Medical Specialties Commons^™

Use Of Befasstt Acronym To Improve Brainstem Stroke Recognition - A Case Study, Marietta Sperry, Amber Wilson

Articles, Abstracts, and Reports

Purpose: To increase recognition of lower brain stem strokes through the use of the BeFASSTT acronym to improve patient outcomes.

Impact Of Integrating A Neonatal Early-Onset Sepsis Risk Calculator Into The Electronic Health Record., Nyles T Fowler, Michael Garcia, Cynthia Hankins

Articles, Abstracts, and Reports

Investigators from Kaiser Permanente developed a risk-assessment calculator as a tool for evaluation of early-onset sepsis (EOS) to narrow antibiotic use for the treatment of EOS. The integration of the EOS risk calculator into an electronic health record will minimize manual calculations and data entry and improve compliance and accuracy through automation.

Methods: We performed a retrospective chart review for neonates ≥34 weeks and 0 days gestational age. We collected data pre-integration and post-integration of the EOS risk calculator. The primary outcome measure is the accuracy of user input into the calculator. Secondary outcomes include compliance with using the EOS …

Precision Medicine In Pancreatic Disease-Knowledge Gaps And Research Opportunities: Summary Of A National Institute Of Diabetes And Digestive And Kidney Diseases Workshop., Mark E Lowe, Dana K Andersen, Richard M Caprioli, Jyoti Choudhary, Zobeida Cruz-Monserrate, Anil K Dasyam, Christopher E Forsmark, Fred S Gorelick, Joe W Gray, Mark Haupt, Kimberly A Kelly, Kenneth P Olive, Sylvia K Plevritis, Noa Rappaport, Holger R Roth, Hanno Steen, S Joshua Swamidass, Temel Tirkes, Aliye Uc, Kirill Veselkov, David C Whitcomb, Aida Habtezion

Articles, Abstracts, and Reports

A workshop on research gaps and opportunities for Precision Medicine in Pancreatic Disease was sponsored by the National Institute of Diabetes and Digestive Kidney Diseases on July 24, 2019, in Pittsburgh. The workshop included an overview lecture on precision medicine in cancer and 4 sessions: (1) general considerations for the application of bioinformatics and artificial intelligence; (2) omics, the combination of risk factors and biomarkers; (3) precision imaging; and (4) gaps, barriers, and needs to move from precision to personalized medicine for pancreatic disease. Current precision medicine approaches and tools were reviewed, and participants identified knowledge gaps and research needs …

Biological Intratumoral Therapy For The High-Grade Glioma Part I: Intratumoral Delivery And Immunotoxins., Joshua Loya, Charlie Zhang, Emily J Cox, Achal Singh Achrol, Santosh Kesari

Articles, Abstracts, and Reports

Management of high-grade gliomas remains a complex challenge. Standard of care consists of microsurgical resection, chemotherapy and radiation, but despite these aggressive multimodality therapies the overall prognosis remains poor. A major focus of ongoing translational research studies is to develop novel therapeutic strategies that can maximize tumor cell eradication while minimizing collateral side effects. Particularly, biological intratumoral therapies have been the focus of new translational research efforts due to their inherent potential to be both dynamically adaptive and target specific. This two-part review will provide an overview of biological intratumoral therapies and summarize key advances and remaining challenges in intratumoral …

Targeting Dna Repair In Metastatic Castration-Resistant Prostate Cancer (Mcrpc): Genomic Screening For A Clinical Trial Of Rucaparib, Alan Bryce, Mitchell E. Gross, Charles Redfern, Nicholas Vogelzang, Matthew Rettig, Thomas Stanton, Julie N. Graff, Sandhya Srinivas, Andrea Loehr, Wassim Abida

Articles, Abstracts, and Reports

Objectives: The high prevalence of men with mCRPC carrying pathogenic mutations in DNA damage repair (DDR) genes may have implications for clinical treatment, as poly(ADP-ribose) polymerase (PARP) inhibitors, such as rucaparib, have shown preliminary evidence of activity in these patients. The ongoing phase 2 TRITON2 study (NCT02952534) is evaluating rucaparib in mCRPC patients harboring a deleterious germline or somatic mutation in BRCA1, BRCA2, ATM, or other DDR gene. Here we present results from genomic screening of tissue and plasma samples from mCRPC patients.

Methods: Comprehensive genomic profiling was performed by Foundation Medicine, Inc., using FFPE tumor tissue …

Biological Intratumoral Therapy For The High-Grade Glioma Part Ii: Vector- And Cell-Based Therapies And Radioimmunotherapy., Joshua Loya, Charlie Zhang, Emily J Cox, Achal Singh Achrol, Santosh Kesari

Articles, Abstracts, and Reports

Management of high-grade gliomas (HGGs) remains a complex challenge with an overall poor prognosis despite aggressive multimodal treatment. New translational research has focused on maximizing tumor cell eradication through improved tumor cell targeting while minimizing collateral systemic side effects. In particular, biological intratumoral therapies have been the focus of novel translational research efforts due to their inherent potential to be both dynamically adaptive and target specific. This two part review will provide an overview of biological intratumoral therapies that have been evaluated in human clinical trials in HGGs, and summarize key advances and remaining challenges in the development of these …

Characterization Of Patients With Both Alcoholic And Nonalcoholic Fatty Liver Disease In A Large United States Cohort., George Khoudari, Amandeep Singh, Mazen Noureddin, Danielle Fritze, Rocio Lopez, Imad Asaad, Eric Lawitz, Fred Poordad, Kris V Kowdley, Naim Alkhouri

Articles, Abstracts, and Reports

BACKGROUND: Nonalcoholic fatty liver disease (NAFLD) is the hepatic manifestation of the metabolic syndrome (MetS) and is characterized by steatosis in the absence of significant alcohol consumption. However, MetS and significant alcohol intake coexist in certain individuals which may lead to the development of BAFLD.

AIM: To assess the clinical characteristics of patients with both alcoholic and NAFLD (BAFLD) in a large cohort in the United States.

METHODS: Adults from the National Health and Nutrition Examination Survey between 2003-2014 were included. NAFLD was diagnosed based on elevated alanine aminotransferase (ALT) and being overweight or obese in the absence of other …

Tct-804 Outcomes Of Trans-Carotid Tavr In A High-Volume Center, Bm Jones, Ethan C Korngold, Robert W. Hodson, Shih-Ting Chiu, V Kumar, Eric B Kirker

Articles, Abstracts, and Reports

Background

Although the preferred route for transcatheter aortic valve replacement is through the femoral artery, alternatives remain necessary for patients with obstructive iliofemoral disease. Our valve team has developed a large experience using the carotid artery as a primary alternative vascular access approach for transcatheter aortic valve replacement (TAVR). We aim to compare short-term outcomes by access route in a single-center, high-volume, transcarotid (TC) TAVR program.

Methods

All patients undergoing TAVR between September 2012 and September 2018 were included in the study. Baseline demographics and outcomes were obtained from data our institution submitted in compliance with TVT (Transcatheter Valve Therapy) …

Association Of Histologic Disease Activity With Progression Of Nonalcoholic Fatty Liver Disease., David E Kleiner, Elizabeth M Brunt, Laura A Wilson, Cynthia Behling, Cynthia Guy, Melissa Contos, Oscar Cummings, Matthew Yeh, Ryan Gill, Naga Chalasani, Brent A Neuschwander-Tetri, Anna Mae Diehl, Srinivasan Dasarathy, Norah Terrault, Kris V Kowdley, Rohit Loomba, Patricia Belt, James Tonascia, Joel E Lavine, Arun J Sanyal

Articles, Abstracts, and Reports

Importance: The histologic evolution of the full spectrum of nonalcoholic fatty liver disease (NAFLD) and factors associated with progression or regression remain to be definitively established.

Objective: To evaluate the histologic evolution of NAFLD and the factors associated with changes in disease severity over time.

Design, Setting, and Participants: A prospective cohort substudy from the Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) NAFLD Database study, a noninterventional registry, was performed at 8 university medical research centers. Masked assessment of liver histologic specimens was performed, using a prespecified protocol to score individual biopsies. Participants included 446 adults with NAFLD enrolled in …

Oregon Region Standard Stroke Neurological Assessment Utilizing Aacn Synergy Model, Natalie Swearingen, Kailey Cox, Diane Clark

Articles, Abstracts, and Reports

No abstract provided.

Mortality Assessment Of Paclitaxel-Coated Balloons: Patient-Level Meta-Analysis Of The Illumenate Clinical Program At 3 Years., William A Gray, Michael R Jaff, Sahil A Parikh, Gary M Ansel, Marianne Brodmann, Prakash Krishnan, Mahmood K Razavi, Frank Vermassen, Thomas Zeller, Roseann White, Kenneth Ouriel, Mark A Adelman, Sean P Lyden

Articles, Abstracts, and Reports

BACKGROUND: A recent summary-level meta-analysis comprising randomized, controlled trials (RCTs) of femoropopliteal paclitaxel-coated balloon and stent intervention identified excess late mortality in the paclitaxel-treated patients.

METHODS: We evaluated the safety of the Stellarex drug-coated balloon (DCB) for femoropopliteal artery disease with an independently performed meta-analysis of patient-level data from all patients in the Stellarex femoropopliteal clinical program. To compare mortality after DCB or uncoated percutaneous transluminal angioplasty (PTA), we aggregated data from 2 RCTs comprising 419 patients treated with DCB and 170 patients treated with PTA. In an additional analysis, data were aggregated from 6 poolable Stellarex DCB studies (2 …

Long-Term Safety And Efficacy Of Subcutaneous Pasireotide In Patients With Cushing's Disease: Interim Results From A Long-Term Real-World Evidence Study., Luca Manetti, Timo Deutschbein, Jochen Schopohl, Kevin C J Yuen, Michael Roughton, Ulrike Kriemler-Krahn, Libuse Tauchmanova, Ricardo Maamari, Carla Giordano

Articles, Abstracts, and Reports

PURPOSE: Clinical trials have demonstrated the favorable efficacy/safety profile of pasireotide in patients with Cushing's disease (CD). We report interim long-term results of an ongoing real-world evidence study of subcutaneous pasireotide in patients with CD.

METHODS: Adults with CD receiving pasireotide, initiated before (prior-use) or at study entry (new-use), were monitored for ≤ 3 years during a multicenter observational study ( http://clinicaltrials.gov identifier NCT02310269). Primary objective was to assess long-term safety of pasireotide alone or with other CD therapies.

RESULTS: At the time of this interim analysis, 127 patients had received pasireotide (new-use, n = 31; prior-use, n = 96). …

Evaluation Of Intervention Aimed At Reducing Alteplase Returns Without Compromising Clinical Care In Acute Ischemic Stroke, Natalie Swearingen, Jillian Myers, Alyx Lesko, Lindsay Lucas, Elizabeth Baraban

Articles, Abstracts, and Reports

No abstract provided.

Big Spine Data Put To Good Use: The Nhs Sets An Example., Jens R Chapman, Jeffrey C Wang, Karsten Wiechert

Articles, Abstracts, and Reports

No abstract provided.

Hemostatic Complications Associated With Ventricular Assist Devices., Talal Hilal, James O Mudd, Thomas G Deloughery

Articles, Abstracts, and Reports

Hemostatic complications are common in patients with ventricular assist devices. The pathophysiologic mechanisms that lead to dysregulated hemostasis involve complex interactions between device surface, sheer stress, and blood flow. These factors lead to various manifestations that require a thorough understanding of the interplay among platelets, coagulation factors, and red cells. In this article, we review the pathophysiology of hematologic complications (bleeding, acquired von Willebrand disease, heparin-induced thrombocytopenia, hemolysis, stroke and pump thrombosis), the clinical manifestations, and the management of each. We summarize the evidence available for management of these entities and provide a pragmatic clinical review.

Tct-142 Clinical Characteristics And Outcomes Of Patients Undergoing Transcatheter Aortic Valve Replacement With Sts Prom Of ≤3%, Jackson King, Morgan Black, Ruyun Jin, Gary Grunkemeier, Brydan Curtis, Robert W. Hodson, Michael Ring

Articles, Abstracts, and Reports

Background

Transcatheter aortic valve replacement (TAVR) is the preferred treatment for most patients with aortic stenosis deemed at intermediate or higher risk for surgical aortic valve replacement (SAVR). Determination of the risk of SAVR is mainly based on the Society of Thoracic Surgeons (STS) risk calculator. However, federal regulations permit the heart team latitude to offer TAVR for patients with ≤3% predicted risk of mortality (PROM) whose perceived risk is not adequately accounted for by the STS risk model. Limited data is available on the clinical characteristics and outcomes of these patients

Methods

The study group involved 2,539 patients who …

Publisher Correction: Shared Heritability And Functional Enrichment Across Six Solid Cancers., Xia Jiang, Hilary K Finucane, Fredrick R Schumacher, Stephanie L Schmit, Jonathan P Tyrer, Younghun Han, Kyriaki Michailidou, Corina Lesseur, Karoline B Kuchenbaecker, Joe Dennis, David V Conti, Graham Casey, Mia M Gaudet, Jeroen R Huyghe, Demetrius Albanes, Melinda C Aldrich, Angeline S Andrew, Irene L Andrulis, Hoda Anton-Culver, Antonis C Antoniou, Natalia N Antonenkova, Susanne M Arnold, Kristan J Aronson, Banu K Arun, Elisa V Bandera, Rosa B Barkardottir, Daniel R Barnes, Jyotsna Batra, Matthias W Beckmann, Javier Benitez, Sara Benlloch, Andrew Berchuck, Sonja I Berndt, Heike Bickeböller, Stephanie A Bien, Carl Blomqvist, Stefania Boccia, Natalia V Bogdanova, Stig E Bojesen, Manjeet K Bolla, Hiltrud Brauch, Hermann Brenner, James D Brenton, Mark N Brook, Joan Brunet, Hans Brunnström, Daniel D Buchanan, Barbara Burwinkel, Ralf Butzow, Gabriella Cadoni, Trinidad Caldés, Maria A Caligo, Ian Campbell, Peter T Campbell, Géraldine Cancel-Tassin, Lisa Cannon-Albright, Daniele Campa, Neil Caporaso, André L Carvalho, Andrew T Chan, Jenny Chang-Claude, Stephen J Chanock, Chu Chen, David C Christiani, Kathleen B M Claes, Frank Claessens, Judith Clements, J Margriet Collée, Marcia Cruz Correa, Fergus J Couch, Angela Cox, Julie M Cunningham, Cezary Cybulski, Kamila Czene, Mary B Daly, Anna Defazio, Peter Devilee, Orland Diez, Manuela Gago-Dominguez, Jenny L Donovan, Thilo Dörk, Eric J Duell, Alison M Dunning, Miriam Dwek, Diana M Eccles, Christopher K Edlund, Digna R Velez Edwards, Carolina Ellberg, D Gareth Evans, Peter A Fasching, Robert L Ferris, Triantafillos Liloglou, Jane C Figueiredo, Olivia Fletcher, Renée T Fortner, Florentia Fostira, Silvia Franceschi, Eitan Friedman, Steven J Gallinger, Patricia A Ganz, Judy Garber, José A García-Sáenz, Simon A Gayther, Graham G Giles, Andrew K Godwin, Mark S Goldberg, David E Goldgar, Ellen L Goode, Marc T Goodman, Gary E Goodman, Kjell Grankvist, Mark H Greene, Henrik Gronberg, Jacek Gronwald, Pascal Guénel, Niclas Håkansson, Per Hall, Ute Hamann, Freddie C Hamdy, Robert J Hamilton, Jochen Hampe, Aage Haugen, Florian Heitz, Rolando Herrero, Peter Hillemanns, Michael Hoffmeister, Estrid Høgdall, Yun-Chul Hong, John L Hopper, Richard Houlston, Peter J Hulick, David J Hunter, David G Huntsman, Gregory Idos, Evgeny N Imyanitov, Sue Ann Ingles, Claudine Isaacs, Anna Jakubowska, Paul James, Mark A Jenkins, Mattias Johansson, Mikael Johansson, Esther M John, Amit D Joshi, Radka Kaneva, Beth Y Karlan, Linda E Kelemen, Tabea Kühl, Kay-Tee Khaw, Elza Khusnutdinova, Adam S Kibel, Lambertus A Kiemeney, Jeri Kim, Susanne K Kjaer, Julia A Knight, Manolis Kogevinas, Zsofia Kote-Jarai, Stella Koutros, Vessela N Kristensen, Jolanta Kupryjanczyk, Martin Lacko, Stephan Lam, Diether Lambrechts, Maria Teresa Landi, Philip Lazarus, Nhu D Le, Eunjung Lee, Flavio Lejbkowicz, Heinz-Josef Lenz, Goska Leslie, Davor Lessel, Jenny Lester, Douglas A Levine, Li Li, Christopher I Li, Annika Lindblom, Noralane M Lindor, Geoffrey Liu, Fotios Loupakis, Jan Lubiński, Lovise Maehle, Christiane Maier, Arto Mannermaa, Loic Le Marchand, Sara Margolin, Taymaa May, Lesley Mcguffog, Alfons Meindl, Pooja Middha, Austin Miller, Roger L Milne, Robert J Macinnis, Francesmary Modugno, Marco Montagna, Victor Moreno, Kirsten B Moysich, Lorelei Mucci, Kenneth Muir, Anna Marie Mulligan, Katherine L Nathanson, David E Neal, Andrew R Ness, Susan L Neuhausen, Heli Nevanlinna, Polly A Newcomb, Lisa F Newcomb, Finn Cilius Nielsen, Liene Nikitina-Zake, Børge G Nordestgaard, Robert L Nussbaum, Kenneth Offit, Edith Olah, Ali Amin Al Olama, Olufunmilayo I Olopade, Andrew F Olshan, Håkan Olsson, Ana Osorio, Hardev Pandha, Jong Y Park, Nora Pashayan, Michael T Parsons, Tanja Pejovic, Kathryn L Penney, Wilbert H M Peters, Catherine M Phelan, Amanda I Phipps, Dijana Plaseska-Karanfilska, Miranda Pring, Darya Prokofyeva, Paolo Radice, Kari Stefansson, Susan J Ramus, Leon Raskin, Gad Rennert, Hedy S Rennert, Elizabeth J Van Rensburg, Marjorie J Riggan, Harvey A Risch, Angela Risch, Monique J Roobol, Barry S Rosenstein, Mary Anne Rossing, Kim De Ruyck, Emmanouil Saloustros, Dale P Sandler, Elinor J Sawyer, Matthew B Schabath, Johanna Schleutker, Marjanka K Schmidt, V Wendy Setiawan, Hongbing Shen, Erin M Siegel, Weiva Sieh, Christian F Singer, Martha L Slattery, Karina Dalsgaard Sorensen, Melissa C Southey, Amanda B Spurdle, Janet L Stanford, Victoria L Stevens, Sebastian Stintzing, Jennifer Stone, Karin Sundfeldt, Rebecca Sutphen, Anthony J Swerdlow, Eloiza H Tajara, Catherine M Tangen, Adonina Tardon, Jack A Taylor, M Dawn Teare, Manuel R Teixeira, Mary Beth Terry, Kathryn L Terry, Stephen N Thibodeau, Mads Thomassen, Line Bjørge, Marc Tischkowitz, Amanda E Toland, Diana Torres, Paul A Townsend, Ruth C Travis, Nadine Tung, Shelley S Tworoger, Cornelia M Ulrich, Nawaid Usmani, Celine M Vachon, Els Van Nieuwenhuysen, Ana Vega, Miguel Elías Aguado-Barrera, Qin Wang, Penelope M Webb, Clarice R Weinberg, Stephanie Weinstein, Mark C Weissler, Jeffrey N Weitzel, Catharine M L West, Emily White, Alice S Whittemore, H-Erich Wichmann, Fredrik Wiklund, Robert Winqvist, Alicja Wolk, Penella Woll, Michael Woods, Anna H Wu, Xifeng Wu, Drakoulis Yannoukakos, Wei Zheng, Shanbeh Zienolddiny, Argyrios Ziogas, Kristin K Zorn, Jacqueline M Lane, Richa Saxena, Duncan Thomas, Rayjean J Hung, Brenda Diergaarde, James Mckay, Ulrike Peters, Li Hsu, Montserrat García-Closas, Rosalind A Eeles, Georgia Chenevix-Trench, Paul J Brennan, Christopher A Haiman, Jacques Simard, Douglas F Easton, Stephen B Gruber, Paul D P Pharoah, Alkes L Price, Bogdan Pasaniuc, Christopher I Amos, Peter Kraft, Sara Lindström

Articles, Abstracts, and Reports

An amendment to this paper has been published and can be accessed via a link at the top of the paper.

Comparison Of Lumbar Laminectomy Alone, Lumbar Laminectomy And Fusion, Stand-Alone Anterior Lumbar Interbody Fusion, And Stand-Alone Lateral Lumbar Interbody Fusion For Treatment Of Lumbar Spinal Stenosis: A Review Of The Literature., Manan Shah, Bradley Kolb, Emre Yilmaz, Dia R Halalmeh, Marc D Moisi

Articles, Abstracts, and Reports

Lumbar spinal stenosis is defined as narrowing of the lumbar spinal canal, which causes compression of the spinal cord and nerves. Spinal stenosis can cause leg pain and potentially back pain that can affect the quality of life. Ultimately, surgical decompression is required to alleviate the symptoms. In this review, we first utilize several important studies to compare lumbar laminectomy alone versus lumbar laminectomy and fusion. We also compare the effectiveness of more novel surgical approaches, stand-alone anterior lumbar interbody fusion (ALIF), and stand-alone lateral lumbar interbody fusion (LLIF). These techniques have their own advantages and disadvantages in which many …

Effect Of 3-Dimensional Virtual Reality Models For Surgical Planning Of Robotic-Assisted Partial Nephrectomy On Surgical Outcomes: A Randomized Clinical Trial., Joseph D Shirk, David D Thiel, Eric M Wallen, Jennifer M Linehan, Wesley M White, Ketan K Badani, James R Porter

Articles, Abstracts, and Reports

Importance: Planning complex operations such as robotic-assisted partial nephrectomy requires surgeons to review 2-dimensional computed tomography or magnetic resonance images to understand 3-dimensional (3-D), patient-specific anatomy.

Objective: To determine surgical outcomes for robotic-assisted partial nephrectomy when surgeons reviewed 3-D virtual reality (VR) models during operative planning.

Design, Setting, and Participants: A single-blind randomized clinical trial was performed. Ninety-two patients undergoing robotic-assisted partial nephrectomy performed by 1 of 11 surgeons at 6 large teaching hospitals were prospectively enrolled and randomized. Enrollment and data collection occurred from October 2017 through December 2018, and data analysis was performed from December 2018 through March …

Preliminary Observations On Soluble Programmed Cell Death Protein-1 As A Prognostic And Predictive Biomarker In Patients With Metastatic Melanoma Treated With Patient-Specific Autologous Vaccines., Robert O Dillman, Gabriel I Nistor, Bryce T Mclelland, Candace Hsieh, Aleksandra J Poole, Andrew N Cornforth, Hans S Keirstead

Articles, Abstracts, and Reports

Because of its role as an immune checkpoint, levels of soluble programmed cell death protein-1 (sPD-1) could be useful as a prognostic biomarker or predictive biomarker in cancer patients treated with vaccines. Very low levels of sPD-1 may indicate lack of an existing anti-cancer immune response; very high levels may indicate an active immune response that is suppressed. In between these extremes, a decrease in PD-1 following injections of an anti-cancer vaccine may indicate an enhanced immune response that has not been suppressed. Blood samples obtained during a randomized trial in patients with metastatic melanoma were tested from 22 patients …

Phase 1 Trial Of Fruquintinib In Patients With Advanced Solid Tumors: Results Of The Dose Escalation Phase., A Wang-Gillam, H Park, H Yeckes-Rodin, Thomas Stanton, M Kosmo, S Fan, N Sauter, M Kanie

Articles, Abstracts, and Reports

Background: Fruquintinib (Fruq) is a potent, highly selective, novel vascular endothelial growth factor receptor (VEGFR) -1, -2, and -3 tyrosine kinase inhibitor. In the Phase III FRESCO trial¹ that led to the drug approval in China, Fruq improved the median overall survival in patients with metastatic colorectal cancer (mCRC) in the third line or later setting when compared to placebo (9.3 vs 6.6 months); hazard ratio 0.65 (95% CI, 0.51-0.83; P < .001),

Methods: This is a Phase 1 open-label, dose escalation/dose expansion study conducted in the US (NCT03251378). The primary objectives are to evaluate the safety and tolerability of Fruq …

No Difference In Quality Of Life (Qol) Among People Who Switched From Injectable To Oral Disease Modifying Treatment (Dmt) Compared To Those Who Stayed On Injectable In A Community Cohort: A Propensity Score Matched (Psm) Analysis, Tamela Stuchiner, Lindsay Lucas, Elizabeth Baraban, Chiayi Chen, Stanley Cohan

Articles, Abstracts, and Reports

Introduction: Since the emergence of oral DMT to the market, few studies have examined patient reported outcomes of quality of life (QoL) after switching to oral DMT.

Objective: The aim of this study was to evaluate the impact of switching from injectable to oral disease modifying treatment (DMT) on QoL among relapsing multiple sclerosis (MS) participants.

Methods: Participants registered in the Pacific Northwest Multiple Sclerosis Registry who completed at least two annual surveys between 2012 and 2018 were included. Two cohorts were identified: “switchers”, those who switched from injectable to oral DMT, and “stayers”, those who stayed on injectable DMT. …

The Art Of Surgery: The Strange World Of The Placebo Response., Joseph R Dettori, Daniel C Norvell, Jens R Chapman

Articles, Abstracts, and Reports

No abstract provided.

Collaborative International Research In Clinical And Longitudinal Experience Study In Nmosd., Lawrence J Cook, John W Rose, Jessica S Alvey, Anna Marie Jolley, Renee Kuhn, Brie Marron, Melissa Pederson, Rene Enriquez, Jeff Yearley, Stephen Mckechnie, May H Han, Anna J Tomczak, Michael Levy, Maureen A Mealy, Jessica Coleman, Jeffrey L Bennett, Ruth Johnson, Myka Barnes-Garcia, Anthony L Traboulsee, Robert L Carruthers, Lisa Eunyoung Lee, Julia J Schubert, Katrina Mcmullen, Ilya Kister, Zoe Rimler, Allyson Reid, Nancy L Sicotte, Sarah M Planchon, Jeffrey A Cohen, Diane Ivancic, Jennifer L Sedlak, Ilana Katz Sand, Pavle Repovic, Lilyana Amezcua, Ana Pruitt, Erika Amundson, Tanuja Chitnis, Devin S Mullin, Eric C Klawiter, Andrew W Russo, Claire S Riley, Kaho B Onomichi, Libby Levine, Katherine E Nelson, Nancy M Nealon, Casey Engel, Mason Kruse-Hoyer, Melanie Marcille, Leticia Tornes, Anne Rumpf, Angela Greer, Megan Kenneally Behne, Renee R Rodriguez, Daniel W Behne, Derek W Blackway, Brian Coords, Terrence F Blaschke, Judy Sheard, Terry J Smith, Jacinta M Behne, Michael R Yeaman

Articles, Abstracts, and Reports

Objective: To develop a resource of systematically collected, longitudinal clinical data and biospecimens for assisting in the investigation into neuromyelitis optica spectrum disorder (NMOSD) epidemiology, pathogenesis, and treatment.

Methods: To illustrate its research-enabling purpose, epidemiologic patterns and disease phenotypes were assessed among enrolled subjects, including age at disease onset, annualized relapse rate (ARR), and time between the first and second attacks.

Results: As of December 2017, the Collaborative International Research in Clinical and Longitudinal Experience Study (CIRCLES) had enrolled more than 1,000 participants, of whom 77.5% of the NMOSD cases and 71.7% of the controls continue in active follow-up. Consanguineous …

Reported Quality Of Life In Those On High Efficacy Compared To First Line Disease Modifying Therapies In A Community Cohort, Tamela Stuchiner, Lindsay Lucas, Elizabeth Baraban, Chiayi Chen, Stanley Cohan

Articles, Abstracts, and Reports

Introduction: In recent years, more high efficacy disease modifying treatments (DMT) have been approved and patients are placed on high efficacy DMT earlier in their disease course. Few studies have assessed the impact of high efficacy DMT on patients’ quality of life (QoL).

Objective: The study objective was to determine whether participants on high efficacy DMT reported greater impact of MS on their physical and psychological QoL than those on first line DMT using survey data from the Pacific Northwest Multiple Sclerosis Registry (PNWMSR).

Methods: This was a cross-sectional study using participants’ last annual follow up survey submitted between 2013 …

Three Pillars Of Expectation Management In Spine Surgery: Trust, Communication, And Patient Education., Karsten Wiechert, Jeffrey C Wang, Jens R Chapman

Articles, Abstracts, and Reports

No abstract provided.

The Impact Of Ocrelizumab On Immunoglobulin Levels And The Risk Of Infection., Kyle Smoot

Articles, Abstracts, and Reports

Background: Ocrelizumab (OCR), a CD20 antibody, was approved in the US in 2017 for the treatment of relapsing MS (RMS) and primary progressive MS (PPMS). In clinical trials, infection rates are higher in patients receiving OCR. Rituximab, which is very similar to OCR, has been reported to be associated with higher risk of infection particularly in patients with low levels of IgM or IgG.

Objective: To determine if low levels of IgM or IgG increase the risk of infection in patients treated with OCR.

Methods: MS patients in the OCR registry with at least one IgM/IgG value at doses 2, …

Liquid Biopsy-Based Single-Cell Metabolic Phenotyping Of Lung Cancer Patients For Informative Diagnostics., Ziming Li, Zhuo Wang, Yin Tang, Xiang Lu, Jie Chen, Yu Dong, Baojun Wu, Chunying Wang, Liu Yang, Zhili Guo, Min Xue, Shun Lu, Wei Wei, Qihui Shi

Articles, Abstracts, and Reports

Accurate prediction of chemo- or targeted therapy responses for patients with similar driver oncogenes through a simple and least-invasive assay represents an unmet need in the clinical diagnosis of non-small cell lung cancer. Using a single-cell on-chip metabolic cytometry and fluorescent metabolic probes, we show metabolic phenotyping on the rare disseminated tumor cells in pleural effusions across a panel of 32 lung adenocarcinoma patients. Our results reveal extensive metabolic heterogeneity of tumor cells that differentially engage in glycolysis and mitochondrial oxidation. The cell number ratio of the two metabolic phenotypes is found to be predictive for patient therapy response, physiological …

Anti-Pd-1 Monoclonal Antibody Medi0680 In A Phase I Study Of Patients With Advanced Solid Malignancies., Aung Naing, Jeffrey Infante, Sanjay Goel, Howard Burris, Chelsea Black, Shannon Marshall, Ikbel Achour, Susannah Barbee, Rena May, Chris Morehouse, Kristen Pollizzi, Xuyang Song, Keith Steele, Nairouz Elgeioushi, Farzana Walcott, Joyson Karakunnel, Patricia Lorusso, Amy Weise, Joseph Eder, Brendan Curti, Michael Oberst

Articles, Abstracts, and Reports

BACKGROUND: The safety, efficacy, pharmacokinetics, and pharmacodynamics of the anti-programmed cell death-1 antibody MEDI0680 were evaluated in a phase I, multicenter, dose-escalation study in advanced solid malignancies.

METHODS: MEDI0680 was administered intravenously once every 2 weeks (Q2W) or once every 3 weeks at 0.1, 0.5, 2.5, 10 or 20 mg/kg. Two cohorts received 20 mg/kg once a week for 2 or 4 weeks, then 20 mg/kg Q2W. All were treated for 12 months or until progression. The primary endpoint was safety. Secondary endpoints were efficacy and pharmacokinetics. Exploratory endpoints included pharmacodynamics.

RESULTS: Fifty-eight patients were treated. Median age was 62.5 …

First-In-Human Study Of Tk-Positive Oncolytic Vaccinia Virus Delivered By Adipose Stromal Vascular Fraction Cells., Boris R Minev, Elliot Lander, John F Feller, Mark Berman, Bernadette M Greenwood, Ivelina Minev, Antonio F Santidrian, Duong Nguyen, Dobrin Draganov, Mehmet O Killinc, Anna Vyalkova, Santosh Kesari, Edward Mcclay, Gabriel Carabulea, Francesco M Marincola, Lisa H Butterfield, Aladar A Szalay

Articles, Abstracts, and Reports

BACKGROUND: ACAM2000, a thymidine kinase (TK)-positive strain of vaccinia virus, is the current smallpox vaccine in the US. Preclinical testing demonstrated potent oncolytic activity of ACAM2000 against several tumor types. This Phase I clinical trial of ACAM2000 delivered by autologous adipose stromal vascular fraction (SVF) cells was conducted to determine the safety and feasibility of such a treatment in patients with advanced solid tumors or acute myeloid leukemia (AML).

METHODS: Twenty-four patients with solid tumors and two patients with AML participated in this open-label, non-randomized dose-escalation trial. All patients were treated with SVF derived from autologous fat and incubated for …