Coagulation Dysfunction Criteria in Critically Ill Children: The PODIUM Consensus Conference.

Context: Previous criteria for coagulation dysfunction in critically ill children were based mainly on expert opinion.

Objective: To evaluate current evidence regarding coagulation tests associated with adverse outcomes in children to inform criteria for coagulation dysfunction during critical illness.

Data Sources: Electronic searches of PubMed and Embase were conducted from January 1992 to January 2020 by using a combination of medical subject heading terms and text words to define concepts of coagulation dysfunction, pediatric critical illness, and outcomes of interest.

Disease Burden Affects Outcomes in Pediatric and Young Adult B-Cell Lymphoblastic Leukemia After Commercial Tisagenlecleucel: A Pediatric Real-World Chimeric Antigen Receptor Consortium Report.

Purpose: Tisagenlecleucel is a CD19-specific chimeric antigen receptor T-cell therapy, US Food and Drug Administration-approved for children, adolescents, and young adults (CAYA) with relapsed and/or refractory (RR) B-cell acute lymphoblastic leukemia (B-ALL). The US Food and Drug Administration registration for tisagenlecleucel was based on a complete response (CR) rate of 81%, 12-month overall survival (OS) of 76%, and event-free survival (EFS) of 50%. We report clinical outcomes and analyze covariates of outcomes after commercial tisagenlecleucel.

Efficacy and Outcomes of Faecal Microbiota Transplantation for Recurrent Clostridioides difficile Infection in Children with Inflammatory Bowel Disease.

Background: Children with inflammatory bowel disease [IBD] are disproportionally affected by recurrent Clostridioides difficile infection [rCDI]. Although faecal microbiota transplantation [FMT] has been used with good efficacy in adults with IBD, little is known about outcomes associated with FMT in paediatric IBD.

Methods: We performed a retrospective review of FMT at 20 paediatric centres in the USA from March 2012 to March 2020.

Transfusion practices for pediatric oncology and hematopoietic stem cell transplantation patients: Data from the National Heart Lung and Blood Institute Recipient Epidemiology and Donor Evaluation Study-III (REDS-III).

Background: To evaluate transfusion practices in pediatric oncology and hematopoietic stem cell transplant (HSCT) patients.

Study Design And Methods: This is a multicenter retrospective study of children with oncologic diagnoses treated from 2013 to 2016 at hospitals participating in the National Heart Lung and Blood Institute Recipient Epidemiology and Donor Evaluation Study-III. Transfusion practices were evaluated by diagnosis codes and pre-transfusion laboratory values.

Frictional melanosis: A pediatric case series.

We present a pair of cases detailing acquired hyperpigmented patches overlying the spinous processes of pediatric patients. These cases are consistent with a condition that has previously been documented in the adult population and is known by many names, including "Davener's dermatosis," "towel melanosis," and "lifa disease." We propose unifying these terms into a single standardized name, "frictional melanosis," when it is encountered in the pediatric setting.

A comparison of newer classifications of bronchopulmonary dysplasia: findings from the Children's Hospitals Neonatal Consortium Severe BPD Group.

Objective: To compare three bronchopulmonary dysplasia (BPD) definitions against hospital outcomes in a referral-based population.

Study Design: Data from the Children's Hospitals Neonatal Consortium were classified by 2018 NICHD, 2019 NRN, and Canadian Neonatal Network (CNN) BPD definitions. Multivariable models evaluated the associations between BPD severity and death, tracheostomy, or length of stay, relative to No BPD references.

GATA4 Controls Epithelial Morphogenesis in the Developing Stomach to Promote Establishment of Glandular Columnar Epithelium.

Background & Aims: The transcription factor GATA4 is broadly expressed in nascent foregut endoderm. As development progresses, GATA4 is lost in the domain giving rise to the stratified squamous epithelium of the esophagus and forestomach (FS), while it is maintained in the domain giving rise to the simple columnar epithelium of the hindstomach (HS). Differential GATA4 expression within these domains coincides with the onset of distinct tissue morphogenetic events, suggesting a role for GATA4 in diversifying foregut endoderm into discrete esophageal/FS and HS epithelial tissues.

Letting Residents Lead: Implementing Resident Admission Triage Call Curriculum and Practice.

Background And Objectives: Graduating residents are expected to be competent in triaging patients to appropriate resources. Before 2017, pediatric residents were not involved in admission triage decisions. In 2017, after implementing an admission triage curriculum (ATC), residents had opportunities to be involved in overnight admission calls with the emergency department (ED), which were initially supervised (joint calls), and as skills progressed, residents conducted calls and admitted patients independently.

Post-Covid-19 Cholangiopathy-A New Indication for Liver Transplantation: A Case Report.

Liver injury is one of the nonpulmonary manifestations described in coronavirus disease 2019 (COVID-19). Post-COVID-19 cholangiopathy is a special entity of liver injury that has been suggested as a variant of secondary sclerosing cholangitis in critically ill patients (SSC-CIP). In the general population, the outcome of SSC-CIP has been reported to be poor without orthotopic liver transplantation (OLT).

Comparison of pediatric brain-dead donors to donation after circulatory death donors in the United States.

In pediatrics, an increasing need for transplantable organs exists. This study aimed to describe the epidemiology of pediatric deceased donors in the United States. This retrospective observational study utilized data from the Organ Procurement and Transplantation Network (OPTN) from 2000 to 2015.

Dominant variants in PRR12 result in unilateral or bilateral complex microphthalmia.

Complex microphthalmia is characterized by small eyes with additional abnormalities that may include anterior segment dysgenesis. While many genes are known, a genetic cause is identified in only 4-30% of microphthalmia, with the lowest rate in unilateral cases. We identified four novel pathogenic loss-of-function alleles in PRR12 in families affected by complex microphthalmia and/or Peters anomaly, including two de novo, the first dominantly transmitted allele, as well as the first splicing variant.

Chimeric antigen receptor T-cell therapy for marrow and extramedullary relapse of infant acute lymphoblastic leukemia.

Chimeric antigen receptor (CAR) T-cells, engineered autologous T-cells that target antigens found in leukemia, have shown durable remissions in relapsed acute lymphoblastic leukemia (ALL). Infant ALL with KMT2A rearrangements (KMT2Ar) is a rare, aggressive form of leukemia associated with extramedullary disease both at diagnosis and at relapse, and overall outcomes for these patients are dismal. Here we report the successful use of tisagenlecleucel, a CAR T-cell product approved for relapsed/refractory ALL, in a patient with KMT2Ar infant ALL who was treated for combined marrow and extramedullary (renal) relapse.

Using a home oxygen weaning protocol and pCO to evaluate outcomes for infants with bronchopulmonary dysplasia discharged on home oxygen.

Background: Predischarge capillary blood gas partial pressure of carbon dioxide (pCO ) has been associated with increased adverse events including readmission. This study aimed to determine if predischarge pCO or 36-week pCO was associated with increased respiratory readmissions or other pulmonary healthcare utilization in the year after neonatal intensive care unit (NICU) discharge for infants with bronchopulmonary dysplasia (BPD) discharged with home oxygen, using a standardized outpatient oxygen weaning protocol.

Methods: This was a secondary cohort analysis of infants born <32 weeks gestational age with BPD, referred to our clinic for home oxygen therapy from either from our level IV NICU or local level III NICUs between 2015 and 2017.

Participation of Children and Adolescents in Live Crisis Drills and Exercises.

Children and adolescents should be included in exercises and drills to the extent that their involvement advances readiness to meet their unique needs in the event of a crisis and/or furthers their own preparedness or resiliency. However, there is also a need to be cautious about the potential psychological risks and other unintended consequences of directly involving children in live exercises and drills. These risks and consequences are especially a concern when children are deceived and led to believe there is an actual attack and not a drill and/or for high-intensity active shooter drills.

Compound heterozygous splicing CDON variants result in isolated ocular coloboma.

Ocular coloboma is caused by failure of optic fissure closure during development and recognized as part of the microphthalmia, anophthalmia, and coloboma (MAC) spectrum. While many genes are known to cause colobomatous microphthalmia, relatively few have been reported in coloboma with normal eye size. Genetic analysis including trio exome sequencing and Sanger sequencing was undertaken in a family with two siblings affected with bilateral coloboma of the iris, retina, and choroid.

Pleuropulmonary blastoma-like peritoneal sarcoma: a newly described malignancy associated with biallelic DICER1 pathogenic variation.

Since the original description of pathogenic germline DICER1 variation underlying pleuropulmonary blastoma (PPB), the spectrum of extrapulmonary neoplasms known to be associated with DICER1 has continued to expand and now includes tumors of the ovary, thyroid, kidney, eye, and brain among other sites. This report documents our experience with another manifestation: a primitive sarcoma that resembles PPB and DICER1-associated sarcoma of the kidney. These tumors are distinguished by their unusual location in the peritoneal cavity, associated with visceral and/or parietal mesothelium.