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TrialR: critical enablers and the need for reusable Rare Disease Clinical Trial infrastructure in Western Australia

Citation: MacDonald B, Burmaz M, Baker S, et al. TrialR: critical enablers and the need for reusable Rare Disease Clinical Trial infrastructure in

Pharmacokinetics of a three-dose regimen of intramuscular benzathine penicillin G in pregnant women with syphilis

Syphilis remains a global public health concern, particularly in pregnancy due to the risk of congenital syphilis. The WHO recommends benzathine penicillin G (BPG) as the standard treatment in pregnant women, whilst the optimal dosing strategy has not been established, particularly in relation to the impact of gestational age. This study sought to characterize the pharmacokinetics (PK) of a three-dose regimen of intramuscular BPG, given at weekly intervals, in pregnant women with syphilis.

A Pragmatic Bayesian Adaptive Trial Design Based on the Value of Information: The Value-Driven Adaptive Design

Clinical trial designs are typically narrowly focused on error control in hypothesis testing, but this approach is inadequate in many contexts, particularly when a decision maker intends to, or must, consider multiple relevant clinical and health economic outcomes under uncertainty. Value-of-information (VoI) metrics can be used to estimate the monetary value of data collection to the decision maker. 

BEAT-BK: An Adaptive, Randomized Controlled Trial to Treat Polyomavirus Infections (BKPyV) in Kidney and Kidney-pancreas Transplantation Recipients (BEAT-BK) Study Protocol

BK polyomavirus (BKPyV) is a common opportunistic infection in kidney transplant recipients, typically reactivating in the context of immunosuppression. Although asymptomatic in immunocompetent individuals, reactivation in transplant recipients can cause BKPyV-associated nephropathy (BKPyVAN), a leading cause of graft dysfunction and loss. BKPyV viremia affects approximately 10%-15% of transplant recipients, and once BKPyVAN is established, the risk of graft failure can exceed 50%.

Higher Promoter Methylation of the Ubiquitin-Associated and SH3 Domain Containing A (UBASH3A) Gene Is Associated With T-Lymphocyte Ontogeny and Reduced Susceptibility to Early-Onset Sepsis

We investigated the genetic and epigenetic regulation of the UBASH3A gene and its association with early-onset sepsis. Using matched whole blood DNA methylation, gene expression, genotypes, and immune cell counts from the EPIC-HIPC newborn cohort, we report that promoter methylation was negatively correlated with ontogenetic changes in UBASH3A gene expression and circulating CD3+ T-cell numbers. 

Fabric contamination and effective laundering for managing skin conditions: A systematic review

Household fabrics, such as clothes, bedding, and towels, are in close contact with the skin and are assumed to play a role in the transmission of skin pathogens/ectoparasites. International public health advice for managing skin conditions therefore usually includes recommendations to wash clothes and bedding. However, such advice is often general and inconsistent between sources.

Understanding parental decisions to decline or delay infant RSV immunisation, nirsevimab, in Western Australia in 2024

In 2024, the government of Western Australia introduced 'nirsevimab', a monoclonal antibody offering protection from respiratory syncytial virus (RSV), for eligible infants. This study explores why parents of infants who were eligible to receive nirsevimab opted to decline or delay the immunisation.

Challenges and insights in self-swab surveillance for asymptomatic Streptococcus pyogenes carriage

Asymptomatic carriage of Streptococcus pyogenes (Strep A) may contribute to transmission, yet its role remains poorly understood and evidence on optimal detection methods is limited. While self-collected throat swabs are used in infectious disease surveillance, their value for identifying asymptomatic Strep A carriage in adults is uncertain. This pilot prospective cohort study, conducted at a Perth medical research institute between August and October 2024, assessed feasibility and acceptability of self-collection, with sensitivity as a secondary objective. 

Modelling Micro-Elimination: Third-Trimester Tenofovir Prophylaxis for Perinatal Transmission of Hepatitis B in the Remote Dolpa District of Nepal

Hepatitis B (HBV) prevalence is very high in pregnant women in the Dolpa district of Nepal, a region characterised by a remote geographic landscape and low vaccination coverage. Using mathematical modelling, we evaluated the impact of third-trimester tenofovir disoproxil fumarate (TDF) prophylaxis on HBV burden and estimated the time required to achieve HBV elimination in Dolpa. 

Safety, tolerability, and pharmacokinetics of a 2 g subcutaneous dose of ceftriaxone as an alternative to intravenous delivery

Subcutaneous delivery of antibiotics is a practical alternative to intravenous administration. Ceftriaxone is commonly used for a variety of infections with limited data on the safety and pharmacokinetics of a 2 g subcutaneous dose. This was a prospective, self-controlled cross-over study in 20 stable inpatients receiving ceftriaxone for their infection. Following an intravenous dose, participants received a single dose of 2 g subcutaneous ceftriaxone, in 50 mL normal saline via gravity feed.