ISCT MSC committee statement on the US FDA approval of allogenic bone-marrow mesenchymal stromal cells.

The December 2024 US Food and Drug Administration (FDA) approval of Mesoblast's Ryoncil (remestemcel-L-rknd)-allogeneic bone marrow mesenchymal stromal cell (MSC(M)) therapy-in pediatric acute steroid-refractory graft-versus-host-disease finally ended a long-lasting drought on approved MSC clinical products in the United States. While other jurisdictions-including Europe, Japan, India, and South Korea-have marketed autologous or allogeneic MSC products, the United States has lagged in its approval. The sponsor's significant efforts and investments, working closely with the FDA addressing concerns regarding clinical efficacy and consistent MSC potency through an iterative process that spanned several years, was rewarded with this landmark approval.

Body Composition in Preterm Infants: Current Insights and Emerging Perspectives.

In recent years, significant advancements in respiratory and nutritional care have markedly improved the survival rates of preterm infants and enhanced long-term health outcomes. Despite these improvements, emerging research highlights the lasting impacts of early growth patterns on an individual's health trajectory. Adults born prematurely face a higher incidence of health issues related to their early birth.

Pediatric Transplant and Cellular Therapy Consortium RESILIENT Conference on Pediatric Chronic Graft-versus-Host Disease Survivorship after Hematopoietic Cell Transplantation: Part II. Organ Dysfunction and Immune Reconstitution Considerations for children with chronic GVHD after Hematopoietic Cell Transplantation.

While highly morbid forms of chronic graft versus host disease (cGVHD) and severe late effects of allogeneic hematopoietic cell transplant (HCT) can impact children and adults alike, unique considerations arise in pediatric cases regarding diagnosis, monitoring, treatment, and likelihood of resolution. As children can present with atypical features of cGVHD, and with more significant disease due to inability to communicate symptoms, they may be at increased risk for highly morbid forms of cGVHD and incur greater subsequent late effects, which may be more pronounced in those with underlying chromosomal breakage syndromes, with higher prevalence in pediatric HCT recipients. The long-term effects of cGVHD and its therapies include impaired immune reconstitution, leading to increased risks of infection and secondary malignant neoplasms.

Antiseizure medications for Lennox-Gastaut Syndrome: Comprehensive review and proposed consensus treatment algorithm.

Lennox-Gastaut syndrome (LGS) is a severe, childhood-onset developmental and epileptic encephalopathy characterized by multiple drug-resistant seizure types, specific electroencephalogram (EEG) patterns, and significant cognitive and behavioral impairments. To date, eight anti-seizure medications (ASMs) have been specifically approved by the U.S.

A Retrospective Analysis of the Impact of Electroconvulsive Therapy on Anxiety Symptoms in Patients With Treatment-Resistant Depression.

Background: Electroconvulsive therapy (ECT) is an effective treatment for treatment-resistant depression (TRD). There are limited data on the improvement of anxiety symptoms in patients receiving ECT for TRD.

Objective: The aim of the study was to examine the extent to which anxiety symptom severity improves, relative to improvements in depressive symptoms, in TRD patients receiving an acute course of ECT.

Circulating and Magnetic Resonance Imaging Biomarkers of Intestinal Fibrosis in Small Bowel Crohn's Disease.

Background: We previously identified circulating and MRI biomarkers associated with the surgical management of Crohn's disease (CD). Here we tested associations between these biomarkers and ileal resection inflammation and collagen content.

Methods: Fifty CD patients undergoing ileal resection were prospectively enrolled at 4 centers.

Worldwide epidemiology of paediatric multiple sclerosis: data from the Multiple Sclerosis International Federation Atlas of MS, third edition.

Background: Limited data are available on the global rates of paediatric multiple sclerosis. Here, we report on the estimated worldwide prevalence of paediatric MS.

Methods: We included paediatric prevalence data in 2020-2022 (Multiple Sclerosis International Federation Atlas of MS) and the prevalence of child neurologists (International Child Neurology Association).

A carnitine transporter at the blood-brain barrier modulates sleep via glial lipid metabolism in .

To regulate brain function, peripheral compounds must traverse the blood-brain barrier (BBB), an interface between the brain and the circulatory system. To determine whether specific transport mechanisms are relevant for sleep, we conducted a BBB-specific inducible RNAi knockdown (iKD) screen for genes affecting sleep in . We observed reduced sleep with knockdown of solute carrier , a carnitine transporter, as determined by isotope flux.

Genomics yields biological and phenotypic insights into bipolar disorder.

Bipolar disorder is a leading contributor to the global burden of disease. Despite high heritability (60-80%), the majority of the underlying genetic determinants remain unknown. We analysed data from participants of European, East Asian, African American and Latino ancestries (n = 158,036 cases with bipolar disorder, 2.

Spleen stiffness in a healthy pediatric population undergoing liver magnetic resonance elastography.

Background: Splenic stiffness is a potential imaging marker of portal hypertension. Normative spleen stiffness values are needed to define diagnostic thresholds.

Objective: To report stiffness measurements of the spleen in healthy children undergoing liver magnetic resonance (MR) elastography across MRI vendors and field strengths.

Healthy Aging and the Gut Microbiome in People With and Without HIV.

Background: Aging-related comorbidities are more common in people with human immunodeficiency virus (HIV) compared to people without HIV. The gut microbiome may play a role in healthy aging; however, this relationship remains unexplored in the context of HIV.

Methods: 16S rRNA gene sequencing was conducted on stool from 1409 women (69% with HIV; 2304 samples) and 990 men (54% with HIV; 1008 samples) in the MACS/WIHS Combined Cohort Study.

Recent advances in the management of pediatric cholestatic liver diseases.

Pediatric cholestatic liver diseases are rare conditions that can result from multiple specific underlying etiologies. Among the most common etiologies of pediatric cholestatic liver diseases are biliary atresia, Alagille syndrome (ALGS), and inherited disorders of bile acid transport. These diseases are characterized by episodic or chronic unremitting cholestasis.

BRAF inhibitor monotherapy in BRAFV600E-mutated pediatric low-grade glioma: a single center's experience.

Background: Pediatric low-grade gliomas (pLGGs) have an overall survival of over 90%; however, patients harboring a BRAF alteration may have worse outcomes, particularly when treated with classic chemotherapy. Combined BRAF/MEK inhibition following incomplete resection demonstrated improved outcome in BRAF altered pLGG compared to combined carboplatin/vincristine chemotherapy and is now considered the standard FDA-approved treatment for this group of tumors. The aim herein was to investigate the efficacy and tolerability of single agent BRAF inhibitor treatment in BRAF altered pLGG.

Pediatric Predictive Artificial Intelligence Implemented in Clinical Practice from 2010-2021: A Systematic Review.

Objective: To review pediatric artificial intelligence (AI) implementation studies from 2010-2021 and analyze reported performance measures.

Methods: We searched PubMed/Medline, Embase CINHAL, Cochrane Library CENTRAL, IEEE and Web of Science with controlled vocabulary.

Inclusion Criteria: AI intervention in a pediatric clinical setting that learns from data (i.

Secondhand vape exposure regulation of CFTR and immune function in cystic fibrosis.

Secondhand smoke exposure (SHSe) is a public health threat for people with cystic fibrosis (CF) and other lung diseases. Primary smoking reduces CFTR channel function, the causative defect in CF. We reported that SHSe worsens respiratory and nutritional outcomes in CF by disrupting immune responses and metabolic signaling.

Evaluating access to psychosocial services for the medicaid-insured children in Georgia.

Background: Evaluating access to psychosocial services can inform policy decision-making on ways to address shortages in the availability of mental health (MH)-specialized providers. The objective of the study was to assess how the mental health (MH)-specialized workforce met the demand for psychosocial services of Medicaid-insured children in Georgia, with direct relevance in establishing quantitative network adequacy.

Methods: We used the 2018 Medicaid (TAF) claims data, the 2018 National Plan and Provider Enumeration System database, and the 2019 Georgia school-based program data to estimate community-level demand and practice-level supply of psychosocial services.

Proposed resources required for a comprehensive program for CCT CHD imaging.

Background: Cardiac Computed Tomography (CCT) is increasingly used for evaluation of congenital heart disease (CHD) in patients of all ages. Pediatric and adult congenital heart disease (ACHD) surgical programs require high quality CCT imaging as part of the multimodality imaging support expected of comprehensive care centers. Despite these expectations, there are no benchmarks or defined programmatic elements specific to the performance of CCT in patients with CHD.

The ACTION VAD registry: A collective five-year experience.

Background: The Advanced Cardiac Therapies Improving Outcomes Network (ACTION) began in 2018 as a collaborative learning health system committed to improving outcomes in pediatric heart failure, including children and adults with congenital heart disease, supported with ventricular assist devices (VADs). This report describes patient and device characteristics, and outcomes through 1-year post-implant.

Methods: The ACTION VAD registry report was created from data submitted to the ACTION learning network from April 2018 to June 2023.

Early developmental trajectories of the impaired hand in infants with unilateral cerebral palsy.

Aim: To identify developmental trajectories of impaired hand function in infants aged 3 to 15 months with unilateral cerebral palsy (CP).

Method: Sixty-three infants (37 male; median gestational age 37 weeks [interquartile range 30-39.1 weeks]) recruited as part of a randomized trial with a confirmed diagnosis of unilateral CP were included.

State laws on intimate partner violence witnessed by children in the United States.

Intimate partner violence (IPV) is common, and almost half of all IPV takes place in relationships with children in the home. We inventoried laws in the 50 states and the District of Columbia in the United States of America (USA) focused on addressing IPV committed in the presence of children, as these laws could help prevent or remediate this critical health and social issue. Using WestLaw, a web-based legal research service, we identified over 1,200 statutes and 500 regulations.

Neutralizing antibody immune correlates in COVAIL trial recipients of an mRNA second COVID-19 vaccine boost.

Neutralizing antibody titer has been a surrogate endpoint for guiding COVID-19 vaccine approval and use, although the pandemic's evolution and the introduction of variant-adapted vaccine boosters raise questions as to this surrogate's contemporary performance. For 985 recipients of an mRNA second bivalent or monovalent booster containing various Spike inserts [Prototype (Ancestral), Beta, Delta, and/or Omicron BA.1 or BA.

Dispensing practices of amoxicillin suspension by community pharmacists.

Background: Amoxicillin suspension is frequently prescribed to children; we hypothesized that prescribing convention system constraints lead to unusual dosing regimens and unnecessary waste of the drug.

Objective: Identify antibiotic dispensing practices by community pharmacists and/or technicians to understand opportunities to decrease wasted amoxicillin liquid and optimize prescribing convention of liquid amoxicillin to children.

Methods: Pilot online survey of Atlanta area and National Community Pharmacists Association pharmacists or pharmacy technicians that self-reported dispensing amoxicillin suspension.

New Index Demonstrates Association between Social Vulnerability, Environmental Burden, and Kidney Failure Risk among Individuals with Glomerular Disease.

Background: The Centers for Disease Control and Prevention (CDC) Environmental Justice Index Social-Environmental Ranking (EJI-SER) combines a Social Vulnerability Module (SV) with an Environmental Burden Module (EB) to characterize cumulative environmental and social burden at the census tract level. This analysis evaluates the association between EJI-SER and kidney outcomes in glomerular disease (GD) patients.

Methods: Cure Glomerulopathy (CureGN) is an observational cohort study of adults and children with biopsy-proven GD.

Multicenter Retrospective Analysis of Pediatric Differentiated Thyroid Carcinoma: Treatment Practices and Outcomes Prior to Pediatric American Thyroid Association Guidelines Implementation.

Background: Differentiated thyroid carcinoma (DTC) is the most common pediatric thyroid malignancy, with papillary thyroid carcinoma (PTC) representing 90% of the cases. In 2015, the American Thyroid Association (ATA) developed management guidelines for pediatric DTC.

Procedure: Patients less than 21 years of age diagnosed with DTC between 2000 and 2015 at Texas Children's Hospital, Seattle Children's Hospital, Children's Healthcare of Atlanta, Children's Hospital Colorado, and Nationwide Children's Hospital were retrospectively analyzed to evaluate treatment practices before the implementation of the ATA guidelines.