Medicine and Health Sciences Commons^™

Longitudinal Predictors Of Health-Related Quality Of Life In Isolated Dystonia, Johanna Junker, Joel S Perlmutter, Et Al.

2020-Current year OA Pubs

OBJECTIVE: To determine longitudinal predictors of health-related quality of life (HR-QoL) in an international multicenter cohort of patients with isolated dystonia.

METHODS: Out of 603 dystonia patients prospectively enrolled in the Natural History Dystonia Coalition study, 155 were assessed three times within 2 years for HR-QoL, symptoms of depression, generalized anxiety disorder (GAD), and social anxiety disorder (SAD), as well as dystonia severity and dystonic tremor. In addition, the impact of botulinum neurotoxin (BoNT) injections on HR-QoL was evaluated after 1 year.

RESULTS: Depressive symptoms at baseline predicted lower HR-QoL on all subscales after 2 years (all p ≤ 0.001). …

Implementation And Outcomes Of A Pilot Collaborative Surgical Hydrocele Training In Côte D'Ivoire, Kevin Montes, Georgina Angoa, Catherine M Bjerum, Adama Guira, Olivier K Kouadio, Allassane F Ouattara, Gary J Weil, Peter U Fischer, Aboulaye Meite, Benjamin G Koudou, Philip J Budge

2020-Current year OA Pubs

Lymphatic filariasis (LF) is a neglected tropical disease that can cause hydrocele and its associated stigma, loss of economic productivity, and depression. Hydrocele surgery is an essential part of LF morbidity management but can be difficult for national programs to implement. To improve access to hydrocele surgeries in Côte d'Ivoire, we provided a WHO-certified surgical training for six surgical teams from five health districts in Côte d'Ivoire. We then evaluated the surgical outcomes and assessed the impact of hydrocele surgery on quality of life of hydrocelectomy patients. Preoperative and operative records were reviewed to describe baseline hydrocele characteristics and operative …

Short-Stay Units Vs Routine Admission From The Emergency Department In Patients With Acute Heart Failure: The Ssu-Ahf Randomized Clinical Trial, Peter S Pang, Douglas Char, Et Al.

2020-Current year OA Pubs

IMPORTANCE: More than 80% of patients who present to the emergency department (ED) with acute heart failure (AHF) are hospitalized. With more than 1 million annual hospitalizations for AHF in the US, safe and effective alternatives are needed. Care for AHF in short-stay units (SSUs) may be safe and more efficient than hospitalization, especially for lower-risk patients, but randomized clinical trial data are lacking.

OBJECTIVE: To compare the effectiveness of SSU care vs hospitalization in lower-risk patients with AHF.

DESIGN, SETTING, AND PARTICIPANTS: This multicenter randomized clinical trial randomly assigned low-risk patients with AHF 1:1 to SSU or hospital admission …

Patient-Reported Treatment Response In Chronic Graft-Versus-Host Disease, Annie Im, Iskra Pusic, Et Al.

2020-Current year OA Pubs

Chronic graft-versus-host disease (GvHD) treatment response is assessed using National Institutes of Health (NIH) Consensus Criteria in clinical trials, and by clinician assessment in routine practice. Patient-reported treatment response is central to the experience of chronic GvHD manifestations as well as treatment benefit and toxicity, but how they correlate with clinician- or NIH-responses has not been well-studied. We aimed to characterize 6-month patientreported response, determine associated chronic GvHD baseline organ features and changes, and evaluate which patientreported quality of life and chronic GvHD symptom burden measures correlated with patient-reported response. From two nationally representative Chronic GVHD Consortium prospective observational studies, …

The Persistency Index: A Novel Screening Tool For Identifying Myofascial Pelvic Floor Dysfunction In Patients Seeking Care For Lower Urinary Tract Symptoms, A Lenore Ackerman, Michele Torosis, Nicholas J Jackson, Ashley T Caron, Melissa R Kaufman, Jerry L Lowder, Jonathan C Routh

2020-Current year OA Pubs

BACKGROUND: Patients with myofascial pelvic floor dysfunction often present with lower urinary tract symptoms, such as urinary frequency, urgency, and bladder pressure. Often confused with other lower urinary tract disorders, this constellation of symptoms, recently termed myofascial urinary frequency syndrome, is distinct from other lower urinary tract symptoms and optimally responds to pelvic floor physical therapy. A detailed pelvic floor myofascial examination performed by a skilled provider is currently the only method to identify myofascial urinary frequency syndrome. Despite a high influence on quality of life, low awareness of this condition combined with no objective diagnostic testing leads to the …

The Persistency Index: A Novel Screening Tool For Identifying Myofascial Pelvic Floor Dysfunction In Patients Seeking Care For Lower Urinary Tract Symptoms, A Lenore Ackerman, Michele Torosis, Nicholas J Jackson, Ashley T Caron, Melissa R Kaufman, Jerry L Lowder, Jonathan C Routh

2020-Current year OA Pubs

BACKGROUND: Patients with myofascial pelvic floor dysfunction often present with lower urinary tract symptoms, such as urinary frequency, urgency, and bladder pressure. Often confused with other lower urinary tract disorders, this constellation of symptoms, recently termed myofascial urinary frequency syndrome, is distinct from other lower urinary tract symptoms and optimally responds to pelvic floor physical therapy. A detailed pelvic floor myofascial examination performed by a skilled provider is currently the only method to identify myofascial urinary frequency syndrome. Despite a high influence on quality of life, low awareness of this condition combined with no objective diagnostic testing leads to the …

Awareness With Paralysis And Symptoms Of Post-Traumatic Stress Disorder Among Mechanically Ventilated Emergency Department Survivors (Ed-Awareness-2 Trial): Study Protocol For A Pragmatic, Multicenter, Stepped Wedge Cluster Randomized Trial, Brian M Fuller, Brian E Driver, Michael B Roberts, Christa A Schorr, Kathryn Thompson, Brett Faine, Julianne Yeary, Nicholas M Mohr, Ryan D Pappal, Robert J Stephens, Yan Yan, Nicholas J Johnson, Brian W Roberts

2020-Current year OA Pubs

BACKGROUND: Awareness with paralysis (AWP) is memory recall during neuromuscular blockade (NMB) and can cause significant psychological harm. Decades of effort and rigorous trials have been conducted to prevent AWP in the operating room, where prevalence is 0.1-0.2%. By contrast, AWP in mechanically ventilated emergency department (ED) patients is common, with estimated prevalence of 3.3-7.4% among survivors given NMB. Longer-acting NMB use is a critical risk for AWP, and we have shown an association between ED rocuronium use and increased AWP prevalence. As NMB are given to more than 90% of ED patients during tracheal intubation, this trial provides a …

A Multi-Institutional Phase 2 Trial Of Ablative 5-Fraction Stereotactic Magnetic Resonance-Guided On-Table Adaptive Radiation Therapy For Borderline Resectable And Locally Advanced Pancreatic Cancer, Parag Jitendra Parikh, Hyun Kim, Lauren E Henke, Alex T Price, Et Al.

2020-Current year OA Pubs

PURPOSE: Magnetic resonance (MR) image guidance may facilitate safe ultrahypofractionated radiation dose escalation for inoperable pancreatic ductal adenocarcinoma. We conducted a prospective study evaluating the safety of 5-fraction Stereotactic MR-guided on-table Adaptive Radiation Therapy (SMART) for locally advanced (LAPC) and borderline resectable pancreatic cancer (BRPC).

METHODS AND MATERIALS: Patients with LAPC or BRPC were eligible for this multi-institutional, single-arm, phase 2 trial after ≥3 months of systemic therapy without evidence of distant progression. Fifty gray in 5 fractions was prescribed on a 0.35T MR-guided radiation delivery system. The primary endpoint was acute grade ≥3 gastrointestinal (GI) toxicity definitely attributed to …

Six-Month Outcomes Of Mechanical Thrombectomy For Treating Deep Vein Thrombosis: Analysis From The 500-Patient Clout Registry, Abdullah Shaikh, Vipul Khetarpaul, Et Al.

2020-Current year OA Pubs

PURPOSE: Mechanical thrombectomy for the treatment of deep vein thrombosis (DVT) is being increasingly utilized to reduce symptoms and prevent postthrombotic syndrome (PTS), but more data on clinical outcomes are needed. Mechanical thrombectomy was studied in the ClotTriever Outcomes (CLOUT) registry with 6-month full analysis outcomes reported herein.

MATERIALS AND METHODS: The CLOUT registry is a prospective, all-comer study that enrolled 500 lower extremity DVT patients across 43 US sites treated with mechanical thrombectomy using the ClotTriever System. Core-lab assessed Marder scores and physician-assessed venous patency by duplex ultrasound, PTS assessment using Villalta score, venous symptom severity, pain, and quality …

Optimizing The Utility Of Anorectal Manometry For Diagnosis And Therapy: A Roundtable Review And Recommendations, Satish S C Rao, Gregory S Sayuk, Et Al.

2020-Current year OA Pubs

BACKGROUND & AIMS: Anorectal manometry (ARM) is a comprehensive diagnostic tool for evaluating patients with constipation, fecal incontinence, or anorectal pain; however, it is not widely utilized for reasons that remain unclear. The aim of this roundtable discussion was to critically examine the current clinical practices of ARM and biofeedback therapy by physicians and surgeons in both academic and community settings.

METHODS: Leaders in medical and surgical gastroenterology and physical therapy with interest in anorectal disorders were surveyed regarding practice patterns and utilization of these technologies. Subsequently, a roundtable was held to discuss survey results, explore current diagnostic and therapeutic …

Feasibility Of The Audio-Visual Assisted Therapeutic Ambience In Radiotherapy (Avatar) System For Anesthesia Avoidance In Pediatric Patients: A Multicenter Trial, Paulina M Gutkin, Stephanie M Perkins, Et Al.

2020-Current year OA Pubs

PURPOSE: The Audio-Visual Assisted Therapeutic Ambience in Radiotherapy (AVATAR) system was the first published radiation therapy (RT)-compatible system to reduce the need for pediatric anesthesia through video-based distraction. We evaluated the feasibility of AVATAR implementation and effects on anesthesia use, quality of life, and anxiety in a multicenter pediatric trial.

METHODS AND MATERIALS: Pediatric patients 3 to 10 years of age preparing to undergo RT at 10 institutions were prospectively enrolled. Children able to undergo at least 1 fraction of RT using AVATAR without anesthesia were considered successful (S). Patients requiring anesthesia for their entire treatment course were nonsuccessful (NS). …

Treatment Preference And Quality Of Life Impact: Ravulizumab Vs Eculizumab For Atypical Hemolytic Uremic Syndrome, Teri J Mauch, Michael R Chladek, Spero Cataland, Shruti Chaturvedi, Bradley P Dixon, Katherine Garlo, Christoph Gasteyger, Anuja Java, Jorge Leguizamo, Lucy Lloyd-Price, Tan P Pham, Tara Symonds, Ioannis Tomazos, Yan Wang

2020-Current year OA Pubs

No abstract provided.

Using The Sociotechnical Model To Conduct A Focused Usability Assessment Of A Breast Reconstruction Decision Tool, Randi Foraker, Crystal Phommasathit, Kaleigh Clevenger, Clara Lee, Jessica Boateng, Napiera Shareef, Mary C Politi

2020-Current year OA Pubs

INTRODUCTION: BREASTChoice is a web-based breast reconstruction decision aid. The previous clinical trial-prior to the adaptation of this refined tool in which we explored usability-measured decision quality, quality of life, patient activation, shared decision making, and treatment choice. The current usability study was designed to elicit patients' and clinicians' perspectives on barriers and facilitators for implementing BREASTChoice into the clinical workflow.

METHODS: We conducted qualitative interviews with patients and clinicians from two Midwestern medical specialty centers from August 2020 to April 2021. Interviews were first double coded until coders achieved a kappa > 0.8 and percent agreement > 95%, then were coded …

Treatment Decisions And The Use Of Mek Inhibitors For Children With Neurofibromatosis Type 1-Related Plexiform Neurofibromas, Amy E Armstrong, Allan J Belzberg, John R Crawford, Angela C Hirbe, Zhihong J Wang

2020-Current year OA Pubs

Neurofibromatosis type 1 (NF1), the most common tumor predisposition syndrome, occurs when NF1 gene variants result in loss of neurofibromin, a negative regulator of RAS activity. Plexiform neurofibromas (PN) are peripheral nerve sheath tumors that develop in patients with NF1 and are associated with substantial morbidity and for which, until recently, the only treatment was surgical resection. However, surgery carries several risks and a proportion of PN are considered inoperable. Understanding the genetic underpinnings of PN led to the investigation of targeted therapies as medical treatment options, and the MEK1/2 inhibitor selumetinib has shown promising efficacy in pediatric patients with …

Symptomatic Benefit Of Momelotinib In Patients With Myelofibrosis: Results From The Simplify Phase Iii Studies, Ruben A Mesa, Stephen T Oh, Et Al.

2020-Current year OA Pubs

BACKGROUND: Myelofibrosis (MF)-associated constitutional symptoms can severely impact health-related quality of life. Clinical trials in MF traditionally measure symptom response to treatment as a landmark endpoint of total symptom score (TSS) reduction ≥50% from baseline. However, this dichotomous assessment provides a limited view of clinically relevant symptomatic changes. Herein we evaluated longitudinal change from baseline in TSS over the continuous 24-week period and individual symptom scores to obtain a more comprehensive understanding of symptom benefits experienced by patients with MF receiving therapy.

METHODS: Longitudinal symptom change was evaluated using mixed-effect model repeated measure (MMRM) methodology with individual item-level analyses to …

Examining Mental Health, Education, Employment, And Pain In Sickle Cell Disease, Kelly M Harris, Liliana Preiss, Taniya Varughese, Anna Bauer, Cecelia L Calhoun, Marsha Treadwell, Rita Masese, Jane S Hankins, Faiz Ahmed Hussain, Jeffrey Glassberg, Cathy L Melvin, Robert Gibson, Allison A King, Sickle Cell Disease Implementation Consortium

2020-Current year OA Pubs

IMPORTANCE: Pain related to sickle cell disease (SCD) is complex and associated with social determinants of health. Emotional and stress-related effects of SCD impact daily quality of life and the frequency and severity of pain.

OBJECTIVE: To explore the association of educational attainment, employment status, and mental health with pain episode frequency and severity among individuals with SCD.

DESIGN, SETTING, AND PARTICIPANTS: This is a cross-sectional analysis of patient registry data collected at baseline (2017-2018) from patients treated at 8 sites of the US Sickle Cell Disease Implementation Consortium. Data analysis was performed from September 2020 to March 2022.

MAIN …

Translingual Neurostimulation Combined With Physical Therapy To Improve Walking And Balance In Multiple Sclerosis (Neuromstrals): Study Protocol For A Randomized Controlled Trial, Michelle Ploughman, Ganeswara Rao Melam, Syamala Buragadda, Keith R Lohse, Fraser Clift, Mark Stefanelli, Michael Levin, Sarah J Donkers

2020-Current year OA Pubs

INTRODUCTION: Physical rehabilitation restores lost function and promotes brain plasticity in people with Multiple Sclerosis (MS). Research groups worldwide are testing the therapeutic effects of combining non-invasive neuromodulation with physical therapy (PT) to further improve functional outcomes in neurological disorders but with mixed results. Whether such devices enhance function is not clear. We present the rationale and study design for a randomized controlled trial evaluating if there is additional benefit to the synergistic pairing of translingual neurostimulation (TLNS) with PT to improve walking and balance in MS.

METHODS AND ANALYSIS: A parallel group [PT + TLNS or PT + Sham], …

Nirogacestat, A Γ-Secretase Inhibitor For Desmoid Tumors, Mrinal Gounder, Brian A Van Tine, Et Al.

2020-Current year OA Pubs

BACKGROUND: Desmoid tumors are rare, locally aggressive, highly recurrent soft-tissue tumors without approved treatments.

METHODS: We conducted a phase 3, international, double-blind, randomized, placebo-controlled trial of nirogacestat in adults with progressing desmoid tumors according to the Response Evaluation Criteria in Solid Tumors, version 1.1. Patients were assigned in a 1:1 ratio to receive the oral γ-secretase inhibitor nirogacestat (150 mg) or placebo twice daily. The primary end point was progression-free survival.

RESULTS: From May 2019 through August 2020, a total of 70 patients were assigned to receive nirogacestat and 72 to receive placebo. Nirogacestat had a significant progression-free survival benefit …

Final Findings From The Control Trial: Strategies To Reduce The Incidence And Severity Of Neratinib-Associated Diarrhea In Patients With Her2-Positive Early-Stage Breast Cancer, Arlene Chan, Ron Bose, Et Al.

2020-Current year OA Pubs

BACKGROUND: Neratinib is an irreversible pan-HER tyrosine kinase inhibitor approved for HER2-positive early-stage and metastatic breast cancer. Diarrhea is the most frequent side effect and the most common reason for early discontinuation. The phase II CONTROL trial investigated antidiarrheal prophylaxis or neratinib dose escalation (DE) for prevention of diarrhea. We present complete study results including final data for two DE strategies.

METHODS: Patients who completed trastuzumab-based adjuvant therapy received neratinib 240 mg/day for 1 year. Early cohorts investigated mandatory prophylaxis with loperamide, then additional budesonide or colestipol. Final cohorts assessed neratinib DE over the first 2 (DE1) or 4 weeks …

Navigating Care For Rare Diseases: Caregiver And Patient Advice For Families And Clinicians Managing Care For Vascular Malformations, Anna M Kerr, Christine Bereitschaft, Kayla M Duty, Bryan A Sisk

2020-Current year OA Pubs

OBJECTIVE: Families affected by rare diseases face many challenges finding adequate care and often report poor communication with clinicians. In the current study, we explore patient and caregiver advice for families and clinicians in the context of complex vascular malformations (VMs), a condition that is frequently misunderstood and misdiagnosed.

METHODS: We performed semi-structured interviews with 21 adult patients with complex VMs and 24 caregivers of children with VMs. We analyzed the transcripts using thematic analysis.

RESULTS: Participants advised patients and caregivers to advocate for care, address mental and emotional well-being, seek social support, and promote self-management and self-care. Participants advised …

Case Report: Insulin Hypersensitivity In Youth With Type 1 Diabetes, Einas H Alkhatib, Jody B Grundman, Anna M Adamusiak, Melena D Bellin, Joel P Brooks, Kevin S Buckley, Erin M Janssen, Maleewan Kitcharoensakkul, Kyle P Mcnerney, Thea L Pfeifer, Brooke I Polk, Brynn E Marks

2020-Current year OA Pubs

OBJECTIVE: Immediate type I, type III, and delayed type IV hypersensitivity reactions to insulin are rare, but potentially serious complications of exogenous insulin administration required for the treatment of type 1 diabetes (T1D).

METHODS: We present four cases of insulin hypersensitivity reactions occurring in youth with T1D and a literature review of this topic.

RESULTS: Insulin hypersensitivity reactions included types I, III, and IV with presentations ranging from localized urticaria, erythematous nodules, and eczematous plaques to anaphylaxis with respiratory distress. Reactions occurred in youth with newly diagnosed T1D and in those with long-standing T1D who were using both injection and …

Health Utilities And Quality-Adjusted Life Years For Patients With Amyotrophic Lateral Sclerosis Receiving Reldesemtiv Or Placebo In Fortitude-Als, Paulos Gebrehiwet, Lisa Meng, Stacy A Rudnicki, Phil Sarocco, Jenny Wei, Andrew A Wolff, Michael Butzner, Adriano Chiò, Jinsy A Andrews, Angela Genge, Dyfrig A Hughes, Carlayne E Jackson, Noah Lechtzin, Timothy M Miller, Jeremy M Shefner

2020-Current year OA Pubs

AIMS: To estimate the health utilities and quality-adjusted life years (QALYs) in patients with amyotrophic lateral sclerosis (ALS) receiving

MATERIALS AND METHODS: We performed a post hoc analysis of clinical trial data from FORTITUDE-ALS (NCT03160898). This Phase IIb, double-blind, randomized, dose-ranging, placebo-controlled, parallel-group, 12-week trial evaluated

RESULTS: The full analysis set consisted of 456 patients (

CONCLUSIONS: This post hoc analysis of FORTITUDE-ALS suggests that

CLINICALTRIALS.GOV IDENTIFIER: NCT03160898.

Case Report: Insulin Hypersensitivity In Youth With Type 1 Diabetes, Einas H Alkhatib, Jody B Grundman, Anna M Adamusiak, Melena D Bellin, Joel P Brooks, Kevin S Buckley, Erin M Janssen, Maleewan Kitcharoensakkul, Kyle P Mcnerney, Thea L Pfeifer, Brooke I Polk, Brynn E Marks

2020-Current year OA Pubs

OBJECTIVE: Immediate type I, type III, and delayed type IV hypersensitivity reactions to insulin are rare, but potentially serious complications of exogenous insulin administration required for the treatment of type 1 diabetes (T1D).

METHODS: We present four cases of insulin hypersensitivity reactions occurring in youth with T1D and a literature review of this topic.

RESULTS: Insulin hypersensitivity reactions included types I, III, and IV with presentations ranging from localized urticaria, erythematous nodules, and eczematous plaques to anaphylaxis with respiratory distress. Reactions occurred in youth with newly diagnosed T1D and in those with long-standing T1D who were using both injection and …

Examining Decisional Needs And Contextual Factors Influencing Fertility Status Assessment Among Young Female Survivors Of Childhood Cancer: A Sequential Mixed Methods Study Protocol, Brooke Cherven, Nataliya V. Ivankova, Jessica B. Spencer, Anne M. Fitzpatrick, Karen C. Burns, Jenna Demedis, Holly R Hoefgen, Ann C. Mertens, James L. Klosky

2020-Current year OA Pubs

INTRODUCTION: Female cancer survivors who received gonadotoxic cancer treatment are at risk for profound diminished ovarian reserve and/or primary ovarian insufficiency with resulting infertility, which can be associated with distress and decreased quality of life.. Despite prioritizing future parenthood, many survivors are unsure of the impact of their treatment on their future fertility, and little is known about the perceived reproductive health needs and factors associated with receipt of a fertility status assessment (FSA). There is a lack of developmentally appropriate reproductive health decisional support interventions available for emerging adult cancer survivors. This study will explore the perceived reproductive health …

Examining Decisional Needs And Contextual Factors Influencing Fertility Status Assessment Among Young Female Survivors Of Childhood Cancer: A Sequential Mixed Methods Study Protocol, Brooke Cherven, Nataliya V. Ivankova, Jessica B. Spencer, Anne M. Fitzpatrick, Karen C. Burns, Jenna Demedis, Holly R. Hoefgen, Ann C. Mertens, James L. Klosky

2020-Current year OA Pubs

INTRODUCTION: Female cancer survivors who received gonadotoxic cancer treatment are at risk for profound diminished ovarian reserve and/or primary ovarian insufficiency with resulting infertility, which can be associated with distress and decreased quality of life.. Despite prioritizing future parenthood, many survivors are unsure of the impact of their treatment on their future fertility, and little is known about the perceived reproductive health needs and factors associated with receipt of a fertility status assessment (FSA). There is a lack of developmentally appropriate reproductive health decisional support interventions available for emerging adult cancer survivors. This study will explore the perceived reproductive health …

Cognition And Education Benefits Of Increased Hemoglobin And Blood Oxygenation In Children With Sickle Cell Disease, Joanna P. Macewan, Allison A. King, Andy Nguyen, Anuj Mubayi, Irene Agodoa, Kim Smith-Whitley

2020-Current year OA Pubs

BACKGROUND: Among individuals with sickle cell disease (SCD), decreased hemoglobin is associated with lower oxygen saturation (SpO2) and increased risk of stroke, both of which are associated with lower intelligence quotient (IQ) scores. Thus, increasing hemoglobin and SpO2 in individuals with SCD may increase IQ and educational attainment.

METHODS: A cohort simulation model was built to determine academic performance and educational attainment based on cognitive function (measured by IQ) of a pediatric SCD cohort randomly assigned to treatment and control groups. The model contained two key stages: childhood (<10 years) and adolescence (≥10 years). In stage 1, increased hemoglobin and increased SpO2 (assigned to the treatment group) were determinants of higher IQ, prevention of IQ deterioration over time. Increased hemoglobin was also a determinant of decreased stroke risk. In stage 2, improvement in adolescent IQ as a result of treatment was a determinant of academic performance.

RESULTS: In a simulated cohort of 2000 children and adolescents with …

American Cochlear Implant Alliance Task Force Guidelines For Clinical Assessment And Management Of Adult Cochlear Implantation For Single-Sided Deafness, Margaret T. Dillon, Armine Kocharyan, Ghazal S. Daher, Matthew L. Carlson, William H. Shapiro, Hillary A. Snapp, Jill B. Firszt

2020-Current year OA Pubs

The indications for cochlear implantation have expanded to include individuals with profound sensorineural hearing loss in the impaired ear and normal hearing (NH) in the contralateral ear, known as single-sided deafness (SSD). There are additional considerations for the clinical assessment and management of adult cochlear implant candidates and recipients with SSD as compared to conventional cochlear implant candidates with bilateral moderate to profound sensorineural hearing loss. The present report reviews the current evidence relevant to the assessment and management of adults with SSD. A systematic review was also conducted on published studies that investigated outcomes of cochlear implant use on …

A Randomized Phase Ii Study Of Sm-88 Plus Methoxsalen, Phenytoin, And Sirolimus In Patients With Metastatic Pancreatic Cancer Treated In The Second Line And Beyond, Marcus S Noel, Semmie Kim, Marion L Hartley, Steve Wong, Vincent J Picozzi, Harry Staszewski, Dae Won Kim, Jan M Van Tornout, Philip Agop Philip, Vincent Chung, Allyson J Ocean, Andrea Wang-Gillam

2020-Current year OA Pubs

BACKGROUND: This trial explores SM-88 used with methoxsalen, phenytoin, and sirolimus (MPS) in pretreated metastatic pancreatic ductal adenocarcinoma (mPDAC) METHODS: Forty-nine patients were randomized to daily 460 or 920 mg oral SM-88 with MPS (SM-88 Regimen). The primary endpoint was objective response rate (RECIST 1.1).

RESULTS: Thirty-seven patients completed ≥ one cycle of SM-88 Regimen (response evaluable population). Disease control rate (DCR), overall survival (OS), and progression-free survival (PFS) did not differ significantly between dose levels. Stable disease was achieved in 9/37 patients (DCR, 24.3%); there were no complete or partial responses. Quality-of-life (QOL) was maintained and trended in favor …

Radiotherapy For Elderly Patients With Glioblastoma: An Assessment Of Hypofractionation And Modern Treatment Techniques, Jennifer K. Matsui, Jiayi Huang, Et Al.

2020-Current year OA Pubs

Glioblastoma (GBM) is a disease with a poor prognosis. For decades, radiotherapy has played a critical role in the management of GBM. The standard of care radiation prescription is 60 Gy in 30 fractions, but landmark trials have historically excluded patients older than 70 years. Currently, there is considerable variation in the management of elderly patients with GBM. Shortened radiation treatment (hypofractionated) regimens have been explored since conventional treatment schedules are lengthy and many elderly patients have functional, cognitive, and social limitations. Clinical trials have demonstrated the effectiveness of hypofractionated radiotherapy (40 Gy in 15 fractions) to treat elderly or …

Health-Related Quality Of Life In Older Patients With Heart Failure From Before To Early After Advanced Surgical Therapies: Findings From The Sustain-It Study, Kathleen L. Grady, Justin Hartupee, Et Al.

2020-Current year OA Pubs

BACKGROUND: Restoring health-related quality of life (HRQOL) is a therapeutic goal for older patients with advanced heart failure. We aimed to describe change in HRQOL in older patients (60-80 years) awaiting heart transplantation (HT) with or without pretransplant mechanical circulatory support (MCS) or scheduled for long-term MCS, if ineligible for HT, from before to 6 months after these surgeries and identify factors associated with change.

METHODS: Patients from 13 US sites completed the EuroQol 5-dimension 3L questionnaire and Kansas City Cardiomyopathy Questionnaire-12 at baseline and 3 and 6 months after HT or long-term MCS. Analyses included univariate comparisons and multivariable …