Medical Specialties Commons^™

The Routine Use And Cost Analysis Of Acid-Fast Bacilli And Fungal Cultures In Foot And Ankle Surgery: A Retrospective Study, Zein S. El-Zein, Scott Diamond, Margaret C. Bohr, Erin A. Baker, Mackenzie M. Fleischer, Corinn Gehrke, Paul T. Fortin, Zachary Vaupel

Conference Presentation Abstracts

This study highlighted low rates of positive AFB/fungal cultures in our foot and ankle surgery department and demonstrated that routine use of such cultures poses substantial costs to the hospital.

Modulation Of The Acute Post-Anterior Cruciate Ligament Rupture Intra-Articular Environment Via Recruitment Of Marrow-Derived Stem Cells To Promote Bone Tunnel Healing In Acl Reconstruction, Kevin C. Baker, Samantha Hartner, Mackenzie M. Fleischer, Michael D. Newton, William B. Acker, Perry Altman, Christopher Bush, Tristan Maerz, Asheesh Bedi

Conference Presentation Abstracts

Acute, post-injury peripheral blood mobilization therapy with a CXCR4 antagonist improved bone tunnel healing and graft integration following ACL reconstruction surgery in a rat model.

Diagnosing Total Joint Arthroplasty-Associated Periprosthetic Joint Infections In Immunocompromised Patient Populations, Philip Zakko, Erin A. Baker, Christian M. Huber, Corinn Gehrke, Brett J. Friedman, Michael A. Flierl

Conference Presentation Abstracts

The 2018 ICM/MSIS classification system was used to assess PJI diagnosis in immunocompromised vs. non-immunocompromised patient populations to determine applicability of the scoring criteria.

A Phase 1b/2a Study Of Soticlestat As Adjunctive Therapy In Participants With Developmental And/Or Epileptic Encephalopathies., Jonathan J. Halford, Michael R. Sperling, Dimitrios Arkilo, Mahnaz Asgharnejad, Celia Zinger, Rengyi Xu, Matthew During, Jacqueline A. French

Department of Neurology Faculty Papers

OBJECTIVE: To evaluate the safety, tolerability, and pharmacokinetics of soticlestat, a first-in-class cholesterol 24-hydroxylase inhibitor, in adults with developmental and/or epileptic encephalopathies (DEE).

METHODS: The study comprised a 30-day, randomized, double-blind, placebo-controlled phase (Part A), followed by a 55-day open-label phase (Part B) (ClinicalTrials.gov ID: NCT03166215) . In Part A, patients with DEE and at least one bilateral motor seizure during the 4-week prospective baseline period were randomized 4:1 to receive soticlestat or placebo, in addition to their usual antiseizure medication. In Part B, all patients received open-label soticlestat. Soticlestat doses were titrated according to tolerability to a maximum of …

Pedsql™ Gastroparesis Symptoms Module Domain And Item Development: Qualitative Methods, James W Varni, Robert J Shulman, Mariella M Self, Liz Febo-Rodriguez, Heather Charron, Kent Williams, Sam Nurko, Rachel L Rosen, Bruno P Chumpitazi

Children’s Nutrition Research Center Staff Publications

Objectives: Like adults, children suffer from gastroparesis; however, there are currently no validated instruments to determine the impact of gastroparesis in pediatric patients. The objective of this study was to develop the items and domains to support the content validity of the new Pediatric Quality of Life Inventory (PedsQL™) Gastroparesis Symptoms Module.

Methods: Patients were recruited from the National Institute of Diabetes and Digestive and Kidney Diseases Pediatric Gastroparesis Registry. The qualitative methods involved an iterative process comprising a literature review of existing questionnaires and gastroparesis clinical research, an expert review panel of pediatric gastroenterologists who provided feedback on the …

Recommendations For The Use Of The Veggie Meter® For Spectroscopy-Based Skin Carotenoid Measurements In The Research Setting, Marcela D Radtke, Mykaela Poe, Jodi Stookey, Stephanie Jilcott Pitts, Nancy E Moran, Matthew J Landry, Lewis P Rubin, Virginia C Stage, Rachel E Scherr

Children’s Nutrition Research Center Staff Publications

Carotenoids are a class of phytochemical compounds found in a variety of fruits and vegetables (F/V) and, therefore, are commonly used as a biomarker for F/V intake. The Veggie Meter® is a noninvasive research-grade instrument that detects and quantifies carotenoids in the skin. To determine current practices and examine variability among users, a survey was administered to researchers using the device (n = 19, response rate = 35.8%) and variation in anatomical site preparation, calibration, number of measurements, measurement site, and documentation was observed. A protocol was developed in partnership with Veggie Meter® users to outline the preparation, calibration, …

Digital Literacy At An Urban Cancer Center: Implications For Technology Use And Vulnerable Patients, Amy Leader, Lisa M. Capparella, L. Waldman, Ba, Rebecca Cammy, Alison Petok, Rebecca Dean, Ayako Shimada, Liana Yocavitch, Kristin L. Rising, Gregory Garber, Brooke Worster, Adam Dicker

Kimmel Cancer Center Faculty Papers

PURPOSE: eHealth literacy, or the ability to seek, find, understand, and appraise health information from electronic sources, has become increasingly relevant in the era of COVID-19, when so many aspects of patient care became dependent on technology. We aimed to understand eHealth literacy among a diverse sample of patients with cancer and discuss ways for health systems and cancer centers to ensure that all patients have access to high-quality care.

METHODS: A cross-sectional survey of patients with cancer and caregivers was conducted at an NCI-designated cancer center to assess access to the Internet, smartphone ownership, use of mobile apps, willingness …

Comparison Of Brachiobasilic Transposition Arteriovenous Fistula Outcomes Among Diabetic And Non-Diabetic Patients: A Case Controlled Study, Zia Ur Rehman, Waryam Muhammad Saleh, Anum Sadruddin Pidani

Department of Surgery Docs

Diabetes is considered a risk factor for arteriovenous fistula failure and increased perioperative complications; but this view is not proven for patients undergoing brachiobasilic transposition arteriovenous fistula (BBT-AVF). Fifty-one (68.9%) diabetic and 23 (31.08%) non-diabetic patients undergoing single-stage BBT-AVF were compared in terms of perioperative complications, access maturation and patency rates at The Aga Khan University Hospital from between January 2016 to December 2017. Diabetics were elder and more obese. The perioperative complications 19 (37.2%) vs. 8 (34.7%), and access maturation rates were compared (93.2% vs. 95.5%) between the two groups. At 6 months, access patency in diabetics was lower …

“Old People Problems”, Uncertainty And Legitimacy: Challenges With Diagnosing Parkinson's Disease In Kenya, Natasha Fothergill-Misbah, Richard Walker, Judith Kwasa, Juzar Hooker, Kate Hampshire

Internal Medicine, East Africa

Very little is known about the experience of people living with Parkinson's disease (PD) in low- and middle-income countries, such as those in sub-Saharan Africa. The number of specialists in the region is low and awareness is limited among the population and healthcare professionals. Drawing on ten months of ethnographic fieldwork in urban and rural Kenya with 55 people living with PD (PwP), 23 family members and 22 healthcare professionals from public and private clinics, we set out to understand the experience of diagnosis among PwP in Kenya. The diagnostic journeys of our study participants were typically long, convoluted and …

Integrating Telepsychiatry Services In A Care Setting In Kenya: A Case Report, Frank Njenga, Linnet Ongeri, Anna Nguithi, Jacqueline Anundo, Maryann Mugane, Zawadi Kimari, Gathoni Mbugua, Cushny Kaigwa, Lukoye Atwoli

Internal Medicine, East Africa

Globally, health providers and patients alike have been forced to adapt rapidly to the use of telemedicine during the COVID-19 pandemic. Although telepsychiatry has been tested and found just as effective as face-to-face care, there still remains little uptake of this form of care provision in sub-Saharan Africa. This case study highlights the use of telepsychiatry in a previously telemedicine-naive private mental health facility in Kenya. We describe the challenges and lessons learned from the experience. We conclude on the need to test the effectiveness and acceptability of this mode of therapy in sub-Saharan Africa.

Differences In Socio-Demographic And Risk Factor Profile, Clinical Presentation, And Outcomes Between Patients With And Without Rhd Heart Failure In Sub-Saharan Africa: Results From The Thesus-Hf Registry, Clovis Nkoke, Albertino Damasceno, Christopher Edwards, Beth Davison, Gad Cotter, Mahmoud Sani, Lauren Gaeta, Okechukwu S. Ogah, Charles Mondo, Gerald Yonga

Internal Medicine, East Africa

Background: Rheumatic heart disease (RHD) was found in the THESUS-HF registry to be the third most common cause of acute heart failure (AHF) in Sub-Saharan Africa.

Methods: One thousand six patients with AHF from 9 Sub-Saharan African countries were recruited in THESUS-HF, of which 143 (14.3%) had RHD-AHF. Clinical characteristics and outcomes in patients with RHD-AHF and non-RHD-AHF were compared. Kaplan-Meier plots for time to all-cause death and/or HF readmission according to the presence of RHD-AHF and non-RHD-AHF were performed and survival distributions compared using the log-rank test. Cox regression was used to determine the hazard ratio of …

Smart Watch Detection Of Supraventricular Tachycardia (Svt): First Case From Tanzania, Nadeem Kassam, Omar Aziz, Eric Aghan, Samina Somji, Hanifa Mbithe, Mustaafa Bapumia, Robert Mvungi, Salim Surani

Internal Medicine, East Africa

Smartwatches like the Apple Watch have been on the rise worldwide and their use is gaining popularity in developing countries. Their ability to detect dysrhythmias is well documented. Present practice discourages the use of these devices as a diagnostic tool. Nevertheless, atypical findings from these devices should be clinically investigated. This case demonstrates an eventual diagnosis of supraventricular tachycardia (SVT) based on an Apple watch alert which was subsequently confirmed by electrophysiological evaluation.

Factors Associated With Mortality Among Hospitalized Adults With Covid-19 Pneumonia At A Private Tertiary Hospital In Tanzania: A Retrospective Cohort Study, Nadeem Kassam, Eric Aghan, Omar Aziz, Hanifa Mbithe, Kamran Hamid, Reena Shah, Salim Surani, James Orwa, Samina Somji

Internal Medicine, East Africa

Background: The emergence of the novel coronavirus disease 2019 (COVID-19) has caused millions of deaths worldwide. There has been paucity of data for hospitalized African patients suffering from COVID-19. This study aimed to identify factors associated with in-hospital mortality in patients suffering from COVID-19 in Tanzania.

Methods: This was a single center, retrospective, observational cohort study in adult patients hospitalized with confirmed COVID-19 infection. Demographics, clinical pattern, laboratory and radiological investigations associated with increased odds of mortality were analyzed.

Results: Of the 157 patients, 107 (68.1%) patients survived and 50 (31.8%) died. Mortality was highest in patients …

Lipocalin 2 Promotes Inflammatory Breast Cancer Tumorigenesis And Skin Invasion, Emilly S. Villodre, Xiaoding Hu, Richard Larson, Pascal Finetti, Kristen Gomez, Wintana Balema, Shane R. Stecklein, Ginette Santiago-Sanchez, Savitri Krishnamurthy, Juhee Song

Health & Biomedical Sciences Faculty Publications

Inflammatory breast cancer (IBC) is an aggressive form of primary breast cancer characterized by rapid onset and high risk of metastasis and poor clinical outcomes. The biological basis for the aggressiveness of IBC is still not well understood and no IBC-specific targeted therapies exist. In this study, we report that lipocalin 2 (LCN2), a small secreted glycoprotein belonging to the lipocalin superfamily, is expressed at significantly higher levels in IBC vs non-IBC tumors, independently of molecular subtype. LCN2 levels were also significantly higher in IBC cell lines and in their culture media than in non-IBC cell lines. High expression was …

Automation Of Radiation Treatment Planning For Cervical Cancer, Dong Joo Rhee

Dissertations and Theses (Open Access)

Cervical cancer is one of the most common cancer in low- and middle-income countries (LMICs). The mortality rate can be reduced if radiation treatment becomes widely available. However, due to the lack of radiation treatment facilities and human resources, many cervical cancer patients in Africa are not able to receive timely treatments or advanced therapies. To increase the availability of radiation treatment in low-and middle-income countries (LMICs) including African countries, many attempts have been made to reduce the cost of medical linear accelerators. However, increasing the number of treatment machines would not instantly resolve the issues, as there would be …

Quantitative Magnetic Resonance Imaging For The Early Prediction Of Treatment Response In Triple Negative Breast Cancer, Benjamin C. Musall

Dissertations and Theses (Open Access)

Triple Negative Breast Cancer (TNBC) is an aggressive subtype of breast cancer which lacks upregulated hormone receptors. Because of this, it is not vulnerable to clinically available targeted therapies. When treated with standard of care neoadjuvant systemic therapy (NAST), TNBC only shows approximately a 40% rate of pathologic complete response (pCR). A biomarker which could predict TNBC response to NAST early during treatment would be useful, as it would allow for non-responders to be triaged to alternative therapies and potentially allow for the treatment of responders to be de-escalated.

Quantitative Magnetic Resonance Imaging (MRI) may be used to probe and …

Advancement Of A 3d Computational Phantom And Its Age Scaling Methodologies For Retrospective Dose Reconstruction Studies, Aashish Gupta

Dissertations and Theses (Open Access)

We have used a 3D age-scalable computational phantom for over two decades for retrospective dose reconstruction studies of childhood cancer survivors (CCS) treated with 2D historic radiotherapy (RT). However, our phantom and its age scaling functions (ASF) must be updated so that it can be used in studies that include survivors treated with contemporary RT. We aimed to implement our phantom and its age scaling functions in DICOM format and determine the feasibility of applying our ASFs to accurately scale the whole-body CT-based anatomies.

In the implementation study, we developed Python scripts that model the phantom and ASFs in a …

The Role Of Ifitm3 In The Immune Response Of Brca-Deficient High Grade Serous Ovarian Carcinoma, Han Cun

Dissertations and Theses (Open Access)

Background: Prior studies showed that BRCA-deficient high grade serous ovarian carcinoma (HGSOC) had increased tumor infiltrating lymphocytes (TILs) compared to BRCA-wildtype (WT). To better understand the underlying immune mechanism in these tumors, a preliminary transcriptome analysis was performed on a set of microdissected HGSOC tumor specimens with BRCA1-mutation, BRCA2-mutation, or WT. This demonstrated an upregulation of IFITM3, an essential gene in modulating immune function. Based on these findings, we hypothesized that BRCA-deficient HGSOC have increased DNA damage leading to upregulation of IFITM3 and subsequent increase in antigen presentation and T-cell activation.

Methods: Following IRB approval, preliminary transcriptome analysis was performed …

Genome-Wide Association Study Of Serum Metabolites In The African American Study Of Kidney Disease And Hypertension, Shengyuan Luo, Elena V Feofanova, Adrienne Tin, Sarah Tung, Eugene P Rhee, Josef Coresh, Dan E Arking, Aditya Surapaneni, Pascal Schlosser, Yong Li, Anna Köttgen, Bing Yu, Morgan E Grams

Faculty, Staff and Student Publications

The genome-wide association study (GWAS) is a powerful means to study genetic determinants of disease traits and generate insights into disease pathophysiology. to date, few GWAS of circulating metabolite levels have been performed in African Americans with chronic kidney disease. Hypothesizing that novel genetic-metabolite associations may be identified in a unique population of African Americans with a lower glomerular filtration rate (GFR), we conducted a GWAS of 652 serum metabolites in 619 participants (mean measured glomerular filtration rate 45 mL/min/1.73m

Management Of Peritonsillar Abscess Within A Local Emergency Department: A Quality Analysis Study., Briana K Ortega, Spencer Short, Bryan G. Kane Md, Robert M. Dedio Md

Department of Emergency Medicine

OBJECTIVE: Peritonsillar abscess (PTA) is the most common deep space infection of the head and neck, affecting thousands of people annually with high treatment costs. The purpose of this project was to determine how in-network emergency departments (EDs) adhere to generally accepted guidelines regarding diagnosis and management of potential PTAs.

METHODS: The authors performed a retrospective chart review to identify patients with PTA in five EDs in one year. Information pertaining to diagnostic tests, treatment, and airway status was also collected. Descriptive analysis was used to assess if EDs were consistent with generally accepted guidelines.

RESULTS: Six hundred twenty-one patient …

Gemcitabine-Loaded Microbubble System For Ultrasound Imaging And Therapy., Lauren J. Delaney, John R. Eisenbrey, David Brown, Jonathan R Brody, Masaya Jimbo, Brian E Oeffinger, Maria Stanczak, Flemming Forsberg, Ji-Bin Liu, Margaret A Wheatley

Department of Radiology Faculty Papers

Ultrasound imaging presents many positive attributes, including safety, real-time imaging, universal accessibility, and cost. However, inherent difficulties in discrimination between soft tissues and tumors prompted development of stabilized microbubble contrast agents. This presents the opportunity to develop agents in which drug is entrapped in the microbubble shell. We describe preparation and characterization of theranostic poly(lactide) (PLA) and pegylated PLA (PEG-PLA) shelled microbubbles that entrap gemcitabine, a commonly used drug for pancreatic cancer (PDAC). Entrapping 6 wt% gemcitabine did not significantly affect drug activity, microbubble morphology, or ultrasound contrast activity compared with unmodified microbubbles. In vitro microbubble concentrations yielding ≥ 500nM …

Institutional Factors In General Surgery Resident Wellness And Burnout: Identification Via A Modified Delphi Technique, Darci C. Foote, Gurjit Sandhu, Kathryn Ziegler, James Lau

Conference Presentation Abstracts

Background: While there has been significant focus on individual and programmatic factors impacting general surgical residents wellbeing and burnout, the effect of the greater institutional macrocosm has not been well studied. Our work seeks to identify organizational factors that may be implicated in resident wellness. Summary of Work: Using a modified Delphi technique, a survey structured around six domains of the Maslach Burnout Theory (workload, control, reward, community, fairness, values) was distributed to wellness stakeholders at two institutions. Participants identified factors that may be implicated in resident wellness and burnout. Two subsequent multiple-choice survey iterations were distributed to stakeholders who …

Treatment And Outcomes Of Soft Tissue Sarcoma Of Groin, Hip And Thigh: A Retrospective Review From A Tertiary Care Hospital, Masood Umer, Javeria Saeed, Zaid Ahmed Shamsi, Muhammad Usman Tariq

Section of Orthopaedic Surgery

Objective: To study the frequency of the thigh, hip and groin soft tissue sarcomas and retrospectively analyse the management, treatment results, and outcomes of these uncommon malignant tumours, in a tertiary care hospital of the city of Karachi.
Methodology: Data of soft tissue tumours registered from 2017-2018 was retrieved during January 2019 to March 2019 from Aga Khan University Hospital, Karachi bone and soft tissue tumour registry. A retrospective review was performed and all soft tissue tumour cases treated with surgical intervention (with adjuvant /neoadjuvant therapy) or palliative intention were included.
Results: Total 119 cases of soft tissue tumours (STS) …

Prevention Of Falls In Hospital: Audit Report From A Tertiary Care Hospital Of Pakistan, Anum Sadruddin Pidani, Tashfeen Ahmad, Nasreen Panjwani, Shahryar Noordin

Department of Surgery Docs

Objective: The study aims to assess the fall incidents in past 5 years and fall assessment practices at the Aga Khan University Hospital, Karachi.
Methods: We performed a single-center retrospective audit at Aga Khan University Hospital from October 2019 to December 2019. A list of all patients admitted to Aga Khan University Hospital under the Musculoskeletal and Sports Medicine Service Line was obtained using the Hospital Information Management System (HIMS) from Jan 2017 to June 2018. Data including fall assessment scores was collected retrospectively from medical record files.
Results: A total of 1499 patients were admitted during this time period, …

Concurrent Primary Cardiac Tumors In A High-Risk Patient Presenting With Tamponade., Jay V Gopal, Lauren Crowley, Shawn M Quinn, Timothy S Misselbeck, Joseph B Zackary

Department of Emergency Medicine

Primary cardiac tumors are rare, particularly in the elderly population. The patient described in this report presented with symptoms of dyspnea on exertion, leg swelling, and weight gain and was found to have two histologically distinct cardiac masses: atrial myxoma with concurrent aortic fibroelastoma. Given her history of cirrhosis and end-stage renal disease, the patient was a poor surgical candidate but opted for excision of both masses. The patient eventually succumbed to her cirrhosis six weeks after presentation. In this report, we advocate for further research into medical management for the unique presentation of concurrent primary cardiac tumors in high-operative-risk …

Synchronous Primary Diffuse Large B-Cell Lymphoma And Papillary Thyroid Carcinoma: A Case Report Highlighting "Lymphoma First Approach", Rahim Dhanani, Ambreen Abdullah Unar, Muhammad Hassan Danish, Hamdan Ahmed Pasha, Ummiya Tahir, Mubasher Ikram

Section of Otolaryngology, Head & Neck Surgery

Papillary thyroid carcinoma (PTC) is the most common type of all thyroid carcinomas and accounts for up to 90% of all thyroid malignancies. It is the most indolent form of the disease and has an excellent prognosis. On the other hand, Primary thyroid lymphoma (PTL) is a rare entity accounting for only about 1% to 5% of all thyroid malignancies and only 2% of extranodal lymphomas. Synchronous PTC and PTL is a very rare condition and only a few cases have been reported in the literature up till now. We report one such case in a 52-year-old lady who was …

Pooled Safety Analysis Of Tisagenlecleucel In Children And Young Adults With B Cell Acute Lymphoblastic Leukemia., John E. Levine, Stephan A. Grupp, Michael A. Pulsipher, Andrew C. Dietz, Susana Rives, Douglas Myers, Keith August, Michael R. Verneris, Jochen Buechner, Theodore W. Laetsch, Henrique Bittencourt, Andre Baruchel, Michael W. Boyer, Barbara De Moerloose, Muna Qayed, Stella M. Davies, Christine L. Phillips, Timothy A. Driscoll, Peter Bader, Krysta Schlis, Patricia A. Wood, Rajen Mody, Lan Yi, Mimi Leung, Lamis K. Eldjerou, Carl H. June, Shannon L. Maude

Manuscripts, Articles, Book Chapters and Other Papers

Background: Tisagenlecleucel, an anti-CD19 chimeric antigen receptor T cell therapy, has demonstrated efficacy in children and young adults with relapsed/refractory B cell acute lymphoblastic leukemia (B-ALL) in two multicenter phase 2 trials (ClinicalTrials.gov, NCT02435849 (ELIANA) and NCT02228096 (ENSIGN)), leading to commercialization of tisagenlecleucel for the treatment of patients up to age 25 years with B-ALL that is refractory or in second or greater relapse.

Methods: A pooled analysis of 137 patients from these trials (ELIANA: n=79; ENSIGN: n=58) was performed to provide a comprehensive safety profile for tisagenlecleucel.

Results: Grade 3/4 tisagenlecleucel-related adverse events (AEs) were reported in 77% of …

Impact Of Genotype, Serum Bile Acids, And Surgical Biliary Diversion On Native Liver Survival In Fic1 Deficiency., Daan B E Van Wessel, Richard J. Thompson, Emmanuel Gonzales, Irena Jankowska, Benjamin L. Shneider, Etienne Sokal, Tassos Grammatikopoulos, Agustina Kadaristiana, Emmanuel Jacquemin, Anne Spraul, Patryk Lipiński, Piotr Czubkowski, Nathalie Rock, Mohammad Shagrani, Dieter Broering, Talal Algoufi, Nejat Mazhar, Emanuele Nicastro, Deirdre Kelly, Gabriella Nebbia, Henrik Arnell, Björn Fischler, Jan B F Hulscher, Daniele Serranti, Cigdem Arikan, Dominique Debray, Florence Lacaille, Cristina Goncalves, Loreto Hierro, Gema Muñoz Bartolo, Yael Mozer-Glassberg, Amer Azaz, Jernej Brecelj, Antal Dezsőfi, Pier Luigi Calvo, Dorothee Krebs-Schmitt, Steffen Hartleif, Wendy L. Van Der Woerd, Jian-She Wang, Li-Ting Li, Özlem Durmaz, Nanda Kerkar, Marianne Hørby Jørgensen, Ryan T. Fischer, Carolina Jimenez-Rivera, Seema Alam, Mara Cananzi, Noémie Laverdure, Cristina Targa Ferreira, Felipe Ordonez, Heng Wang, Valerie Sency, Kyung Mo Kim, Huey-Ling Chen, Elisa Carvalho, Alexandre Fabre, Jesus Quintero Bernabeu, Estella M. Alonso, Ronald J. Sokol, Frederick J. Suchy, Kathleen M. Loomes, Patrick J. Mckiernan, Philip Rosenthal, Yumirle Turmelle, Girish S. Rao, Simon Horslen, Binita M. Kamath, Maria Rogalidou, Wikrom W. Karnsakul, Bettina Hansen, Henkjan J. Verkade, Natural Course And Prognosis Of Pfic And Effect Of Biliary Diversion Consortium

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND AND AIMS: Mutations in ATPase phospholipid transporting 8B1 (ATP8B1) can lead to familial intrahepatic cholestasis type 1 (FIC1) deficiency, or progressive familial intrahepatic cholestasis type 1. The rarity of FIC1 deficiency has largely prevented a detailed analysis of its natural history, effects of predicted protein truncating mutations (PPTMs), and possible associations of serum bile acid (sBA) concentrations and surgical biliary diversion (SBD) with long-term outcome. We aimed to provide insights by using the largest genetically defined cohort of patients with FIC1 deficiency to date.

APPROACH AND RESULTS: This multicenter, combined retrospective and prospective study included 130 patients with compound …

Association Of New And/Or Progressive Multiple Organ Dysfunction Syndrome With Mortality And New Morbidity Among Children Encountering Septic Shock, Rachel L. Waworuntu

Capstone Experience: Master of Public Health

Despite reductions in sepsis mortality, there remains a substantial morbidity burden among children who survive sepsis. Furthermore, the majority develop multiple organ ‘group has historically been analyzed as a single cohort. Using the Life After Pediatric Sepsis Evaluation (LAPSE) investigation database, we studied the association of new and progressive multiple organ dysfunction syndrome (NPMODS) with morbidity and mortality among children with septic shock. We hypothesized that NPMODS represents a distinct phenotype based on evolving organ dysfunction. There was evidence of an association but it did not reach the 0.05 level of significance. We also hypothesized and confirmed that children with …

Esr And Crp Testing May Not Be Useful In Monitoring Patients With Native Vertebral Osteomyelitis, Abigail Heilenman, Amy Slenker Md

Research Scholars

No abstract provided.