586 results match your criteria: "and Boston Children's Hospital.[Affiliation]"

Background: Although systemic inequities, broadly defined, are associated with health disparities in adults, there is a dearth of research linking contextual measures of exclusionary policies or prejudicial attitudes to health impairments in children, particularly among Latino populations. In this study, we examined a composite measure of systemic inequities in relation to the cooccurrence of multiple health problems in Latino children in the United States.

Methods: Participants included 17 855 Latino children aged 3 to 17 years from the National Survey of Children's Health (2016-2020).

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Purpose: To evaluate the cost-effectiveness of the treatment of geography atrophy (GA) with intravitreal pegcetacoplan and to identify utility-measurement surrogates.

Design: Cost analysis based on data from a published study.

Subjects: None; based on data from published sham control compared with 2 treatment groups in the index study.

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Helicobacter pylori ( H pylori ) eradication rates have declined globally, stressing the importance of antimicrobial susceptibility testing to inform treatment. Molecular tests such as next-generation sequencing (NGS) provide susceptibility data for the antibiotics used in the treatment of H pylori in a noninvasive, effective, and rapid manner. We obtained stool susceptibility testing using a novel NGS-based analysis and compared results with the current "gold standard" of gastric biopsy culture via agar dilution in 20 pediatric patients with evidence of H pylori in gastric biopsies.

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Abdominal pain is a common symptom of several debilitating conditions (eg, inflammatory bowel disease, irritable bowel syndrome, and endometriosis) and affects individuals throughout their lifespan. Quantitative sensory testing (QST) reference values exist for many body sites but not the abdomen. Using a QST battery adapted from the German Research Network on Neuropathic Pain, we collected QST data on the upper and lower abdomen in 181 pain-free participants, ages 12 to 50 years, to establish reference values by age and biological sex.

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What We Know About Intracranial Hypertension in Children With Syndromic Craniosynostosis.

J Craniofac Surg

October 2023

Sophia Children's Hospital, Dutch Craniofacial Center, Department of Plastic and Reconstructive Surgery and Hand Surgery, Erasmus MC, University Medical Center.

Article Synopsis
  • The study reviews literature on intracranial hypertension (ICH) in patients with syndromic craniosynostosis (sCS) and whether cognitive and behavioral outcomes are more affected by genetic factors than ICH itself.
  • The review analyzed 1893 search results, focusing on defining ICH and examining its mechanisms based on the Monro-Kellie doctrine, concluding that invasive measures for ICH are the most reliable, with some noninvasive alternatives being explored.
  • Ultimately, the findings suggest that while ICH results from various factors in sCS, cognitive and behavioral issues are primarily influenced by genetics, whereas ICH contributes to cortical thinning and visual problems.
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FISH-Flow to quantify nascent and mature ribosomal RNA in mouse and human cells.

STAR Protoc

September 2023

Division of Hematology and Oncology, Department of Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA 19104, USA; Department of Cell and Developmental Biology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA 19104, USA; Abramson Family Cancer Research Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA 19104, USA. Electronic address:

FISH-Flow (fluorescence in situ hybridization-flow cytometry) involves hybridizing fluorescent oligos to RNA and quantifying fluorescence at a single-cell level using flow cytometry. Here, we present a FISH-Flow protocol to quantify nascent 47S and mature 18S and 28S rRNAs in mouse and human cells, including rRNA quantification across cell cycle stages using DNA staining. We describe steps for cell preparation, hybridization of fluorescent probes against rRNA, and DNA staining.

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Adolescent and young adults (AYAs) with acute lymphoblastic leukemia (ALL) treated with asparaginase-containing pediatric regimens are commonly overweight or obese. We studied the association of body mass index (BMI) on outcomes of 388 AYAs aged 15 to 50 years treated on Dana-Farber Cancer Institute (DFCI) consortium regimens (2008-2021). BMI was normal in 207 (53.

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Background: As implementation science in global health continues to evolve, there is a need for valid and reliable measures that consider diverse linguistic and cultural contexts. A standardized, reproducible process for multilingual measure development may improve accessibility and validity by participants in global health settings. To address this need, we propose a rigorous methodology for multilingual measurement development.

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Objective: Social determinants of health (SDoH), such as poverty, are associated with increased burden and severity of rheumatic and musculoskeletal diseases. This study was undertaken to study the prevalence and documentation of SDoH-related needs in electronic health records (EHRs) of individuals with these conditions.

Methods: We randomly selected individuals with ≥1 International Classification of Diseases, Ninth/Tenth Revision (ICD-9/10) code for a rheumatic/musculoskeletal condition enrolled in a multihospital integrated care management program that coordinates care for medically and/or psychosocially complex individuals.

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Clinical profiling of specific diagnostic subgroups of women with chronic pelvic pain.

Front Reprod Health

May 2023

Oxford Endometriosis Centre, Nuffield Department of Women's and Reproductive Health, University of Oxford, Oxford, United Kingdom.

Introduction: Chronic pelvic pain (CPP) is a common condition affecting up to 26.6% of women, with many suffering for several years before diagnosis and/or treatment. Its clinical presentation is varied and there are frequently comorbid conditions both within and outside the pelvis.

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Concomitant cannabidiol does not impact safety and effectiveness of diazepam nasal spray for seizure clusters: Post hoc analysis of a phase 3 safety study.

Epilepsy Behav

July 2023

Neurelis, Inc., 3430 Carmel Mountain Road, Suite 300, San Diego, CA 92121, USA; John A. Burns School of Medicine, University of Hawaii, Honolulu, HI, USA.

People with epilepsy may experience episodes of frequent seizure activity (seizure clusters, acute repetitive seizures), and benzodiazepines are the cornerstone of rescue treatment. Cannabidiol (CBD) can be used as an adjunctive treatment for epilepsy, and it may interact with other antiseizure drugs, such as benzodiazepines. Here, we examined the safety and effectiveness of intermittent use of diazepam nasal spray in patients with seizure clusters who also received CBD treatment.

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Construction and validation of customized genomes for human and mouse ribosomal DNA mapping.

J Biol Chem

June 2023

Division of Hematology and Oncology, Department of Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA; Department of Cell and Developmental Biology, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA; Abramson Family Cancer Research Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA. Electronic address:

rRNAs are transcribed from ribosomal DNA (rDNA) repeats, the most intensively transcribed loci in the genome. Due to their repetitive nature, there is a lack of genome assemblies suitable for rDNA mapping, creating a vacuum in our understanding of how the most abundant RNA in the cell is regulated. Our recent work revealed binding of numerous mammalian transcription and chromatin factors to rDNA.

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Objective: The present study was undertaken to evaluate high-quality care delivery in the context of provider goal-setting activities and a multidisciplinary care model using an electronic health record (EHR)-enabled pediatric lupus registry. We then determined associations between care quality and prednisone use among youth with systemic lupus erythematosus (SLE).

Methods: We implemented standardized EHR documentation tools to autopopulate a SLE registry.

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Rapid-kinetics degron benchmarking reveals off-target activities and mixed agonism-antagonism of MYB inhibitors.

bioRxiv

April 2023

Cancer and Blood Disorders Center, Dana-Farber Cancer Institute and Boston Children's Hospital, Harvard Medical School, Boston, MA, 02215, USA.

Attenuating aberrant transcriptional circuits holds great promise for the treatment of numerous diseases, including cancer. However, development of transcriptional inhibitors is hampered by the lack of a generally accepted functional cellular readout to characterize their target specificity and on-target activity. We benchmarked the direct gene-regulatory signatures of six agents reported as inhibitors of the oncogenic transcription factor MYB against targeted MYB degradation in a nascent transcriptomics assay.

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Apical extracellular matrix (aECM) constitutes the interface between every tissue and the outside world. It is patterned into diverse tissue-specific structures through unknown mechanisms. Here, we show that a male-specific genetic switch in a single glial cell patterns the aECM into a ∼200 nm pore, allowing a male sensory neuron to access the environment.

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Lineage-defining transcription factors form densely interconnected circuits in chromatin occupancy assays, but the functional significance of these networks remains underexplored. We reconstructed the functional topology of a leukemia cell transcription network from the direct gene-regulatory programs of eight core transcriptional regulators established in pre-steady state assays coupling targeted protein degradation with nascent transcriptomics. The core regulators displayed narrow, largely non-overlapping direct transcriptional programs, forming a sparsely interconnected functional hierarchy stabilized by incoherent feed-forward loops.

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Neighborhood Opportunity and Mortality Among Children and Adults in Their Households.

Pediatrics

April 2023

The Heller School for Social Policy and Management, Institute for Child, Youth and Family Policy, Brandeis University, Waltham Massachusetts.

Article Synopsis
  • Research indicates a connection between the neighborhood opportunity and health outcomes, specifically focusing on children's mortality risk and their caregivers over an 11-year period.
  • A study involving over 1 million children found that those living in low opportunity neighborhoods had significantly higher mortality risks, both for themselves and for their caregivers.
  • The findings highlight the negative effects of neighborhood inequalities on child well-being and suggest that addressing these disparities through targeted policies could improve health outcomes for children and their families.
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Objective: There is no validated tool to assess iatrogenic opioid withdrawal in preterm infants in the newborn intensive care unit (NICU).

Study Design: The Neonatal Withdrawal Assessment Tool (NWAT) was developed to address this gap in clinical practice. In this pilot study, the NWAT was assessed for inter-rater reliability (IRR) and content validity.

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Mitochondrial fusion is a therapeutic vulnerability of acute myeloid leukemia.

Leukemia

April 2023

Translational Research Center for Hemato-Oncology, Faculty of Medicine, University of Geneva, Geneva, Switzerland.

Article Synopsis
  • Mitochondrial metabolism is crucial for acute myeloid leukemia (AML) and is regulated by proteins that manage mitochondrial shape through fusion and fission processes.* -
  • Research indicates that targeting mitochondrial fusion presents a new vulnerability in AML cells, demonstrated through studies using patient-derived xenograft (PDX) models.* -
  • Disruption of mitochondrial fusion through genetic depletion or pharmacological inhibition significantly impairs AML cell growth by affecting respiration and causing cell cycle arrest, suggesting a potential therapeutic strategy.*
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Background: Continuous electroencephalography (cEEG) is commonly used for neuromonitoring in pediatric intensive care units (PICU); however, there are barriers to real-time interpretation of EEG data. Quantitative EEG (qEEG) transforms the EEG signal into time-compressed graphs, which can be displayed at the bedside. A survey was designed to understand current PICU qEEG use.

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Background: Biomarkers may enhance diagnostic capability for common paediatric infections, especially in low- and middle-income countries (LMICs) where standard diagnostic modalities are frequently unavailable, but disease burden is high. A comprehensive understanding of the diagnostic capability of commonly available biomarkers for neonatal sepsis in LMICs is lacking. Our objective was to systematically review evidence on biomarkers to understand their diagnostic performance for neonatal sepsis in LMICs.

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Therapeutic adenine base editing of human hematopoietic stem cells.

Nat Commun

January 2023

Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China.

In β-thalassemia, either γ-globin induction to form fetal hemoglobin (α2γ2) or β-globin repair to restore adult hemoglobin (α2β2) could be therapeutic. ABE8e, a recently evolved adenine base editor variant, can achieve efficient adenine conversion, yet its application in patient-derived hematopoietic stem cells needs further exploration. Here, we purified ABE8e for ribonucleoprotein electroporation of β-thalassemia patient CD34 hematopoietic stem and progenitor cells to introduce nucleotide substitutions that upregulate γ-globin expression in the BCL11A enhancer or in the HBG promoter.

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