Pediatrics Commons^™

Anti-Inflammatory Diet And Probiotic Supplementation As Strategies To Modulate Immune Dysregulation In Autism Spectrum Disorder, Carlos Andrés Naranjo-Galvis, Diana María Trejos-Gallego, Cristina Correa-Salazar, Jessica Triviño-Valencia, Marysol Valencia-Buitrago, Andrés Felipe Ruiz-Pulecio, Luisa Fernanda Méndez-Ramírez, Jovanny Zabaleta, Miguel Andres Meñaca-Puentes, Carlos Alberto Ruiz-Villa, Marcela Orjuela-Rodriguez, Juan Carlos Carmona-Hernández, Luisa Matilde Salamanca-Duque

School of Medicine Faculty Publications

Background/Objectives: Autism spectrum disorder (ASD) is a neurodevelopmental condition associated with behavioral and cognitive impairments. Increasing evidence also links ASD with systemic immune dysregulation, including abnormal cytokine profiles and chronic low-grade inflammation. Emerging evidence suggests that targeted dietary strategies and probiotic supplementation may modulate immune responses and gut–brain interactions in patients with ASD. This study aimed to evaluate the immunomodulatory effects of a structured anti-inflammatory diet (NeuroGutPlus) compared to multi-strain probiotics in children with ASD. NeuroGutPlus is a nutritionally complete anti-inflammatory dietary protocol that targets gut integrity, inflammation, and mitochondrial function. It includes a diet low in gluten, FODMAPs, casein, …

Molecular Inflammatory Expression Profiles Associated With The Frequency Of Pain In Individuals With Sickle Cell Disease, Lana Mucalo Katunaric, Shuang Jia, Ashima Singh, Mark F. Roethle, Julie A. Panepinto, David C. Brousseau, Martin J. Hessner, Amanda M. Brandow

Department of Pediatrics Faculty Papers

Pain is the most common complication of sickle cell disease (SCD). The underlying biology of SCD pain is not well understood, which is a barrier to novel, effective analgesic and preventive therapies. A wide variability in the phenotypic expression of pain exists among individuals with SCD, despite the inheritance of a similar defective hemoglobin gene. This interindividual pain variability further complicates the ability to understand the biology and effectively treat pain. We sought to discover a biological signature comprising differentially expressed genes unique to SCD that could differentiate between individuals with varied pain frequency. We conducted plasma-induced transcription analysis from …

Botulinum Toxin For The Treatment Of Motor Tics In Children: A Case Series, Ethan Edmondson, Mariam Hull, Sukru Aras, Mered Parnes

Faculty, Staff and Students Publications

Tic disorders, including Tourette syndrome, are characterized by brief, repetitive, and patterned movements or vocalizations. Botulinum neurotoxin (BoNT) has emerged as a therapeutic option for motor tics, particularly when oral medications are insufficient or poorly tolerated. This retrospective cohort study evaluated the safety and effectiveness of botulinum neurotoxin injections in 50 pediatric patients (aged 7-18 years) with motor tic disorders treated at a tertiary pediatric movement disorders clinic between May 2019 and January 2024. Botulinum neurotoxin injections were tailored to individual symptomatology. Overall, 64% of patients reported improvement in tic severity, 36% noted no change, and 6% worsened. Response did …

Survival To Hospital Discharge In The Fetus With Moderate To Severe Mitral Regurgitation Either In Isolation Or In Combination With Aortic Valve Stenosis Or Atresia, A Fetal Heart Society Collaborative Study, Betul Yilmaz Furtun, Kavitha Pundi, Anita Moon-Grady, Nicole Cresalia, Asela Nieuwsma, Sarah K Gelehrter, Bhawna Arya, Mary T Donofrio, Christopher Statile, Ann Kavanaugh-Mchugh, Lindsay Freud, Elijah Bolin, Megan Warner, Sowmya Balasubramanian, James Strainic, Carol Wittlieb-Weber, Nelangi M Pinto, Lisa Hornberger, Shaine A Morris

Faculty, Staff and Students Publications

Background: Case series report high mortality after fetal diagnosis of moderate to severe mitral regurgitation in combination with left atrial dilation, hydrops, or restrictive atrial septum. However, overall outcomes and specific high-risk features remain unclear.

Methods: We performed a multicenter retrospective cohort study of fetuses with ≥moderate mitral regurgitation and normal cardiac connections evaluated January 1, 2005 to January 31, 2016. Fetuses undergoing fetal cardiac intervention (FCI) were described separately. We assessed associations between fetal echocardiographic features and discharge mortality using mixed logistic regression and classification and regression tree modeling, stratified by studies performed < 28 and ≥28 weeks gestational age (GA).

Results: Of 67 fetuses, 96% had …

Leishmania In Texas: A Contemporary One Health Scoping Review Of Vectors, Reservoirs, And Human Health, Morgan H Jibowu, Richard Chung, Nina L Tang, Sarah Guo, Leigh-Anne Lawton, Brendan J Sullivan, Dawn M Wetzel, Sarah M Gunter

Faculty, Staff and Students Publications

Leishmaniasis, a vector-borne neglected tropical disease, affects over 6.2 million people globally. Case acquisition is increasingly recognized in the United States, and in Texas, most reported cases are locally acquired and speciated to Leishmania mexicana. We conducted a scoping literature review to systematically assess contemporary research on Leishmania in humans, animals, reservoir hosts, or vectors in Texas after 2000. Out of 22 eligible studies, the most prevalent themes were case reports, followed by studies on domestic animals, reservoirs, and vectors, with several studies bridging multiple disciplines. Climate change, urbanization, and habitat encroachment appear to be driving the northward expansion …

Provider Attitudes And Perspectives On Rehabilitation For Pediatric Cancer Patients, Maria C Swartz, Eduardo Gonzalez Villarreal, Keri Schadler, Donna Kelly, Alakh P Rajan, Clark Andersen, Shiming Zhang, Stephanie J Wells, Amy Heaton, Karen M Moody

Faculty, Staff and Student Publications

PurposeTwenty percent of childhood cancer survivors experience physical function impairments, and ∼75% develop a chronic health condition. Physical and occupational therapists (PT/OTs) can mitigate these late effects, yet few children receive cancer rehabilitation (CR). This research aimed to identify provider attitudes and perspectives towards CR services for children across inpatient and outpatient settings at a cancer center.MethodsThree cardiac rehabilitation instruments were adapted to evaluate knowledge, attitudes, and perceptions regarding CR delivery. Descriptive statistics were used to summarize participant survey results.ResultsTwenty administrators, 20 physicians/advanced practice providers (APPs), and 20 PT/OTs completed surveys. All disciplines strongly agreed on the value of CR …

A Clinic-Based Healthcare Transition Preparation Program For Adolescents And Young Adults With Type 1 Diabetes: Study Protocol For The Shift Randomized Clinical Trial, Laura J Caccavale, Randi Streisand, Jessica Gokee Larose, Edmond P Wickham, Marisa E Hilliard, Laura M Thornton, Melanie K Bean

Faculty, Staff and Students Publications

Background: A striking 83 % of adolescents and young adults (AYA) with type 1 diabetes (T1D) have glycemic outcomes outside the target range, placing them at risk for acute and chronic complications. Identification of evidence-based strategies to enhance T1D management in AYAs, prior to the transition from pediatric to adult healthcare, is needed to optimize AYA health and adequately prepare them for independence. We present the design of Supporting Health Improvement for Transition in T1D (SHIFT), a randomized clinical trial of a clinic-based transition preparation intervention for AYAs with T1D.

Methods: Participants will be 50 AYAs with T1D (age 16-22 …

Outcomes Of Suprapatellar Intramedullary Nail Fixation Of Tibial Shaft Fractures In Skeletally Immature Patients, Carlos D Pargas-Colina, Tori J Coble, Sara E Davis, David D Spence, Jonathan K Rowland, Derek M Kelly, Benjamin W Sheffer

Faculty, Staff and Students Publications

Background: Tibial shaft fractures in children require individualized treatment approaches, including elastic nails, plates and screws, intramedullary rigid nails (IMN), and external fixators, with selection based on fracture characteristics and patient factors. IMN risks damaging the physis, but can provide enhanced stability and immediate weight bearing. While both infrapatellar and suprapatellar approaches exist for IMN placement, studies in adults have demonstrated that the suprapatellar approach offers higher patient satisfaction, less anterior knee pain, and lower radiation exposure. However, there is a lack of data on IMN for pediatric tibial fractures.

Methods: This single-center, retrospective cohort study analyzed patients aged 11-16 …

Addressing Anxiety And Depression In The Allergy Clinic Through Motivational Interviewing, Brief Cognitive Behavioral Therapy, And Curious Questions, Marylee Verdi, Dominic Candido, Juliette Madan, Jonathan A Bernstein, Don Bukstein, Aikaterini Anagnostou, Matthew Greenhawt, S Shahzad Mustafa, Julie Wang, Anna Nowak-Wegrzyn, Elissa M Abrams, John Oppenheimer, Alexandra E Conway, Andrew A White, Taylor A Doherty, Richard P Morse, Marcus Shaker

Faculty, Staff and Students Publications

Allergic diseases, particularly food allergy, can be associated with significant psychosocial impairment. Allergist-immunologists can provide evidence-based information to correct misperceptions and misunderstandings regarding food allergy that may perpetuate cycles of fear and anxiety. Whereas motivational interviewing can be an empathetic approach to empower patients to actualize health goals, cognitive behavioral therapy (CBT) aims to provide disease-state insight to facilitate an understanding of risk that can transform health beliefs and behaviors. Brief CBT is a clinical approach that can help address ineffective management paradigms through conversations that begin with curious questions. In this review, we discuss evidence supporting implementation of an …

Anaphylaxis Definition, Overview, And Clinical Support Tool: 2024 Consensus Report-A Ga2len Project, Timothy E Dribin, Antonella Muraro, Carlos A Camargo, Paul J Turner, Julie Wang, Graham Roberts, Aikaterini Anagnostou, Susanne Halken, Jay Liebermann, Margitta Worm, Torsten Zuberbier, Hugh A Sampson, Ga(2)Len Anaphylaxis Study Team

Faculty, Staff and Students Publications

Background: The 2006 National Institute of Allergy and Infectious Disease/Food Allergy and Anaphylaxis Network anaphylaxis criteria are widely used in clinical care and research. In 2020, the World Allergy Organization published modified criteria that have not been uniformly adopted. Different criteria contribute to inconsistent care and research outcomes.

Objective: We sought to develop a consensus anaphylaxis definition, overview, and clinical support tool.

Methods: A 12-member writing group developed draft outputs modified with input from a 46-member international expert panel, 31 medical stakeholder organizations, and 15 patient advocacy organizations. The expert panel participated in a modified Delphi process to seek consensus …

Novel Parent Survey Measures Sensory Behaviors Incorporating Sensory Modality And Stimulus Intensity, Connor Yee, Ellie Rather, Sakib Hoque, Joon Lee, Stephanie Marton, Jill Roth, Frederick L Oswald, Kenneth J Nobleza, Ankona Banerjee, Holly Harris, Ariel M Lyons-Warren

Faculty, Staff and Students Publications

Objective: Sensory behaviors are common, clinically relevant features of many neurodevelopmental disorders, including autism. However, existing assessments lack granularity in evaluating hypersensitivity and hyposensitivity, particularly in relation to stimulus intensity. We address this gap with our Sensory Response Questionnaire Phenotyper (SRQP), a parent-questionnaire that comprehensively assesses sensory behaviors across five sensory modalities. The purpose of this study was to validate the SRQP against established tools and assess its efficacy in identifying nuanced sensory processing patterns in children with autism.

Methods: A convenience sample of 317 participants (57 with autism, 260 typically developing) aged 3-17 years were enrolled in a cross-sectional …

Use Of Granulocyte-Colony Stimulating Factor For Beta-Lactam Induced Neutropenia In Children With Bacterial Meningitis, Cheng Yu Yen, Sheldon L Kaplan, Debra L Palazzi, Grant Stimes

Faculty, Staff and Students Publications

Drug induced neutropenia is an uncommon but potentially serious side effect in children receiving prolonged β-lactam antibiotic therapy. Management of β-lactam induced neutropenia in children remains challenging and often requires antibiotic therapy interruption or modification. There are limited data in pediatric patients about use of granulocyte-colony stimulating factor (G-CSF) for the treatment of drug induced neutropenia. We report the use of G-CSF for β-lactam induced neutropenia in four pediatric patients between the ages of 3 months and 18 years with bacterial meningitis in this case series.

Common Cast Complications, Jessica A Mcgraw-Heinrich, Jon C Wall, Scott B Rosenfeld

Faculty, Staff and Students Publications

Complications can arise from the application, wear, and removal of casts and splints. This text reviews common complications, risk factors, and prevention strategies while exploring the medical-legal implications of these complications. Being aware of common pitfalls can help reduce risk and promote optimal patient outcomes.

Key concepts: (1)Proper and judicious casting and splinting techniques are critical to prevent complications.(2)Thermal injuries can occur due to excessive curing heat and cast saw burns.(3)Poor cast molding can lead to skin sores and loss of fracture reduction.(4)Patients should be comfortable in their cast, and uncontrolled pain warrants investigation.

The 2024 Think Tank On Prevention Of Sudden Cardiac Death In The Young: Pathway To Survival A Report From The Cardiac Safety Research Consortium, Jordan E Ezekian, Charles C Anderson, Peter F Aziz, Samia Baluch, Stuart Berger, Martha Lopez-Anderson, Vincent Miller, Yoshihide Mitani, Silvana Molossi, Valarie Morrow, Victoria L Vetter, Elizabeth Vickers Saarel, Bhavya Trivedi, Salim F Idriss, 2024 Think Tank Participants

Faculty, Staff and Students Publications

Sudden cardiac arrest and death in the young is a critical public health issue. It occurs in children of any age, sex, racial or ethnic demographic, or socioeconomic status. Importantly, it can affect any individual-athlete and nonathlete alike. Prevention of sudden death in the young is of high importance not only because of the loss of a young life but also because of the substantial impact to families and to society at large. This White Paper summarizes the proceedings of a third national Think Tank on prevention of sudden cardiac death in the young. The Think Tank, which convened on …

Semaphorin 3e-Plexin-D1 Pathway Downstream Of The Luteinizing Hormone Surge Regulates Ovulation, Granulosa Cell Luteinization, And Ovarian Angiogenesis In Mice, Hanxue Zhang, Jimmy Dhillon, Paul D Soloway, Bo Shui, Seoyeon Lee, Jennifer K Grenier, Paul R Munn, M Cecilia Ljungberg, Rebecca B Williams, Rainer B Lanz, Yu-Hsiang Liao, Yi A Ren

Faculty, Staff and Students Publications

Ovulation is induced by the luteinizing hormone (LH) surge and accompanied by granulosa cell luteinization and ovarian angiogenesis. Semaphorin 3E (Sema3E)-Plexin-D1 pathway regulates angiogenesis in other tissues, but its role in the ovary is unknown. Evidence indicates that Sema3E-Plexin-D1 pathway plays an important role in the mouse ovary. The expression of Sema3E and its receptor, Plexin-D1, is dynamically regulated in the mouse ovary downstream of the LH surge. This regulation requires the modulation of chromatin accessibility by CCAAT/enhancer-binding proteins α and β. Intraovarian injection of recombinant Sema3E results in reduced ovulation, impaired corpus luteum formation, and aberrant ovarian angiogenesis. These …

Improving Pediatric Heart Failure Outcomes Through Collaboration And Innovation: The Current Status Of The Advanced Cardiac Therapies Improving Outcomes Network (Action), Lydia K Wright, David M Peng, Joseph A Spinner, Jenna M Murray, Matthew J O'Connor, Christina J Vanderpluym, David N Rosenthal, Lauren Smyth, Angela Lorts

Faculty, Staff and Students Publications

The Advanced Cardiac Therapies Improving Outcomes Network (ACTION) was founded in 2017 as a learning health care system to bring together all stakeholders with the aim of improving outcomes for pediatric and congenital heart disease patients with heart failure. From an initial focus on the ventricular assist device (VAD) population, work in the network has expanded to encompass the scope of heart failure in these patients. Through the collection and use of real-world data, the field has gained greater understanding of best practices in care of these patients, as well as improved access to devices with pediatric labeling of devices …

High Risk Of Drug-Resistant Tuberculosis In Igra-Negative Contacts: Should Preventive Treatment Be Considered?, Thomas Theo Brehm, Niklas Köhler, Hans-Peter Grobbel, Jürgen Welling, Anna Maria Mandalakas, Vinicius Fava, Erwin Schurr, Christoph Lange

Faculty, Staff and Students Publications

Purpose: Deciding whether to provide preventive treatment to contacts of individuals with multidrug-resistant (MDR) tuberculosis is complex.

Methods: We present the diagnostic pathways, clinical course and outcome of tuberculosis treatment in eight siblings from a single family. Tuberculosis disease was diagnosed by Mycobacterium tuberculosis culture and molecular detection of M. tuberculosis-specific DNA from bronchopulmonary specimens using GeneXpert® MTB/RIF. M. tuberculosis infection was diagnosed by an interferon-gamma release assay (IGRA; QuantiFERON®-TB Gold Plus). Whole exome sequencing for genetic predisposition to mycobacterial infection was performed in one patient.

Results: Six of eight siblings aged 16-20 years from a migrant family of Somali …

Impact Of Resources And Structure On Scholarly Productivity In Pediatric Critical Care Fellowship, Andrew G Smith, Danny Castro, Caroline Andy, Angela S Czaja, Donald L Boyer, Erika L Abramson, Pnina Weiss, Richard B Mink

Faculty, Staff and Students Publications

Background: Pediatric critical care medicine (PCCM) fellowship structures vary widely among programs and have not been examined since 2006. The development of scholarly knowledge and skills is an important part of fellowship training. However, the optimal PCCM fellowship structure to enable fellows' scholarly productivity is unknown. Objective: We examined PCCM fellowship time structure and resources to better understand their association with scholarly productivity.

Methods: This is a secondary analysis of cross-sectional survey data from PCCM fellowship program directors. We defined highly productive programs as those in which greater than 75% of fellows published papers from their scholarly projects in the …

Respiratory Failure In Patients With Intracerebral Hemorrhage And Intraventricular Hemorrhage Extension: A Retrospective Study, Min Cheol Chang, Michael Y Lee, Sang Gyu Kwak, Ah Young Lee

Faculty, Staff and Students Publications

Background/Objectives: This study aimed to identify the risk factors for respiratory failure in patients with intracerebral hemorrhage (ICH) accompanied by intraventricular hemorrhage (IVH) extension.

Methods: We retrospectively included 208 patients with ICH accompanied by IVH extension. Respiratory failure was defined as carbon dioxide levels > 45 mmHg with a pH < 7.35 in arterial blood gas analysis (ABGA) or the application of a ventilator due to respiratory dysfunction. We measured the severity of IVH extension using the Graeb scale, and ICH volume was assessed for each patient.

Results: Of the 208 included patients, 83 had respiratory failure. There were no significant differences in age, sex ratio, or Graeb scale score between patients with and without respiratory failure (p > 0.05). However, ICH volume was significantly larger in patients with respiratory failure (42.0 ± 42.5 mL) than …

Xpert Mtb/Rif Ultra Assay For Pulmonary Tuberculosis And Rifampicin Resistance In Adults And Adolescents, David J Horne, Jerry S Zifodya, Adrienne E Shapiro, Elizabeth Chandler Church, Jonah S Kreniske, Alexander W Kay, Katie Scandrett, Karen R Steingart, Yemisi Takwoingi

Faculty, Staff and Students Publications

Background: Xpert MTB/RIF Ultra (Xpert Ultra) is a molecular World Health Organization (WHO)-recommended rapid diagnostic test that simultaneously detects tuberculosis and rifampicin resistance. This review updates a comparative accuracy Cochrane review of Xpert MTB/RIF and Xpert Ultra as Xpert Ultra has replaced Xpert MTB/RIF.

Objectives: To determine the diagnostic accuracy of Xpert MTB/RIF Ultra (Xpert Ultra) for detecting pulmonary tuberculosis and rifampicin resistance in adults and adolescents with presumptive tuberculosis based on signs or symptoms or with an abnormal chest x-ray suggestive of tuberculosis.

Search methods: We searched seven databases including CENTRAL, MEDLINE, and Embase, plus two trial registers (ClinicalTrials.gov …

The Feasibility Of Passively Tracking Children’S Tv Viewing And Mobile Device Use In Naturalistic Settings, Teresia M O'Connor, Tatyana Garza, Uzair Alam, Anil Kumar Vadathya, Jennette P Moreno, Alicia Beltran, Samah Haidar, Nimah Haidar, Sheryl O Hughes, Debbe Thompson, Salma M A Musaad, Tom Baranowski, Jason A Mendoza, Joseph Young, Akane Sano, Ashok Veeraraghavan

Faculty, Staff and Students Publications

Research on children's technology and digital media (TDM) is hampered by a lack of robust approaches for assessing TDM use. This study assessed the feasibility of passively measuring children's TV screens and mobile devices (TDM) in a naturalistic setting. In the three-day feasibility study, FLASH-TV was set up on one to two TVs the child (5-12 year olds) typically used in the home (n=20). Children's mobile device use was assessed with either the Chronicle App or ScreenTime screenshots. Parents completed three TDM diaries. An exit interview with the parent explored their perceptions of the assessments and the child's TDM use …

Genome Report: Whole-Genome Assembly Of The Relapsing Fever Tick Ornithodoros Turicata Dugès (Acari: Argasidae), Mackenzie Tietjen, Amanda R Stahlke, David Luecke, Perot Saelao, Sheina B Sim, Scott M Geib, Brian E Scheffler, Anna K Childers, Alexander R Kneubehl, Pete D Teel, Job E Lopez

Faculty, Staff and Students Publications

The soft tick family Argasidae contains vectors of medical and veterinary importance, but few molecular resources are available compared to hard ticks (Ixodidae). One example is Ornithodoros turicata, a recognized vector of Borrelia turicatae, causal agent of human relapsing fever, and a putative vector of African swine fever virus. To address the current lack of molecular resources for the Argasidae, we generated a chromosome-level genome assembly for O. turicata using PacBio sequencing in conjunction with an Illumina Hi-C library. The resulting reference genome has a total of 1.1 Gb in length and was assembled into 10 chromosomes and 368 unplaced …

Clinically Important Endpoints In Individuals With Leukodystrophy: A Multisite Study, Emma R Kotes, Sarah Woidill, Russell D'Aiello, Amina Khan, Jacob Mccann, Mark Ramos, Francesco Gavazzi, Stephanie Keller, Keith Van Haren, Ali Fatemi, Florian Eichler, Joshua Bonkowsky, Jamie Fraser, Lisa Emrick, Omar Sherbini, Ashley Hackett, Jeilo Gauna, Dandre Amos, Jordan Goodman, Amena Smith Fine, Amanda Nagy, Seungil Lee, Nicole Page, Johanna Schmidt, Amy Pizzino, Kayla Muirhead, Mariko Bennett, Amy Waldman, Justine Shults, Laura Adang, Robert Grundmeier, Adeline Vanderver

Faculty, Staff and Students Publications

Importance: Leukodystrophies are a diverse group of rare disorders that disrupt central myelination. These disorders present with a broad spectrum of neurologic severity and are associated with a range of potential secondary complications, such as scoliosis and failure of independent feeding.

Objective: we explore real-world data of leukodystrophy complications to inform future evidence-based care guidelines across these rare diseases.

Design: In a cross-sectional observational study, we use a leukodystrophy-specific research consortium and the availability of electronic health records (EHR) to capture a cross-section of real-world data.

Setting: Subjects were identified using EHR data from five hospital systems with established expertise …

Development And Validation Of A Novel Clinical Risk Score To Predict Hypoxaemia In Children With Pneumonia Using The Who Prepare Dataset, Rainer Tan, Arjun Chandna, Tim Colbourn, Shubhada Hooli, Carina King, Norman Lufesi, Eric D Mccollum, Charles Mwansambo, Joseph L Mathew, Clare L Cutland, Shabir A Madhi, Marta Nunes, Sudha Basnet, Tor A Strand, Kerry-Ann F O'Grady, Brad Gessner, Emmanuel Addo-Yobo, Noel Chisaka, Patricia Hibberd, Prakash M Jeena, Juan M Lozano, William B Maleod, Archana Patel, Donald M Thea, Ngoc Tuong Vy Nguyen, Marilla Lucero, Syed Mohammad Akram Uz Zaman, Shinjini Bhatnagar, Nitya Wadhwa, Rakesh Lodha, Satinder Aneja, Mathuram Santosham, Shally Awasthi, Ashish Bavdekar, Monidarin Chou, Pagbajabyn Nymadawa, Jean William Pape, Glaucia Paranhos-Baccala, Valentina S Picot, Mala Rakoto-Andrianarivelo, Vanessa Rouzier, Graciela Russomando, Mariam Sylla, Philippe Vanhems, Jianwei Wang, Romina Libster, Alexey W Clara, Fenella Beynon, Gillian Levine, Chris A Rees, Mark I Neuman, Shamim Qazi, Yasir Bin Nisar, World Health Organization Prepare Study Group

Faculty, Staff and Students Publications

Background: Hypoxaemia predicts mortality at all levels of care, and appropriate management can reduce preventable deaths. However, pulse oximetry and oxygen therapy remain inaccessible in many primary care health facilities. We aimed to develop and validate a simple risk score comprising commonly evaluated clinical features to predict hypoxaemia in 2-59-month-old children with pneumonia.

Methods: Data from seven studies conducted in five countries from the Pneumonia Research Partnership to Assess WHO Recommendations (PREPARE) dataset were included. Readily available clinical features and demographic variables were used to develop a multivariable logistic regression model to predict hypoxemia (oxygen saturation < 90%) at presentation to care. The adjusted log coefficients were transformed to derive the PREPARE hypoxemia risk score and its diagnostic value was assessed in a held-out, temporal validation dataset. The model and risk score were analysed by evaluating the area under the receiver operating characteristic curve (AUC), sensitivity and specificity.

Results: We included 14 …

Serological Analysis Indicating The Exposure Of Two Volunteers To Relapsing Fever Borrelia Sp. In Nayarit, Mexico, Sofía L Luna-Rojas, Edwin Vázquez-Guerrero, Jose A Hernández-Martínez, Jeanet Serafín-López, Fernando Martínez-Hernández, José Alejandro Martinez-Ibarra, Rigoberto Hernández-Castro, Job E López, J Antonio Ibarra

Faculty, Staff and Students Publications

Relapsing fever caused by Borrelia sp. is a neglected disease in Mexico and more studies are needed to update its presence in multiple regions of the country. Here, recombinant proteins glycerophosphodiester phosphodiesterase (GlpQ) and three Borrelia immunogenic protein A (BipA) variants were used for serosurveillance in 142 human serum samples. Results showed that two persons were positive to Borrelia extracts and GlpQ but negative to all three BipA, suggesting they were infected by relapsing fever spirochetes of a yet to be determined species. This shows that testing for GlpQ and Borrelia extracts are a useful tool for potential infections and …

A Phase 2 Trial Of Short-Term Intravenous N-Acetylcysteine In Biliary Atresia After Kasai Portoenterostomy, Sanjiv Harpavat, Kristin A Borovsky, Michael E Scheurer, Laurel Cavallo, Franca E Erhiawarie, Sanjeev Vasudevan, Adam M Vogel, Dana Cerminara, Elizabeth M Tessier, Kalyani R Patel, Sridevi Devaraj, Benjamin L Shneider

Center for Medical Ethics and Health Policy Staff Publications

Background: For infants with biliary atresia, the only treatment that can establish bile flow and delay need for liver transplant is the Kasai portoenterostomy (KP). Unfortunately, the KP has variable success. In this study, we hypothesized that intravenous N-acetylcysteine (IV NAC) treatment following KP would improve bile flow.

Methods: This was a phase 2 study following the two-stage "minimax" trial design. Participants received IV NAC (150 mg/kg/day) for 7 days after KP, and the primary endpoint was achieving total serum bile acids (TSBA) ≤10 μmol/L within 24 weeks of KP. Secondary endpoints were clinical markers and the occurrence of sentinel …

Identification Of Distinct Biological Groups Of Patients With Cryptogenic Norse Via Inflammatory Profiling, Martin Guillemaud, Mario Chavez, Firas Kobeissy, Annamaria Vezzani, Anthony D Jimenez, Maysaa Merhi Basha, Ayush Batra, Sophie Demeret, Onome Eka, Krista Eschbach, Brandon Foreman, Nicolas Gaspard, Elizabeth E Gerard, Teneille Emma Gofton, Hiba A Haider, Stephen T Hantus, Charles L Howe, Amy Jongeling, Mariel Kalkach-Aparicio, Padmaja Kandula, Karnig Kazazian, Minjee Kim, Yi-Chen Lai, Clémence Marois, Andrew Mellor, Wazim Mohamed, Mikaela Morales, Cederic M Pimentel, Alexandra M Ramirez, Claude Steriade, Aaron F Struck, Olga Taraschenko, Nathan Torcida Sedano, Mark S Wainwright, Ji Yeoun Yoo, Kevin K W Wang, Vincent Navarro, Lawrence J Hirsch, Aurélie Hanin

Faculty, Staff and Students Publications

Background and objectives: Emerging evidence suggests that immune dysregulation plays a pivotal role in triggering cryptogenic new-onset refractory status epilepticus (c-NORSE), prompting a consensus on early initiation of immunotherapy. However, despite similar timing of administration, responses to immunotherapies have been varied and unpredictable, suggesting the presence of heterogeneous underlying mechanisms. The aim of this study was to identify distinct inflammatory response subtypes in patients with c-NORSE by analyzing their cytokine profiles. Insights into underlying mechanisms were sought to understand the pathophysiology and guide personalized therapies to improve patient outcomes.

Methods: Sixty-two patients with c-NORSE were included. A comprehensive panel …

Short-Term Neurologic Outcomes In Pediatric Extracorporeal Membrane Oxygenation Are Proportional To Bleeding Severity Graded By A Novel Bleeding Scale, Katherine Doane, Danielle Guffey, Laura L Loftis, Trung C Nguyen, Matthew A Musick, Amanda Ruth, Ryan D Coleman, Jun Teruya, Christine Allen, Melania M Bembea, Brian Boville, Jamie Furlong-Dillard, Santosh Kaipa, Mara Leimanis, Matthew P Malone, Lindsey K Rasmussen, Ahmed Said, Marie E Steiner, Deanna T Tzanetos, Heather Viamonte, Linda Wallenkamp, Arun Saini

Faculty, Staff and Students Publications

Patients admitted to a pediatric intensive care unit (PICU) need individualized nutrition support that is tailored to their particular disease severity, nutritional status, and therapeutic interventions. We aim to evaluate how calories and proteins are provided during the first seven days of hospitalization for children in critical condition with organ dysfunction (OD). A single-center retrospective cohort study of children aged 2–18 years, mechanically ventilated > 48 h, and admitted > 7 days to a PICU from 2016 to 2017 was carried out. Nutrition support included enteral and parenteral nutrition. We calculated scores for the Pediatric Sequential Organ Failure Assessment (pSOFA) on days …

Reporting Abcd1 Variants As Actionable Secondary Findings On Exome And Genome Sequencing, Carlos A Dominguez Gonzalez, Nancy B Spinner, Rebecca C Ahrens-Nicklas, Lisa R Young, Laura A Voss, Sara L Reichert, Daniel J Gallo, Julie S Cohen, Joshua L Bonkowsky, Stephanie R Keller, Mariko L Bennett, Amy M Pizzino, Meghan Swantkowski, Kaley Arnold, Jamie L Fraser, Felicity J Emerson, Kelly Miettunen, Ali Fatemi, Keith P Van Haren, Laura Adang, Amy Waldman, Lisa Emrick, Florian Eichler, Adeline Vanderver

Faculty, Staff and Students Publications

No abstract provided.

Metabolomic Data May Support The Development Of Personalized Nutrition Approaches To Type 2 Diabetes Management, Danielle J Lee, Shragvi Balaji, Jerome I Rotter, Alexis C Wood

Faculty, Staff and Students Publications

Diet is a major modifiable risk factor for type 2 diabetes (T2D), and suboptimal diets continue to contribute substantially to the population burden of T2D. Personalized nutrition proponents argue that specializing recommendations to interindividual differences in diet-health relationships will yield reductions in disease risk. However, how to personalize diet, and to whom, to reduce T2D risk remains unclear. Metabolites offer promise in this respect, as they capture (in part) dietary intake after the processes of digestion, processing, and absorption. The incorporation of metabolite data into diet-health studies therefore offers the opportunity to examine how the effects of food on the …