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2025

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Full-Text Articles in Pediatrics

Multimodal Imaging Directs Decision Making In Intraseptal L-Aaoca, Joseph Burns, Lindsay F Eilers, Stephen J Dolgner, Edward J Hickey, Silvana Molossi Jun 2025

Multimodal Imaging Directs Decision Making In Intraseptal L-Aaoca, Joseph Burns, Lindsay F Eilers, Stephen J Dolgner, Edward J Hickey, Silvana Molossi

Faculty, Staff and Students Publications

Background: Congenital coronary artery abnormalities represent an uncommon class of congenital heart disease with an associated risk of sudden cardiac death. There is a paucity of data for the evaluation and management of affected patients with an intraseptal course.

Case summary: A 27-year-old woman was found to have a single coronary trunk arising from the right sinus with a 2.7 cm intraseptal course of the left main coronary artery. She underwent stress cardiac magnetic resonance and catheterization, to assess for inducible ischemia before surgical repair with transconal unroofing. Her recovery was uneventful, and she remains free of symptoms.

Discussion: Management …


Modeling Reemergence Of Vaccine-Eliminated Infectious Diseases Under Declining Vaccination In The Us, Mathew V Kiang, Kate M Bubar, Yvonne Maldonado, Peter J Hotez, Nathan C Lo Jun 2025

Modeling Reemergence Of Vaccine-Eliminated Infectious Diseases Under Declining Vaccination In The Us, Mathew V Kiang, Kate M Bubar, Yvonne Maldonado, Peter J Hotez, Nathan C Lo

Faculty, Staff and Students Publications

Importance: Widespread childhood vaccination has eliminated many infectious diseases in the US. However, vaccination rates are declining, and there are ongoing policy debates to reduce the childhood vaccine schedule, which may risk reemergence of previously eliminated infectious diseases.

Objective: To estimate the number of cases and complications in the US under scenarios of declining childhood vaccination for measles, rubella, poliomyelitis, and diphtheria.

Design, setting, and participants: A simulation model was used to assess the importation and dynamic spread of vaccine-preventable infectious diseases across 50 US states and the District of Columbia. The model was parameterized with data on area-specific estimates …


Nosocomial Sepsis Triggered By Pluralibacter Gergoviae In A Pediatric Intensive Care Unit: A Case Report And Literature Review, Abdulrahman Özel, Nurettin Onur Kutlu, Servet Yüce, Cenk Özdalgiçoğlu, Meltem Erol Jun 2025

Nosocomial Sepsis Triggered By Pluralibacter Gergoviae In A Pediatric Intensive Care Unit: A Case Report And Literature Review, Abdulrahman Özel, Nurettin Onur Kutlu, Servet Yüce, Cenk Özdalgiçoğlu, Meltem Erol

Journal of Pediatric Infectious Diseases

Pluralibacter gergoviae is a rare opportunistic gram-negative bacillus in humans and exhibits resistance to preservatives in cosmetic products. In this case report, we present nosocomial P. gergoviae sepsis that developed through contamination from a cleaning product in a previously healthy child without known chronic diseases, pediatric intensive care unit care. Contamination with the same microorganism was detected even in unused samples of the cleaning product, leading to a recall of these items. This report highlights the case of hospital-acquired P. gergoviae sepsis to emphasize the importance of adherence to antiseptic protocols by health care work as well as the necessity …


New Insights On The Clinical Significance Of Mycoplasma Pneumoniae-Dna Load In Mycoplasma Pneumoniae Pneumonia, Chen Yu, Lin Chenxi, Huang Rui, Chen Qi, Zhang Min, Chen Ling, Lai Xingqian Jun 2025

New Insights On The Clinical Significance Of Mycoplasma Pneumoniae-Dna Load In Mycoplasma Pneumoniae Pneumonia, Chen Yu, Lin Chenxi, Huang Rui, Chen Qi, Zhang Min, Chen Ling, Lai Xingqian

Journal of Pediatric Infectious Diseases

Objective: This study aimed to assess the consistency of MP-DNA load in the respiratory tracts, its correlation with Mycoplasma pneumoniae pneumonia (MPP) manifestations, and its predictive value for refractory M. pneumoniae pneumonia (RMPP).

Methods: A retrospective study was performed on a cohort of MPP cases, including 215 patients with positive nasopharyngeal aspirate (NPA) MP-DNA and 59 with positive bronchoalveolar lavage fluid (BALF) MP-DNA. Patients were categorized into two groups based on MP-DNA load: low-load (≤106/mL) and high-load (>106/mL). The consistency of MP-DNA load in NPA and BALF was determined by Spearman’s correlation coefficient. Clinical, laboratory, and radiological data were …


Comparison Of Delta Neutrophil Index And Inflammatory Markers In The Diagnosis Of Late-Onset Neonatal Sepsis In Preterm Infants, Aybuke Yazici, Hayriye Gozde Kanmaz Kutman, Gulsum Kadioglu Simsek, Betul Siyah Bilgin, Fuat Emre Canpolat, Bedia Dinc, Serife Suna Oguz Jun 2025

Comparison Of Delta Neutrophil Index And Inflammatory Markers In The Diagnosis Of Late-Onset Neonatal Sepsis In Preterm Infants, Aybuke Yazici, Hayriye Gozde Kanmaz Kutman, Gulsum Kadioglu Simsek, Betul Siyah Bilgin, Fuat Emre Canpolat, Bedia Dinc, Serife Suna Oguz

Journal of Pediatric Infectious Diseases

Objective: To assess the performance of the relatively new hematological marker delta neutrophil index (DNI) and other inflammatory markers in proven sepsis of the very preterm infant.

Methods: Infants with a gestational age of <32 weeks and examined for suspected late-onset sepsis (LOS) were evaluated retrospectively. Demographic characteristics, laboratory findings, and neonatal morbidity and mortality were compared between infants with blood culture-confirmed sepsis and clinical sepsis.

Results: The study included 358 infants, 90 with proven sepsis and 268 with clinical sepsis. The mean gestational ages in these groups were 28.2±2 and 28.2±2.1 weeks, and the mean birth weight was 1139±372 and 1148±370 g, respectively (p>0.05). The proven sepsis group had higher DNI (median 7.1% [range, 0.1-74] vs. 0.6% [range, 0.1-24.7%], p<0.01], CRP (median 5 [range, 1-38] mg/L vs. 1 [range, 1-15] mg/L, p<0.001), interleukin 6 (median 620 [range, 6.9-5500] pg/mL vs. 42.2 [range, 2.2-5500] pg/mL, p<0.01), and procalcitonin (median 6.2 [range, 0.26-86] μg/L vs. 0.6 [range, 0.06-25.7] μg/L, p=0.002). According to ROC analysis, interleukin 6 had the highest area under the curve (AUC) at 0.74 (95% CI 0.60-0.88, p<0.01). The AUC values were 0.73 (95% CI 0.59-0.88, p=0.01) for procalcitonin, 0.62 (95% CI 0.44-0.80, p=0.18) for DNI, and 0.66 (95% CI 0.50-0.82, p=0.05) for CRP.

Conclusion: The delta neutrophil index showed a statistically significant elevation …


Diagnostic Value Of Lncrna Fgd5-As1 Sponge Mir-223-3p In Infantile Pneumonia And Its Prognostic Effect On Rehabilitation, Hong-Ping Ren, Hong-Lin Wen, Ya-Nan Liu, Lin Cai Jun 2025

Diagnostic Value Of Lncrna Fgd5-As1 Sponge Mir-223-3p In Infantile Pneumonia And Its Prognostic Effect On Rehabilitation, Hong-Ping Ren, Hong-Lin Wen, Ya-Nan Liu, Lin Cai

Journal of Pediatric Infectious Diseases

Objective: This study aimed to uncover the value of long non-coding RNA FGD5-AS1 (lncRNA FGD5-AS1) in the diagnosis of infantile pneumonia and explore its pathological mechanism in lung fibroblasts. This research may provide a potential biomarker for diagnosing patients and predicting their rehabilitation outcomes.

Methods: This study included 92 children with infantile pneumonia as the research object, and an equal number of healthy children were introduced as controls. The FGD5-AS1 content was detected using real-time quantitative polymerase chain reaction (RT-qPCR) assay. The diagnostic value of FGD5-AS1 was evaluated by receiver operating characteristic (ROC) and logistic analysis. The molecular mechanism of …


Hematological Parameters And Systemic Inflammatory Markers In Children With Viral Acute Lower Respiratory Tract Infections, Sadiye Sert, Fatma Sargin, Şerife Karaçal Say, Berna Erayman, Ebru Buldu Jun 2025

Hematological Parameters And Systemic Inflammatory Markers In Children With Viral Acute Lower Respiratory Tract Infections, Sadiye Sert, Fatma Sargin, Şerife Karaçal Say, Berna Erayman, Ebru Buldu

Journal of Pediatric Infectious Diseases

Objective: Viral acute lower respiratory tract infections (ALRTIs) are a leading cause of morbidity and mortality in children, particularly those under 5 years. Accurate diagnosis and timely management are crucial to improve outcomes. This study aimed to address the lack of comparative analysis between hematological and systemic inflammatory markers concerning established disease severity scores in children with viral ALRTIs. We sought to evaluate the correlations between these markers and severity scores to identify which parameters provide the most reliable predictive value for assessing the severity of ALRTI diagnoses in this population.

Methods: This retrospective cross-sectional study included 191 children hospitalized …


Evaluation Of Gastrointestinal Pathogens In Children With Inflammatory Bowel Disease Using Multiplex Polymerase Chain Reaction, Yeliz Çağan Appak, Özgür Appak, Betül Aksoy, Büşra Emi̇r, Ayça Arzu Sayiner, Maşallah Baran Jun 2025

Evaluation Of Gastrointestinal Pathogens In Children With Inflammatory Bowel Disease Using Multiplex Polymerase Chain Reaction, Yeliz Çağan Appak, Özgür Appak, Betül Aksoy, Büşra Emi̇r, Ayça Arzu Sayiner, Maşallah Baran

Journal of Pediatric Infectious Diseases

Objective: Impaired gastrointestinal mucosa and immunosuppressant therapies increase the risk of secondary infection in patients with inflammatory bowel disease (IBD). This study evaluated the detection of pathogens in children with IBD using a gastrointestinal panel (GP). This is the first study to compare this method with clinical data from pediatric IBD patients.

Methods: Children with newly diagnosed IBD or experiencing disease flares were included. Demographic data, clinical and laboratory findings, treatments, treatment durations, and disease activity were analyzed. Stool samples were assessed using multiplex real-time polymerase chain reaction with QIAstat-Dx GP®. Results were compared between groups.

Results: Thirty-five patients with …


Abdominal Tuberculosis In Children, Melike Emiroglu, Gulsum Alkan, Meltem Kiymaz, Sadiye Kubra Tuter Oz, Hatice Turk Dagi, Mehmet Öztürk, Zeliha Esin Çelik Jun 2025

Abdominal Tuberculosis In Children, Melike Emiroglu, Gulsum Alkan, Meltem Kiymaz, Sadiye Kubra Tuter Oz, Hatice Turk Dagi, Mehmet Öztürk, Zeliha Esin Çelik

Journal of Pediatric Infectious Diseases

Objective: To provide an insight into the presentation, diagnosis and management of gastrointestinal tuberculosis in children.

Methods: We reviewed the medical records of children who were diagnosed with gastrointestinal tuberculosis, between October 2013 and October 2023. The analysis was performed using descriptive statistics.

Results: During the study period, 11 of 76 tuberculosis pediatric patients (14.5%) were diagnosed with gastrointestinal tuberculosis. Six of 11 patients (54.5%) were female. The median age of the patients was 60 months (51-205 months). Eight of 11 patients had intra-abdominal lymph node involvement, 3 of 11 patients had intestinal tuberculosis and 2 of 11 patients had …


Urgent Implementation Of Point-Of-Care Testing For Group A Streptococcal Infection In A Uk Children’S Emergency Department, Ruth Shorrocks, Philip J. Turner, Thomas Fanshawe, Umasha Ukwatte, Jim Gray, James Taylor, Liliana Graveto, Gail Hayward, Chris Bird Jun 2025

Urgent Implementation Of Point-Of-Care Testing For Group A Streptococcal Infection In A Uk Children’S Emergency Department, Ruth Shorrocks, Philip J. Turner, Thomas Fanshawe, Umasha Ukwatte, Jim Gray, James Taylor, Liliana Graveto, Gail Hayward, Chris Bird

Journal of Pediatric Infectious Diseases

Objective: In late 2022, Western Europe and the United States experienced increased incidences of scarlet fever and life-threatening invasive group A strepto- coccal (iGAS) infections, especially among children. The outbreak was widely reported in the media in the United Kingdom, leading to increased emergency department (ED) attendances and threatened supplies of antibiotics. Our ED rapidly introduced point-of-care testing for GAS pharyngitis with the aim of rationalizing antibiotic prescribing.

Methods: We rapidly implemented the use of a molecular-based (polymerase chain reaction) point-of-care test for all children scoring 3 or higher on the McIsaac clinical prediction rule for GAS pharyngitis in children …


Epidemiology Of Acute Infectious Diarrhea-Associated Viruses In Children In Hangzhou, China, 2023, Ya-Jun Guo, Lin Li, Qin-Rui Lai, Wei Li Jun 2025

Epidemiology Of Acute Infectious Diarrhea-Associated Viruses In Children In Hangzhou, China, 2023, Ya-Jun Guo, Lin Li, Qin-Rui Lai, Wei Li

Journal of Pediatric Infectious Diseases

Objective: To investigate the epidemiological characteristics of children infected with diarrheal virus in Hangzhou, China, 2023.

Methods: From January 2023 to December 2023, 20,939 stool samples from children with acute infectious diarrhea were collected for detection of rotavirus A and human adenovirus, using Latex agglutination detection kits; 7584 stool samples were collected for detection of norovirus, using real‐time PCR reagent.

Results: 639 (3.0%, 639/20,939) tested positive for rotavirus A, and 1,201 (5.7%, 1,201/20,939) positive samples were detected by Colloidal gold method. The positive rates of norovirus were 42.2% (3,203/7,585). Among all age groups, the rotavirus A positive detection rate was …


Evaluation Of Pediatric Patients Diagnosed With Measles: A Retrospective Study In Istanbul, Zehra Yazi, Nahide Haykir Zehi̇r, Merve Akçay, Ufuk Yükselmiş, Ceren Çetin, Ayşe Karaaslan, Yasemin Akin Jun 2025

Evaluation Of Pediatric Patients Diagnosed With Measles: A Retrospective Study In Istanbul, Zehra Yazi, Nahide Haykir Zehi̇r, Merve Akçay, Ufuk Yükselmiş, Ceren Çetin, Ayşe Karaaslan, Yasemin Akin

Journal of Pediatric Infectious Diseases

Objective: Measles, predominantly impacting the pediatric population, manifests symptoms encompassing fever, rash, rhinorrhea, cough, conjunctivitis, accompanied by infrequent yet plausible life-threatening complications.

Methods: This retrospective study conducted between January 2022 and July 2023 at a 1105-bed tertiary hospital in Istanbul, Türkiye, was focused on children diagnosed with measles and aged(PCR) were defined as definite measles cases.

Results: A total of 78 pediatric patients were diagnosed with measles, of whom 40 were evaluated as definite (51.3%) cases. Among the definite cases, 29 (72.5%) and 11 (27.5%) were girls and boys, respectively. Their median age was 78 (42–158) months. It was observed …


Rcpneoperu Trial: A Cluster Randomized Pilot Trial To Assess Traditional Neonatal Resuscitation Compared To Partially Virtual Training In Remote Areas., Carlos A Delgado, Enrique Gómez Pomar, Pablo Velásquez, Víctor Sánchez, Roberto Shimabuku, Luis Huicho Jun 2025

Rcpneoperu Trial: A Cluster Randomized Pilot Trial To Assess Traditional Neonatal Resuscitation Compared To Partially Virtual Training In Remote Areas., Carlos A Delgado, Enrique Gómez Pomar, Pablo Velásquez, Víctor Sánchez, Roberto Shimabuku, Luis Huicho

Pediatrics Faculty Publications

OBJECTIVE: To assess the effects of a neonatal resuscitation training program using traditional training and partial distance learning.

METHOD: Through an open cluster-randomized trial, the authors compared a traditional approach involving face-to-face theory and practice sessions using information and communication technology to offer theory and distance examination, followed by face-to-face practice. Twelve health facilities were allocated by blocked randomization. Comparisons were made adjusting for clustering in qualitative and quantitative data. The primary outcome was the percentage of infants with heart rates ≥100 per minute at the second minute after birth. The authors performed a cluster-level analysis for cluster randomized trials, …


Risk Factors For Serious Bacterial Infections Among Young Infants With Hypothermia: Protocol For A Multicenter, Retrospective Case-Control Study, Sriram Ramgopal, Paul Aronson, Douglas Lorenz, Alexander Joseph Rogers, Andrea Tania Cruz Jun 2025

Risk Factors For Serious Bacterial Infections Among Young Infants With Hypothermia: Protocol For A Multicenter, Retrospective Case-Control Study, Sriram Ramgopal, Paul Aronson, Douglas Lorenz, Alexander Joseph Rogers, Andrea Tania Cruz

Faculty, Staff and Students Publications

Background: Hypothermia in young infants presenting to the emergency department (ED) may indicate a serious bacterial infection (SBI) such as a urinary tract infection, bacteremia, or bacterial meningitis. Improved understanding of the epidemiology of SBI in infants with hypothermia and the development of prediction models can help avoid unnecessary invasive procedures and antimicrobial exposure.

Objective: The aim of the study is to (1) describe the epidemiology of SBI and herpes simplex virus (HSV) among infants with hypothermia, (2) assess the role of biomarkers in predicting SBI, and (3) derive and internally validate a multivariable predictive model for SBI among infants …


Early Recurrent Midgut Volvulus Post Ladd's Procedure In A Newborn: A Case Report, Nicole Clarke, Joanna Sajdlowska, John Paul Bustamante, Nishith Bhattacharyya Jun 2025

Early Recurrent Midgut Volvulus Post Ladd's Procedure In A Newborn: A Case Report, Nicole Clarke, Joanna Sajdlowska, John Paul Bustamante, Nishith Bhattacharyya

Rowan-Virtua School of Osteopathic Medicine Departmental Research

Ladd's procedure is the standard surgical intervention for intestinal malrotation. Although generally curative, rare cases of early postoperative complications, including adhesions or recurrent volvulus, can necessitate reoperation. We present the case of a full-term newborn girl who underwent emergent Ladd's procedure on Day 3 of life for malrotation with volvulus. She was discharged on postoperative Day 17 but returned the following day with recurrent bilious emesis. Imaging suggested partial obstruction. Exploratory laparotomy revealed recurrent volvulus with two twists at the duodenojejunal junction, which was successfully reduced. The patient recovered well and was discharged on postoperative Day 8. This case underscores …


Education Improves Perceived Control But Not Risk Identification In Adolescents Regarding Fentanyl, Christine Bakos-Block, Francine R Vega, Marylou Cardenas-Turanzas, Bhanumathi Gopal, Tiffany Champagne-Langabeer Jun 2025

Education Improves Perceived Control But Not Risk Identification In Adolescents Regarding Fentanyl, Christine Bakos-Block, Francine R Vega, Marylou Cardenas-Turanzas, Bhanumathi Gopal, Tiffany Champagne-Langabeer

Faculty, Staff and Student Publications

Background/objectives: In 2022, 2.2 million adolescents were diagnosed with substance use disorders, including 265,000 with opioid use disorder. The National Survey on Drug Use and Health revealed that 130,000 adolescents misused prescription pain medications, often obtaining them from friends or relatives. This age group perceives weekly heroin use as less risky than those younger or older.

Methods: A questionnaire was developed for 7th to 12th graders in a rural Texas school district as part of a fentanyl awareness curriculum. The questionnaire included Likert scale, multiple choice, and yes/no questions. The participants were categorized into younger (grades 7th and 8th) and …


X-Linked Hypophosphatemia Management In Children: An International Working Group Clinical Practice Guideline., Dalal S. Ali, Thomas O. Carpenter, Erik A. Imel, Leanne M. Ward, Natasha M. Appelman-Dijkstra, Catherine Chaussain, Suzanne M. Jan De Beur, Pablo Florenzano, Hajar Abu Alrob, Rana Aldabagh, R Todd Alexander, Farah Alsarraf, Signe Sparre Beck-Nielsen, Martin Biosse-Duplan, Rachel K. Crowley, Karel Dandurand, Guido Filler, Lisa Friedlander, Seiji Fukumoto, Claudia Gagnon, Paul Goodyer, Corinna Grasemann, Chelsey Grimbly, Salma Hussein, Muhammad K. Javaid, Sarah Khan, Aneal Khan, Anna Lehman, Willem F. Lems, E Michael Lewiecki, Ciara Mcdonnell, Reza D. Mirza, Emmett Morgante, Archibald Morrison, Anthony A. Portale, Christina Rao, Yumie Rhee, Eric T. Rush, Heide Siggelkow, Sotirios Tetradis, Laura Tosi, Gordon Guyatt, Maria Luisa Brandi, Aliya A. Khan Jun 2025

X-Linked Hypophosphatemia Management In Children: An International Working Group Clinical Practice Guideline., Dalal S. Ali, Thomas O. Carpenter, Erik A. Imel, Leanne M. Ward, Natasha M. Appelman-Dijkstra, Catherine Chaussain, Suzanne M. Jan De Beur, Pablo Florenzano, Hajar Abu Alrob, Rana Aldabagh, R Todd Alexander, Farah Alsarraf, Signe Sparre Beck-Nielsen, Martin Biosse-Duplan, Rachel K. Crowley, Karel Dandurand, Guido Filler, Lisa Friedlander, Seiji Fukumoto, Claudia Gagnon, Paul Goodyer, Corinna Grasemann, Chelsey Grimbly, Salma Hussein, Muhammad K. Javaid, Sarah Khan, Aneal Khan, Anna Lehman, Willem F. Lems, E Michael Lewiecki, Ciara Mcdonnell, Reza D. Mirza, Emmett Morgante, Archibald Morrison, Anthony A. Portale, Christina Rao, Yumie Rhee, Eric T. Rush, Heide Siggelkow, Sotirios Tetradis, Laura Tosi, Gordon Guyatt, Maria Luisa Brandi, Aliya A. Khan

Manuscripts, Articles, Book Chapters and Other Papers

CONTEXT: An International Working Group (IWG) developed new guidelines on the diagnosis, evaluation, management, and monitoring of X-linked hypophosphatemia (XLH) in children. Over the past 5 years, important advances have occurred in our understanding of the presentation, complications, and treatment of XLH.

METHODS: A group of 50 international experts in XLH from Canada, the United States, Europe, Asia, and South America, along with methodology experts and a patient partner, held 18 teleconference meetings in 2023-2024. These meetings addressed key issues regarding diagnosing, evaluating, managing, and monitoring XLH in children. Two systematic reviews were conducted to examine the impact of burosumab …


In Vivo Direct Lentiviral Gene Therapy Improves Disease Pathology In A Mucopolysaccharidosis Iva Murine Model, Betul Celik, Estera Rintz, Shaukat Khan, Andrés F. Leal, Fnu Nidhi, Shunji Tomatsu Jun 2025

In Vivo Direct Lentiviral Gene Therapy Improves Disease Pathology In A Mucopolysaccharidosis Iva Murine Model, Betul Celik, Estera Rintz, Shaukat Khan, Andrés F. Leal, Fnu Nidhi, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Mucopolysaccharidosis IVA (MPS IVA) is an autosomal recessive disorder that causes the accumulation of keratan sulfate (KS) and chondroitin-6-sulfate in bone and cartilage. This results in progressive skeletal dysplasia, with no effective treatment available. Our study hypothesized that direct lentiviral vector (LV) gene therapy could produce active enzymes from transduced cells, impacting bone and cartilage lesions in MPS IVA. We developed LVs carrying the GALNS gene under three promoters: ubiquitous (CBh), collagen type II (COL2A1), and Mac-1 integrin subunit (CD11b). At the newborn stage and 4 weeks, Galns-knockout mice received intravenous injections at different doses (5 × 109 …


Performance Of A Novel Stool Quantitative Polymerase Chain Reaction Assay For Pediatric Tuberculosis Detection In Sub-Saharan Africa, Anca Vasiliu, Lucia Carratala-Castro, Abigail Seeger, Joanna Ehrlich, Babongile Nkala, Tara Ness, Miguel Mario Cumbe, Durbbin Mulengwa, Shilzia Munguambe, Bariki Mtafya, Edson Mambuque, Nosisa Shiba, Sozinho Acacio, Lilian Komba, Clement Gascua Adu-Gyamfi, H Lester Kirchner, Christoph Lange, Andrew R Dinardo, Alberto L Garcia-Basteiro, Anna Maria Mandalakas, Alexander Kay Jun 2025

Performance Of A Novel Stool Quantitative Polymerase Chain Reaction Assay For Pediatric Tuberculosis Detection In Sub-Saharan Africa, Anca Vasiliu, Lucia Carratala-Castro, Abigail Seeger, Joanna Ehrlich, Babongile Nkala, Tara Ness, Miguel Mario Cumbe, Durbbin Mulengwa, Shilzia Munguambe, Bariki Mtafya, Edson Mambuque, Nosisa Shiba, Sozinho Acacio, Lilian Komba, Clement Gascua Adu-Gyamfi, H Lester Kirchner, Christoph Lange, Andrew R Dinardo, Alberto L Garcia-Basteiro, Anna Maria Mandalakas, Alexander Kay

Faculty, Staff and Students Publications

Background: Children have paucibacillary tuberculosis and cannot provide expectorated sputum. Invasive specimen collection, by gastric aspiration or sputum induction, has a low diagnostic yield. In this study, we aimed to evaluate the diagnostic performance and additive yield of a novel stool-based assay in children diagnosed with tuberculosis in sub-Saharan Africa.

Methods: We conducted a prospective case-control study from October 2020 to June 2023 in Eswatini, Mozambique, and Tanzania. Children under 15 years newly diagnosed with tuberculosis completed clinical examination, chest radiography, culture, sputum Xpert Ultra, stool Xpert Ultra, and stool-based quantitative polymerase chain reaction (stool qPCR) assessment. Stool qPCR sensitivity …


Applying The Nonadoption, Abandonment, Scale-Up, Spread, And Sustainability (Nasss) Framework To Adapt The Champ App For Pediatric Feeding Tube Weaning: Application And Case Report., Dana Bakula, Alexandra Zax, Sarah T. Edwards, Kristina Nash, April Escobar, Rachel Graham, Amy Ricketts, Ryan Thompson, Sarah Bullard, Julianne Brogren, Leah Shimmens, Lori A. Erickson Jun 2025

Applying The Nonadoption, Abandonment, Scale-Up, Spread, And Sustainability (Nasss) Framework To Adapt The Champ App For Pediatric Feeding Tube Weaning: Application And Case Report., Dana Bakula, Alexandra Zax, Sarah T. Edwards, Kristina Nash, April Escobar, Rachel Graham, Amy Ricketts, Ryan Thompson, Sarah Bullard, Julianne Brogren, Leah Shimmens, Lori A. Erickson

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Evidence-based tube feeding (TF) weaning involves reducing the volume of tube feeds to induce hunger, with interdisciplinary monitoring to allow for proactive medical, nutritional, and behavioral intervention as needed. This can be done outpatient; however, the current standard of care requires a high degree of medical monitoring and care coordination, which can be challenging to implement. The CHAMP App is a mobile app designed for remote patient monitoring of children born with congenital heart conditions who are at high risk for medical morbidity and mortality. The CHAMP App remote patient monitoring program would be ideally suited to improve medical …


Prognosis After Switching To E-Cigarettes: Methods Raise Doubt About Conclusion, Harold J Farber Jun 2025

Prognosis After Switching To E-Cigarettes: Methods Raise Doubt About Conclusion, Harold J Farber

Faculty, Staff and Students Publications

No abstract provided.


Evaluation Of Aav Vectors With Tissue-Specific Or Ubiquitous Promoters In A Mouse Model Of Mucopolysaccharidosis Type Iva, Shaukat A. Khan, Jose Victor Álvarez, F. N. U. Nidhi, Eliana Benincore-Florez, Shunji Tomatsu Jun 2025

Evaluation Of Aav Vectors With Tissue-Specific Or Ubiquitous Promoters In A Mouse Model Of Mucopolysaccharidosis Type Iva, Shaukat A. Khan, Jose Victor Álvarez, F. N. U. Nidhi, Eliana Benincore-Florez, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Mucopolysaccharidosis type IVA (MPS IVA) is caused by a deficiency of N-acetyl-galactosamine-6-sulfate sulfatase (GALNS), leading to the accumulation of keratan sulfate and chondroitin-6-sulfate and development of severe skeletal dysplasia. Enzyme replacement therapy and hematopoietic stem cell transplantation are current treatment options but have limited impact on bone lesions. In this study, we investigated adeno-associated virus (AAV)8 or AAV9 vectors with liver-specific thyroxine-binding globulin or liver-specific promoter-a modification of hAAT (LSPX), liver-muscle tandem (LMTP), liver-bone tandem (LBTP), and ubiquitous cytomegalovirus early enhancer/chicken β-actin (CAG) promoters in MPS IVA mice to compare therapeutic efficacy on biochemical markers and bone pathology. All vectors …


Parallel Use Of Low-Complexity Automated Nucleic Acid Amplification Tests On Respiratory And Stool Samples With Or Without Lateral Flow Lipoarabinomannan Assays To Detect Pulmonary Tuberculosis Disease In Children, Laura Olbrich, Bada Yang, Hayley Poore, Alia Razid, Brittney Sweetser, Mathias Weis Damkjær, Alexander W Kay, Johanna Åhsberg, Ruvandhi R Nathavitharana, Ian Schiller, Nandini Dendukuri, Andreas Lundh, Maunank Shah, Stephanie Bjerrum, Devan Jaganath Jun 2025

Parallel Use Of Low-Complexity Automated Nucleic Acid Amplification Tests On Respiratory And Stool Samples With Or Without Lateral Flow Lipoarabinomannan Assays To Detect Pulmonary Tuberculosis Disease In Children, Laura Olbrich, Bada Yang, Hayley Poore, Alia Razid, Brittney Sweetser, Mathias Weis Damkjær, Alexander W Kay, Johanna Åhsberg, Ruvandhi R Nathavitharana, Ian Schiller, Nandini Dendukuri, Andreas Lundh, Maunank Shah, Stephanie Bjerrum, Devan Jaganath

Faculty, Staff and Students Publications

Background: Low-complexity automated nucleic acid amplification tests (LC-aNAATs) are molecular assays widely used to diagnose tuberculosis disease in children. The lateral flow urine lipoarabinomannan assay (LF-LAM) is recommended for use amongst children with HIV. Previous systematic reviews have assessed the diagnostic accuracy of LC-aNAATs and LF-LAM separately in children, but in clinical practice the tests may be used concurrently, i.e. in 'parallel'.

Objectives: To compare the diagnostic accuracy of the parallel use of LC-aNAAT on respiratory and stool specimens in children, and with LF-LAM on urine amongst children with HIV, versus each assay alone for detecting pulmonary tuberculosis disease.

Search …


Hematopoietic Stem Cell Transplantation Outcomes For High-Risk Aml: A Report From The Children's Oncology Group., Benjamin J. Huang, Lauren K. Meyer, Todd A. Alonzo, Yi-Cheng Wang, Adam J. Lamble, Rhonda E. Ries, Weijie Wang, Betsy Hirsch, Gordana Raca, Xiaotu Ma, Alan S. Gamis, Richard Aplenc, E Anders Kolb, Todd M. Cooper, Katherine Tarlock, Michael R. Loken, Soheil Meshinchi, Joseph H. Chewning, William G. Woods, John T. Horan Jun 2025

Hematopoietic Stem Cell Transplantation Outcomes For High-Risk Aml: A Report From The Children's Oncology Group., Benjamin J. Huang, Lauren K. Meyer, Todd A. Alonzo, Yi-Cheng Wang, Adam J. Lamble, Rhonda E. Ries, Weijie Wang, Betsy Hirsch, Gordana Raca, Xiaotu Ma, Alan S. Gamis, Richard Aplenc, E Anders Kolb, Todd M. Cooper, Katherine Tarlock, Michael R. Loken, Soheil Meshinchi, Joseph H. Chewning, William G. Woods, John T. Horan

Manuscripts, Articles, Book Chapters and Other Papers

PURPOSE: Hematopoietic stem cell transplantation (HSCT) is used as consolidation for pediatric patients with high-risk AML in first complete remission (CR1). The definition of high-risk AML has evolved considerably over the past two decades with the successive identification of new unfavorable risk factors. We conducted a cross-study analysis to determine whether HSCT improves the outcomes of patients with contemporarily defined high-risk AML.

METHODS: We combined data from AAML0531 and AAML1031, the last two phase III clinical trials completed by the Children's Oncology Group (COG). These two trials established the prognostic importance of measurable residual disease (MRD) and several high-risk cryptic …


Parallel Use Of Low-Complexity Automated Nucleic Acid Amplification Tests And Lateral Flow Urine Lipoarabinomannan Assays To Detect Tuberculosis Disease In Adults And Adolescents Living With Hiv, Stephanie Bjerrum, Bada Yang, Johanna Åhsberg, Laura Olbrich, Mathias Weis Damkjær, Ruvandhi R Nathavitharana, Tobias Broger, Ioana Diana Olaru, Brittney Sweetser, Hayley Poore, Alia Razid, Alexander W Kay, Claudia M Denkinger, Ian Schiller, Nandini Dendukuri, Devan Jaganath, Andreas Lundh, Maunank Shah Jun 2025

Parallel Use Of Low-Complexity Automated Nucleic Acid Amplification Tests And Lateral Flow Urine Lipoarabinomannan Assays To Detect Tuberculosis Disease In Adults And Adolescents Living With Hiv, Stephanie Bjerrum, Bada Yang, Johanna Åhsberg, Laura Olbrich, Mathias Weis Damkjær, Ruvandhi R Nathavitharana, Tobias Broger, Ioana Diana Olaru, Brittney Sweetser, Hayley Poore, Alia Razid, Alexander W Kay, Claudia M Denkinger, Ian Schiller, Nandini Dendukuri, Devan Jaganath, Andreas Lundh, Maunank Shah

Faculty, Staff and Students Publications

Background: Low-complexity automated nucleic acid amplification tests (LC-aNAATs) are molecular World Health Organization (WHO)-recommended rapid diagnostic tests (also known as mWRDs) widely used to diagnose tuberculosis disease. The lateral flow urine lipoarabinomannan assay (LF-LAM) is recommended by the WHO to assist in diagnosing tuberculosis disease amongst people with HIV. Previous systematic reviews have assessed the diagnostic accuracy of LC-aNAATs and LF-LAM used in isolation for the detection of tuberculosis, but in clinical practice the tests may be used in parallel (i.e. LC-aNAAT in combination with LF-LAM).

Objectives: To compare the diagnostic accuracy of the parallel use of LC-aNAAT on respiratory …


Real-World Outcomes For Young Adult Patients Receiving Cd19 Car T-Cell Therapy., Hannah Lust, Liora M. Schultz, Soyang Kwon, Gregory W. Roloff, Ibrahim Aldoss, Christina Baggott, Samuel John, Jenna E. Rossoff, Kevin O. Mcnerney, Vanessa A. Fabrizio, Julie-An Talano, Amy Moskop, Kevin J. Curran, Christine L. Phillips, Nicole Karras, Susanne H C Baumeister, Stacy L. Cooper, Michelle Hermiston, Prakash Satwani, Muna Qayed, Sunil S. Raikar, Margaret Macmillan, Erin Hall, Khanh Nguyen, Ryan D. Cassaday, Noam E. Kopmar, Vamsi K. Kota, John Mathews, Paul Shaughnessy, Marc S. Schwartz, Abdullah Ladha, George Yaghmour, Muthu V. Kumaran, Veronika Bachanova, Sean Tracy, Tamer Othman, Marlise R. Luskin, Evan C. Chen, Anjani S. Advani, Nikeshan Jeyakumar, Katharine Miller, Amy Zhang, Katherine C. Sutherland, Bijal D. Shah, Lori Muffly, Rawan Faramand Jun 2025

Real-World Outcomes For Young Adult Patients Receiving Cd19 Car T-Cell Therapy., Hannah Lust, Liora M. Schultz, Soyang Kwon, Gregory W. Roloff, Ibrahim Aldoss, Christina Baggott, Samuel John, Jenna E. Rossoff, Kevin O. Mcnerney, Vanessa A. Fabrizio, Julie-An Talano, Amy Moskop, Kevin J. Curran, Christine L. Phillips, Nicole Karras, Susanne H C Baumeister, Stacy L. Cooper, Michelle Hermiston, Prakash Satwani, Muna Qayed, Sunil S. Raikar, Margaret Macmillan, Erin Hall, Khanh Nguyen, Ryan D. Cassaday, Noam E. Kopmar, Vamsi K. Kota, John Mathews, Paul Shaughnessy, Marc S. Schwartz, Abdullah Ladha, George Yaghmour, Muthu V. Kumaran, Veronika Bachanova, Sean Tracy, Tamer Othman, Marlise R. Luskin, Evan C. Chen, Anjani S. Advani, Nikeshan Jeyakumar, Katharine Miller, Amy Zhang, Katherine C. Sutherland, Bijal D. Shah, Lori Muffly, Rawan Faramand

Manuscripts, Articles, Book Chapters and Other Papers

Tisagenlecleucel (tisa-cel) and brexucabtagene autoleucel (brexu-cel) are approved CD19 chimeric antigen receptor T-cell therapy (CAR T) products for young adults (YA) with relapsed/refractory B-cell acute lymphoblastic leukemia. A distinct analysis of YAs receiving commercial CD19 CAR T has not been reported. Using retrospective data from the Pediatric Real-World CAR T Consortium and the Real-World Outcomes of CAR T in Adult ALL collaboration, we describe the efficacy and safety of tisa-cel and brexu-cel in 70 YAs (18-26 years; tisa-cel, n = 50; brexu-cel, n = 20). Cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were observed more frequently …


Adolescent Scoliosis In Autism Spectrum Disorder: Is It Idiopathic Or Syndromic??, Ravi Rajendra, Matthew B. Bratton, Claudia Leonardi, R. Carter Clement Jun 2025

Adolescent Scoliosis In Autism Spectrum Disorder: Is It Idiopathic Or Syndromic??, Ravi Rajendra, Matthew B. Bratton, Claudia Leonardi, R. Carter Clement

School of Medicine Faculty Publications

Purpose: Minimal data exists on whether adolescents with both autism spectrum disorder (ASD) and scoliosis exhibit distinct curve types or require varying surgical management compared to neurotypical adolescents. Our study at a high-volume scoliosis center compares patterns of scoliosis in ASD patients with those of neurotypical patients. We hypothesized adolescents with ASD would present with atypical scoliosis curve characteristics compared to neurotypical adolescents. Methods: Using ICD-10 codes, we constructed an electronic database of adolescents aged 10 to 18 with diagnoses of both scoliosis and ASD. To avoid confounding with syndromic conditions, adolescents with a syndrome independently associated with scoliosis were …


Childhood Gastroesophageal Reflux Disease: A Comprehensive Review Of Disease, Diagnosis, And Therapeutic Management, Daniyal Raza, Farhan Mohiuddin, Muhammad Haris Khan, Maheen Fawad, Syed Musa Raza Jun 2025

Childhood Gastroesophageal Reflux Disease: A Comprehensive Review Of Disease, Diagnosis, And Therapeutic Management, Daniyal Raza, Farhan Mohiuddin, Muhammad Haris Khan, Maheen Fawad, Syed Musa Raza

School of Medicine Faculty Publications

Gastroesophageal reflux disease (GERD) affects both adults and children, although the symptoms differ significantly between these groups. While adults typically experience heartburn and regurgitation, children may present with more subtle signs, such as failure to thrive, chronic cough, wheezing, and Sandifer syndrome. Diagnosing GERD in children necessitates a multifaceted approach due to the diverse symptomatology and challenges in communication. Clinical assessment serves as the cornerstone of diagnosis, supported by tools like pH monitoring, esophageal impedance testing, and upper gastrointestinal endoscopy. Imaging studies, such as barium swallow, can also provide valuable insights into anatomical abnormalities and the extent of reflux. Treatment …


Oxygen Saturation Targeting In The Neonatal Intensive Care Unit, Faeq Almudares, Bheru Gandhi, Jonathan Davies, Xanthi Couroucli, Natalie Villafranco, Nidhy Paulose Varghese, Milenka Cuevas Guaman, Charleta Guillory, Binoy Shivanna Jun 2025

Oxygen Saturation Targeting In The Neonatal Intensive Care Unit, Faeq Almudares, Bheru Gandhi, Jonathan Davies, Xanthi Couroucli, Natalie Villafranco, Nidhy Paulose Varghese, Milenka Cuevas Guaman, Charleta Guillory, Binoy Shivanna

Faculty, Staff and Students Publications

Oxygen (O2) is vital for cellular development, function, proliferation, and repair, underscoring its critical role in organogenesis. Both hypoxia (reduced tissue O2) and hyperoxia (excess tissue O2), when prolonged, can trigger inflammation and oxidative stress, contributing to acute and long-term cardiopulmonary and neurodevelopmental morbidity. In sick neonates, immature defense mechanisms and coexisting morbidities demand nuanced and sometimes opposing strategies for O2 saturation targets and therapeutic titration. Most current neonatal O2 targeting guidelines are based on animal models or small clinical studies, resulting in recommendations with limited evidence. This narrative review aims to provide an updated overview of the physiological roles …


Emerging Therapeutic Potential Of Fisetin For Nephrotoxicity, Kidney Injury, And Nephropathy: A Systematic Review, Saeed Mohajeri, Alizamen Salehifard Jouneghani, Saeid Heidari-Soureshjani, Catherine M.T. Sherwin, Faramarz Beigi Jun 2025

Emerging Therapeutic Potential Of Fisetin For Nephrotoxicity, Kidney Injury, And Nephropathy: A Systematic Review, Saeed Mohajeri, Alizamen Salehifard Jouneghani, Saeid Heidari-Soureshjani, Catherine M.T. Sherwin, Faramarz Beigi

Pediatrics Faculty Publications

INTRODUCTION/OBJECTIVE: Kidney diseases cause high morbidity and mortality worldwide. This study investigated the mechanistic effects of Fisetin (FIS) on nephrotoxicity, kidney injury, and nephropathy induced by drugs, toxic chemicals, diabetes, lupus, diet, ureteral obstruction, and ischemic situations.

METHODS: To identify pertinent articles published before Oct 1, 2024, a comprehensive electronic search was performed across several databases, including MEDLINE/PubMed, Embase, Cochrane Library, Web of Science, and Scopus. After establishing clear inclusion and exclusion criteria, studies that met the research objectives were selected. Data were extracted and analyzed, documenting study characteristics, methodologies, and biological mechanisms.

RESULTS: Antioxidant benefits were evident with increased …