The Research Agenda for Perinatal Innovation and Digital Health Project: Human-Centered Approach to Multipartner Research Agenda Codevelopment.

Background: Digital health innovations provide an opportunity to improve access to care, information, and quality of care during the perinatal period, a critical period of health for mothers and infants. However, research to develop perinatal digital health solutions needs to be informed by actual patient and health system needs in order to optimize implementation, adoption, and sustainability.

Objective: Our aim was to co-design a research agenda with defined research priorities that reflected health system realities and patient needs.

Characterization of Factors Associated With Death in Deceased Patients With Mitochondrial Disorders: A Multicenter Cross-Sectional Survey.

Background And Objectives: Mitochondrial disorders are multiorgan disorders resulting in significant morbidity and mortality. We aimed to characterize death-associated factors in an international cohort of deceased individuals with mitochondrial disorders.

Methods: This cross-sectional multicenter observational study used data provided by 26 mitochondrial disease centers from 8 countries from January 2022 to March 2023.

Intraprocedural Three-Dimensional Imaging Registration Optimizes Magnetic Resonance Imaging-Guided Focused Ultrasound and Facilitates Novel Applications.

Background And Objective: Transcranial magnetic resonance-guided focused ultrasound (MRgFUS) has revolutionized ablative treatment of essential tremor in recent years. However, limitations in precision targeting may account for suboptimal efficacy and significant side effects. We describe a simple intraprocedural three-dimensional image-guided lesion shaping technique that can improve overall outcomes of MRgFUS for essential tremor and facilitate expansion to novel indications.

Current Evidence on the Care of Young Infants With Hypothermia in the Emergency Department.

The presence of hypothermia among young infants in the emergency department may be a sign of serious or invasive bacterial infections, or invasive herpes simplex viral infection. However, hypothermia may also occur due to a variety of other infectious and noninfectious conditions or environmental exposure. In some settings, hypothermia may represent a protective, energy-conserving response to illness.

Homeobox protein MSX-1 restricts hepatitis B virus by promoting ubiquitin-independent proteasomal degradation of HBx protein.

Hepatitis B virus (HBV) X protein (HBx) is a key factor for regulating viral transcription and replication. We recently characterized homeobox protein MSX-1 (MSX1) as a host restriction factor that inhibits HBV gene expression and genome replication by directly binding to HBV enhancer II/core promoter (EnII/Cp) and suppressing its promoter and enhancer activities. Notably, HBx expression was observed to be repressed more drastically by MSX1 compared to other viral antigens.

Limited research investigating the value of MRI in predicting future cognitive morbidity in survivors of paediatric brain tumours: A systematic-review and call to action for clinical neuroimaging researchers.

Survivors of pediatric brain tumours are at a high risk of cognitive morbidity. Reliable individual-level predictions regarding the likelihood, degree, and affected domains of cognitive impairment would be clinically beneficial. While established risk factors exist, quantitative MRI analysis may enhance predictive value, above and beyond current clinical risk models.

The Management of Chronic Insomnia in Young Children: A Survey of UK General Practice.

Objectives: Chronic insomnia (CI), the most common sleep disorder in young children, is associated with negative outcomes for the child and family. This study explores the beliefs, current practice, and understanding of UK primary health care professionals (PCPs) regarding CI in children under 5 years.

Method: An online survey of UK PCPs (e.

Bone Health and Linear Growth in Children with Familial Hypoparathyroidism Treated with Human Parathyroid Hormone 1-34.

Context: Our study explores the impact of human PTH 1-34 injections (PTH therapy) on growth, areal bone mineral density (BMD), and bone quality (measured by trabecular bone score, TBS) in hypoparathyroidism due to autoimmune polyendocrine syndrome type 1 (APS-1) or an activating variant of the calcium sensing receptor (CaR).

Objective: To assess associations of 1) age and PTH therapy duration with age-standardized Z-scores for height (HAZ), BMD (BMD-Z), and TBS (TBS-Z) in CaR or APS-1, and 2) APS-1 disease severity with BMD-Z and TBS-Z.

Methods: This secondary analysis pooled linear growth and lumbar spine (LS) DXA data from studies of hypoparathyroidism with mean baseline age of 13.

Revised Process for ACNS Guidelines Development.

The development of clinical practice guidelines is an evolving field. In response to the need for consistent, evidence-based medical practice, the American Clinical Neurophysiology Society identified the need to update the Society's guideline development process. The American Clinical Neurophysiology Society Guidelines Committee created an action plan with the goal of improving transparency and rigor for future guidelines and bringing existing guidelines to current standards.

Sacral Alar-Iliac (SAI) screw diameter is not associated with pelvic fixation failure for neuromuscular scoliosis patients.

Purpose: Determine if Sacral Alar-Iliac (SAI) screw diameter is associated with pelvic fixation failure in pediatric patients with neuromuscular scoliosis (NMS) treated with posterior spinal fusion (PSF).

Methods: NMS patients from a single institution who underwent PSF with bilateral SAI screw fixation from 2010 to 2021 were retrospectively reviewed. Clinical parameters, SAI screw sizes, and radiographic outcomes were analyzed.

Adenine base editor corrected ADPKD point mutations in hiPSCs and kidney organoids.

Autosomal dominant polycystic kidney disease (ADPKD) is a dominant genetic disorder caused primarily by mutations in the PKD1 gene, resulting in the formation of numerous cysts and eventually kidney failure. However, there are currently no gene therapy studies aimed at correcting PKD1 gene mutations. In this study, we identified two mutation sites associated with ADPKD, c.

The ability of CD4CD8 T cells to distinguish between Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis and pediatric infectious mononucleosis.

Epstein-Barr virus (EBV)-related hemophagocytic lymphohistiocytosis (EBV-HLH) and infectious mononucleosis (IM) are characterized by fever, hepatomegaly, and splenomegaly, but HLH has a 50% lethality rate. Therefore, this study aimed to compare the laboratory findings in differentiating EBV-HLH children from IM children who have fever, hepatomegaly, or splenomegaly. A total of 131 IM patients and 29 EBV-HLH pediatric patients with fever, hepatomegaly, or splenomegaly were enrolled in our study.

Ultrasound evaluation of gastric emptying for two different quantities of milk in elementary school-aged children.

Unlabelled: Current guidelines recommend 6-h milk fasting in periprocedural settings; however, recent evidence suggests potential overconservativeness and supports more liberal pediatric fasting protocols. This study assessed the gastric emptying of two different milk quantities in elementary school-age children using gastric ultrasonography. This prospective crossover trial involved 30 healthy children who fasted overnight on two separate occasions within one month, consuming either 5 or 10 mL/kg of milk.

FOXP3 as a prognostic marker and therapeutic target in immunogenic cell death modulation for clear cell renal cell carcinoma.

Background: Clear cell renal cell carcinoma (ccRCC) remains a challenging cancer type due to its resistance to standard treatments. Immunogenic cell death (ICD) has the potential to activate anti-tumor immunity, presenting a promising avenue for ccRCC therapies.

Methods: We analyzed data from GSE29609, TCGA-KIRC, and GSE159115 to identify ICD-related prognostic genes in ccRCC.

Higher bone mineral density at six years of age in very preterm-born infants fed human milk compared to formula feeding. A secondary analysis of an RCT.

Unlabelled: In very preterm-born infants, nutritional intake is important to reduce the risk of severe metabolic bone disease including the risk of a lower bone mineral density (BMD). The aim of this study was to evaluate bone mineral content (BMC) and BMD (measured as BMC per bone area (BA)) at six years of age in very preterm-born infants fed different diets post-discharge. Data on this topic so far is insufficient, and with this study we aim to supply more useful data.

European central hypoventilation syndrome consortium description of congenital central hypoventilation syndrome neonatal onset.

Unlabelled: It is known that in most cases of congenital central hypoventilation syndrome (CCHS), apnoeas and hypoventilation occur at birth. Nevertheless, a detailed description of initial symptoms, including pregnancy events and diagnostic tests performed, is warranted in infants with neonatal onset of CCHS, that is, in the first month of life. The European Central Hypoventilation Syndrome Consortium created an online patient registry from which 97 infants (44 females) with CCHS of neonatal onset and PHOX2B mutation from 10 countries were selected.

Clinical applicability of 2023 International Pediatric Nephrology Association recommended limited therapeutic drug monitoring formulae to assess mycophenolic acid exposure.

Background: The 2023 IPNA guidelines recommended a 12-h mycophenolic acid (MPA) area under the curve (AUC) estimation for managing pediatric nephrotic syndrome and MPA AUC > 50 mg * h/L for an optimal therapeutic response to mycophenolate mofetil (MMF). The IPNA guidelines endorsed two limited AUC formulae based on three-point MPA measurements to predict 12-h MPA AUC. The relative performance of these two limited AUC formulae has not been tested.

Retinoid-impregnated nanoparticles enable control of bone growth by site-specific modulation of endochondral ossification in mice.

Growth-plate (GP) injures in limbs and other sites can impair GP function and cause deceleration of bone growth, leading to progressive bone lengthening imbalance, deformities and/or physical discomfort, decreased motion and pain. At present, surgical interventions are the only means available to correct these conditions by suppressing the GP activity in the unaffected limb and/or other bones in the ipsilateral region. Here, we aimed to develop a pharmacologic treatment of GP growth imbalance that involves local application of nanoparticles-based controlled release of a selective retinoic acid nuclear receptor gamma (RARγ) agonist drug.

A survey of current trends in postgraduate year 2 pediatric pharmacy residencies and growth of pediatric ambulatory care pharmacy practice.

Disclaimer: In an effort to expedite the publication of articles, AJHP is posting manuscripts online as soon as possible after acceptance. Accepted manuscripts have been peer-reviewed and copyedited, but are posted online before technical formatting and author proofing. These manuscripts are not the final version of record and will be replaced with the final article (formatted per AJHP style and proofed by the authors) at a later time.

Sickle Cell Trait Does Not Cause "Sickle Cell Crisis" Leading to Exertion-Related Death: A Systematic Review.

Globally, an estimated 300 million individuals have sickle cell trait (SCT), the carrier state for sickle cell disease. While sickle cell disease (SCD) is associated with increased morbidity and shortened lifespan, SCT has a lifespan comparable to that of the general population. However, "sickle cell crisis" has been used as a cause of death for decedents with SCT in reports of exertion-related death in athletes, military personnel, and individuals in police custody.