Medicine and Health Sciences Commons^™

Skin Soft Tissue Infection: Antibiotics For Abscesses, Children's Mercy Kansas City

Clinical Critically Appraised Topics

No abstract provided.

Severe Hypertriglyceridemia: Care For The Non-Diabetic Patient, Children's Mercy Kansas City

Clinical Pathways (Formally Known As Care Process Models)

No abstract provided.

Gastric Sleeve Eras - Use Of Transverse Abdominal Plane (Tap) Blocks And Reduction Of Pain In Laparoscopic Gastric Bypass Patient, Children's Mercy Kansas City

Clinical Critically Appraised Topics

No abstract provided.

Pharmacogenetic Testing In Patients With Autism Spectrum Disorder Evaluated In A Pediatric Precision Medicine Clinic, Rachel Goodson, Cy Nadler, Jennifer A. Wagner, Sarah Soden, Sarah Nyp, Tracy L. Sandritter

Posters

Pharmacogenetic Testing In Patients with Autism Spectrum Disorder Evaluated in a Pediatric Precision Medicine Clinic Purpose The purpose of this study is to investigate the demographic and presentation profiles of children with autism spectrum disorder (ASD) who present for evaluation in a pediatric precision medicine clinic. Methods: This retrospective, observational cohort study utilized data extracted from a pediatric precision medicine clinic database between 2010 and 2021 with recorded ICD9/10 codes of Autism Spectrum Disorder, Autistic Disorder, Pervasive Developmental Disorder, or Asperger’s Syndrome. Extracted variables included demographic data, presenting medication regimens and concerns to be addressed by precision medicine. Results: A …

Assessing Early Use And Complications Of Gastrostomy Blended Feeds., James Fraser, Kristen L. Sayers, Amy L. Pierce, Beth A. Orrick, Wendy Jo Svetanoff, Tolulope A. Oyetunji Md Mph, Shawn D. St Peter

Posters

Providers are hesitant to recommend using blended tube feeds (BF) after gastrostomy tube (GT) placement due to increased risk of bacterial contamination, nutrition inadequacy, tube blockages, and lack of data addressing clinical outcomes. Caregivers often feel that BF are more natural, better tolerated, and more cost-effective. We studied early use of BF, potential complications, and satisfaction among caregivers.

Severe Diastolic Dysfunction Following Prolonged Extracorporeal Membrane Oxygenation In A Pediatric Burn Patient., Cara Holton, Johanna I. Orrick, Igor Areinamo, Jenna Miller

Posters

A previously healthy 3-year-old female was admitted following a 22% TBSA scald burn. She was initially hemodynamically stable on room air. However, one week into hospitalization she developed ARDS with catecholamine-refractory shock requiring VA-ECMO. She was enterovirus positive, grew MRSA in respiratory cultures and was treated with prolonged antibiotics. The patient spent 26 days on VA-ECMO followed by 61 days on VV- ECMO after failing decannulation due to refractory hypoxia. She developed anuric renal failure requiring CRRT during her second ECMO course. Following VV-ECMO decannulation, she required mechanical ventilation with high settings and had severe recurrent pulmonary hemorrhage requiring FFP, …

Short And Long Term Outcomes Of Using Cryoablation For Post-Operative Pain Control In Patients After Pectus Excavatum Repair, James Fraser, Kayla B. Briggs, Wendy Jo Svetanoff, David Juang, Pablo Aguayo, Jason D. Fraser, Charles L. Snyder, Tolulope A. Oyetunji, Shawn D. St. Peter Md

Posters

Background: Previous prospective trials have demonstrated that intercostal cryoablation reduces length of stay and postoperative opioid consumption compared with thoracic epidural and patient controlled analgesia (PCA) modalities. We report the findings of a 3-year prospective, observational study to elucidate long-term pain control, symptoms, and complications with attention to postoperative short and long-term pain control associated with the cryoablation technique.

Methods: Following IRB approval, we prospectively collected data on patients who underwent bar placement for pectus excavatum with thoracoscopic intercostal cryoablation by six surgeons at our institution from 2017 to 2021. Patients and their parents completed surveys regarding pain scores, narcotic …

Descriptive Study Of The Safety Behaviors And Attitudes Of Portable Pool Owners, Kristyn Jeffries, Kathy W. Monroe, Alicia Webb, Kristin L. Chancellor, Justina C. Goldman, David C. Schwebel

Posters

Background Drowning is the leading cause of injury death for children 1-4 years old and the second leading cause for children 5-9 years old. Most prior epidemiology work has focused on submersions in below-ground swimming pools and natural bodies of water. Portable pools pose a new and emerging risk for drowning due to their affordability, convenience, and easy assembly. Successful drowning prevention consumer products, such as 4-sided fencing, may prove more difficult to implement with portable pools, and currently are not marketed for such use. Furthermore, parental perceptions and knowledge of drowning risks associated with portable pools has not yet …

Racial Disparities In Testicular Torsion, Kayla B. Briggs, Obiyo O. Osuchukwu, Chris E. Roberts, James Fraser, Wendy Jo Svetanoff, Tolulope A. Oyetunji, Hanna Alemayehu

Posters

Racial Disparities in Testicular Torsion Introduction: Testicular torsion (TT) is a surgical emergency requiring prompt intervention to preserve testicular function. Race and insurance status are thought to be associated with higher rates of gonadal loss; however, reports in the literature are conflicting. We sought to determine the effect of race on the treatment and outcomes of testicular torsion. Methods: Following IRB approval, a retrospective review was conducted of patients <18 years of age who presented to 2 institutions (hereby referred to as H1 and H2) with acute scrotal pain between December 2017 and September 2019. Demographic data, clinical history, imaging results, diagnosis, and surgical outcomes were recorded. Social vulnerability index (SVI), as determined by zip code, was obtained from the Centers for Disease Control and dichotomized using the 75th percentile. Higher SVI denotes increased social vulnerability. Primary outcome was diagnosis of TT. Secondary outcomes included orchiectomy rates. A p-value <0.05 was considered significant. Results: A total of 515 patients (H1 85%, H2 15%) were included in the final analysis. There were no difference in median age, BMI, TT diagnosis and orchiectomy rate between the two institutions. H2 treated more black patients (43% vs. 14%, p=<0.01) and patients with public insurance (57% vs. 41%, p=0.03) compared to H1. In multivariate analysis, older age was associated with TT (median age TT 14.12 years [12.13,15.69]) vs. no TT 9.86 years [5,13.05], OR 1.35, 95% CI 1.18-1.55, p=<0.01). Black patients were ~4 times more likely (OR 4.05, 95% CI 2.13-7.69, p=<0.01) than white patients to be diagnosed with TT when controlled for dichotomized SVI, insurance, and age. In the same model, Hispanic patients were ~2 times more likely (OR 2.20, 95% CI 1.10-4.40, p=0.03) to be diagnosed with TT when compared to white patients. Despite more black and Hispanic patients having dichotomized SVI >75th percentile than white patients (70% and 82%, respectively vs. 16%, p=<0.01), dichotomized SVI was not found to be associated with higher rates of TT or orchiectomy, respectively. Conclusion: At the two centers, more black and Hispanic children had SVI >75th percentile, however, when controlling for this in multivariate analysis, they were significant more likely to be diagnosed with …

A Proposal For Developing Academic Partnerships Between American Clinical Institutions And Ngos, Anik Patel, Nahreen Ahmed, Alfredo Mena Lora, Riley Jones

Posters

"Humanitarian and health-focused non-governmental organizations, such as MedGlobal, and American clinical institutions can mutually benefit from the formation of academic partnerships. MedGlobal has a longitudinal presence in ongoing humanitarian disasters that can provide rich clinical experiences for academic institutions looking to provide more diverse and equity-driven training for its residents and fellows. American institutions can provide resources, personnel with special skills and knowledge, as well as research assistance to MedGlobal and its partner sites as it continues to promote evidence-based clinical care for refugees. We propose an innovative model that addresses 3 areas of opportunities: education, research/QI, and clinical care. …

Organophosphate Pesticides And Progression Of Chronic Kidney Disease Among Children: A Prospective Cohort Study., Melanie H. Jacobson, Yinxiang Wu, Mengling Liu, Kurunthachalam Kannan, Adela Jing Li, Morgan Robinson, Bradley A. Warady, Susan Furth, Howard Trachtman, Leonardo Trasande

Manuscripts, Articles, Book Chapters and Other Papers

Background: Growing evidence suggests that exposure to environmental chemicals, such as pesticides, impacts renal function and chronic kidney disease (CKD). However, it is not clear if pesticides may affect CKD progression and no studies exist in children.

Objectives: The objective of this study was to examine associations between serially measured urinary OP pesticide metabolites and clinical and laboratory measures of kidney function over time among children with CKD.

Methods: This study used data on 618 participants enrolled in the CKD in Children study (CKiD), a cohort study of pediatric CKD patients from the US and Canada. Children were followed over …

Data-Driven Clustering Identifies Features Distinguishing Multisystem Inflammatory Syndrome From Acute Covid-19 In Children And Adolescents., Alon Geva, Manish M. Patel, Margaret M. Newhams, Cameron C. Young, Mary Beth F. Son, Michele Kong, Aline B. Maddux, Mark W. Hall, Becky J. Riggs, Aalok R. Singh, John S. Giuliano, Charlotte V. Hobbs, Laura L. Loftis, Gwenn E. Mclaughlin, Stephanie P. Schwartz, Jennifer E. Schuster, Christopher J. Babbitt, Natasha B. Halasa, Shira J. Gertz, Sule Doymaz, Janet R. Hume, Tamara T. Bradford, Katherine Irby, Christopher L. Carroll, John K. Mcguire, Keiko M. Tarquinio, Courtney M. Rowan, Elizabeth H. Mack, Natalie Z. Cvijanovich, Julie C. Fitzgerald, Philip C. Spinella, Mary A. Staat, Katharine N. Clouser, Vijaya L. Soma, Heda Dapul, Mia Maamari, Cindy Bowens, Kevin M. Havlin, Peter M. Mourani, Sabrina M. Heidemann, Steven M. Horwitz, Leora R. Feldstein, Mark W. Tenforde, Jane W. Newburger, Kenneth D. Mandl, Adrienne G. Randolph, Overcoming Covid-19 Investigators

Manuscripts, Articles, Book Chapters and Other Papers

Background: Multisystem inflammatory syndrome in children (MIS-C) consensus criteria were designed for maximal sensitivity and therefore capture patients with acute COVID-19 pneumonia.

Methods: We performed unsupervised clustering on data from 1,526 patients (684 labeled MIS-C by clinicians)clusters, followed by recursive feature elimination to identify characteristics of potentially misclassified MIS-C-labeled patients.

Findings: Of 94 clinical features tested, 46 were retained for clustering. Cluster 1 patients (N = 498; 92% labeled MIS-C) were mostly previously healthy (71%), with mean age 7·2 ± 0·4 years, predominant cardiovascular (77%) and/or mucocutaneous (82%) involvement, high inflammatory biomarkers, and mostly SARS-CoV-2 PCR negative (60%). Cluster …

Aberrantly Low Stat3 And Stat5 Responses Are Associated With Poor Outcome And An Inflammatory Gene Expression Signature In Pediatric Acute Myeloid Leukemia., P Narayanan, T-K Man, R B Gerbing, R Ries, A M Stevens, Y-C Wang, X Long, A S. Gamis, T Cooper, S Meshinchi, T A Alonzo, M S Redell

Manuscripts, Articles, Book Chapters and Other Papers

The relapse rate for children with acute myeloid leukemia is nearly 40% despite aggressive chemotherapy and often stem cell transplant. We sought to understand how environment-induced signaling responses are associated with clinical response to treatment. We previously reported that patients whose AML cells showed low G-CSF-induced STAT3 activation had inferior event-free survival compared to patients with stronger STAT3 responses. Here, we expanded the paradigm to evaluate multiple signaling parameters induced by a more physiological stimulus. We measured STAT3, STAT5 and ERK1/2 responses to G-CSF and to stromal cell-conditioned medium for 113 patients enrolled on COG trials AAML03P1 and AAML0531. Low …

Longitudinal Associations Between Family Conflict, Parent Engagement, And Metabolic Control In Children With Recent-Onset Type 1 Diabetes., Hannah Case, David D. Williams, Shideh Majidi, Diana Ferro, Mark A. Clements, Susana R. Patton

Manuscripts, Articles, Book Chapters and Other Papers

Introduction: We prospectively investigated the associations between diabetes-related family conflict, parent engagement in child type 1 diabetes (T1D) care, and child glycated hemoglobin (HbA1c) in 127 families of school-age children who we recruited within the first year of their T1D diagnosis.

Research design and methods: Parents completed the Diabetes Family Conflict Scale-Revised (DFCS-R) to assess for diabetes-related family conflict and the Diabetes Self-Management Questionnaire-Brief (DSMQ-Brief) to assess parent engagement in child T1D care at the initial study visit (T1) and at 12 (T2) and 27 (T3) months later. We also collected child HbA1c at these time points. Our analyses included …

Initiative To Increase The Rate Of Emergency Department Physician Preprocedure Time-Out Documentation., Arjun Sarin, Nikita Sharma, Shobhit Jain

Manuscripts, Articles, Book Chapters and Other Papers

The preprocedure time-out is an important safety measure to verify patient identity and accuracy of a planned procedure. The time-out is an institutional and Joint Commission requirement. However, physicians in our emergency departments (EDs) document it inconsistently. We aimed to improve physician preprocedure time-out documentation for deep sedation (ketamine and/or propofol) from 75% to 90%, and separately for cutaneous abscess incision and drainage (I&D) from 94% to 98% by June 2020.

Methods: We analyzed 1 year of baseline data and weekly electronic medical record (EMR) reports from November 2019 through June 2020. Our outcome measures were the rate of physician …

Brief Musculoskeletal Screen And Patient Education For Down Syndrome-Associated Arthritis., Jordan T. Jones, Chelsey Smith, Nasreen Talib

Manuscripts, Articles, Book Chapters and Other Papers

No abstract provided.

Pulmonary Embolism, Intermediate/High Risk, Children's Mercy Kansas City

Clinical Pathways (Formally Known As Care Process Models)

No abstract provided.

Gastric Sleeve Eras - Ketorolac In Gastric Bypass Patients And Risk Of Bleeding, Children's Mercy Kansas City

Clinical Critically Appraised Topics

No abstract provided.

Umbilical Access In Laparoscopic Surgery In Infants Less Than 3 Months: Single Institution Retrospective Review, James Fraser, Kayla B. Briggs, Wendy Jo Svetanoff, Rebecca M. Rentea, Pablo Aguayo, David Juang, Jason D. Fraser, Charles L. Snyder, Richard J. Hendrickson, Shawn D. St Peter, Tolulope A. Oyetunji

Presentations

PURPOSE: Umbilical access in laparoscopic surgery has been cited as a potential factor for increased complications in low birth weight infants and those less than three months old. Previous series noted a self-reported complication rate of 10.6% among 329 pediatric surgeons via anonymous survey, citing carbon dioxide (CO2) embolism as the most common complication. We report four-year outcomes with blunt transumbilical laparoscopic access to examine the safety of this technique. METHODS: Following IRB approval, a retrospective database of patients less than three months of age who underwent laparoscopic pyloromyotomy or inguinal hernia repair from 2016-2019 at a tertiary care academic …

Improving Skin And Soft Tissue Antibiotic Duration Concordance With National Guidelines In Pediatric Urgent Care Clinics, Megan Hamner, Amanda Nedved, Holly Austin, Donna Wyly, Alaina N. Burns, Diana King, Brian Lee, Rana El Feghaly

Posters

Introduction: Skin and soft tissue infections (SSTIs) are the second most common diagnosis leading to pediatric antibiotic prescriptions in the outpatient setting after respiratory diagnoses. Children with SSTIs often receive >7 days of antibiotics, although current guidelines recommend 5-7 days for most diagnoses. At CMH urgent care clinics (UCCs), only 58% patients received the recommended 5-7 days of antibiotics. We aimed to increase the percentage of patients receiving 5-7 days of oral antibiotics for SSTIs from 58% to 75% by December 31st, 2021. Methods: We formed a multidisciplinary team in April 2020. A provider survey assessed factors influencing prescribing habits. …

Reconsidering Perioperative Antibiotic Use In Elective Laparoscopic Cholecystectomy, Kayla B. Briggs, James Fraser, Wendy Jo Svetanoff, Charles L. Snyder, Pablo Aguayo, David Juang, Rebecca M. Rentea, Jason D. Fraser, Shawn D. St. Peter Md, Tolulope A. Oyetunji

Posters

Introduction: Prophylactic preoperative antibiotics (PPA) are questionable in cases with a low rate of surgical site infection (SSI). We report institutional PPA usage and SSI rates after elective laparoscopic cholecystectomy in a children’s hospital. Methods: Children <18 years old who underwent outpatient laparoscopic cholecystectomy between 7/2010 and 8/2020 were included. SSI was defined as clinical signs of infection, requiring antibiotics, within 30 days of surgery. Results: 502 patients met inclusion criteria; 50% were pre-operatively diagnosed with symptomatic cholelithiasis, 47% biliary dyskinesia, 2% hyperkinetic gallbladder, and 1% gallbladder polyp(s). The majority were female (78%) and Caucasian (80%). 60% (n=301) of patients received PPA while 40% (n=201) did not; 1.3% (n=4) of those who received PPA developed SSI compared to 5.5% (n=11) of those who did not (p=0.01). Though PPA use was associated with an 84% reduction in risk of SSI on multivariate analysis (p=0.01), all SSIs were superficial. One child required readmission for intravenous antibiotics while the remainder were treated with outpatient antibiotics. Gender, age, BMI, ethnicity, and preoperative diagnosis did not influence the likelihood of receiving PPA (Table 1). Conclusion: Given the relatively low morbidity of the superficial SSI, conservative use of PPA should be carefully considered in outpatient laparoscopic cholecystectomy to avoid contributing to antibiotic-related complications.

Are Body Surface Area Based Estimates Of Liver Volume Applicable To Children With Overweight Or Obesity? An In Vivo Validation Study., Chelsea Hosey-Cojocari, Sherwin S. Chan, Chance S. Friesen, Amie L. Robinson, Veronica Williams, Erica Swanson, Daniel O'Toole, Jansynn Radford, Neil J. Mardis, Trevor N. Johnson, J Steven Leeder, Valentina Shakhnovich

Manuscripts, Articles, Book Chapters and Other Papers

The liver is the primary organ responsible for clearing most drugs from the body and thus determines systemic drug concentrations over time. Drug clearance by the liver appears to be directly related to organ size. In children, organ size changes as children age and grow. Liver volume has been correlated with body surface area (BSA) in healthy children and adults and has been estimated by functions of BSA. However, these relationships were derived from "typical" populations and it is unknown whether they extend to estimations of liver volumes for population "outliers," such as children with overweight or obesity, who today …

Mortality In Children Treated With Maintenance Peritoneal Dialysis: Findings From The International Pediatric Peritoneal Dialysis Network Registry., Sophie Ploos Van Amstel, Marlies Noordzij, Dagmara Borzych-Duzalka, Nicholas C. Chesnaye, Hong Xu, Lesley Rees, Il-Soo Ha, Zenaida L. Antonio, Nakysa Hooman, William Wong, Karel Vondrak, Yok Chin Yap, Hiren Patel, Maria Szczepanska, Sara Testa, Monica Galanti, Jameela A. Kari, Charlotte Samaille, Sevcan A. Bakkaloglu, Wai-Ming Lai, Luisa Fernanda Rojas, Mabel Sandoval Diaz, Biswanath Basu, Alicia Neu, Bradley A. Warady, Kitty J. Jager, Franz Schaefer

Manuscripts, Articles, Book Chapters and Other Papers

RATIONALE & OBJECTIVE: Research on pediatric kidney replacement therapy (KRT) has primarily focused on Europe and North America. In this study, we describe the mortality risk of children treated with maintenance peritoneal dialysis (MPD) in different parts of the world and characterize the associated demographic and macroeconomic factors.

STUDY DESIGN: Prospective cohort study.

SETTING & PARTICIPANTS: Patients younger than 19 years at inclusion into the International Pediatric Peritoneal Dialysis Network registry, who initiated MPD between 1996 and 2017.

EXPOSURE: Region as primary exposure (Asia, Western Europe, Eastern Europe, Latin America, North America, and Oceania). Other demographic, clinical, and macroeconomic (4 …

Identifying The Content And Context Of Pain Within Paediatric Rheumatology Healthcare Professional Curricula In The Uk: A Summative Content Analysis., Rebecca Rachael Lee, Janet E. Mcdonagh, Mark Connelly, Sarah Peters, Lis Cordingley

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: The curriculum for professionals working in paediatric rheumatology should include pain but it is unclear to what extent this currently occurs. The aim of this study was to identify pain-related curriculum content and the context in which pain is presented in educational and training documentation for healthcare professionals in this clinical speciality.

METHODS: Core curricula documents from UK based professional organisations were identified in partnership with healthcare professionals. Documents were analysed using a summative content analysis approach. Key pain terms were quantified and weighted frequencies were used to explore narrative pain themes. Latent content was interpreted qualitatively to explore …

Rumination Syndrome In Children And Adolescents: A Mini Review., Marc Martinez, Sandeep Rathod, Hunter J. Friesen, John Rosen, Craig A. Friesen, Jennifer Verrill Schurman

Manuscripts, Articles, Book Chapters and Other Papers

Introduction: Rumination syndrome involves recurrent regurgitation of food and is believed to be underdiagnosed with patients experiencing long delays in diagnosis. It can be associated with significant social consequences, high rates of school absenteeism, and medical complications such as weight loss. The primary aims of the current review are to assess the literature regarding prevalence, pathophysiology, and treatment outcomes with a focus on neurotypical children and adolescents.

Results: Population studies in children/adolescents, 5 years of age or older, range from 0 to 5.1%. There are fewer studies in clinical settings, but the prevalence appears to be higher in patients with …

Ability Of Video Telemetry To Predict Unplanned Hospital Admissions For Single Ventricle Infants., Doaa Aly, Lori Erickson, Hayley S. Hancock, Johnathan Apperson, Monica Gaddis, Girish S. Shirali, Suma Goudar

Manuscripts, Articles, Book Chapters and Other Papers

Background: Our Cardiac High Acuity Monitoring Program (CHAMP) uses home video telemetry (HVT) as an adjunct to monitor infants with single ventricle during the interstage period. This study describes the development of an objective early warning score using HVT, for identification of infants with single ventricle at risk for clinical deterioration and unplanned hospital admissions (UHA).

Methods and Results: Six candidate scoring parameters were selected to develop a pragmatic score for routine evaluation of HVT during the interstage period. We evaluated the individual and combined ability of these parameters to predict UHA. All infants with single ventricle monitored at home …

Ventricular Fibrillation Due To A Likely Pathogenic Sos1 Variant: An Unrecognized Etiology Of Infantile Sudden Death?, Christopher Follansbee, Lindsey Malloy-Walton

Manuscripts, Articles, Book Chapters and Other Papers

We present the case of a female infant presenting after a ventricular fibrillation arrest found to have ectopic atrial tachycardia (EAT). Evaluation revealed a likely pathogenic variant in SOS1 not previously reported in affected individuals. SOS1 variants are associated with Noonan syndrome, which belongs to a family of related genetic syndromes affecting the RAS/MAPK signaling pathway. To date, this is the first case reported of a ventricular fibrillation arrest in a patient with a RASopathy-related variant prior to development of the typically associated structural cardiac phenotype and may represent a previously unrecognized etiology of sudden death during infancy.

Atypical Presentation Of Systemic Arthritis In A Toddler With Down Syndrome., Emily Worley, Weijie Li, Jordan T. Jones

Manuscripts, Articles, Book Chapters and Other Papers

Systemic juvenile idiopathic arthritis (sJIA) is a chronic, inflammatory disease of childhood, which is characterized by the combination of arthritis, serositis, daily, high-spiking fevers, and evanescent macular rash and can present with the life-threatening complication of macrophage activation syndrome (MAS). Children with Down syndrome (DS) have complex medical challenges related to abnormalities in their immune system, which can cause a broad spectrum of disease manifestations, which can occur atypically. Children with DS are at increased risk for arthritis and interstitial lung disease (ILD) associated with sJIA that has high mortality. This case report outlines an atypical presentation of sJIA in …

An Update On The Assessment And Management Of Pediatric Abdominal Pain., Craig A. Friesen, Jennifer Colombo, Amanda D. Deacy, Jennifer Verrill Schurman

Manuscripts, Articles, Book Chapters and Other Papers

Chronic abdominal pain is very common in children and adolescent and results in high personal and social costs. Most youth with chronic abdominal pain fulfill criteria for a functional abdominal pain disorder (FAPD) as defined by Rome criteria. These are complex conditions with a wide array of biological, psychological, and social factors contributing to the experience of pain. The purpose of the current review is to provide an overview of the pathophysiology of FAPDs and an up-to-date summary of the literature related to FAPDs in children and adolescents, with additional focus on several areas (eg, diet and probiotics) where patients …

Pooled Safety Analysis Of Tisagenlecleucel In Children And Young Adults With B Cell Acute Lymphoblastic Leukemia., John E. Levine, Stephan A. Grupp, Michael A. Pulsipher, Andrew C. Dietz, Susana Rives, Douglas Myers, Keith August, Michael R. Verneris, Jochen Buechner, Theodore W. Laetsch, Henrique Bittencourt, Andre Baruchel, Michael W. Boyer, Barbara De Moerloose, Muna Qayed, Stella M. Davies, Christine L. Phillips, Timothy A. Driscoll, Peter Bader, Krysta Schlis, Patricia A. Wood, Rajen Mody, Lan Yi, Mimi Leung, Lamis K. Eldjerou, Carl H. June, Shannon L. Maude

Manuscripts, Articles, Book Chapters and Other Papers

Background: Tisagenlecleucel, an anti-CD19 chimeric antigen receptor T cell therapy, has demonstrated efficacy in children and young adults with relapsed/refractory B cell acute lymphoblastic leukemia (B-ALL) in two multicenter phase 2 trials (ClinicalTrials.gov, NCT02435849 (ELIANA) and NCT02228096 (ENSIGN)), leading to commercialization of tisagenlecleucel for the treatment of patients up to age 25 years with B-ALL that is refractory or in second or greater relapse.

Methods: A pooled analysis of 137 patients from these trials (ELIANA: n=79; ENSIGN: n=58) was performed to provide a comprehensive safety profile for tisagenlecleucel.

Results: Grade 3/4 tisagenlecleucel-related adverse events (AEs) were reported in 77% of …