Medicine and Health Sciences Commons^™

Initial Laparotomy Versus Peritoneal Drainage In Extremely Low Birthweight Infants With Surgical Necrotizing Enterocolitis Or Isolated Intestinal Perforation: A Multicenter Randomized Clinical Trial, Martin L Blakely, Jon E Tyson, Kevin P Lally, Susan R Hintz, Barry Eggleston, David K Stevenson, Gail E Besner, Abhik Das, Robin K Ohls, William E Truog, Leif D Nelin, Brenda B Poindexter, Claudia Pedroza, Michele C Walsh, Barbara J Stoll, Rachel Geller, Kathleen A Kennedy, Reed A Dimmitt, Waldemar A Carlo, C Michael Cotten, Abbot R Laptook, Krisa P Van Meurs, Kara L Calkins, Gregory M Sokol, Pablo J Sanchez, Myra H Wyckoff, Ravi M Patel, Ivan D Frantz, Seetha Shankaran, Carl T D'Angio, Bradley A Yoder, Edward F Bell, Kristi L Watterberg, Colin A Martin, Carroll M Harmon, Henry Rice, Arlet G Kurkchubasche, Karl Sylvester, James C Y Dunn, Troy A Markel, Diana L Diesen, Amina M Bhatia, Alan Flake, Walter J Chwals, Rebeccah Brown, Kathryn D Bass, Shawn D St Peter, Christina M Shanti, Walter Pegoli, David Skarda, Joel Shilyansky, David G Lemon, Ricardo A Mosquera, Myriam Peralta-Carcelen, Ricki F Goldstein, Betty R Vohr, Isabell B Purdy, Abbey C Hines, Nathalie L Maitre, Roy J Heyne, Sara B Demauro, Elisabeth C Mcgowan, Kimberly Yolton, Howard W Kilbride, Girija Natarajan, Kelley Yost, Sarah Winter, Tarah T Colaizy, Matthew M Laughon, Satyanarayana Lakshminrusimha, Rosemary D Higgins, Eunice Kennedy Shriver National Institute Of Child Health And Human Development Neonatal Research Network

Faculty, Staff and Student Publications

OBJECTIVE: The aim of this study was to determine which initial surgical treatment results in the lowest rate of death or neurodevelopmental impairment (NDI) in premature infants with necrotizing enterocolitis (NEC) or isolated intestinal perforation (IP).

SUMMARY BACKGROUND DATA: The impact of initial laparotomy versus peritoneal drainage for NEC or IP on the rate of death or NDI in extremely low birth weight infants is unknown.

METHODS: We conducted the largest feasible randomized trial in 20 US centers, comparing initial laparotomy versus peritoneal drainage. The primary outcome was a composite of death or NDI at 18 to 22 months corrected …

Outcomes Of Primary Versus Revisional Robotically Assisted Laparoscopic Roux-En-Y Gastric Bypass: A Multicenter Analysis Of Ten-Year Experience, Pouya Iranmanesh, John Fam, Thomas Nguyen, David Talarico, Kavita D Chandwani, Kulvinder S Bajwa, Melissa M Felinski, Leon V Katz, Sheilendra S Mehta, Stephan R Myers, Brad E Snyder, Peter A Walker, Todd D Wilson, Angielyn R Rivera, Connie L Klein, Shinil K Shah, Erik B Wilson

Faculty, Staff and Student Publications

BACKGROUND: Postoperative morbidity after laparoscopic bariatric surgery is considered higher for patients undergoing revisional versus primary procedures. The objective of this retrospective cohort study was to compare outcomes between patients undergoing primary versus revisional robotically assisted laparoscopic (RAL) Roux-en-Y gastric bypass (RYGB).

METHODS: Data of all patients who underwent RAL primary and revisional RYGB between 2009 and 2019 at two accredited, high-volume bariatric surgery centers-the Memorial Hermann - Texas Medical Center, Houston, TX, and the Tower Health, Reading Hospital, Reading, PA, were analyzed. Primary outcomes were early (< 30 days) and overall postoperative complications. Secondary outcomes included intraoperative complications, operative times, conversions to laparotomy, length of hospital stay, early (< 30 days) postoperative readmissions and deaths.

RESULTS: Data of 1072 patients were analyzed, including 806 primary and 266 …

Alveolar Socket Healing In 5-Lipoxygenase Knockout Aged Female Mice Treated Or Not With High Dose Of Zoledronic Acid, Ramez H Mahmoud, Claudia C Biguetti, Gustavo B Simionato, Isabela C Custódio, Raquel B P Silva, Marco A H Duarte, Leonardo P Faverani, Edilson Ervolino, Walid D Fakhouri, Mariza A Matsumoto

Faculty, Staff and Student Publications

This study investigated the role 5-lypoxigenase (5-LO) on alveolar socket healing in aged female mice treated with zoledronic acid (ZL). Forty 129/Sv female mice (64-68 weeks old), 20 wild type (WT) and 20 5-LO knockout (5LOKO) were equally distributed according to ZL treatment: WT Control, WT ZL, 5LOKO Control, and 5LOKO ZL. ZL groups were treated with an intraperitoneal injection of 250 µg/Kg of ZL, while controls were treated with saline. Treatments were administered once a week, starting four weeks before surgery for tooth extraction and until 7 and 21 days post-surgery. Mice were euthanized for a comprehensive microscopic analysis …

4-Dimensional Intracardiac Echocardiography In Transcatheter Mitral Valve Repair With The Mitraclip System., Carlos E Sanchez, Steven J Yakubov, Gagan Singh, Jason H Rogers, Nathan H Kander, Gilbert H L Tang

Heart and Vascular Articles

No abstract provided.

Adjuvant Platinum-Based Chemotherapy In Radically Resected Adrenocortical Carcinoma: A Cohort Study, Otilia Kimpel, Sara Bedrose, Felix Megerle, Alfredo Berruti, Massimo Terzolo, Matthias Kroiss, Knut Mai, Olaf M Dekkers, Mouhammed Amir Habra, Martin Fassnacht

Faculty, Staff and Students Publications

BACKGROUND: After radical resection, patients with adrenocortical carcinoma (ACC) frequently experience recurrence and, therefore, effective adjuvant treatment is urgently needed. The aim of the study was to investigate the role of adjuvant platinum-based therapy.

METHODS: In this retrospective multicentre cohort study, we identified patients treated with adjuvant platinum-based chemotherapy after radical resection and compared them with patients without adjuvant chemotherapy. Recurrence-free and overall survival (RFS/OS) were investigated in a matched group analysis and by applying a propensity score matching using the full control cohort (n = 268). For both approaches, we accounted for immortal time bias.

RESULTS: Of the 31 …

Transcatheter Aortic Valve Replacement With Self-Expandable Supra-Annular Valves For Degenerated Surgical Bioprostheses: Insights From Transcatheter Valve Therapy Registry., Luis Augusto P Dallan, John K Forrest, Michael J Reardon, Wilson Y Szeto, Isaac George, Susheel Kodali, Neal S Kleiman, Steven J Yakubov, Kendra J Grubb, Fang Liu, Cristian Baeza, Guilherme F Attizzani

Heart and Vascular Articles

Background Transcatheter aortic valve replacement with supra-annular transcatheter heart valves has been adopted in patients with degenerated surgical aortic valves. The next generation self-expanding Evolut PRO valve has not been evaluated in patients with surgical valve failure. Methods and Results Patients undergoing transcatheter aortic valve replacement in degenerated surgical aortic valve procedures using the Evolut R or Evolut PRO transcatheter heart valves in the Society of Thoracic Surgeons and American College of Cardiology Transcatheter Valve Therapy Registry between April 2015 and June 2019 were evaluated. Transcatheter valve performance was evaluated by clinical site echocardiography. In-hospital, 30-day, and 1-year clinical outcomes …

Surgical And Percutaneous Revascularization Outcomes Based On Syntax I, Ii, And Residual Scores: A Long-Term Follow-Up Study, Eduardo Bello Martins, Whady Hueb, David L Brown, Thiago Luis Scudeler, Eduardo Gomes Lima, Paulo Cury Rezende, Paulo Rogério Soares, Cibele Larrosa Garzillo, Jaime Paula Pessoa Linhares Filho, Daniel Valente Batista, Jose Antonio Franchini Ramires, Roberto Kalil Filho

2020-Current year OA Pubs

BACKGROUND: The objective of this study was to evaluate the association of SYNTAX scores I, II, and residual with cardiovascular outcomes of patients undergoing coronary artery bypass grafting (CABG) or percutaneous coronary intervention (PCI) and compare both procedures in a long-term follow-up.

METHODS: This is a retrospective single-center study from the MASS registry at the Heart Institute of the University of São Paulo, Brazil in which 969 patients with stable coronary artery disease undergoing CABG (559) or PCI (410) were included. We assessed the SYNTAX scores I, II and residual in both interventions. Clinical endpoints were the first occurrence of …

Predicting Long-Term Hepatocellular Carcinoma Response To Transarterial Radioembolization Using Contrast-Enhanced Ultrasound: Initial Experiences., Lauren J. Delaney, M. Tantawi, Corinne Wessner, Priscilla Machado, Flemming Forsberg, Andrej Lyshchik, Patrick O'Kane, Ji-Bin Liu, Jesse M. Civan, Alison Tan, Kevin Anton, Colette Shaw, John R. Eisenbrey

Department of Radiology Faculty Papers

Conventional cross-sectional imaging done shortly after radioembolization of hepatocellular carcinoma (HCC) does not reliably predict long-term response to treatment. This study evaluated whether quantitative contrast-enhanced ultrasound (CEUS) can predict the long-term response of HCC to yttrium-90 (Y-90) treatment. Fifteen patients underwent CEUS at three time points: immediately following treatment and 1 and 2 wk post-treatment. Response 3-6 mo after treatment was categorized on contrast-enhanced magnetic resonance imaging by two experienced radiologists using the Modified Response Evaluation Criteria in Solid Tumors. CEUS data were analyzed by quantifying tumor perfusion and residual fractional vascularity using time-intensity curves. Patients with stable disease on …

Continuous-Flow Left Ventricular Assist Device Support In Patients With Ischemic Versus Nonischemic Cardiomyopathy, Brendan P Chou, Andre Critsinelis, Harveen K Lamba, Gregory Long, Andrew B Civitello, Reynolds M Delgado, Subhasis Chatterjee

Faculty, Staff and Students Publications

To determine whether the cause of cardiomyopathy affects outcomes in patients who undergo continuous-flow left ventricular assist device support, we compared postimplant adverse events and survival between patients with ischemic and nonischemic cardiomyopathy. The inclusion criteria for the ischemic group were a history of myocardial infarction or revascularization (coronary artery bypass grafting or percutaneous coronary intervention), ≥75% stenosis of the left main or proximal left anterior descending coronary artery, or ≥75% stenosis of ≥2 epicardial vessels. From November 2003 through March 2016, 526 patients underwent device support: 256 (48.7%) in the ischemic group and 270 (51.3%) in the nonischemic group. …

Use Of Convalescent Plasma In Covid-19 Patients With Immunosuppression, Jonathon W Senefeld, Jeffrey P Henderson, Et Al.

2020-Current year OA Pubs

In the absence of effective countermeasures, human convalescent plasma has been widely used to treat severe acute respiratory syndrome coronavirus 2, the causative agent of novel coronavirus disease 19 (COVID-19), including among patients with innate or acquired immunosuppression. However, the association between COVID-19-associated mortality in patients with immunosuppression and therapeutic use of convalescent plasma is unknown. We review 75 reports, including one large matched-control registry study of 143 COVID-19 patients with hematological malignancies, and 51 case reports and 23 case series representing 238 COVID-19 patients with immunosuppression. We review clinical features and treatment protocols of COVID-19 patients with immunosuppression after …

Integrated Analysis Of A Phase 2 Study Of Cemiplimab In Advanced Cutaneous Squamous Cell Carcinoma: Extended Follow-Up Of Outcomes And Quality Of Life Analysis, Danny Rischin, Leonel Hernandez-Aya, Et Al

2020-Current year OA Pubs

BACKGROUND: To provide pooled longer term data from three groups of a phase 2 study of cemiplimab in patients with advanced cutaneous squamous cell carcinoma (CSCC), and to determine duration of response (DOR) and impact on quality of life (QoL).

METHODS: Patients received cemiplimab 3 mg/kg every 2 weeks (group 1, metastatic CSCC [mCSCC], n=59; group 2, locally advanced CSCC, n=78) or cemiplimab 350 mg every 3 weeks (group 3, mCSCC, n=56). Primary endpoint was objective response rate (ORR) per independent central review (ICR). QoL was repeatedly measured at day 1 of each treatment cycle (groups 1 and 2: 8 …

Efficacy Of Neratinib Plus Capecitabine In The Subgroup Of Patients With Central Nervous System Involvement From The Nala Trial, Sara A Hurvitz, Ron Bose, Et Al

2020-Current year OA Pubs

BACKGROUND: Neratinib has efficacy in central nervous system (CNS) metastases from HER2-positive metastatic breast cancer (MBC). We report outcomes among patients with CNS metastases at baseline from the phase III NALA trial of neratinib plus capecitabine (N + C) versus lapatinib plus capecitabine (L + C).

MATERIALS AND METHODS: NALA was a randomized, active-controlled trial in patients who received two or more previous HER2-directed regimens for HER2-positive MBC. Patients with asymptomatic/stable brain metastases (treated or untreated) were eligible. Patients were assigned to N + C (neratinib 240 mg per day, capecitabine 750 mg/m

RESULTS: Of 621 patients enrolled, 101 (16.3%) …

Predictors Of Device-Related Thrombus Following Percutaneous Left Atrial Appendage Occlusion., Trevor Simard, Richard G Jung, Kyle Lehenbauer, Kerstin Piayda, Radoslaw Pracoń, Gregory G Jackson, Eduardo Flores-Umanzor, Laurent Faroux, Kasper Korsholm, Julian K R Chun, Shaojie Chen, Moniek Maarse, Kristi Montrella, Zakeih Chaker, Jocelyn N Spoon, Luigi E Pastormerlo, Felix Meincke, Abhishek C Sawant, Carmen M Moldovan, Mohammed Qintar, Mehmet K Aktas, Luca Branca, Andrea Radinovic, Pradhum Ram, Rayan S El-Zein, Thomas Flautt, Wern Yew Ding, Bassel Sayegh, Tomás Benito-González, Oh-Hyun Lee, Solomon O Badejoko, Christina Paitazoglou, Nabeela Karim, Ahmed M Zaghloul, Himanshu Agrawal, Rachel M Kaplan, Oluseun Alli, Aamir Ahmed, Hussam S Suradi, Bradley P Knight, Venkata M Alla, Sidakpal S Panaich, Tom Wong, Martin W Bergmann, Rashaad Chothia, Jung-Sun Kim, Armando Pérez De Prado, Raveen Bazaz, Dhiraj Gupta, Miguel Valderrabano, Carlos E Sanchez, Mikhael F El Chami, Patrizio Mazzone, Marianna Adamo, Fred Ling, Dee Dee Wang, William O'Neill, Wojtek Wojakowski, Ashish Pershad, Sergio Berti, Daniel Spoon, Akram Kawsara, George Jabbour, Lucas V A Boersma, Boris Schmidt, Jens Erik Nielsen-Kudsk, Josep Rodés-Cabau, Xavier Freixa, Christopher R Ellis, Laurent Fauchier, Marcin Demkow, Horst Sievert, Michael L Main, Benjamin Hibbert, David R Holmes, Mohamad Alkhouli

Heart and Vascular Articles

BACKGROUND: Device-related thrombus (DRT) has been considered an Achilles' heel of left atrial appendage occlusion (LAAO). However, data on DRT prediction remain limited.

OBJECTIVES: This study constructed a DRT registry via a multicenter collaboration aimed to assess outcomes and predictors of DRT.

METHODS: Thirty-seven international centers contributed LAAO cases with and without DRT (device-matched and temporally related to the DRT cases). This study described the management patterns and mid-term outcomes of DRT and assessed patient and procedural predictors of DRT.

RESULTS: A total of 711 patients (237 with and 474 without DRT) were included. Follow-up duration was similar in the …

Statistical Analysis Of Two Arm Randomized Pre-Post Designs With One Post-Treatment Measurement, Fei Wan

2020-Current year OA Pubs

BACKGROUND: Randomized pre-post designs, with outcomes measured at baseline and after treatment, have been commonly used to compare the clinical effectiveness of two competing treatments. There are vast, but often conflicting, amount of information in current literature about the best analytic methods for pre-post designs. It is challenging for applied researchers to make an informed choice.

METHODS: We discuss six methods commonly used in literature: one way analysis of variance ("ANOVA"), analysis of covariance main effect and interaction models on the post-treatment score ("ANCOVAI" and "ANCOVAII"), ANOVA on the change score between the baseline and post-treatment scores ("ANOVA-Change"), repeated measures …

Analysis Of Pretreatment Factors Associated With Stability In Early Class Iii Treatment, Yasuko Inoue, Toru Deguchi, James K. Hartsfield Jr., Wakako Tome, Noriyuki Kitai

Oral Health Science Faculty Publications

BACKGROUND: The purpose of this study was to identify pretreatment factors associated with the stability of early class III treatment, since most orthodontists start the treatment with their uncertain hypotheses and/or predictions. Subjects consisted of 75 patients with a class III skeletal relationship (ANB < 2° and overjet < 0 mm) who had been consecutively treated with rapid maxillary expansion and facemask and followed until their second phase treatment. The patients were divided into two groups according to whether they showed relapse in follow-up. The stable group maintained their positive overjet (n = 55), and the unstable group experienced relapse with a zero or negative overjet (n = 20). Two general, three dental, and 13 cephalometric pretreatment factors were investigated to determine which factors were associated with stability.

RESULTS: Sex, pretreatment age, and anteroposterior functional shift, which were hypothesized as associated factors, were not related to …

A Machine Learning Approach To First Pass Reperfusion In Mechanical Thrombectomy: Prediction And Feature Analysis., Lohit Velagapudi, Nikolaos Mouchtouris, Richard F Schmidt, David Vuong, Omaditya Khanna, Ahmad Sweid, Bryan Sadler, Fadi Al-Saiegh, M Reid Gooch, Pascal Jabbour, Robert H Rosenwasser, Stavropoula Tjoumakaris

Department of Neurosurgery Faculty Papers

INTRODUCTION: Novel machine learning (ML) methods are being investigated across medicine for their predictive capabilities while boasting increased adaptability and generalizability. In our study, we compare logistic regression with machine learning for feature importance analysis and prediction in first-pass reperfusion.

METHODS: We retrospectively identified cases of ischemic stroke treated with mechanical thrombectomy (MT) at our institution from 2012-2018. Significant variables used in predictive modeling were demographic characteristics, medical history, admission NIHSS, and stroke characteristics. Outcome was binarized TICI on first pass (0-2a vs 2b-3). Shapley feature importance plots were used to identify variables that strongly affected outcomes.

RESULTS: Accuracy for …

Sustained Use Of The Impella 50 Heart Pump Enables Bridge To Clinical Decisions In 34 Patients, Daniel W Nelson, Sakthi Sundararajan, Evan Klein, Lyle D Joyce, Lucian A Durham, David L Joyce, Asim A Mohammed

The Texas Heart Institute Journal

We studied whether sustained hemodynamic support (>7 d) with the Impella 5.0 heart pump can be used as a bridge to clinical decisions in patients who present with cardiogenic shock, and whether such support can improve their outcomes. We retrospectively reviewed cases of patients who had Impella 5.0 support at our hospital from August 2017 through May 2019. Thirty-four patients (23 with cardiogenic shock and 11 with severely decompensated heart failure) underwent sustained support for a mean duration of 11.7 ± 9.3 days (range, ≤48 d). Of 29 patients (85.3%) who survived to next therapy, 15 were weaned from …

Delayed Treatment Of Acute Myocardial Infarction With Ventricular Septal Rupture Due To Patient Fear During The Covid-19 Pandemic, Minar Chhetry, Reema Bhatt, Nathan H Tehrani, Dimitrios V Avgerinos, Charles A Mack, Samuel J Lang, Xuming Dai

The Texas Heart Institute Journal

During the coronavirus disease 2019 (COVID-19) pandmic, more patients are presenting with complications late after acute myocardial infarction. We report the case of a 71-year-old man who delayed seeking medical care for 2 weeks, despite progressive shortness of breath, cough, and tactile fever, for fear of contracting COVID-19 in the hospital. Clinical and echocardiographic evaluation revealed a ventricular septal rupture secondary to acute myocardial infarction. The patient underwent urgent cardiac catheterization, followed by successful saphenous vein grafting to the left anterior descending coronary artery and open surgical repair of the ventricular septal rupture with a bovine pericardial patch. This case …

Oral Once-Daily Berotralstat For The Prevention Of Hereditary Angioedema Attacks: A Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial, Bruce Zuraw, H James Wedner, Et Al.

2020-Current year OA Pubs

BACKGROUND: Berotralstat (BCX7353) is an oral, once-daily inhibitor of plasma kallikrein in development for the prophylaxis of hereditary angioedema (HAE) attacks.

OBJECTIVE: Our aim was to determine the efficacy, safety, and tolerability of berotralstat in patients with HAE over a 24-week treatment period (the phase 3 APeX-2 trial).

METHODS: APeX-2 was a double-blind, parallel-group study that randomized patients at 40 sites in 11 countries 1:1:1 to receive once-daily berotralstat in a dose of 110 mg or 150 mg or placebo (Clinicaltrials.gov identifier NCT03485911). Patients aged 12 years or older with HAE due to C1 inhibitor deficiency and at least 2 …

Active Surveillance Of Metastatic Renal Cell Carcinoma: Results From A Prospective Observational Study (Marcc), Michael R Harrison, Russell K Pachynski, Et Al.

2020-Current year OA Pubs

BACKGROUND: Systemic therapy (ST) can be deferred in patients who have metastatic renal cell carcinoma (mRCC) and slow-growing metastases. Currently, this subset of patients managed with active surveillance (AS) is not well described in the literature.

METHODS: This was a prospective observational study of patients with mRCC across 46 US community and academic centers. The objective was to describe baseline characteristics and demographics of patients with mRCC initially managed by AS, reasons for AS, and patient outcomes. Descriptive statistics were used to characterize demographics, baseline characteristics, and patient-related outcomes. Wilcoxon 2-sample rank-sum tests and χ

RESULTS: Of 504 patients, mRCC …

Left Ventricular Recovery With Explantation Of Continuous-Flow Left Ventricular Assist Device After 5 Years Of Support, Andre Critsinelis, Chitaru Kurihara, Masashi Kawabori, Tadahisa Sugiura, Andrew B Civitello, Jeffrey A Morgan

Faculty, Staff and Students Publications

Mechanical circulatory support may result in sufficient myocardial recovery to allow for explantation of the left ventricular assist device (LVAD). The duration of support associated with left ventricular recovery has generally been 6-12 months. In this report, we present a patient in whom the left ventricle recovered after 5 years of support with a LVAD. Our report demonstrates that long-term monitoring for left ventricular recovery is prudent and may allow for late device explantation.

Covid-19 Treatment Combinations And Associations With Mortality In A Large Multi-Site Healthcare System., Dagan Coppock, Michael Baram, Anna Marie Chang, Patricia Henwood, Alan Kubey, Ross Summer, John Zurlo, Michael Li, Bryan Hess

Division of Infectious Diseases and Environmental Medicine Faculty Papers

INTRODUCTION: During the early months of the COVID-19 pandemic, mortality associated with the disease declined in the United States. The standard of care for pharmacological interventions evolved during this period as new and repurposed treatments were used alone and in combination. Though these medications have been studied individually, data are limited regarding the relative impact of different medication combinations. The objectives of this study were to evaluate the association of COVID-19-related mortality and observed medication combinations and to determine whether changes in medication-related practice patterns and measured patient characteristics, alone, explain the decline in mortality seen early in the COVID-19 …

Continuous-Flow Left Ventricular Assist Device Explantation After More Than 5 Years Of Circulatory Support And Ventricular Reconditioning, George V Letsou, Andrew C W Baldwin, Andrew B Civitello, William E Cohn, O H Frazier

The Texas Heart Institute Journal

Continuous-flow left ventricular assist devices have proved to be effective, durable, life-saving tools in patients with end-stage heart failure. However, because of the risks associated with mechanical circulatory support (including stroke, infection, gastrointestinal bleeding, and device malfunction), the optimal goal of device therapy is myocardial recovery and device removal. Ventricular reconditioning and pump explantation after continuous-flow support have been reported; however, little is known about variables that govern the pace and degree of myocardial response in patients who experience such recovery. We describe our long-term pump-weaning strategy for a 25-year-old man who had a continuous-flow device implanted and then needed …

Evaluation Of Diazepam Nasal Spray In Patients With Epilepsy Concomitantly Using Maintenance Benzodiazepines: An Interim Subgroup Analysis From A Phase 3, Long-Term, Open-Label Safety Study., Eric B Segal, Daniel Tarquinio, Ian Miller, James W Wheless, Dennis Dlugos, Victor Biton, Gregory D Cascino, Jay Desai, R Edward Hogan, Kore Liow, Michael R Sperling, Blanca Vazquez, David F Cook, Adrian L Rabinowicz, Enrique Carrazana

Department of Neurology Faculty Papers

OBJECTIVE: Diazepam nasal spray (Valtoco), indicated for acute treatment of frequent seizure activity (seizure clusters) in patients with epilepsy ≥6 years of age, is designed to be a rapid, noninvasive, socially acceptable route of administration. This interim analysis evaluated the safety profile of diazepam nasal spray in patients with and without concomitant use of benzodiazepines, with use of a second dose for a seizure cluster as a proxy for effectiveness.

METHODS: A long-term, phase 3, open-label safety study enrolled patients with epilepsy who had seizures despite a stable antiseizure medication regimen.

RESULTS: Among 175 patients enrolled by October 31, 2019, …

Outcomes And Risk Factors Associated With Sars-Cov-2 Infection In A North American Registry Of Patients With Multiple Sclerosis, Amber Salter, Robert J Fox, Scott D Newsome, June Halper, David K B Li, Pamela Kanellis, Kathleen Costello, Bruce Bebo, Kottil Rammohan, Gary R Cutter, Anne H Cross

2020-Current year OA Pubs

Importance: Emergence of SARS-CoV-2 causing COVID-19 prompted the need to gather information on clinical outcomes and risk factors associated with morbidity and mortality in patients with multiple sclerosis (MS) and concomitant SARS-CoV-2 infections.

Objective: To examine outcomes and risk factors associated with COVID-19 clinical severity in a large, diverse cohort of North American patients with MS.

Design, Setting, and Participants: This analysis used deidentified, cross-sectional data on patients with MS and SARS-CoV-2 infection reported by health care professionals in North American academic and community practices between April 1, 2020, and December 12, 2020, in the COVID-19 Infections in MS Registry. …

Randomized Trial Of The Efficacy And Safety Of Berotralstat (Bcx7353) As An Oral Prophylactic Therapy For Hereditary Angioedema: Results Of Apex-2 Through 48 Weeks (Part 2), H James Wedner, Et Al.

2020-Current year OA Pubs

BACKGROUND: Berotralstat (BCX7353) is a recently approved, oral, once-daily kallikrein inhibitor for hereditary angioedema (HAE) prophylaxis. In the APeX-2 trial, berotralstat reduced HAE attack rates over 24 weeks, with a favorable safety and tolerability profile.

OBJECTIVE: Evaluate berotralstat safety, tolerability, and effectiveness over 48 weeks.

METHODS: APeX-2 is a phase 3, parallel-group, multicenter trial (NCT03485911) in patients with HAE due to C1 esterase inhibitor deficiency. Part 1 was double-blind and placebo-controlled, with patients randomized to 24 weeks of berotralstat 150 mg, 110 mg, or placebo. In part 2, patients continued berotralstat the same dose or, if initially randomized to placebo, …

Safety And Efficacy Of Umbilical Cord-Derived Wharton's Jelly Compared To Hyaluronic Acid And Saline For Knee Osteoarthritis: Study Protocol For A Randomized, Controlled, Single-Blind, Multi-Center Trial, Ashim Gupta, Nicola Maffulli, Hugo C Rodriguez, Eric W Carson, Randa A Bascharon, Kristin Delfino, Howard J Levy, Saadiq F El-Amin

2020-Current year OA Pubs

BACKGROUND: Osteoarthritis (OA) is the most common joint disorder in the United States of America (USA) with a fast-rising prevalence. Current treatment modalities are limited, and total knee replacement surgeries have shown disadvantages, especially for grade II/III OA. The interest in the use of biologics, including umbilical cord (UC)-derived Wharton's jelly (WJ), has grown in recent years. The results from a preliminary study demonstrated the presence of essential components of regenerative medicine, namely growth factors, cytokines, hyaluronic acid (HA), and extracellular vesicles, including exosomes, in WJ. The proposed study aims to evaluate the safety and efficacy of intra-articular injection of …

Long Term Safety, Tolerability, And Efficacy Of Intracutaneous Zolmitriptan (M207) In The Acute Treatment Of Migraine., Stephanie J. Nahas, Nada Hindiyeh, Deborah I. Friedman, Nada Elbuluk, Donald J. Kellerman, Pamela K. Foreman, Peter Schmidt

Department of Neurology Faculty Papers

OBJECTIVE: To determine the long-term safety and tolerability profile of M207 in the acute treatment of migraine.

BACKGROUND: M207 is an investigational microneedle-based system for intracutaneous delivery of zolmitriptan for the treatment of migraine attacks. Following on the positive results of a Phase 2/3 placebo-controlled efficacy study (ZOTRIP), this study was designed to evaluate the safety of this novel product during repeated use for the treatment of migraine attacks.

METHODS: In this 6-12 month open-label, multicenter observational study, participants used an eDiary to record headache symptoms and adverse events at specified intervals up to 48 h following treatment of a …

One-Year Urinary And Sexual Outcome Trajectories Among Prostate Cancer Patients Treated By Radical Prostatectomy: A Prospective Study, Lin Yang, Jung Ae Lee, Emily Heer, Claire Pernar, Graham A Colditz, Ratna Pakpahan, Kellie R Imm, Eric H Kim, Robert L Grubb 3rd, Kathleen Y Wolin, Adam S Kibel, Siobhan Sutcliffe

2020-Current year OA Pubs

BACKGROUND: To examine one-year trajectories of urinary and sexual outcomes, and correlates of these trajectories, among prostate cancer patients treated by radical prostatectomy (RP).

METHODS: Study participants were recruited from 2011 to 2014 at two US institutions. Self-reported urinary and sexual outcomes were measured at baseline before surgery, and 5 weeks, 6 months and 12 months after surgery, using the modified Expanded Prostate Cancer Index Composite-50 (EPIC-50). Changes in EPIC-50 scores from baseline were categorized as improved (beyond baseline), maintained, or impaired (below baseline), using previously-reported minimum clinically important differences.

RESULTS: Of the 426 eligible participants who completed the baseline …

Tofacitinib, An Oral Janus Kinase Inhibitor: Analysis Of Malignancy (Excluding Nonmelanoma Skin Cancer) Events Across The Ulcerative Colitis Clinical Program, Gary R Lichtenstein, Gerhard Rogler, Matthew A Ciorba, Chinyu Su, Gary Chan, Ronald D Pedersen, Nervin Lawendy, Daniel Quirk, Chudy I Nduaka, Andrew J Thorpe, Julian Panés

2020-Current year OA Pubs

BACKGROUND: Tofacitinib is an oral, small molecule Janus kinase inhibitor for the treatment of ulcerative colitis (UC). Here, we performed an integrated analysis of malignancy events from the tofacitinib phase 3 UC clinical development program (excluding nonmelanoma skin cancer [NMSC]).

METHODS: Data (up to May 2019) were pooled from two phase 3 induction studies, a phase 3 maintenance study, and an ongoing, open-label, long-term extension (OLE) study, and analyzed as 3 cohorts: induction (N = 1139), maintenance (N = 592), and overall (induction, maintenance, and ongoing OLE study; N = 1124). Proportions and incidence rates (IRs; unique patients with events …