Leptomeningeal metastatic disease: new frontiers and future directions.

Leptomeningeal metastatic disease (LMD), encompassing entities of 'meningeal carcinomatosis', neoplastic meningitis' and 'leukaemic/lymphomatous meningitis', arises secondary to the metastatic dissemination of cancer cells from extracranial and certain intracranial malignancies into the leptomeninges and cerebrospinal fluid. The clinical burden of LMD has been increasing secondary to more sensitive diagnostics, aggressive local therapies for discrete brain metastases, and improved management of extracranial disease with targeted and immunotherapeutic agents, resulting in improved survival. However, owing to drug delivery challenges and the unique microenvironment of LMD, novel therapies against systemic disease have not yet translated into improved outcomes for these patients.

Development of Prediction Models for Severe Pain and Urinary Symptoms After Ureteroscopy With Ureteral Stent Placement: Results From the STENTS Study and Initial Validation of Pain Interference.

Purpose: We developed prediction models for severe pain and urinary symptoms after ureteroscopy with ureteral stent placement.

Materials And Methods: The development cohort included 424 adults and adolescents enrolled in the multicenter STENTS prospective cohort study who underwent ureteroscopy with stent placement for urinary stones. The validation cohort was an independent prospective cohort of 115 adults.

Endoscopic endonasal resection of a craniopharyngioma in a 23-month-old patient: illustrative case.

Background: Treatment of pediatric craniopharyngioma requires a multidisciplinary approach to counsel patients and families on the spectrum of treatment options, including biopsy, radiation, and/or resection. Gross-total resection can avoid radiation and its long-term comorbidities. In very young patients, this is of particular importance but is especially challenging because of anatomical considerations.

Respiratory Syncytial Virus Disease Burden and Nirsevimab Effectiveness in Young Children From 2023-2024.

Importance: During the 2023-2024 respiratory syncytial virus (RSV) season in the United States, 2 new RSV prevention products were recommended to protect infants in their first RSV season: nirsevimab and Pfizer's maternal RSV vaccine. Postlicensure studies are needed to assess prevention product impact and effectiveness.

Objective: To compare the epidemiology and disease burden of medically attended RSV-associated acute respiratory illness (ARI) among children younger than 5 years during the 2023-2024 RSV season with 3 prepandemic RSV seasons (2017-2020), estimate nirsevimab effectiveness against medically attended RSV-associated ARI, and compare nirsevimab binding site mutations among circulating RSV in infants with and without nirsevimab receipt.

How Can Implementation Science Advance Behavioral Interventions in Preschool? A Scoping Review and Recommendations.

Behavioral interventions delivered in preschools can help young children who need support for their behavior. However, preschool teachers face barriers to implementing behavioral interventions, leading to a research-to-practice gap. To better understand how to support preschool teachers, we conducted a scoping review of determinants (i.

Blinatumomab in Standard-Risk B-Cell Acute Lymphoblastic Leukemia in Children.

Background: B-cell acute lymphoblastic leukemia (B-cell ALL) is the most common childhood cancer. Despite a high overall cure rate, relapsed B-cell ALL remains a leading cause of cancer-related death among children. The addition of the bispecific T-cell engager molecule blinatumomab (an anti-CD19 and anti-CD3 single-chain molecule) to therapy for newly diagnosed standard-risk (as defined by the National Cancer Institute) B-cell ALL in children may improve outcomes.

Initiation of a Pediatric Movement Clinic in an Urban Setting.

Physical activity is an essential component to overall health and wellness. Disparities and barriers to equitable access to physical activity exist and are more common in urban, under resourced and minority communities. Health care providers play an essential role in supporting patients to reach ideal physical activity goals.

Diagnostic Test Characteristics of Ultrasound-Based Hydronephrosis for Chronic Kidney Disease in Children and Adolescents With Myelomeningocele: Results From the UMPIRE and NSBPR Cohort Studies.

Purpose: Renal ultrasounds are performed in patients with myelomeningocele to screen for markers of kidney health, including hydronephrosis. We evaluated the diagnostic accuracy of hydronephrosis to screen for low kidney function defined by estimated glomerular filtration rate (eGFR).

Materials And Methods: We performed a retrospective cross-sectional study using data from 2 cohorts of children and youth with myelomeningocele.

Digital divide: Differences between digital access, preference, and use among parents of pediatric research participants.

Objective: Healthcare delivery and research are moving increasingly towards digital engagement, which could inequitably exclude some populations due to differential technology access and uptake. We sought to evaluate study-related digital access, preferences, and use among parents of patients with Medicaid insurance enrolled in research in a pediatric emergency department (ED).

Methods: We conducted a secondary analysis within a randomized trial at an academic pediatric ED.

High precision newborn screening for mucopolysaccharidosis type I by enzymatic activity followed by endogenous, non-reducing end glycosaminoglycan analysis.

Measurement of enzymatic activity in newborn dried blood spots (DBS) is the preferred first-tier method in newborn screening (NBS) for mucopolysaccharidosis (MPS) disorders. However, false positives are observed due mainly to the presence of pseudodeficiencies. Our previous publications on glycosaminoglycan (GAG) biomarker levels in dried blood spots (DBS) for mucopolysaccharidoses demonstrated that second-tier GAG biomarker analysis can dramatically reduce the false positive rate in NBS.

Qualitative assessment of provider approaches to dilation following vaginoplasty.

Study Objective: Most surgical vaginoplasties are followed post-operatively, when age appropriate, by patient-led dilation to maintain patency, but there is a lack of both standardized protocols and research comparing dilation practices within and across surgical routes. This study's objective is to determine surgeon attitudes about dilation and learn what protocols they use and endorse to take steps towards addressing the literature gap around dilation practices and outcomes.

Methods: We performed semi-structured interviews with ten providers (nine surgeons, one nurse) involved in vaginoplasty at a single tertiary care children's hospital about their dilation practices.

Disclosure and non-disclosure of childhood sexual abuse in Australia: Results from a national survey.

Background: Little population-based evidence exists about prevalence of lifetime disclosure and non-disclosure of child sexual abuse (CSA). Evidence is lacking about disclosure by girls and women compared with boys and men, and gender diverse individuals. It is unclear if disclosure is more common in contemporary society, and if disclosure is influenced by abuse severity and perpetrator type.

Identifying Plasmodium P36 and P52 antigens for coadministration with circumsporozoite protein to enhance vaccine efficacy.

Vaccines targeting the complex pre-erythrocytic stage of Plasmodium parasites may benefit from the inclusion of multiple antigens. However, discerning protective effects can be difficult because newer candidates may not be as protective as leading antigens like the circumsporozoite protein (CSP) in the conventional pre-clinical mouse model. We developed a modified mouse model challenge strategy that maximizes the contribution of T cells induced by novel candidate antigens at the sporozoite challenge time point and used this approach to test Plasmodium P36 and P52 vaccine candidates alone and in concert with non-protective doses of CSP.

Association Between Food Insecurity and Inability to Obtain Provider-Recommended Medications, Multidisciplinary Services, and Technology in Youth and Young Adults With Diabetes: A Cross-Sectional Study.

Objective: We assessed if food insecurity (FI) is associated with not obtaining recommended diabetes medications, technology, and multidisciplinary services, and explored the most common reasons for not obtaining recommended treatments in youth and young adults (YYA) with diabetes.

Methods: In this cross-sectional study, among 911 YYA with type 1 diabetes (T1D) and 144 with type 2 diabetes (T2D) from the SEARCH Food Security Cohort Study Follow-up 1 (2018-2021), FI (≥ 3 items affirmed from the 18-item Household Food Security Survey module), and inability to obtain recommended treatments were self-reported.

Results: Almost 30% of YYA with T1D and FI and 20% of YYA with T2D and FI did not obtain 1 or more recommended treatments.

The Natural History of Dermatosparaxis Ehlers Danlos Syndrome: An Adult Case Series.

Dermatosparaxis Ehlers Danlos syndrome (dEDS) is a very rare monogenic EDS that occurs due to biallelic pathogenic variants in ADAMTS2. Fifteen individuals with dEDS have been reported in the literature, with the oldest being 19 years at follow-up. Given the lack of information regarding adults with dEDS, our aim was to describe adults with dEDS to inform management recommendations in adulthood.

The prognostic value of single and repeated ≥10% FEV declines for chronic lung allograft dysfunction.

There is a paucity of data reporting the positive and negative predictive values (PPV, NPV) of acute declines in lung function on chronic lung allograft dysfunction (CLAD). We sought to define the predictive ability of single or repeated forced expiratory volume in the first second (FEV) declines for at least 3 weeks on the development of CLAD or death by 1-year. We analyzed 340 subjects with at least 3 years of follow-up data from two lung transplant centers.