Digital Commons Network^™

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 …

Sexual Coloration And Performance Capacity In Male Side-Blotched Lizards (Uta Stansburiana), Forrest Jensen, Spencer B. Hudson, Susannah S. French

Biology Posters

In many species of lizards, males fight over territories that allow them access to females, and during these interactions males will display sexual signals to competing individuals. Social signals such as coloration, are seemingly favored by sexual selection as they are thought to provide information about individual quality, such as performance capacity. Side-blotched Lizards, Uta stansburiana, exhibit throat color polymorphism (i.e. orange, yellow, and blue badges), which signal alternative life history strategies to conspecifics. Although morphological, behavioral and physiological characteristics have been shown to differ across male morphs, color variation within each morph has not been compared to aspects of …

Do Female Guppies Really Know What They Want?, Jennica Blasi, Abby Keuhl-Coe, Hanna Kirkland, Michael Larson

Biology Posters

• Hypothesis: Female guppies do not influence each other’s choice of male guppy

• Predictions: If female guppies do not influence each other’s choice in males, then most females guppies will choose the same male whether alone or in the presence of other females

• Choice is defined as which pane of glass the female interacts more with from 1 to 3 minutes after being introduced to the tank

• 34 Females were used, 17 small and 17 large

Guppies: Sexy And You Know It?, Leslie Bredthauer, Katelin Boettcher, Landon Keller, Kim Sullivan

Biology Posters

We were curious to know if female guppies are more interested in the quality or quantity of males.

Efficacy And Safety Of Pembrolizumab In Patients Enrolled In Keynote-030 In The United States: An Expanded Access Program., Tara C Gangadhar, Wen-Jen Hwu, Michael A Postow, Omid Hamid, Adil Daud, Roxana Dronca, Richard Joseph, Steven J O'Day, F S Hodi, Anna C Pavlick, Harriet Kluger, Romina P Oxborough, Aiming Yang, Mihaela Gazdoiu, Debra A Kush, Scot Ebbinghaus, April K S Salama

Articles, Abstracts, and Reports

KEYNOTE-030 (ClinicalTrials.gov ID, NCT02083484) was a global expanded access program that allowed access to pembrolizumab, an antiprogrammed death 1 antibody, for patients with advanced melanoma before its regulatory approval. Patients with unresectable stage III/IV melanoma that progressed after standard-of-care therapy, including ipilimumab and, if BRAF mutant, a BRAF inhibitor, were eligible to receive pembrolizumab 2 mg/kg every 3 weeks. Response was assessed by immune-related response criteria by investigator review. Adverse events (AEs) were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events, version 4.0. In the United States, 979 patients enrolled between April and September 2014. …

Pro-Inflammatory Chemokines And Cytokines Dominate The Blister Fluid Molecular Signature In Patients With Epidermolysis Bullosa And Affect Leukocyte And Stem Cell Migration., Vitali Alexeev, Julio Cesar Salas-Alanis, Francis Palisson, Lila Mukhtarzada, Giulio Fortuna, Jouni Uitto, Andrew P. South, Olga Igoucheva

Department of Dermatology and Cutaneous Biology Faculty Papers

Hereditary epidermolysis bullosa (EB) is associated with skin blistering and the development of chronic nonhealing wounds. Although clinical studies have shown that cell-based therapies improve wound healing, the recruitment of therapeutic cells to blistering skin and to more advanced skin lesions remains a challenge. Here, we analyzed cytokines and chemokines in blister fluids of patients affected by dystrophic, junctional, and simplex EB. Our analysis revealed high levels of CXCR1, CXCR2, CCR2, and CCR4 ligands, particularly dominant in dystrophic and junctional EB. In vitro migration assays demonstrated the preferential recruitment of CCR4⁺ lymphocytes and CXCR1⁺, CXCR2⁺, …

Predictors Of Sleepiness In Obstructive Sleep Apnoea At Baseline And After 6 Months Of Continuous Positive Airway Pressure Therapy., Rohit Budhiraja, Clete A Kushida, Deborah A Nichols, James K Walsh, Richard D Simon, Daniel J Gottlieb, Stuart F Quan

Articles, Abstracts, and Reports

We evaluated factors associated with subjective and objective sleepiness at baseline and after 6 months of continuous positive airway pressure (CPAP) therapy in patients with obstructive sleep apnoea (OSA).We analysed data from the Apnoea Positive Pressure Long-term Efficacy Study (APPLES), a prospective 6-month multicentre randomised controlled trial with 1105 subjects with OSA, 558 of who were randomised to active CPAP. Epworth sleepiness scale (ESS) scores and the mean sleep latency (MSL) on the maintenance of wakefulness test at baseline and after 6 months of CPAP therapy were recorded.Excessive sleepiness (ESS score >10) was present in 543 (49.1%) participants. Younger age, …

Second Primary Malignant Neoplasms And Survival In Adolescent And Young Adult Cancer Survivors., Theresa H M Keegan, Archie Bleyer, Aaron S Rosenberg, Qian Li, Melanie Goldfarb

Articles, Abstracts, and Reports

Importance: Although the increased incidence of second primary malignant neoplasms (SPMs) is a well-known late effect after cancer, few studies have compared survival after an SPM to survival of the same cancer occurring as first primary malignant neoplasm (PM) by age.

Objective: To assess the survival impact of SPMs in adolescents and young adults (AYAs) (15-39 years) compared with that of pediatric (<15 >years) and older adult (≥40 years) patients with the same SPMs.

Design, Setting, and Participants: This was a population-based, retrospective cohort study of patients with cancer in 13 Surveillance, Epidemiology and End Results regions in the United …

Characteristics Associated With Requests By Pathologists For Second Opinions On Breast Biopsies., Berta M Geller, Heidi D Nelson, Donald L Weaver, Paul D Frederick, Kimberly H Allison, Tracy Onega, Patricia A Carney, Anna N A Tosteson, Joann G Elmore

Articles, Abstracts, and Reports

AIMS: Second opinions in pathology improve patient safety by reducing diagnostic errors, leading to more appropriate clinical treatment decisions. Little objective data are available regarding the factors triggering a request for second opinion despite second opinion consultations being part of the diagnostic system of pathology. Therefore we sought to assess breast biopsy cases and interpreting pathologists characteristics associated with second opinion requests.

METHODS: Collected pathologist surveys and their interpretations of 60 test set cases were used to explore the relationships between case characteristics, pathologist characteristics and case perceptions, and requests for second opinions. Data were evaluated by logistic regression and …

Rapid Rest/Stress Regadenoson Ungated Perfusion Cmr For Detection Of Coronary Artery Disease In Patients With Atrial Fibrillation., Erik T Bieging, I Haider, G Adluru, L Chang, P Suksaranjit, D Likhite, A Shaaban, L Jensen, B D Wilson, C J Mcgann, E Dibella

Articles, Abstracts, and Reports

Cardiovascular magnetic resonance (CMR) perfusion has been established as a useful imaging modality for the detection of coronary artery disease (CAD). However, there are several limitations when applying standard, ECG-gated stress/rest perfusion CMR to patients with atrial fibrillation (AF). In this study we investigate an approach with no ECG gating and a rapid rest/stress perfusion protocol to determine its accuracy for detection of CAD in patients with AF. 26 patients with AF underwent a rapid rest/regadenoson stress CMR perfusion imaging protocol, and all patients had X-ray coronary angiography. An ungated radial myocardial perfusion sequence was used. Imaging protocol included: rest …

Organic Solvent Exposure And Depressive Symptoms Among Licensed Pesticide Applicators In The Agricultural Health Study, Miriam Siegel, Sarah E. Starks, Wayne T. Sanderson, Freya Kamel, Jane A. Hoppin, Fred Gerr

Epidemiology and Environmental Health Faculty Publications

Purpose

Although organic solvents are often used in agricultural operations, neurotoxic effects of solvent exposure have not been extensively studied among farmers. The current analysis examined associations between questionnaire-based metrics of organic solvent exposure and depressive symptoms among farmers.

Methods

Results from 692 male Agricultural Health Study participants were analyzed. Solvent type and exposure duration were assessed by questionnaire. An “ever-use” variable and years of use categories were constructed for exposure to gasoline, paint/lacquer thinner, petroleum distillates, and any solvent. Depressive symptoms were ascertained with the Center for Epidemiologic Studies Depression Scale (CES-D); scores were analyzed separately as continuous (0–60) …

Outlier Identification In Radiation Therapy Knowledge-Based Planning: A Study Of Pelvic Cases., Yang Sheng, Yaorong Ge, Lulin Yuan, Taoran Li, Fang-Fang Yin, Qingrong Jackie Wu

Department of Radiation Oncology Faculty Papers

PURPOSE: The purpose of this study was to apply statistical metrics to identify outliers and to investigate the impact of outliers on knowledge-based planning in radiation therapy of pelvic cases. We also aimed to develop a systematic workflow for identifying and analyzing geometric and dosimetric outliers.

METHODS: Four groups (G1-G4) of pelvic plans were sampled in this study. These include the following three groups of clinical IMRT cases: G1 (37 prostate cases), G2 (37 prostate plus lymph node cases) and G3 (37 prostate bed cases). Cases in G4 were planned in accordance with dynamic-arc radiation therapy procedure and include 10 …

A Rural/Urban Comparison Of Privacy And Confidentiality Concerns Associated With Providing Sensitive Location Information In Epidemiologic Research Involving Persons Who Use Drugs, Abby E. Rudolph, April M. Young, Jennifer R. Havens

Epidemiology and Environmental Health Faculty Publications

Background—Analyses that link contextual factors with individual-level data can improve our understanding of the "risk environment"; however, the accuracy of information provided by participants about locations where illegal/stigmatized behaviors occur may be influenced by privacy/confidentiality concerns that may vary by setting and/or data collection approach.

Methods—We recruited thirty-five persons who use drugs from a rural Appalachian town and a Mid-Atlantic city to participate in in-depth interviews. Through thematic analyses, we identified and compared privacy/confidentiality concerns associated with two survey methods that (1) collect self-reported addresses/cross-streets and (2) use an interactive web-based map to find/confirm locations in rural and …

Multisite Evaluation Of The Bd Max Extended Enteric Bacterial Panel For Detection Of Yersinia Enterocolitica, Enterotoxigenic Escherichia Coli, Vibrio, And Plesiomonas Shigelloides From Stool Specimens., Patricia J Simner, Margret Oethinger, Kathleen A Stellrecht, Dylan R Pillai, Ram Yogev, Helene Leblond, Joel Mortensen

Articles, Abstracts, and Reports

The purpose of this study was to perform a multisite evaluation to establish the performance characteristics of the BD Max extended enteric bacterial panel (xEBP) assay directly from unpreserved or Cary-Blair-preserved stool specimens for the detection of Yersinia enterocolitica, enterotoxigenic Escherichia coli (ETEC), Vibrio, and Plesiomonas shigelloides The study included prospective, retrospective, and prepared contrived specimens from 6 clinical sites. BD Max xEBP results were compared to the reference method, which included standard culture techniques coupled with alternate PCR and sequencing, except for ETEC, for which the reference method was two alternate PCRs and sequencing. Alternate PCR was …

Partner Relationships And Injection Sharing Practices Among Rural Appalachian Women, Michele Staton, Justin C. Strickland, Martha Tillson, Carl Leukefeld, J. Matthew Webster, Carrie B. Oser

Behavioral Science Faculty Publications

Background—The role of relationships in initiating and maintaining women’s risk behaviors has been established. However, understanding factors that may underlie partner relationships and women’s risky drug use, particularly in rural contexts, is limited. This study is the first to examine the association between injecting partners and women’s risky injection practices as a function of relationship power perception.

Methods—Female participants were recruited from three rural jails in the Appalachian region. Women were randomly selected, provided informed consent, and screened for study eligibility criteria. This cross-sectional analysis focuses on women who inject drugs (WWID) during the year before entering jail …

Cerebral Amyloid Angiopathy In Down Syndrome And Sporadic And Autosomal-Dominant Alzheimer's Disease, María Carmona-Iragui, Mircea Balasa, Bessy Benejam, Daniel Alcolea, Susana Fernández, Laura Videla, Isabel Sala, María Belén Sánchez-Saudinós, Estrella Morenas-Rodriguez, Roser Ribosa-Nogué, Ignacio Illán-Gala, Sofía Gonzalez-Ortiz, Jordi Clarimón, Frederick A. Schmitt, David K. Powell, Beatriz Bosch, Albert Lladó, Michael S. Rafii, Elizabeth Head, José Luis Molinuevo, Rafael Blesa, Sebastián Videla, Alberto Lleó, Raquel Sánchez-Valle, Juan Fortea

Sanders-Brown Center on Aging Faculty Publications

Introduction—We aimed to investigate if cerebral amyloid angiopathy (CAA) is more frequent in genetically determined than in sporadic early-onset forms of Alzheimer's disease (AD) (early-onset AD [EOAD]).

Methods—Neuroimaging features of CAA, APOE, and cerebrospinal fluid-Aβ40 levels were studied in subjects with Down syndrome (DS, n = 117), autosomal-dominant AD (ADAD, n = 29), sporadic EOAD (n = 42), and healthy controls (n = 68).

Results—CAA was present in 31%, 38%, and 12% of cognitively impaired DS, symptomatic ADAD, and sporadic EOAD subjects and in 13% and 4% of cognitively unimpaired DS individuals and healthy controls, respectively. …

Clinical Assessment Of Cognitive Function In Patients With Head And Neck Cancer: Prevalence And Correlates, Amy M. Williams, Jamie Lindholm, Farzan Siddiqui, Tamer Ghanem, Steven S. Chang

Otolaryngology Articles

Objective: Identify the prevalence and clinical correlates of cognitive impairment in patients presenting for treatment of head and neck cancer (HNC) using brief screening within a multidisciplinary care team.

Study Design: A case series with planned data collection of cognitive function, quality of life (QoL), and psychosocial variables.

Setting: Urban Midwest academic medical center.

Subjects and Methods: In total, 209 consecutive patients with a diagnosis of HNC between August 2015 and September 2016 who had a pretreatment assessment with a clinical health psychologist. At pretreatment assessment, the Montreal Cognitive Assessment (MoCA), a brief screening tool for cognitive function, was administered …

Dynamic Range Of Frontoparietal Functional Modulation Is Associated With Working Memory Capacity Limitations In Older Adults, Jonathan G. Hakun, Nathan F. Johnson

Physical Therapy Faculty Publications

Older adults tend to over-activate regions throughout frontoparietal cortices and exhibit a reduced range of functional modulation during WM task performance compared to younger adults. While recent evidence suggests that reduced functional modulation is associated with poorer task performance, it remains unclear whether reduced range of modulation is indicative of general WM capacity-limitations. In the current study, we examined whether the range of functional modulation observed over multiple levels of WM task difficulty (N-Back) predicts in-scanner task performance and out-of-scanner psychometric estimates of WM capacity. Within our sample (60–77 years of age), age was negatively associated with frontoparietal modulation range. …

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 …

Bimodal Coupling Of Ripples And Slower Oscillations During Sleep In Patients With Focal Epilepsy., Inkyung Song, Iren Orosz, Inna Chervoneva, Zachary J. Waldman, Itzhak Fried, Chengyuan Wu, Ashwini Sharan, Noriko Salamon, Richard Gorniak, Sandra Dewar, Anatol Bragin, Jerome Engel, Michael R. Sperling, Richard Staba, Shennan A. Weiss

Department of Neurology Faculty Papers

OBJECTIVE: Differentiating pathologic and physiologic high-frequency oscillations (HFOs) is challenging. In patients with focal epilepsy, HFOs occur during the transitional periods between the up and down state of slow waves. The preferred phase angles of this form of phase-event amplitude coupling are bimodally distributed, and the ripples (80-150 Hz) that occur during the up-down transition more often occur in the seizure-onset zone (SOZ). We investigated if bimodal ripple coupling was also evident for faster sleep oscillations, and could identify the SOZ.

METHODS: Using an automated ripple detector, we identified ripple events in 40-60 min intracranial electroencephalography (iEEG) recordings from 23 …

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. …

Suppurative Complications Of Acute Hematogenous Osteomyelitis In Children, Jennifer J. Johnston, Cristina Murray-Krezan, Walter N. Dehority

Pediatrics Research and Scholarship

We carried out a case-control study in children with acute hematogenous osteomyelitis (AHO) with and without suppurative complications discharged from our institution over an 11-year period to test the hypothesis that abscess formation was associated with a delayed presentation to care. Of 102 children with AHO, 54 abscesses were documented in 46 patients (25 bone, 29 muscle). A delay in presentation was not associated with abscess formation (6.5 vs. 5.0 days, P=0.26). Overall, 78% of all bone abscesses were visible on initial MRI. Consistent use of MRI at presentation may identify children with suppurative complications of AHO.

Vitamin D, Heidi D Moretti, Vincent J Colucci, Bradley D Berry

Articles, Abstracts, and Reports

BACKGROUND: Vitamin D status may influence heart failure (HF) patient outcomes by affecting b-type natriuretic peptide (BNP), parathyroid hormone (PTH), and enhancing cardiac contractility. Vitamin D deficiency is associated with morbidity and mortality in HF patients. The objective of this study was to determine if vitamin D3 at a comparatively high dose would replete 25-hydroxyvitamin D (25(OH)D) stores, improve BNP, PTH, cardiopulmonary function, reduce inflammatory markers, and improve quality of life (QOL) in HF patients.

METHODS: This was a 6 month, parallel group, double-blind, placebo-controlled, single clinic center, randomized trial of supplemental vitamin D3 using a dose of 10,000 IU …

Lessons From The Field: The Conduct Of Randomized Controlled Trials In Botswana., Janice M. Bonsu, Rosemary Frasso, Allison E. Curry

College of Population Health Faculty Papers

BACKGROUND: The conduct of randomized controlled trials (RCTs) in low-resource settings may present unique financial, logistic, and process-related challenges. Middle-income countries that have comparable disease burdens to low-income countries, but greater availability of resources, may be conducive settings for RCTs. Indeed, the country of Botswana is experiencing a rapid increase in the conduct of RCTs. Our objective was to explore the experiences of individuals conducting RCTs in Botswana to gain an understanding of the challenges and adaptive strategies to their work.

METHODS: We conducted in-depth interviews with 14 national and international individuals working on RCTs in Botswana. Participants included principal …

Ufm1 Founder Mutation In The Roma Population Causes Recessive Variant Of H-Abc., Eline M C Hamilton, Enrico Bertini, Luba Kalaydjieva, Bharti Morar, Dana Dojčáková, Adeline Vanderver, Recessive H-Abc Research Group.

Neurology Faculty Publications

OBJECTIVE: To identify the gene defect in patients with hypomyelination with atrophy of the basal ganglia and cerebellum (H-ABC) who are negative for TUBB4A mutations.

METHODS: We performed homozygosity mapping and whole exome sequencing (WES) to detect the disease-causing variant. We used a Taqman assay for population screening. We developed a luciferase reporter construct to investigate the effect of the promoter mutation on expression.

RESULTS: Sixteen patients from 14 families from different countries fulfilling the MRI criteria for H-ABC exhibited a similar, severe clinical phenotype, including lack of development and a severe epileptic encephalopathy. The majority of patients had a …

Association Of Metabolic Syndrome And Change In Unified Parkinson's Disease Rating Scale Scores., Maureen Leehey, Sheng Luo, Saloni Sharma, Anne-Marie A. Wills, Jacquelyn L. Bainbridge, Pei Shieen Wong, David K. Simon, Jay S Schneider, Yunxi Zhang, Adriana Pérez, Rohit Dhall, Chadwick W. Christine, Carlos Singer, Franca Cambi, James T Boyd

Department of Pathology, Anatomy, and Cell Biology Faculty Papers

OBJECTIVE: To explore the association between metabolic syndrome and the Unified Parkinson's Disease Rating Scale (UPDRS) scores and, secondarily, the Symbol Digit Modalities Test (SDMT).

METHODS: This is a secondary analysis of data from 1,022 of 1,741 participants of the National Institute of Neurological Disorders and Stroke Exploratory Clinical Trials in Parkinson Disease Long-Term Study 1, a randomized, placebo-controlled trial of creatine. Participants were categorized as having or not having metabolic syndrome on the basis of modified criteria from the National Cholesterol Education Program Adult Treatment Panel III. Those who had the same metabolic syndrome status at consecutive annual visits …

Tnf-Α Promotes Nuclear Enrichment Of The Transcription Factor Tonebp/Nfat5 To Selectively Control Inflammatory But Not Osmoregulatory Responses In Nucleus Pulposus Cells., Zariel I. Johnson, Alexandra C. Doolittle, Joseph W. Snuggs, Irving M. Shapiro, Christine L. Le Maitre, Makarand V. Risbud

Department of Orthopaedic Surgery Faculty Papers

Intervertebral disc degeneration (IDD) causes chronic back pain and is linked to production of proinflammatory molecules by nucleus pulposus (NP) and other disc cells. Activation of tonicity-responsive enhancer-binding protein (TonEBP)/NFAT5 by non-osmotic stimuli, including proinflammatory molecules, occurs in cells involved in immune response. However, whether inflammatory stimuli activate TonEBP in NP cells and whether TonEBP controls inflammation during IDD is unknown. We show that TNF-α, but not IL-1β or LPS, promoted nuclear enrichment of TonEBP protein. However, TNF-α-mediated activation of TonEBP did not cause induction of osmoregulatory genes. RNA sequencing showed that 8.5% of TNF-α transcriptional responses were TonEBP-dependent and …

Intravesical Rad-Ifnα/Syn3 For Patients With High-Grade, Bacillus Calmette-Guerin-Refractory Or Relapsed Non-Muscle-Invasive Bladder Cancer: A Phase Ii Randomized Study., Neal D Shore, Stephen A Boorjian, Daniel J Canter, Kenneth Ogan, Lawrence I Karsh, Tracy M Downs, Leonard G Gomella, Ashish M Kamat, Yair Lotan, Robert S Svatek, Trinity J Bivalacqua, Robert L Grubb, Tracey L Krupski, Seth P Lerner, Michael E Woods, Brant A Inman, Matthew I Milowsky, Alan Boyd, F Peter Treasure, Gillian Gregory, David G Sawutz, Seppo Yla-Herttuala, Nigel R Parker, Colin P N Dinney

Department of Medicine Faculty Papers

Purpose Many patients with high-risk non-muscle-invasive bladder cancer (NMIBC) are either refractory to bacillus Calmette-Guerin (BCG) treatment or may experience disease relapse. We assessed the efficacy and safety of recombinant adenovirus interferon alfa with Syn3 (rAd-IFNα/Syn3), a replication-deficient recombinant adenovirus gene transfer vector, for patients with high-grade (HG) BCG-refractory or relapsed NMIBC. Methods In this open-label, multicenter (n = 13), parallel-arm, phase II study ( ClinicalTrials.gov identifier: NCT01687244), 43 patients with HG BCG-refractory or relapsed NMIBC received intravesical rAd-IFNα/Syn3 (randomly assigned 1:1 to 1 × 10(11) viral particles (vp)/mL or 3 × 10(11) vp/mL). Patients who responded at months 3, …

Characterization Of Pulmonary Metastases In Children With Hepatoblastoma Treated On Children's Oncology Group Protocol Ahep0731 (The Treatment Of Children With All Stages Of Hepatoblastoma): A Report From The Children's Oncology Group., Allison F. O'Neill, Alexander J. Towbin, Mark D. Krailo, Caihong Xia, Yun Gao, M. Beth Mccarville, Rebecka L. Meyers, Eugene D. Mcgahren, Greg M. Tiao, Stephen P. Dunn, Max R. Langham, Christopher B. Weldon, Milton J. Finegold, Sarangarajan Ranganathan, Wayne L. Furman, Marcio Malogolowkin, Carlos Rodriguez-Galindo, Howard M. Katzenstein

Department of Pediatrics Faculty Papers

Purpose To determine whether the pattern of lung nodules in children with metastatic hepatoblastoma (HB) correlates with outcome. Methods Thirty-two patients with metastatic HB were enrolled on Children's Oncology Group Protocol AHEP0731 and treated with vincristine and irinotecan (VI). Responders to VI received two additional cycles of VI intermixed with six cycles of cisplatin/fluorouracil/vincristine/doxorubicin (C5VD), and nonresponders received six cycles of C5VD alone. Patients were imaged after every two cycles and at the conclusion of therapy. All computed tomography scans and pathology reports were centrally reviewed, and information was collected regarding lung nodule number, size, laterality, timing of resolution, and …

Effect Of Oral Semaglutide Compared With Placebo And Subcutaneous Semaglutide On Glycemic Control In Patients With Type 2 Diabetes: A Randomized Clinical Trial., Melanie Davies, Thomas R. Pieber, Marie-Louise Hartoft-Nielsen, Oluf K.H. Hansen, Serge Jabbour, Julio Rosenstock

Department of Medicine Faculty Papers

Importance: Glucagon-like peptide-1 (GLP-1) receptor agonists are effective therapies for the treatment of type 2 diabetes and are all currently available as an injection.

Objectives: To compare the effects of oral semaglutide with placebo (primary) and open-label subcutaneous semaglutide (secondary) on glycemic control in patients with type 2 diabetes.

Design, Setting, and Patients: Phase 2, randomized, parallel-group, dosage-finding, 26-week trial with 5-week follow-up at 100 sites (hospital clinics, general practices, and clinical research centers) in 14 countries conducted between December 2013 and December 2014. Of 1106 participants assessed, 632 with type 2 diabetes and insufficient glycemic control using diet and …