The modern use of hydroxyurea for children with sickle cell anemia.

Over the past 40 years, the introduction and refinement of hydroxyurea therapy has led to remarkable progress for the care of individuals with sickle cell anemia (SCA). From initial small proof-of-principle studies to multi-center Phase 3 controlled clinical trials and then numerous open-label studies, the consistent benefits of once-daily oral hydroxyurea have been demonstrated across the lifespan. Elevated fetal hemoglobin (HbF) serves as the most important treatment response, as HbF delays sickle hemoglobin polymerization and reduces erythrocyte sickling.

A Comparison of Peripherally Inserted Central Catheter Materials.

Background: New catheter materials for peripherally inserted central catheters (PICCs) may reduce the risk of device failure due to infectious, thrombotic, and catheter occlusion events. However, data from randomized trials comparing these catheters are lacking.

Methods: We conducted a randomized, controlled, superiority trial in three Australian tertiary hospitals.

Diagnostic accuracy of early neonatal MRI in predicting adverse motor outcomes in children born preterm: Systematic review and meta-analysis.

Aim: To examine the diagnostic accuracy of Early structural and diffusion-weighted magnetic resonance imaging (MRI) (acquired at < 36 weeks postmenstrual age) to detect cerebral palsy (CP) or other adverse motor outcomes at or beyond 3 years corrected age in infants born preterm.

Method: In this systematic review and meta-analysis, we searched the CINAHL, Embase, PubMed, and Web of Science databases for studies without language restrictions and a prospectively registered protocol up to October 2023. We extracted the study details, associations presented, and meta-analyses conducted with pooled sensitivity and specificity.

High Detection Rates for Prostate-specific Membrane Antigen-avid Prostate Cancer Recurrence at Low Prostate-specific Antigen levels on Extended Axial Field-of-view Positron Emission Tomography/Computed Tomography.

Background And Objective: Although prostate-specific membrane antigen (PSMA) positron emission tomography/computed tomography (PET/CT) has impacted the investigation and management of biochemical recurrence (BCR) of prostate cancer, negative scans are common at low rising prostate-specific antigen (PSA) levels. PET/CT devices with an extended axial field-of-view, such as the Siemens Biograph Vision Quadra (Quadra) scanner, have substantially higher sensitivity than conventional field-of-view scanners. Our aim was to assess whether the enhanced signal-to-noise ratios achieved on the Quadra scanner improve detection of low-volume disease and thereby increase detection of PC at low PSA levels.

Diagnostic accuracy of neonatal structural MRI scores to predict 6-year motor outcomes of children born very preterm.

Aims: This study aimed to (1) evaluate associations between Early and Term structural MRI (sMRI) brain abnormality scores and adverse motor outcomes at 6-years corrected age (CA), (2) determine their diagnostic accuracy in predicting adverse motor outcomes and cerebral palsy (CP) at 6-years CA.

Methods: Infants born < 31-weeks gestational age (GA) returning for 6-year follow-up were included. Early and Term sMRI were scored using a validated method, deriving white matter, cortical grey matter, deep grey matter, cerebellar and global brain abnormality scores (GBAS).

The Voxelotor Effect: Decreased Affinity for New Drugs for Sickle Cell Disease?

Voxelotor (OXBRYTA) was abruptly withdrawn from the global market in September 2024. Clinicians and patients were not prepared for this, and the sudden discontinuation has caused much consternation, uncertainty, and loss of trust.

Hydroxyurea for Children and Adults with Hemoglobin SC Disease.

Background: Hemoglobin SC (HbSC) is a common sickle hemoglobinopathy that causes acute complications, chronic organ damage, and early death with no established disease-modifying treatment. In this trial, we examined the safety and efficacy of hydroxyurea treatment in patients with HbSC.

Methods: Prospective Identification of Variables as Outcomes for Treatment (PIVOT) was a double-blind, randomized, placebo-controlled, non-inferiority phase 2 trial in which we assigned children and adults with HbSC in Ghana to 12 months of hydroxyurea or placebo.

The impact of fluconazole use on the fungal and bacterial microbiomes in recurrent Vulvovaginal Candidiasis (RVVC): a pilot study of vaginal and gastrointestinal site interplay.

Purpose: Recurrent Vulvovaginal Candidiasis (RVVC) is a problematic clinical condition for which fluconazole treatment is commonly prescribed. This study investigated the interkingdom vaginal and gastrointestinal microbiomes of RVVC patients who use fluconazole intermittently or as longer-term maintenance therapy for symptom management and compared them to healthy controls.

Methods: Vaginal swabs and fecal samples were collected.

Does a Polyherbal Medicine Enhance Cognition in Healthy Older Adults? An Exploratory Open-Label Study.

This 12-week open-label study evaluated a multi-herbal tablet containing , , , , and , for potential improvement of cognitive function in 25 healthy older adults (50-75 years). The primary outcome, cognition, was assessed using the Cambridge Neuropsychological Test Automated Battery. Significant improvement in working memory was observed.

Baseline characteristics of Ghanaian children and adults enrolled in PIVOT, a randomised clinical trial of hydroxyurea in HbSC disease in sub-Saharan Africa.

HbSC disease is a common form of sickle cell disease with significant morbidity and early mortality. Whether hydroxyurea is beneficial for HbSC disease is unknown. Prospective Identification of Variables as Outcomes for Treatment (PIVOT, Trial ID PACTR202108893981080) is a double-blind, randomised, placebo-controlled phase II trial of hydroxyurea for people with HbSC, age 5-50 years, in Ghana.

Influenza Epidemiology and Vaccine Effectiveness Following Funded Influenza Vaccine in Queensland, Australia, 2022.

Background: In 2022, publicly funded influenza vaccine was made available to all residents of Queensland, Australia. This study compared influenza epidemiology in 2022 with previous years (2017-2021) and estimated influenza vaccine effectiveness (VE) during 2022.

Methods: The study involved a descriptive analysis of influenza notifications and a case-control study to estimate VE.

Total-Body PET/CT: Pros and Cons.

PET/CT devices with an axial field-of-view (FOV) of 1 m allow simultaneous imaging from the head to the upper thighs, the typical axial extent of many "whole-body" oncological studies acquired by moving a patient sequentially through a conventional FOV device, or rapid total-body imaging using the same approach. Increasing the FOV to around 2 m provides true simultaneous total-body imaging. Either approach dramatically increases the sensitivity for detection of annihilation events arising within the body.

Long guidewire peripheral intravenous catheters in emergency departments for management of difficult intravenous access: A multicenter, pragmatic, randomized controlled trial.

Background: A quarter of patients who present to emergency departments (EDs) have difficult intravenous access (DIVA), making it challenging for clinicians to successfully place a peripheral intravenous catheter (PIVC). Some literature suggests that guidewire PIVC improves first-insertion success rate.

Aim: The aim was to determine the clinical and cost-effectiveness of a novel long PIVC (5.

Oximetry and carbon dioxide screening for ventilatory requirements in children with spinal muscular atrophy type 1-3.

Introduction: Despite disease modifying treatments (DMT), assisted ventilation is commonly required in children with Spinal Muscular Atrophy (SMA). Guidelines suggest screening with oximetry and transcutaneous carbon dioxide (TcCO) for sleep disordered breathing (SDB).

Aim: To determine the utility of pulse oximetry and TcCO as a screen for SDB and the need for Non-Invasive Ventilation (NIV) in children with SMA type 1-3.

Securement to Prevent Noncuffed Central Venous Catheter Dislodgement in Pediatrics: The SECURED Superiority Randomized Clinical Trial.

Importance: Catheter dislodgement is a common complication for children with tunneled or peripherally inserted noncuffed central venous catheters (CVCs). A subcutaneous anchor securement system (SASS) may reduce this risk compared with traditional adhesive securement.

Objective: To compare dislodgement of noncuffed CVCs secured with SASS with dislodgement of noncuffed CVCs secured with sutureless securement devices (SSDs).

A pharmacokinetic-pharmacodynamic analysis of l-glutamine for the treatment of sickle cell disease: Implications for understanding the mechanism of action and evaluating response to therapy.

The mechanisms of action of l-glutamine for the treatment of sickle cell disease (SCD) are not well understood and there are no validated clinical biomarkers to assess response. We conducted a three-week, dose-ascending trial of glutamine and measured the pharmacokinetic (PK) exposure parameters, peak concentration (C) and area under the curve (AUC). We used a panel of biomarkers to investigate the pharmacodynamics (PD) of glutamine and studied PK-PD relationships.

Sensitivity and specificity of the Neonatal Visual Assessment to predict motor and cognitive outcomes in infants born very preterm.

Background: Very preterm infants are at increased risk of neurodevelopmental impairments. The Neonatal Visual Assessment (NVA) assesses visual function and outcomes and has been used to assess early neurodevelopmental outcomes. This study aimed to compare NVA results of very preterm and term-born infants and to calculate the sensitivity and specificity of the NVA at term equivalent age (TEA) and three months corrected age (CA) to predict motor and cognitive outcomes at 12 months CA in very preterm infants.

Twenty-four Month Outcomes of Extended- Versus Standard-course Antibiotic Therapy in Children Hospitalized With Pneumonia in High-risk Settings: A Randomized Controlled Trial.

Background: Pediatric community-acquired pneumonia (CAP) can lead to long-term respiratory sequelae, including bronchiectasis. We determined if an extended (13-14 days) versus standard (5-6 days) antibiotic course improves long-term outcomes in children hospitalized with CAP from populations at high risk of chronic respiratory disease.

Methods: We undertook a multicenter, double-blind, superiority, randomized controlled trial involving 7 Australian, New Zealand, and Malaysian hospitals.