 months of ICT), late TFL (transformation >24 months of ICT).

Predictive performance of the clinicopathologic gene expression profile (CP-GEP) in identifying cutaneous melanoma patients for whom sentinel lymph node biopsy is unnecessary: A systematic review and meta-analysis.

Context & Aim: Sentinel lymph node biopsy (SLNB) is an invasive procedure that detects microscopic nodal metastasis, crucial for accurate staging and optimal management. In melanoma, most patients who undergo the procedure have no sentinel lymph node (SLN) metastasis detected. The CP-GEP model (Merlin Assay) was developed to identify patients who do not have SLN metastases and who may therefore safely forgo SLNB, based upon clinicopathologic and gene expression features of the primary tumour.

Do BRAF-targeted therapies have a role in the era of immunotherapy?

Over half of cutaneous melanomas have BRAF mutations, with this mutation being more prevalent in younger patients who often present with more aggressive disease. BRAF-targeted therapy and checkpoint inhibitor immunotherapy have led to marked improvements in outcomes for patients with BRAF-mutant melanoma. Despite these advances, novel combinatorial strategies are vital given that more than half of advanced melanoma patients will still die due to melanoma.

Patient Preferences for Treatment Attributes in Inflammatory Bowel Disease: A Discrete Choice Experiment Among Patients in Five Non-Western Countries.

Introduction: Understanding patient preferences is important for facilitating informed decision-making in managing patients with inflammatory bowel disease (IBD). This study explored the preferences of patients with Crohn's disease (CD) and ulcerative colitis (UC) regarding advanced therapy treatment attributes in five non-Western countries: Argentina, Australia, Brazil, Saudi Arabia and Taiwan.

Methods: Adult patients with self-reported CD or UC treated for ≥ 6 months participated in this cross-sectional online survey.