Medicine and Health Sciences Commons^™

Onasemnogene Abeparvovec For Presymptomatic Infants With Three Copies Of Smn2 At Risk For Spinal Muscular Atrophy: The Phase Iii Spr1nt Trial, Kevin A Strauss, Craig M Zaidman, Et Al

2020-Current year OA Pubs

Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular atrophy (SMA) type 2. SPR1NT ( NCT03505099 ), a Phase III, multicenter, single-arm trial, investigated the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated within six postnatal weeks. Of 15 children with three SMN2 copies treated before symptom onset, all stood independently before 24 months (P < 0.0001; 14 within normal developmental window), and 14 walked independently (P < 0.0001; 11 within normal developmental window). All survived without permanent ventilation at 14 months; ten (67%) maintained body weight (≥3rd WHO percentile) without feeding support through 24 months; and none required nutritional or respiratory support. No serious adverse events were considered treatment-related by the investigator. Onasemnogene abeparvovec was effective and well-tolerated for presymptomatic infants at risk of SMA type 2, underscoring the urgency of early identification and intervention.

Assessment Of Ethno-Racial And Insurance-Based Disparities In Pediatric Forearm And Tibial Fracture Care In The United States, Andrew J Landau, Afolayan K Oladeji, Pooya Hosseinzadeh

2020-Current year OA Pubs

INTRODUCTION: Despite growing attention to healthcare disparities and interventions to improve inequalities, additional identification of disparities is needed, particularly in the pediatric population. We used state and nationwide databases to identify factors associated with the surgical treatment of pediatric forearm and tibial fractures.

METHODS: The Healthcare Cost and Utilization Project State Inpatient, Emergency Department, and Ambulatory Surgery and Services Databases from four US states and the Nationwide Emergency Department Sample database were quarried using International Classification of Diseases codes to identify patients from 2006 to 2015. Multivariable regression models were used to determine factors associated with surgical treatment.

RESULTS: State …

Onasemnogene Abeparvovec For Presymptomatic Infants With Two Copies Of Smn2 At Risk For Spinal Muscular Atrophy Type 1: The Phase Iii Spr1nt Trial, Kevin A Strauss, Craig M Zaidman, Et Al

2020-Current year OA Pubs

SPR1NT ( NCT03505099 ) was a Phase III, multicenter, single-arm study to investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated at ≤6 weeks of life. Here, we report final results for 14 children with two copies of SMN2, expected to develop spinal muscular atrophy (SMA) type 1. Efficacy was compared with a matched Pediatric Neuromuscular Clinical Research natural-history cohort (n = 23). All 14 enrolled infants sat independently for ≥30 seconds at any visit ≤18 months (Bayley-III item #26; P < 0.001; 11 within the normal developmental window). All survived without permanent ventilation at 14 months as per protocol; 13 maintained body weight (≥3rd WHO percentile) through 18 months. No child used nutritional or respiratory support. No serious adverse events were considered related to treatment by the investigator. Onasemnogene abeparvovec was effective and well-tolerated for children expected to develop SMA type 1, highlighting the urgency for universal newborn screening.

Comparative Effectiveness Of Diversion Of Cerebrospinal Fluid For Children With Severe Traumatic Brain Injury, Michael J Bell, Jose Pineda, Et Al.

2020-Current year OA Pubs

Importance: Diversion of cerebrospinal fluid (CSF) has been used for decades as a treatment for children with severe traumatic brain injury (TBI) and is recommended by evidenced-based guidelines. However, these recommendations are based on limited studies.

Objective: To determine whether CSF diversion is associated with improved Glasgow Outcome Score-Extended for Pediatrics (GOS-EP) and decreased intracranial pressure (ICP) in children with severe TBI.

Design, Setting, and Participants: This observational comparative effectiveness study was performed at 51 clinical centers that routinely care for children with severe TBI in 8 countries (US, United Kingdom, Spain, the Netherlands, Australia, New Zealand, South Africa, and …

Developing Shelf-Stable Microbiota Directed Complementary Food (Mdcf) Prototypes For Malnourished Children: Study Protocol For A Randomized, Single-Blinded, Clinical Study, Ishita Mostafa, Shah Mohammad Fahim, Subhasish Das, Md Amran Gazi, Md Mehedi Hasan, Kazi Nazmus Saqeeb, Mustafa Mahfuz, Hannah B Lynn, Michael J Barratt, Jeffrey I Gordon, Tahmeed Ahmed

2020-Current year OA Pubs

BACKGROUND: Childhood undernutrition is a major public health concern that needs special attention to achieve 2025 global nutrition targets. Moderate acute malnutrition (MAM), manifest as wasting (low weight-for-height), affects 33 million children under 5, yet there are currently no global guidelines for its treatment. We recently performed a randomized-controlled clinical study of a microbiota-directed complementary food formulation (MDCF-2) in 12-18-month-old Bangladeshi children with MAM. The results revealed that MDCF-2, freshly prepared each day, produced a significantly greater improvement in ponderal growth than a standard ready-to-use supplementary food (RUSF), an effect that is associated with repair of the disrupted gut microbial …

Changes In Adiposity Over The Life Course And Gene Expression In Postmenopausal Women, Yunan Han, Graham A Colditz, Adetunji T Toriola

2020-Current year OA Pubs

BACKGROUND: Early life adiposity and changes in adiposity over the life course are associated with mammographic breast density among postmenopausal women. However, the underlying mechanisms are unknown; therefore, we comprehensively examined the associations of early life body mass index (BMI) and changes in BMI from ages 10, 18 to age at mammogram with growth factor, RANK pathway, and sex hormone gene expression in 372 postmenopausal women.

METHODS: We estimated early life BMI at age 10 using the validated 9-level Stunkard pictogram. We calculated BMI at other ages (18, 30, and current age at mammogram) by dividing weight in kilograms at …

Safety Of Onasemnogene Abeparvovec For Patients With Spinal Muscular Atrophy 8.5 Kg Or Heavier In A Global Managed Access Program, Deepa H Chand, Susan Mitchell, Rui Sun, Nicole Lamarca, Sandra P Reyna, Thao Sutter

2020-Current year OA Pubs

BACKGROUND: Spinal muscular atrophy is a rare, neurodegenerative disorder caused by biallelic deletions in the survival motor neuron (SMN1) gene. Onasemnogene abeparvovec is a one-time, intravenous gene replacement therapy designed to deliver the SMN1 transgene. Although available in many geographies, it is not approved globally. The Global Managed Access Program (GMAP) expanded treatment access to patients in countries where treatment was not approved. Previous onasemnogene abeparvovec clinical trials included patients with body weight <8.5 kg. Through GMAP, children weighing ≥8.5 kg received onasemnogene abeparvovec. We describe safety data for heavier patients in GMAP.

METHODS: GMAP records were reviewed to identify patients weighing ≥8.5 kg at onasemnogene abeparvovec dosing. To obtain corresponding adverse event (AE) data, the Novartis ARGUS safety database …

Mechanisms Of Transcranial Doppler Ultrasound Phenotypes In Paediatric Cerebral Malaria Remain Elusive., Nicole F O'Brien, Yudy Fonseca, Hunter C Johnson, Douglas Postels, Gretchen L Birbeck, Yamikani Chimalizeni, Karl B Seydel, Montfort Bernard Gushu, Tusekile Phiri, Sylvester June, Karen Chetcuti, Lorenna Vidal, Manu S Goyal, Terrie E Taylor

2020-Current year OA Pubs

BACKGROUND: Cerebral malaria (CM) results in significant paediatric death and neurodisability in sub-Saharan Africa. Several different alterations to typical Transcranial Doppler Ultrasound (TCD) flow velocities and waveforms in CM have been described, but mechanistic contributors to these abnormalities are unknown. If identified, targeted, TCD-guided adjunctive therapy in CM may improve outcomes.

METHODS: This was a prospective, observational study of children 6 months to 12 years with CM in Blantyre, Malawi recruited between January 2018 and June 2021. Medical history, physical examination, laboratory analysis, electroencephalogram, and magnetic resonance imaging were undertaken on presentation. Admission TCD results determined phenotypic grouping following a …

What Is The State Of Children's Participation In Qualitative Research On Health Interventions?: A Scoping Study, Jean M Hunleth, Julie S Spray, Corey Meehan, Colleen Walsh Lang, Janet Njelesani

2020-Current year OA Pubs

BACKGROUND: Children are the focus of numerous health interventions throughout the world, yet the extent of children's meaningful participation in research that informs the adaptation, implementation, and evaluation of health interventions is not known. We examine the type, extent, and meaningfulness of children's participation in research in qualitative health intervention research.

METHOD: A scoping study was conducted of qualitative published research with children (ages 6-11 years) carried out as part of health intervention research. Following Arksey and O'Malley's scoping study methodology and aligned with the PRISMA-ScR guidelines on the reporting of scoping reviews, the authors searched, charted, collated, and summarized …

Care Levels For Fetal Therapy Centers, Ahmet A Baschat, Barbara B Warner, Et Al

2020-Current year OA Pubs

Fetal therapies undertaken to improve fetal outcome or to optimize transition to neonate life often entail some level of maternal, fetal, or neonatal risk. A fetal therapy center needs access to resources to carry out such therapies and to manage maternal, fetal, and neonatal complications that might arise, either related to the therapy per se or as part of the underlying fetal or maternal condition. Accordingly, a fetal therapy center requires a dedicated operational infrastructure and necessary resources to allow for appropriate oversight and monitoring of clinical performance and to facilitate multidisciplinary collaboration between the relevant specialties. Three care levels …

Accuracy Of Chest Computed Tomography In Distinguishing Cystic Pleuropulmonary Blastoma From Benign Congenital Lung Malformations In Children, Abigail J Engwall-Gill, Jacqueline M Saito, Rebecca L Hulett Bowling, Et Al.

2020-Current year OA Pubs

Importance: The ability of computed tomography (CT) to distinguish between benign congenital lung malformations and malignant cystic pleuropulmonary blastomas (PPBs) is unclear.

Objective: To assess whether chest CT can detect malignant tumors among postnatally detected lung lesions in children.

Design, Setting, and Participants: This retrospective multicenter case-control study used a consortium database of 521 pathologically confirmed primary lung lesions from January 1, 2009, through December 31, 2015, to assess diagnostic accuracy. Preoperative CT scans of children with cystic PPB (cases) were selected and age-matched with CT scans from patients with postnatally detected congenital lung malformations (controls). Statistical analysis was performed …

Synthesizing Pseudo-T2w Images To Recapture Missing Data In Neonatal Neuroimaging With Applications In Rs-Fmri, Sydney Kaplan, Dimitrios Alexopoulos, Jeanette K Kenley, Deanna M Barch, Jeffrey J Neil, Cynthia E Rogers, Aristeidis Sotiras, Christopher D Smyser, Et Al.

2020-Current year OA Pubs

T1- and T2-weighted (T1w and T2w) images are essential for tissue classification and anatomical localization in Magnetic Resonance Imaging (MRI) analyses. However, these anatomical data can be challenging to acquire in non-sedated neonatal cohorts, which are prone to high amplitude movement and display lower tissue contrast than adults. As a result, one of these modalities may be missing or of such poor quality that they cannot be used for accurate image processing, resulting in subject loss. While recent literature attempts to overcome these issues in adult populations using synthetic imaging approaches, evaluation of the efficacy of these methods in pediatric …

The H3k27m Mutation Alters Stem Cell Growth, Epigenetic Regulation, And Differentiation Potential, N Kfoury-Beaumont, R Prakasam, S Pondugula, J S Lagas, S Matkovich, P Gontarz, L Yang, H Yano, A H Kim, J B Rubin, K L Kroll

2020-Current year OA Pubs

BACKGROUND: Neurodevelopmental disorders increase brain tumor risk, suggesting that normal brain development may have protective properties. Mutations in epigenetic regulators are common in pediatric brain tumors, highlighting a potentially central role for disrupted epigenetic regulation of normal brain development in tumorigenesis. For example, lysine 27 to methionine mutation (H3K27M) in the H3F3A gene occurs frequently in Diffuse Intrinsic Pontine Gliomas (DIPGs), the most aggressive pediatric glioma. As H3K27M mutation is necessary but insufficient to cause DIPGs, it is accompanied by additional mutations in tumors. However, how H3K27M alone increases vulnerability to DIPG tumorigenesis remains unclear.

RESULTS: Here, we used human …

A Genetically Distinct Pediatric Subtype Of Primary Cns Large B-Cell Lymphoma Is Associated With Favorable Clinical Outcome, Ekin Güney, Sonika Dahiya, Et Al.

2020-Current year OA Pubs

No abstract provided.

Belimumab For Systemic Lupus Erythematosus - Focus On Lupus Nephritis, Marlene Plüß, Silvia Piantoni, Björn Tampe, Alfred H J Kim, Peter Korsten

2020-Current year OA Pubs

In recent years, advances in the treatment and management of patients with systemic lupus erythematosus (SLE) have improved their life expectancy and quality of life. However, lupus nephritis (LN) still represents a major life-threatening complication of the disease. Belimumab (BEL), a fully human monoclonal IgG1λ antibody neutralizing soluble B cell activating factor, was approved more than ten years ago as add-on therapy in adults and pediatric patients with a highly active, autoantibody-positive disease despite standard of care (SoC). Recently, the superiority of the addition of BEL to SoC was also demonstrated in LN. In this review, we provide a comprehensive …

Reliability And Stability Challenges In Abcd Task Fmri Data, James T. Kennedy, Michael P. Harms, Ozlem Korucuoglu, Serguei V. Astafiev, Deanna M. Barch, Wesley K. Thompson, James M. Bjork, Andrey P. Anokhin

2020-Current year OA Pubs

Trait stability of measures is an essential requirement for individual differences research. Functional MRI has been increasingly used in studies that rely on the assumption of trait stability, such as attempts to relate task related brain activation to individual differences in behavior and psychopathology. However, recent research using adult samples has questioned the trait stability of task-fMRI measures, as assessed by test-retest correlations. To date, little is known about trait stability of task fMRI in children. Here, we examined within-session reliability and long-term stability of individual differences in task-fMRI measures using fMRI measures of brain activation provided by the adolescent …

Machine Learning Derivation Of Four Computable 24-H Pediatric Sepsis Phenotypes To Facilitate Enrollment In Early Personalized Anti-Inflammatory Clinical Trials, Yidi Qin, John C Lin, Allan Doctor, Et Al.

2020-Current year OA Pubs

BACKGROUND: Thrombotic microangiopathy-induced thrombocytopenia-associated multiple organ failure and hyperinflammatory macrophage activation syndrome are important causes of late pediatric sepsis mortality that are often missed or have delayed diagnosis. The National Institutes of General Medical Science sepsis research working group recommendations call for application of new research approaches in extant clinical data sets to improve efficiency of early trials of new sepsis therapies. Our objective is to apply machine learning approaches to derive computable 24-h sepsis phenotypes to facilitate personalized enrollment in early anti-inflammatory trials targeting these conditions.

METHODS: We applied consensus, k-means clustering analysis to our extant PHENOtyping sepsis-induced Multiple …

Iv Sotalol Use In Pediatric And Congenital Heart Patients: A Multicenter Registry Study, Lindsey E Malloy-Walton, Nicholas H Von Bergen, Seshadri Balaji, Peter S Fischbach, Jason M Garnreiter, S Yukiko Asaki, Jeffrey P Moak, Luis A Ochoa, Philip M Chang, Hoang H Nguyen, Akash R Patel, Christa Kirk, Ashley K Sherman, Jennifer N Avari Silva, J Philip Saul

2020-Current year OA Pubs

Background There is limited information regarding the clinical use and effectiveness of IV sotalol in pediatric patients and patients with congenital heart disease, including those with severe myocardial dysfunction. A multicenter registry study was designed to evaluate the safety, efficacy, and dosing of IV sotalol. Methods and Results A total of 85 patients (age 1 day-36 years) received IV sotalol, of whom 45 (53%) had additional congenital cardiac diagnoses and 4 (5%) were greater than 18 years of age. In 79 patients (93%), IV sotalol was used to treat supraventricular tachycardia and 4 (5%) received it to treat ventricular arrhythmias. …

Association Of Inappropriate Outpatient Pediatric Antibiotic Prescriptions With Adverse Drug Events And Health Care Expenditures, Anne M Butler, Derek S Brown, Michael J Durkin, John M Sahrmann, Katelin B Nickel, Caroline A O'Neil, Margaret A Olsen, David Y Hyun, Rachel M Zetts, Jason G Newland

2020-Current year OA Pubs

Importance: Nonguideline antibiotic prescribing for the treatment of pediatric infections is common, but the consequences of inappropriate antibiotics are not well described.

Objective: To evaluate the comparative safety and health care expenditures of inappropriate vs appropriate oral antibiotic prescriptions for common outpatient pediatric infections.

Design, Setting, and Participants: This cohort study included children aged 6 months to 17 years diagnosed with a bacterial infection (suppurative otitis media [OM], pharyngitis, sinusitis) or viral infection (influenza, viral upper respiratory infection [URI], bronchiolitis, bronchitis, nonsuppurative OM) as an outpatient from April 1, 2016, to September 30, 2018, in the IBM MarketScan Commercial Database. …

Demographic And Clinical Characteristics Associated With The Failure Of Nonoperative Management Of Uncomplicated Appendicitis In Children: Secondary Analysis Of A Nonrandomized Clinical Trial, Peter C Minneci, Jacqueline M Saito, Et Al.

2020-Current year OA Pubs

Importance: The factors associated with the failure of nonoperative management of appendicitis and the differences in patient-reported outcomes between successful and unsuccessful nonoperative management remain unknown.

Objectives: To investigate factors associated with the failure of nonoperative management of appendicitis and compare patient-reported outcomes between patients whose treatment succeeded and those whose treatment failed.

Design, Setting, and Participants: This study was a planned subgroup secondary analysis conducted in 10 children's hospitals that included 370 children aged 7 to 17 years with uncomplicated appendicitis enrolled in a prospective, nonrandomized clinical trial between May 1, 2015, and October 31, 2018, with 1-year follow-up …

American Society For Transplantation And Cellular Therapy Series: #5-Management Of Clostridioides Difficile Infection In Hematopoietic Cell Transplant Recipients, Carolyn D Alonso, Gabriela Maron, Mini Kamboj, Paul A Carpenter, Arun Gurunathan, Kathleen M Mullane, Erik R Dubberke

2020-Current year OA Pubs

The Practice Guidelines Committee of the American Society for Transplantation and Cellular Therapy partnered with its Transplant Infectious Disease Special Interest Group to update its 2009 compendium-style infectious disease guidelines for hematopoietic cell transplantation (HCT). A completely new approach was taken with the goal of better serving clinical providers by publishing each standalone topic in the infectious disease series as a concise format of frequently asked questions (FAQ), tables, and figures. Adult and pediatric infectious disease and HCT content experts developed and then answered FAQs and finalized topics with harmonized recommendations that were made by assigning an A through E …

Functional Analysis Of A Novel De Novo Variant In Ppp5c Associated With Microcephaly, Seizures, And Developmental Delay, Sara M Fielder, Jill A Rosenfeld, Lindsay C Burrage, Lisa Emrick, Seema Lalani, Ruben Attali, Joshua N Bembenek, Hieu Hoang, Dustin Baldridge, Gary A Silverman, Undiagnosed Diseases Network, Tim Schedl, Stephen C Pak

2020-Current year OA Pubs

We describe a proband evaluated through the Undiagnosed Diseases Network (UDN) who presented with microcephaly, developmental delay, and refractory epilepsy with a de novo p.Ala47Thr missense variant in the protein phosphatase gene, PPP5C. This gene has not previously been associated with a Mendelian disease, and based on the population database, gnomAD, the gene has a low tolerance for loss-of-function variants (pLI = 1, o/e = 0.07). We functionally evaluated the PPP5C variant in C. elegans by knocking the variant into the orthologous gene, pph-5, at the corresponding residue, Ala48Thr. We employed assays in three different biological processes where pph-5 was …

Products Of Gut Microbial Toll/Interleukin-1 Receptor Domain Nadase Activities In Gnotobiotic Mice And Bangladeshi Children With Malnutrition, James S Weagley, Mark Zaydman, Siddarth Venkatesh, Yo Sasaki, Neha Damaraju, Alex Yenkin, William Buchser, Dmitry A Rodionov, Andrei Osterman, Tahmeed Ahmed, Michael J Barratt, Aaron Diantonio, Jeffrey Milbrandt, Jeffrey I Gordon

2020-Current year OA Pubs

Perturbed gut microbiome development has been linked to childhood malnutrition. Here, we characterize bacterial Toll/interleukin-1 receptor (TIR) protein domains that metabolize nicotinamide adenine dinucleotide (NAD), a co-enzyme with far-reaching effects on human physiology. A consortium of 26 human gut bacterial strains, representing the diversity of TIRs observed in the microbiome and the NAD hydrolase (NADase) activities of a subset of 152 bacterial TIRs assayed in vitro, was introduced into germ-free mice. Integrating mass spectrometry and microbial RNA sequencing (RNA-seq) with consortium membership manipulation disclosed that a variant of cyclic-ADPR (v-cADPR-x) is a specific product of TIR NADase activity and a …

Sex-Based Differences In Functional Brain Activity During Working Memory In Survivors Of Pediatric Acute Lymphoblastic Leukemia, Kellen Gandy, Matthew A Scoggins, Nicholas Phillips, Ellen Van Der Plas, Slim Fellah, Lisa M Jacola, Ching-Hon Pui, Melissa M Hudson, Wilburn E Reddick, Ranganatha Sitaram, Kevin R Krull

2020-Current year OA Pubs

BACKGROUND: Long-term survivors of pediatric acute lymphoblastic leukemia are at elevated risk for neurocognitive deficits and corresponding brain dysfunction. This study examined sex-based differences in functional neuroimaging outcomes in acute lymphoblastic leukemia survivors treated with chemotherapy alone.

METHODS: Functional magnetic resonance imaging (fMRI) and neurocognitive testing were obtained in 123 survivors (46% male; median [min-max] age = 14.2 years [8.3-26.5 years]; time since diagnosis = 7.7 years [5.1-12.5 years]) treated on the St. Jude Total XV treatment protocol. Participants performed the n-back working memory task in a 3 T scanner. Functional neuroimaging data were processed (realigned, slice time corrected, normalized, …

Care Team And Practice-Level Implementation Strategies To Optimize Pediatric Collaborative Care: Study Protocol For A Cluster-Randomized Hybrid Type Iii Trial, David J Kolko, Elizabeth A Mcguier, Renee Turchi, Eileen Thompson, Satish Iyengar, Shawna N Smith, Kimberly Hoagwood, Celeste Liebrecht, Ian M Bennett, Byron J Powell, Kelly Kelleher, Maria Silva, Amy M Kilbourne

2020-Current year OA Pubs

BACKGROUND: Implementation facilitation is an effective strategy to support the implementation of evidence-based practices (EBPs), but our understanding of multilevel strategies and the mechanisms of change within the "black box" of implementation facilitation is limited. This implementation trial seeks to disentangle and evaluate the effects of facilitation strategies that separately target the care team and leadership levels on implementation of a collaborative care model in pediatric primary care. Strategies targeting the provider care team (TEAM) should engage team-level mechanisms, and strategies targeting leaders (LEAD) should engage organizational mechanisms.

METHODS: We will conduct a hybrid type 3 effectiveness-implementation trial in a …

Sustained Efficacy And Safety Of Burosumab, A Monoclonal Antibody To Fgf23, In Children With X-Linked Hypophosphatemia, Agnès Linglart, Erik A Imel, Michael P Whyte, Anthony A Portale, Wolfgang Högler, Annemieke M Boot, Raja Padidela, William Van't Hoff, Gary S Gottesman, Angel Chen, Alison Skrinar, Mary Scott Roberts, Thomas O Carpenter

2020-Current year OA Pubs

PURPOSE: In X-linked hypophosphatemia (XLH), excess fibroblast growth factor-23 causes hypophosphatemia and low calcitriol, leading to musculoskeletal disease with clinical consequences. XLH treatment options include conventional oral phosphate with active vitamin D, or monotherapy with burosumab, a monoclonal antibody approved to treat children and adults with XLH. We have previously reported outcomes up to 64 weeks, and here we report safety and efficacy follow-up results up to 160 weeks from an open-label, multicenter, randomized, dose-finding trial of burosumab for 5- to 12-year-old children with XLH.

METHODS: After 1 week of conventional therapy washout, patients were randomized 1:1 to burosumab every …

Fractality Of Tics As A Quantitative Assessment Tool For Tourette Syndrome, Payton Beeler, Nicholas O Jensen, Soyoung Kim, Amy Robichaux-Viehoever, Bradley L Schlaggar, Deanna J Greene, Kevin J Black, Rajan K Chakrabarty

2020-Current year OA Pubs

Tics manifest as brief, purposeless and unintentional movements or noises that, for many individuals, can be suppressed temporarily with effort. Previous work has hypothesized that the chaotic temporal nature of tics could possess an inherent fractality, that is, have neighbour-to-neighbour correlation at all levels of timescale. However, demonstrating this phenomenon has eluded researchers for more than two decades, primarily because of the challenges associated with estimating the scale-invariant, power law exponent-called the fractal dimension

Validation Of Actigraphy For Sleep Measurement In Children With Cerebral Palsy, Bing Xue, Amy Licis, Jill Boyd, Catherine R Hoyt, Yo-El S Ju

2020-Current year OA Pubs

OBJECTIVES: Sleep issues are common in children with cerebral palsy (CP), although there are challenges in obtaining objective data about their sleep patterns. Actigraphs measure movement to quantify sleep but their accuracy in children with CP is unknown. Our goals were to validate actigraphy for sleep assessment in children with CP and to study their sleep patterns in a cross-sectional cohort study.

METHODS: We recruited children with (N = 13) and without (N = 13) CP aged 2-17 years (mean age 9 y 11mo [SD 4 y 10mo] range 4-17 y; 17 males, 9 females; 54% spastic quadriplegic, 23% spastic …

Importance Of The Intersection Of Age And Sex To Understand Variation In Incidence And Survival For Primary Malignant Gliomas, Gi-Ming Wang, Gino Cioffi, Nirav Patil, Kristin A Waite, Robert Lanese, Quinn T Ostrom, Carol Kruchko, Michael E Berens, James R Connor, Justin D Lathia, Joshua B Rubin, Jill S Barnholtz-Sloan

2020-Current year OA Pubs

BACKGROUND: Gliomas are the most common type of malignant brain and other CNS tumors, accounting for 80.8% of malignant primary brain and CNS tumors. They cause significant morbidity and mortality. This study investigates the intersection between age and sex to better understand variation of incidence and survival for glioma in the United States.

METHODS: Incidence data from 2000 to 2017 were obtained from CBTRUS, which obtains data from the NPCR and SEER, and survival data from the CDC's NPCR. Age-adjusted incidence rate ratios (IRR) per 100 000 were generated to compare male-to-female incidence by age group. Cox proportional hazard models …

Grandi Byen-Supporting Child Growth And Development Through Integrated, Responsive Parenting, Nutrition And Hygiene: Study Protocol For A Randomized Controlled Trial, Patricia L Kohl, Emmanuel A Gyimah, Jenna Diaz, F Matthew Kuhlmann, Sherlie Jean-Louis Dulience, Fithi Embaye, Derek S Brown, Shenyang Guo, Joan L Luby, Jennifer L Nicholas, Jay Turner, Melissa Chapnick, Joseline Marhone Pierre, Jacques Boncy, Rony St Fleur, Maureen M Black, Lora L Iannotti

2020-Current year OA Pubs

BACKGROUND: Poor child growth and development outcomes stem from complex relationships encompassing biological, behavioral, social, and environmental conditions. However, there is a dearth of research on integrated approaches targeting these interwoven factors. The Grandi Byen study seeks to fill this research gap through a three-arm longitudinal randomized controlled trial which will evaluate the impact of an integrated nutrition, responsive parenting, and WASH (water, sanitation and hygiene) intervention on holistic child growth and development.

METHODS: We will recruit 600 mother-infant dyads living in Cap-Haitien, Haiti and randomize them equally into one of the following groups: 1) standard well-baby care; 2) nutritional …