Medicine and Health Sciences Commons^™

The Regulation Of Clinical Research: What's Love Got To Do With It?, John Lantos

Manuscripts, Articles, Book Chapters and Other Papers

The central philosophical pillar of the current system of research regulation in the United States today is that clinical investigators cannot and should not be trusted to protect the interests of the people whom they recruit to participate in research. That distrust of researchers is coupled with a starry-eyed idealism about trustworthiness of clinicians. In my opinion, the distrust of researchers and the complacency about clinicians are both misplaced. The result of these twin errors is that people are overprotected in research studies and inadequately protected in clinical care. Patients outside of research studies are exposed to many types of …

Successful Reversal Of Furosemide-Induced Secondary Hyperparathyroidism With Cinacalcet., Tarak Srivastava, Shahryar Jafri, William E. Truog, Judith Sebestyen Vansickle, Winston M. Manimtim, Uri S. Alon

Manuscripts, Articles, Book Chapters and Other Papers

Secondary hyperparathyroidism (SHPT) is a rare complication of furosemide therapy that can occur in patients treated with the loop diuretic for a long period of time. We report a 6-month-old 28-weeks premature infant treated chronically with furosemide for his bronchopulmonary dysplasia, who developed hypocalcemia and severe SHPT, adversely affecting his bones. Discontinuation of the loop diuretic and the addition of supplemental calcium and calcitriol only partially reversed the SHPT, bringing serum parathyroid hormone level down from 553 to 238 pg/mL. After introduction of the calcimimetic Cinacalcet, we observed a sustained normalization of parathyroid hormone concentration at 27 to 63 pg/mL …

Automated Ecological Assessment Of Physical Activity: Advancing Direct Observation., Jordan A. Carlson, Bo Liu, James F. Sallis, Jacqueline Kerr, J Aaron Hipp, Vincent S. Staggs, Amy Papa, Kelsey Dean, Nuno M. Vasconcelos

Manuscripts, Articles, Book Chapters and Other Papers

Technological advances provide opportunities for automating direct observations of physical activity, which allow for continuous monitoring and feedback. This pilot study evaluated the initial validity of computer vision algorithms for ecological assessment of physical activity. The sample comprised 6630 seconds per camera (three cameras in total) of video capturing up to nine participants engaged in sitting, standing, walking, and jogging in an open outdoor space while wearing accelerometers. Computer vision algorithms were developed to assess the number and proportion of people in sedentary, light, moderate, and vigorous activity, and group-based metabolic equivalents of tasks (MET)-minutes. Means and standard deviations (SD) …

Hypotonia And Intellectual Disability Without Dysmorphic Features In A Patient With Pign-Related Disease., Isabelle Thiffault, Britton Zuccarelli, Holly Welsh, Xuan Yuan, Emily Farrow, Lee Zellmer, Neil Miller, Sarah Soden, Ahmed Abdelmoity, Robert A Brodsky, Carol Saunders

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Defects in the human glycosylphosphatidylinositol anchor biosynthetic pathway are associated with inherited glycosylphosphatidylinositol (GPI)-deficiencies characterized by a broad range of clinical phenotypes including multiple congenital anomalies, dysmorphic faces, developmental delay, hypotonia, and epilepsy. Biallelic variants in PIGN, encoding phosphatidylinositol-glycan biosynthesis class N have been recently associated with multiple congenital anomalies hypotonia seizure syndrome.

CASE PRESENTATION: Our patient is a 2 year old male with hypotonia, global developmental delay, and focal epilepsy. Trio whole-exome sequencing revealed heterozygous variants in PIGN, c.181G > T (p.Glu61*) and c.284G > A (p.Arg95Gln). Analysis of FLAER and anti-CD59 by flow-cytometry demonstrated a shift in this patient's …

Diabetes Conflict Outstrips The Positive Impact Of Self-Efficacy On Youth Adherence And Glycemic Control In Type 1 Diabetes., Amy E. Noser, Lindsay Huffhines, Mark A. Clements, Susana R. Patton

Manuscripts, Articles, Book Chapters and Other Papers

Objective: To examine whether self-efficacy buffers the deleterious consequences of diabetes-specific family conflict on self-monitoring blood glucose (SMBG) and glycated hemoglobin (HbA1c) in youth with type 1 diabetes mellitus (T1DM).

Methods: A total of 129 youth with T1DM (aged 10-16 years) completed measures of diabetes-specific family conflict and self-efficacy for diabetes management, and their blood glucose meter data and HbA1c were extracted from the electronic medical record. We preformed moderation analyses to examine whether self-efficacy moderated the association that diabetes-specific family conflict had with SMBG and HbA1c. We used simple slopes analyses to probe significant interactions.

Results: Our results indicated …

A Nationwide Study On The Prevalence Of Functional Gastrointestinal Disorders In School-Children., Miguel Saps, Jairo Enrique Moreno-Gomez, Carmen Rossy Ramírez-Hernández, John M. Rosen, C A. Velasco-Benitez

Manuscripts, Articles, Book Chapters and Other Papers

Introduction: Functional gastrointestinal disorders (FGIDs) are highly prevalent worldwide and are thought to result from the interplay of multiple factors that can vary from region to region. Nationwide studies can help understand the regional epidemiology and the pathogenesis of FGIDs. The objectives of this study were to determine the prevalence of FGIDs in school-children of Colombia and assess associated factors.

Methods: A cross-sectional study was carried out at twelve private and public schools in ten cities distributed through the four main geographic regions of Colombia. School-children completed a validated questionnaire to assess functional gastrointestinal disorders according to Rome III criteria. …

Hyperfiltration-Associated Biomechanical Forces In Glomerular Injury And Response: Potential Role For Eicosanoids., Mukut Sharma, Ram Sharma, Ellen T. Mccarthy, Virginia J. Savin, Tarak Srivastava

Manuscripts, Articles, Book Chapters and Other Papers

Hyperfiltration is a well-known risk factor in progressive loss of renal function in chronic kidney disease (CKD) secondary to various diseases. A reduced number of functional nephrons due to congenital or acquired cause(s) results in hyperfiltration in the remnant kidney. Hyperfiltration-associated increase in biomechanical forces, namely pressure-induced tensile stress and fluid flow-induced shear stress (FFSS) determine cellular injury and response. We believe the current treatment of CKD yields limited success because it largely attenuates pressure-induced tensile stress changes but not the effect of FFSS on podocytes. Studies on glomerular podocytes, tubular epithelial cells and bone osteocytes provide evidence for a …

Clinician Attitudes About Health Care Services Addressing Adverse Childhood Experiences, Chris C. Sexton, Briana A. Woods-Jaeger, Bridget Cho, Kathy Goggin

Manuscripts, Articles, Book Chapters and Other Papers

Objectives

Study objectives were to elicit feedback from clinical experts in areas with demonstrated health disparities to assess: (1) Perceptions of the relations among Adverse Childhood Experiences (ACEs), parenting, and childhood health disparities; and (2) Recommendations about pediatric health care services to address ACEs and toxic stress.

Methods

Qualitative interviews were conducted with clinical experts (n = 5) in pediatric primary care, asthma and allergy, obesity, and trauma. Interviews were transcribed and coded through iterative thematic extraction.

Results

Two major themes emerged: (1) Need for improved health care access; and (2) Need for family-centered care. Clinicians reported several health care …

Blood Pressure And Visit-To-Visit Blood Pressure Variability Among Individuals With Primary Proteinuric Glomerulopathies., Christine B. Sethna, Kevin E C Meyers, Laura H. Mariani, Kevin J. Psoter, Crystal A. Gadegbeku, Keisha L. Gibson, Tarak Srivastava, Matthias Kretzler, Tammy M. Brady

Manuscripts, Articles, Book Chapters and Other Papers

Hypertension and blood pressure variability (BPV; SD and average real variability) in primary proteinuric glomerulopathies are not well described. Data were from 433 participants in the NEPTUNE (Nephrotic Syndrome Study Network). Hypertensive BP status was defined as previous history of hypertension or BP ≥140/90 mm Hg for adults/≥95th percentile for children at baseline. BPV was measured in participants with ≥3 visits in the first year. Two-hundred ninety-six adults (43 years [interquartile range, 32-57.8 years], 61.5% male) and 147 children (11 years [interquartile range, 5-14 years], 57.8% male) were evaluated. At baseline, 64.8% of adults and 46.9% of children were hypertensive. …

Driving Organizational Change From The Bedside: The Aacn Clinical Scene Investigator Academy., Susan R. Lacey, Caryl Goodyear-Bruch, Adrienne Olney, Dave Hanson, Marian S. Altman, Natasha S. Varn-Davis, Debbie Brinker, Ramón Lavandero, Karen S. Cox

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Staff nurses are pivotal in leading change related to quality improvement efforts, although many lack skills to steer change from the bedside. The American Association of Critical-Care Nurses (AACN) staff nurse leadership program, Clinical Scene Investigator (CSI) Academy, teaches and empowers staff nurses in leadership skills and change concepts to translate evidence into practice affecting patient outcomes.

OBJECTIVES: To describe the curriculum of the AACN CSI Academy that provides staff nurses with the leadership skills required to create unit-based change projects that positively impact patient/family outcomes.

METHODS: The curriculum of the Academy included leadership topics, communication, change concepts, quality …

In Vivo Evaluation Of Stem Cell Aggregates On Osteochondral Regeneration., Banupriya Sridharan, Amy D. Laflin, Michael A. Holtz, Donna M. Pacicca, Nicholas K. Wischmeier, Michael S. Detamore

Manuscripts, Articles, Book Chapters and Other Papers

To date, many osteochondral regenerative approaches have utilized varied combinations of biocompatible materials and cells to engineer cartilage. Even in cell-based approaches, to date, no study has utilized stem cell aggregates alone for regenerating articular cartilage. Thus, the purpose of this study was to evaluate the performance of a novel stem cell-based aggregate approach in a fibrin carrier to regenerate osteochondral defects in the Sprague-Dawley rat trochlear groove model. Two different densities of rat bone marrow mesenchymal stem cell (rBMSC) aggregates were fabricated by the hanging drop technique. At 8 weeks, the cell aggregates supported the defects and served as …

Two-Year Antibody Persistence In Children Vaccinated At 12-15 Months With A Measles-Mumps-Rubella Virus Vaccine Without Human Serum Albumin., Andrea A. Berry, Remon Abu-Elyazeed, Clemente Diaz-Perez, Maurice A. Mufson, Christopher J. Harrison, Michael Leonardi, Jerry D. Twiggs, Christopher Peltier, Stanley Grogg, Antonio Carbayo, Steven Shapiro, Michael Povey, Carmen Baccarini, Bruce L. Innis, Ouzama Henry

Manuscripts, Articles, Book Chapters and Other Papers

One combined measles-mumps-rubella (MMR) vaccine without Human Serum Albumin (HSA) is currently licensed in the USA (M-M-R II; Merck, USA) and another has been developed (Priorix™ [MMR-RIT, GSK, Belgium]). In this follow-up study, children from USA or Puerto Rico, who had received one dose of M-M-R II or MMR-RIT at 12-15 months of age in the primary study (NCT00861744), were followed-up for 2 y post-vaccination. Anti-measles and anti-rubella antibodies were measured using Enzyme-Linked Immunosorbent Assay (ELISA), and anti-mumps antibodies using ELISA and plaque reduction neutralization (PRN) assays. Serious adverse events (SAEs) were recorded during the entire follow-up. The according-to-protocol (ATP) …

Re-Tubularization Of Highly-Ischemic Anti-Mesenteric Border (Rehab): A Novel Bowel Preservation Technique In Complex Gastroschisis., Richard J. Hendrickson, Ashwini S. Poola, Katherine W. Gonzalez, Joel Lim, Tolulope A. Oyetunji

Manuscripts, Articles, Book Chapters and Other Papers

Complex gastroschisis with bowel necrosis poses an operative challenge. Surgeons must weigh the decision between resection versus preservation of ischemic bowel. As one of the leading causes of short bowel syndrome, aggressive resection in complicated gastroschisis subjects children to prolonged dependence on parenteral nutrition and its attendant complications. Herein, we describe a novel technique aimed towards bowel preservation in complex gastroschisis patients with severe bowel ischemia with the ultimate goal for enteral autonomy.

Efficacy And Safety Of Sparsentan Compared With Irbesartan In Patients With Primary Focal Segmental Glomerulosclerosis: Randomized, Controlled Trial Design (Duet)., Radko Komers, Debbie S. Gipson, Peter Nelson, Sharon Adler, Tarak Srivastava, Vimal K. Derebail, Kevin E. Meyers, Pablo Pergola, Meghan E. Macnally, Jennifer L. Hunt, Alvin Shih, Howard Trachtman

Manuscripts, Articles, Book Chapters and Other Papers

Introduction: Primary focal segmental glomerulosclerosis (FSGS) is a leading cause of nephrotic syndrome and end-stage renal disease. There are no US Food and Drug Administration-approved therapies for FSGS, and treatment often fails to reduce proteinuria. Endothelin is an important factor in the pathophysiology of podocyte disorders, including FSGS. Sparsentan is a first-in-class, orally active, dual-acting angiotensin receptor blocker (ARB) and highly selective endothelin Type A receptor antagonist. This study is designed to evaluate whether sparsentan lowers proteinuria compared with an ARB alone and has a favorable safety profile in patients with FSGS.

Methods: DUET is a phase 2, randomized, active-control, …

Clinical Pharmacogenetics Implementation Consortium Guideline (Cpic) For Cyp2d6 And Cyp2c19 Genotypes And Dosing Of Tricyclic Antidepressants: 2016 Update., J K. Hicks, K Sangkuhl, J J. Swen, V L. Ellingrod, D J. Müller, K Shimoda, J R. Bishop, E D. Kharasch, T C. Skaar, Andrea Gaedigk, H M. Dunnenberger, T E. Klein, K E. Caudle, J C. Stingl

Manuscripts, Articles, Book Chapters and Other Papers

No abstract provided.

Neonatal Graves' Disease With Maternal Hypothyroidism., Gangaram Akangire, Alain Cuna, Charisse I. Lachica, Ryan T. Fischer, Sripriya Raman, Venkatesh Sampath

Manuscripts, Articles, Book Chapters and Other Papers

Neonatal Graves' disease presenting as conjugated hyperbilirubinemia is a diagnostic challenge because the differential includes a gamut of liver and systemic diseases. We present a unique case of neonatal Graves' disease in a premature infant with conjugated hyperbilirubinemia born to a mother with hypothyroidism during pregnancy and remote history of Graves' disease. Infant was treated with a combination of methimazole, propranolol, and potassium iodide for 4 weeks. Thyroid function improved after 8 weeks of treatment with full recovery of thyroid function, disappearance of thyroid-stimulating antibodies, and resolution of failure to thrive and conjugated hyperbilirubinemia. This case provides several clinical vignettes …

Term Neonate With Atypical Hypoxic-Ischemic Encephalopathy Presentation: A Case Report., Nick Townley, Emily Mcnellis, Venkatesh Sampath

Manuscripts, Articles, Book Chapters and Other Papers

We describe a case of atypical hypoxic-ischemic encephalopathy (HIE) in a neonate following a normal pregnancy and delivery who was found to have an umbilical vein thrombosis. The infant arrived to our center with continuous bicycling movement of her lower extremities. She had a continuous electroencephalogram that showed burst suppression and magnetic resonance imaging of the brain showed diffusely abnormal cerebral cortical/subcortical diffusion restriction which may be secondary hypoxic-ischemic injury. Interestingly, a pathology report noted a focal umbilical vein thrombosis appearing to have compressed an umbilical artery with associated arterial dissection and hematoma. Our case illustrates how umbilical venous or …

Congenital Left Ventricle-To-Coronary Sinus Fistula: A Rare Isolated Anomaly Of The Coronary Sinus., Doaa M. Aly, Nikki M. Singh, Sanket Shah

Manuscripts, Articles, Book Chapters and Other Papers

Congenital anomalies of the coronary sinus (CS) are very uncommon. They can occur either in isolation or in association with other congenital heart defects. 1 Among those anomalies, congenital fistulae connecting the left ventricle and the CS represent a very rare entity. Most reported cases of CS fistula generally originate in the right coronary artery or left anterior descending coronary artery system. 2 We describe an extremely rare case of an isolated high-pressure congenital left ventricle–to–CS fistula as an anomaly of the CS in an otherwise asymptomatic child. The initial clinical presentation and follow-up, imaging studies, and plan for management …

Antibiotic Prophylaxis Is Associated With Subsequent Resistant Infections In Children With An Initial Extended-Spectrum-Cephalosporin-Resistant Enterobacteriaceae Infection., Sibani Das, Amanda L. Adler, Arianna Miles-Jay, Matthew P. Kronman, Xuan Qin, Scott J. Weissman, C A. Burnham, Alexis Elward, Jason G. Newland, Rangaraj Selvarangan, Kaede V. Sullivan, Theoklis Zaoutis, Danielle M. Zerr

Manuscripts, Articles, Book Chapters and Other Papers

The objective of this study was to assess the association between previous antibiotic use, particularly long-term prophylaxis, and the occurrence of subsequent resistant infections in children with index infections due to extended-spectrum-cephalosporin-resistant Enterobacteriaceae We also investigated the concordance of the index and subsequent isolates. Extended-spectrum-cephalosporin-resistant Escherichia coli and Klebsiella spp. isolated from normally sterile sites of patients aged species, resistance determinants, and fumC-fimH (E. coli) or tonB (Klebsiella pneumoniae) type were identical to those of the index isolate. In total, 323 patients had 396 resistant isolates; 45 (14%) patients had ≥1 subsequent resistant infection, totaling 73 …

A Novel Method Of Measuring Fractional Exhaled Nitric Oxide In Tracheostomized Ventilator-Dependent Children., Vydehi R. Murthy, Hugo Escobar, Michael Norberg, Charisse I. Lachica, Linda L. Gratny, Ashley K. Sherman, William E Truog, Winston M. Manimtim

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: The lower airway concentration of fractional exhaled nitric oxide (FENO) is unknown in children with chronic lung disease of infancy who have tracheostomy for long-term mechanical ventilation. We aimed to evaluate an online method of measuring FENO in a cohort of ventilator-dependent children with a tracheostomy and to explore the relationship between the peak FENO concentration (FENO peak) and the degree of respiratory support using the respiratory severity score.

METHODS: We conducted a prospective cross-sectional study in 31 subjects who were receiving long-term respiratory support through a tracheostomy. We measured the FENO peak and FENO plateau concentration from the …

The Interactive Effect Of Diabetes Family Conflict And Depression On Insulin Bolusing Behaviors For Youth., Genevieve Maliszewski, Susana R. Patton, L Kurt Midyett, Mark A. Clements

Manuscripts, Articles, Book Chapters and Other Papers

OBJECTIVE: Adherence to type 1 diabetes management declines as children enter adolescence. For youth, psychosocial variables including mood and interpersonal relationships play a large role in diabetes maintenance. The current study assessed the unique and interactive roles diabetes family conflict and depression have on insulin bolusing behaviors for youth ages 10-16 years.

METHODS: Ninety-one youth-parent dyads completed a survey assessing family conflict and depression. Mean daily blood glucose levels, mealtime insulin bolus scores ( BOLUS), and glycated hemoglobin (HbA1c) were collected from the medical record as outcome variables.

RESULTS: Parent-reported diabetes-related family conflict and youths' endorsed depression both significantly predicted …

Role Of Biomechanical Forces In Hyperfiltration-Mediated Glomerular Injury In Congenital Anomalies Of The Kidney And Urinary Tract., Tarak Srivastava, Ganesh Thiagarajan, Uri S. Alon, Ram Sharma, Ashraf El-Meanawy, Ellen T. Mccarthy, Virginia J. Savin, Mukut Sharma

Manuscripts, Articles, Book Chapters and Other Papers

Congenital anomalies of the kidney and urinary tract (CAKUT) including solitary kidney constitute the main cause of progressive chronic kidney disease (CKD) in children. Children born with CAKUT develop signs of CKD only during adolescence and do not respond to renin-angiotensin-aldosterone system blockers. Early cellular changes underlying CKD progression to end-stage renal disease by early adulthood are not well understood. The mechanism of maladaptive hyperfiltration that occurs from loss of functional nephrons, including solitary kidney, is not clear. We re-examine the phenomenon of hyperfiltration in the context of biomechanical forces with special reference to glomerular podocytes. Capillary stretch exerts tensile …

Within-Person Associations Of Young Adolescents' Physical Activity Across Five Primary Locations: Is There Evidence Of Cross-Location Compensation?, Jordan A. Carlson, Tarrah B. Mitchell, Brian E. Saelens, Vincent S. Staggs, Jacqueline Kerr, Lawrence D. Frank, Jasper Schipperijn, Terry L. Conway, Karen Glanz, Jim E. Chapman, Kelli L. Cain, James F. Sallis

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Youth are active in multiple locations, but it is unknown whether more physical activity in one location is associated with less in other locations. This cross-sectional study examines whether on days with more physical activity in a given location, relative to their typical activity in that location, youth had less activity in other locations (i.e., within-person associations/compensation).

METHODS: Participants were 528 adolescents, ages 12 to 16 (M = 14.12, SD = 1.44, 50% boys, 70% White non-Hispanic). Accelerometer and Global Positioning System devices were used to measure the proportion of time spent in moderate-to-vigorous physical activity (MVPA) in five …

Exome Analysis Of Rare And Common Variants Within The Nod Signaling Pathway., Gaia Andreoletti, Valentina Shakhnovich, Kathy Christenson, Tracy Coelho, Rachel Haggarty, Nadeem A. Afzal, Akshay Batra, Britt-Sabina Petersen, Matthew Mort, R Mark Beattie, Sarah Ennis

Manuscripts, Articles, Book Chapters and Other Papers

Pediatric inflammatory bowel disease (pIBD) is a chronic heterogeneous disorder. This study looks at the burden of common and rare coding mutations within 41 genes comprising the NOD signaling pathway in pIBD patients. 136 pIBD and 106 control samples underwent whole-exome sequencing. We compared the burden of common, rare and private mutation between these two groups using the SKAT-O test. An independent replication cohort of 33 cases and 111 controls was used to validate significant findings. We observed variation in 40 of 41 genes comprising the NOD signaling pathway. Four genes were significantly associated with disease in the discovery cohort …

Nicotinamide Phosphoribosyltransferase Promotes Pulmonary Vascular Remodeling And Is A Therapeutic Target In Pulmonary Arterial Hypertension., Jiwang Chen, Justin R Sysol, Sunit Singla, Shuangping Zhao, Aya Yamamura, Daniela Valdez-Jasso, Taimur Abbasi, Krystyna M. Shioura, Sakshi Sahni, Vamsi Reddy, Arvind Sridhar, Hui Gao, Jaime Torres, Sara M. Camp, Haiyang Tang, Shui Q. Ye, Suzy Comhair, Raed Dweik, Paul Hassoun, Jason X-J Yuan, Joe G N Garcia, Roberto F. Machado

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Pulmonary arterial hypertension is a severe and progressive disease, a hallmark of which is pulmonary vascular remodeling. Nicotinamide phosphoribosyltransferase (NAMPT) is a cytozyme that regulates intracellular nicotinamide adenine dinucleotide levels and cellular redox state, regulates histone deacetylases, promotes cell proliferation, and inhibits apoptosis. We hypothesized that NAMPT promotes pulmonary vascular remodeling and that inhibition of NAMPT could attenuate pulmonary hypertension.

METHODS: Plasma, mRNA, and protein levels of NAMPT were measured in the lungs and isolated pulmonary artery endothelial cells from patients with pulmonary arterial hypertension and in the lungs of rodent models of pulmonary hypertension.Nampt+/- mice were …

Novel Genetic Variants Associated With Child Refractory Esophageal Stricture With Food Allergy By Exome Sequencing., Min Yang, Min Xiong, Huan Chen, Lanlan Geng, Peiyu Chen, Jing Xie, Shui Qing Ye, Ding-You Li, Sitang Gong

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Refractory esophageal stricture (RES) may be attributed to food allergy. Its etiology and pathogenesis are not fully understood. Identification of novel genetic variants associated with this disease by exome sequencing (exome-seq) may provide new mechanistic insights and new therapeutic targets.

METHODS: To identify new and novel disease-associating variants, whole-exome sequencing was performed on an Illumina NGS platform in three children with RES as well as food allergy.

RESULTS: A total of 91,024 variants were identified. By filtering out 'normal variants' against those of the 1000 Genomes Project, we identified 12,741 remaining variants which are potentially associated with RES plus …

Fate Of Neural Progenitor Cells Transplanted Into Jaundiced And Nonjaundiced Rat Brains., Fu-Chen Yang, Sean M. Riordan, Michelle Winter, Li Gan, Peter G. Smith, Jay L. Vivian, Steven Shapiro, John A. Stanford

Manuscripts, Articles, Book Chapters and Other Papers

High levels of bilirubin in infants can cause kernicterus, which includes basal ganglia damage and dystonia. Stem cell transplantation may be an effective treatment for this disease. In this study, we transplanted human neural progenitor cells differentiated toward propriospinal interneurons into the striatum of 20-day-old spontaneously jaundiced (jj) Gunn rats and nonjaundiced (Nj) littermates. Using immunohistochemical methods, we found that grafted cells survived and grew fibers in jj and Nj brains 3 weeks after transplantation. Grafted cells had a higher survival rate in jj than in Nj brains, suggesting that slightly elevated bilirubin may protect graft survival due to its …

Pilot Study Comparing The Childhood Arthritis & Rheumatology Research Alliance (Carra) Systemic Juvenile Idiopathic Arthritis Consensus Treatment Plans., Yukiko Kimura, Sriharsha Grevich, Timothy Beukelman, Esi Morgan, Peter A. Nigrovic, Kelly Mieszkalski, T Brent Graham, Maria Ibarra, Norman Ilowite, Marisa Klein-Gitelman, Karen Onel, Sampath Prahalad, Marilynn Punaro, Sarah Ringold, Dana Toib, Heather Van Mater, Jennifer E. Weiss, Pamela F. Weiss, Laura E. Schanberg, Carra Registry Investigators

Manuscripts, Articles, Book Chapters and Other Papers

OBJECTIVES: To assess the feasibility of studying the comparative effectiveness of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) consensus treatment plans (CTPs) for systemic Juvenile Idiopathic Arthritis (JIA) using an observational registry.

METHODS: Untreated systemic JIA patients enrolled in the CARRA Registry were begun on one of 4 CTPs chosen by the treating physician and patient/family (glucocorticoid [GC] alone; methotrexate [MTX] ± GC; IL1 inhibitor [IL1i] ± GC; IL6 inhibitor [IL6i] ± GC). The primary outcome of clinical inactive disease (CID) without current GC use was assessed at 9 months.

TRIAL REGISTRATION: clinicaltrials.gov NCT01697254; first registered 9/28/12 (retrospectively enrolled). …

Seroprevalence Of Poliovirus Antibodies In The Kansas City Metropolitan Area, 2012-2013., Gregory S. Wallace, Barbara A. Pahud, William C. Weldon, Aaron T. Curns, M Steven Oberste, Christopher J. Harrison

Manuscripts, Articles, Book Chapters and Other Papers

No indigenous cases of poliomyelitis have occurred in the US since 1979; however the risk of importation persists until global eradication is achieved. The seropositivity rate for different age cohorts with exposures to different poliovirus vaccine types and wild virus in the US are not presently known. A convenience sample was conducted in the Kansas City metropolitan area during 2012-2103 with approximately 100 participants enrolled for each of 5 age cohorts categorized based on vaccine policy changes over time in the US. Immunization records for poliovirus vaccination were required for participants90.7%, 94.4%, and 83.3%, for types 1, 2, and 3, …

Diet And Polycystic Kidney Disease: A Pilot Intervention Study., Jacob M. Taylor, Jill M. Hamilton-Reeves, Debra K. Sullivan, Cheryl A. Gibson, Catherine Creed, Susan E. Carlson, Donald E. Wesson, Jared J. Grantham

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND & AIMS: Dietary sodium, protein, acid precursors, and water have been linked to cyst growth in polycystic kidney disease; yet, no studies in patients have examined the feasibility of using a dietary intervention that controls all of these factors. The aim of this study was to determine if a diet, appropriate for persons of most ages, reduces the excretion of sodium, urea, acid, and decreases mean urine osmolality while gaining acceptance by patients with autosomal dominant polycystic kidney disease (ADPKD).

METHODS: Twelve adults with ADPKD enrolled in a pre-post pilot feasibility study and served as their own controls. Individuals …