Perspectives of pediatric oncologists on referral for CAR-T therapy: a mixed methods pilot study.

Background: Receipt of chimeric antigen receptor T-cell (CAR-T) therapy at an institution different from the primary oncologist's institution is a complex, multistep process. Referral by oncologists plays an important role in the process but may be susceptible to bias.

Methods: Oncologists who previously referred patients for CAR-T therapy at 5 pediatric hospitals were sent surveys by email exploring their CAR-T referral practices.

Immigrant and Refugee Health Curricula in US-Based Medical Training: A Scoping Review to Inform Integration Into Pediatric Residency Programs.

Objective: Despite increases in the US foreign-born population, medical education opportunities in immigrant and refugee health (IRH) remain limited. We summarize findings for published IRH curricula and offer recommendations for integrating IRH into pediatric residency programs.

Methods: We performed a literature review of articles describing the design, implementation, or assessment of IRH curricula for US-based undergraduate and graduate medical trainees.

COVID-19 mRNA vaccination responses in individuals with sickle cell disease: an ASH RC Sickle Cell Research Network Study.

Children and adults with sickle cell disease (SCD) have increases in morbidity and mortality with COVID-19 infections. The American Society of Hematology Research Collaborative Sickle Cell Disease Research Network performed a prospective COVID-19 vaccine study to assess antibody responses and analyze whether messenger RNA (mRNA) vaccination precipitated any adverse effects unique to individuals with SCD. Forty-one participants received 2 doses of the Pfizer-BioNTech vaccine and provided baseline blood samples before vaccination and 2 months after the initial vaccination for analysis of immunoglobulin G (IgG) reactivity against the receptor binding domain (RBD) of the severe acute respiratory syndrome coronavirus 2 spike protein.

Resource and Service Use after Discharge Among Infants 22-25 Weeks Estimated Gestational Age at the First High-Risk Infant Follow-Up Visit in California.

Objective: To examine resource and service use after discharge among infants born extraordinarily preterm in California who attended high-risk infant follow-up (HRIF) clinic by 12 months corrected age.

Study Design: We included infants born 2010-2017 between 22 + 0/7 and 25 + 6/7 weeks' gestational age in the California Perinatal Quality Care Collaborative and California Perinatal Quality Care Collaborative-California Children's Services HRIF databases. We evaluated rates of hospitalization, surgeries, medications, equipment, medical service and special service use, and referrals.

Pediatric Palliative Epilepsy Surgery: A Report From the Pediatric Epilepsy Research Consortium (PERC) Surgery Database.

Background: Epilepsy surgery is an underutilized resource for children with drug-resistant epilepsy. Palliative and definitive surgical options can reduce seizure burden and improve quality of life. Palliative epilepsy surgery is often seen as a "last resort" compared to definitive surgical options.

Understanding the Burden of Pediatric Traumatic Injury in Uganda: A Multicenter, Prospective Study.

Introduction: Traumatic injury is responsible for eight million childhood deaths annually. In Uganda, there is a paucity of comprehensive data describing the burden of pediatric trauma, which is essential for resource allocation and surgical workforce planning. This study aimed to ascertain the burden of non-adolescent pediatric trauma across four Ugandan hospitals.

Non-invasive prenatal testing of beta-hemoglobinopathies using next generation sequencing, in-silico sequence size selection, and haplotyping.

Aim: To develop a non-invasive prenatal test for beta-hemoglobinopathies based on analyzing maternal plasma by using next generation sequencing.

Methods: We applied next generation sequencing (NGS) of maternal plasma to the non-invasive prenatal testing (NIPT) of autosomal recessive diseases, sickle cell disease and beta-thalassemia. Using the Illumina MiSeq, we sequenced plasma libraries obtained via a Twist Bioscience probe capture panel covering 4 Kb of chromosome 11, including the beta-globin (HBB) gene and >450 genomic single-nucleotide polymorphisms (SNPs) used to estimate the fetal fraction (FF).

Nonoperative Versus Operative Treatment of Z-Type Comminuted Clavicle Fractures in Adolescents: A Prospective Substratified Cohort Analysis.

Objectives: To compare the clinical, radiographic, and patient-reported outcomes of nonoperative and operative treatment of adolescents with comminuted "Z-type" midshaft clavicle fractures.

Design: Prospective observational cohort.

Setting: Eight tertiary care pediatric centers.

Single-level laminoplasty approach to selective dorsal rhizotomy with conus localization by rapid spine MRI.

Introduction: While selective dorsal rhizotomy (SDR) was originally described as a multilevel approach, single-level approaches are now popularized. Conus localization is beneficial for operative planning in single-level selective dorsal rhizotomy. Our approach to SDR involves minimal exposure for a single-level laminoplasty, preserving one attached interspinous ligament.

Pathobiological signatures of dysbiotic lung injury in pediatric patients undergoing stem cell transplantation.

Hematopoietic cell transplantation (HCT) uses cytotoxic chemotherapy and/or radiation followed by intravenous infusion of stem cells to cure malignancies, bone marrow failure and inborn errors of immunity, hemoglobin and metabolism. Lung injury is a known complication of the process, due in part to disruption in the pulmonary microenvironment by insults such as infection, alloreactive inflammation and cellular toxicity. How microorganisms, immunity and the respiratory epithelium interact to contribute to lung injury is uncertain, limiting the development of prevention and treatment strategies.

Emergency department pediatric readiness of United States trauma centers in 2021: Trauma center facility characteristics and opportunities for improvement.

Background: Emergency department (ED) pediatric readiness has been associated with lower mortality for injured children but has historically been suboptimal in nonpediatric trauma centers. Over the past decade, the National Pediatric Readiness Project (NPRP) has invested resources in improving ED pediatric readiness. This study aimed to quantify current trauma center pediatric readiness and identify associations with center-level characteristics to target further efforts to guide improvement.

Concurrent Validity of PROMIS and LD-SRS Scores in Pediatric Patients With Lower Limb Differences.

Background: We sought to assess the concurrent validity of select Patient Reported Outcomes Measurement Information System (PROMIS) domains and Limb Deformity-Scoliosis Research Society (LD-SRS) scores.

Methods: We prospectively administered PROMIS - 25 (including anxiety, depression, fatigue, pain interference, peer relationships, and physical function) and LD-SRS questionnaires to 46 consecutive pediatric patients with lower limb differences, presenting to a single surgeon for reconstruction. Concurrent validity between various subdomains of the 2 outcome measures was assessed through Pearson's correlation, with significance defined as P <0.

Durability of Original Monovalent mRNA Vaccine Effectiveness Against COVID-19 Omicron-Associated Hospitalization in Children and Adolescents - United States, 2021-2023.

Pediatric COVID-19 vaccination is effective in preventing COVID-19-related hospitalization, but duration of protection of the original monovalent vaccine during SARS-CoV-2 Omicron predominance merits evaluation, particularly given low coverage with updated COVID-19 vaccines. During December 19, 2021-October 29, 2023, the Overcoming COVID-19 Network evaluated vaccine effectiveness (VE) of ≥2 original monovalent COVID-19 mRNA vaccine doses against COVID-19-related hospitalization and critical illness among U.S.

NKp44/HLA-DP-dependent regulation of CD8 effector T cells by NK cells.

Although natural killer (NK) cells are recognized for their modulation of immune responses, the mechanisms by which human NK cells mediate immune regulation are unclear. Here, we report that expression of human leukocyte antigen (HLA)-DP, a ligand for the activating NK cell receptor NKp44, is significantly upregulated on CD8 effector T cells, in particular in human cytomegalovirus (HCMV) individuals. HLA-DP CD8 T cells expressing NKp44-binding HLA-DP antigens activate NKp44 NK cells, while HLA-DP CD8 T cells not expressing NKp44-binding HLA-DP antigens do not.

The Adverse Event Landscape of Stem Cell Transplant: Evidence for AGVHD Driving Early Transplant Associated Toxicities.

Although unrelated-donor (URD) hematopoietic cell transplantation (HCT) is associated with many toxicities, a detailed analysis of adverse events, as defined by the Common Terminology Criteria for Adverse Events (CTCAE), has not previously been curated. This represents a major unmet need, especially as it relates to assessing the safety of novel agents. We analyzed a detailed AE database from the "ABA2" randomized, double-blind, placebo-controlled clinical trial of abatacept for acute graft-versus-host disease (AGVHD) prevention, for which the FDA mandated a detailed AE assessment through Day +180, and weekly neutrophil and platelet counts through Day +100.

Riociguat in patients with sickle cell disease and hypertension or proteinuria (STERIO-SCD): a randomised, double-blind, placebo controlled, phase 1-2 trial.

Background: Although nitric oxide based therapeutics have been shown in preclinical models to reduce vaso-occlusive events and improve cardiovascular function, a clinical trial of a phosphodiesterase 5 inhibitor increased rates of admission to hospital for pain. We aimed to examine if riociguat, a direct stimulator of the nitric oxide receptor soluble guanylate cyclase, causes similar increases in vaso-occlusive events.

Methods: This was a phase 1-2, randomised, double blind, placebo-controlled trial.

Clinical and biological landscape of constitutional mismatch-repair deficiency syndrome: an International Replication Repair Deficiency Consortium cohort study.

Background: Constitutional mismatch repair deficiency (CMMRD) syndrome is a rare and aggressive cancer predisposition syndrome. Because a scarcity of data on this condition contributes to management challenges and poor outcomes, we aimed to describe the clinical spectrum, cancer biology, and impact of genetics on patient survival in CMMRD.

Methods: In this cohort study, we collected cross-sectional and longitudinal data on all patients with CMMRD, with no age limits, registered with the International Replication Repair Deficiency Consortium (IRRDC) across more than 50 countries.

The Utility of Serum Alpha-fetoprotein for Monitoring for Relapse of Alpha-fetoprotein-Positive Hepatoblastoma.

Hepatoblastoma is the most common liver malignancy in children. Treatment typically involves surgery and cisplatin-based chemotherapy. After therapy completion, children undergo repetitive surveillance imaging to screen for relapse, which occurs in <12% of cases.