Medical Specialties Commons^™

A Systematic Review On Belimumab’S Effectiveness, Improved Health Outcomes And Quality Of Life In Patients With Lupus Syndromes, Emily Meale, Alexandra Fontaine

Rowan-Virtua Research Day

Autoimmune diseases are prevalent disorders that many individuals within the United States suffer from. Systemic lupus erythematosus (SLE) is a common autoimmune disease that has a high incidence in women of child-bearing age. Management for lupus depends on the anatomical location SLE manifests, the severity of disease, and the tolerance that one has for certain adverse effects. Glucocorticoids are commonly used to manage lupus. Currently, long term glucocorticoid usage is associated with devastating adverse effects, so current treatment options aim to mitigate corticosteroids use to improve quality of life while maintaining favorable health outcomes. New management for SLE include biologics …

Preventing Postoperative Cognitive Dysfunction Through Preoperative Exercise, John Andre Socci

Rowan-Virtua Research Day

Introduction: Postoperative cognitive dysfunction (POCD) is a common problem in the elderly population after undergoing anesthesia that results in sometimes irreversible cognitive and physical impairment that can increase mortality and decrease quality of life.

Methods: To see the relationship between preoperative fitness capacity and how this relates to postoperative cognitive ability a literature review was performed. Studies that were included involved populations older than sixty years old that had undergone anesthesia. Additionally, animal studies were included to show inflammatory marker levels and how they increased in relation to physical activity.

Results: Various fitness scales were used such as the Timed …

The Efficacy Of Baricitinib As A Treatment Option For Alopecia, In Terms Of Promoting Hair Regrowth And Improving Patient Reported Outcomes, Bhaumik Patel, Abhiram Gollapudi, Samrat Gollapudi, Srinidhi Banala, Brian Joseph Mathew, Prince Patel

Rowan-Virtua Research Day

Background: Alopecia, an autoimmune condition characterized by hair loss, significantly impacts millions globally, often leading to psychological distress such as anxiety and depression. Current treatments are variably effective and frequently associated with adverse effects that restrict long-term use. This study evaluates the efficacy of Baricitinib, a Janus Kinase inhibitor, in treating severe alopecia, focusing on hair regrowth and patient-reported outcomes.

Methods: This research synthesized data from recent publications (2018-2023) including clinical trials, reviews, meta-analyses, and systematic reviews, sourced from databases such as PubMed, Springer, and Elsevier. The inclusion criteria focused on recent studies in English evaluating Baricitinib's effectiveness in severe …

Interprofessional Team Collaboration To Study Integrated Behavioral Health Services In Primary Care, Priya Srivastava, Sindhura Nemani, Danielle Schweitzer, Anne C. Jones

Rowan-Virtua Research Day

• In Rowan Family Medicine (Sewell, NJ), an interprofessional team of medical and clinical psychology professionals and trainees collaborate to investigate the integration of behavioral health in patients’ primary care visits.

• We screen patients for PTSD symptoms using the DSM CAPS-5 during their primary care visits

• The PTSD Coach app is then encouraged to use 3x a day

• By making mental health care more accessible to patients, we strive to help meet the increased demand and alleviate the strain our healthcare system is currently experiencing.

• The team composed of Medicine and Clinical Psychology allows students to gain invaluable experience in interdisciplinary …

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Effects Of Obesity On Patients Undergoing Cardiomems Procedure: Retrospective Cohort Study, Tony Elias, Mena Elmalh, Priya Brahmbhatt, Kyrillos Girgis, Taha Syed, Michael Valderrama, Rafail Beshai

Rowan-Virtua Research Day

★ Remote hemodynamic monitoring (CardioMEMs) is becoming increasingly important for management of chronic heart failure patients.

★ There is limited data looking specifically for obese patients who undergo CardioMEMs implantation.

★ We sought to examine the national inpatient sample database to describe in-hospital outcomes for this demographic

Sex Differences In Outcomes Among Patients With High-Output Heart Failure: A Retrospective Cohort Study, Tony Elias, Mena Elmalh, Priya Brahmbhatt, Kyrillos Girgis, Taha Syed, Michael Valderrama, Rafail Beshai

Rowan-Virtua Research Day

❖ There is limited data specifically looking for sex differences in patients with high output heart failure (HOHF).

❖ We sought to examine the national inpatient sample database to describe in-hospital outcomes among female patients.

Clinical Outcomes For Impella Procedure In Octogenarians, Nonagenarians, And Centenarians: A Retrospective Cohort Study, Tony Elias, Sonika Vatsa, Kyrillos Girgis, Michael Valderrama, Rafail Beshai

Rowan-Virtua Research Day

The Impella, a vital ventricular assist device, has seen extensive use in managing severe heart failure and cardiogenic shock. However, the impact of this procedure on older individuals remains poorly understood. To address this gap, we scrutinized the National Inpatient Sample Database from 2019 to 2020 to elucidate in-hospital outcomes among older patients who underwent the Impella procedure. Among the 8233 patients who underwent Impella insertion, 1389 (16.8%) were in the older population, aged 80 years or older. This subgroup exhibited a higher prevalence of comorbidities such as hypertension, diabetes mellitus, and chronic kidney disease compared to younger patients. Alarmingly, …

Examining The Differences In Functional Outcomes For Patients With Traumatic Brain Injury After Acute Rehabilitation And Subacute Rehabilitation: A Review Of The Literature, Michael Rangel, Ashley Kakkanatt

Rowan-Virtua Research Day

BACKGROUND/PURPOSE

Traumatic brain injury (TBI) is one of the major causes of morbidity in the US [1-4]. Studies suggest TBI patients’ enrollment in a rehabilitation program strongly predicts social reintegration. The structure and outcomes of inpatient rehabilitation programs, including acute and subacute rehabilitation, are not well-delineated [5-7].

DESIGN

A structured review of current literature was conducted. Articles were screened using the search string: (post-traumatic brain injury OR post-TBI OR traumatic brain injury OR TBI) AND (acute rehabilitation OR subacute rehabilitation) AND (outcomes OR functional outcomes) NOT (outpatient OR outpatient populations) NOT (pediatric OR pediatric populations). Articles were limited to 1990 …

A Literature Review Of Current Treatments For The Hypermobility Subtype Of Ehlers-Danlos Syndrome, John Gericke, Mary Zsolway, Chelsea Reyes, Pooja Patel, Saad Ahmed, Julia Hwang, Venkateswar Venkataraman

Rowan-Virtua Research Day

Purpose: The purpose of this study is to compare pharmacologic intervention, neurocognitive therapy, physical therapy, and orthotics in treating the hypermobility subtype of Ehlers-Danlos Syndrome (hEDS) and determine which has the most positive effect on symptoms.

Introduction: Ehlers-Danlos Syndrome is an inheritable connective tissue disorder which results from a genetic mutation that alters the body’s ability to produce collagen. The most common subtype of Ehlers-Danlos Syndrome is hEDS, which leads to hypermobility and hyperextensibility and can cause frequent joint dislocations.

Methods: A review of literature was performed to compare each treatment based on reported results. The types of studies reviewed …

Comparative Analysis Of Health Outcomes In Gastric Cancer: Robotic Vs. Laparoscopic Gastrectomy, Maftuna Kurbonnazarova

Rowan-Virtua Research Day

Exploring the most effective surgical approach for gastrectomy is crucial for determining superior health outcomes in patients. This research compares robotic and laparoscopic techniques with the aim of pinpointing the optimal method, offering valuable insights for improved postoperative well-being.

How Psychological And Nonpharmacological Treatment Modalities Reduce The Disease Burden Of Amplified Musculoskeletal Pain Syndromes In Pediatrics, Aalia Narvel, Sabrina Gmuca

Rowan-Virtua Research Day

The prevalence of pediatric chronic musculoskeletal pain ranges from 8.5% to 40%.¹ This literature review examined the treatment of amplified musculoskeletal pain syndrome (AMPS), which is a general term that represents a spectrum of pain disorders for patients who may not satisfy specific diagnostic criteria.² Specifically, sources were analyzed to compare the efficacy of individual components in the nonpharmacological, interdisciplinary treatment of AMPS against pain medications. These components included cognitive behavioral psychotherapy (CBT), physical therapy (PT), as well as occupational therapy (OT). There is limited information that assesses these components individually, but there is ample evidence that when …

Neoadjuvant Versus Adjuvant Therapy For Stage Iiib-Iiid Melanoma, Bhumik Patel, Sangnya Upadhyaya

Rowan-Virtua Research Day

The treatment landscape for advanced stage melanoma is rapidly evolving due to advancements in our understanding of melanoma biology and the emergence of novel therapies. This necessitates a comprehensive review to guide clinicians in adopting evidence based and patient centric approaches to treat stage IIIB-IIID melanoma. A literature review was conducted to synthesize current information on the most optimal treatment available. Data available from different clinical trials found that neoadjuvant therapy was a more effective treatment compared to adjuvant therapies alone. Furthermore, neoadjuvant therapy with combination therapy was more efficacious in producing a complete pathological response compared to monotherapy. A …

Comprehensive Atrial Fibrillation Burden And Symptom Reduction Post-Ablation: Insights From Decaaf Ii, Charbel Noujaim, Ala Assaf, Chanho Lim, Han Feng, Hadi Younes, Mario Mekhael, Nour Chouman, Ghaith Shamaileh, Abdel Hadi El Hajjar, Tarek Ayoub, Nino Isakadze, Mihail G Chelu, Nassir Marrouche, Eoin Donnellan

Faculty, Staff and Students Publications

Aims: Traditional atrial fibrillation (AF) recurrence after catheter ablation is reported as a binary outcome. However, a paradigm shift towards a more granular definition, considering arrhythmic or symptomatic burden, is emerging. We hypothesize that ablation reduces AF burden independently of conventional recurrence status in patients with persistent AF, correlating with symptom burden reduction.

Methods and results: Ninety-eight patients with persistent AF from the DECAAF II trial with pre-ablation follow-up were included. Patients recorded daily single-lead electrocardiogram (ECG) strips, defining AF burden as the proportion of AF days among total submitted ECG days. The primary outcome was atrial arrhythmia recurrence. The …

Response Of Treatment-Naive Brain Metastases To Stereotactic Radiosurgery, Chibawanye I Ene, Christina Abi Faraj, Thomas H Beckham, Jeffrey S Weinberg, Clark R Andersen, Ali S Haider, Ganesh Rao, Sherise D Ferguson, Christopher A Alvarez-Brenkenridge, Betty Y S Kim, Amy B Heimberger, Ian E Mccutcheon, Sujit S Prabhu, Chenyang Michael Wang, Amol J Ghia, Susan L Mcgovern, Caroline Chung, Mary Frances Mcaleer, Martin C Tom, Subha Perni, Todd A Swanson, Debra N Yeboa, Tina M Briere, Jason T Huse, Gregory N Fuller, Frederick F Lang, Jing Li, Dima Suki, Raymond E Sawaya

Faculty, Staff and Student Publications

With improvements in survival for patients with metastatic cancer, long-term local control of brain metastases has become an increasingly important clinical priority. While consensus guidelines recommend surgery followed by stereotactic radiosurgery (SRS) for lesions >3 cm, smaller lesions (≤3 cm) treated with SRS alone elicit variable responses. To determine factors influencing this variable response to SRS, we analyzed outcomes of brain metastases ≤3 cm diameter in patients with no prior systemic therapy treated with frame-based single-fraction SRS. Following SRS, 259 out of 1733 (15%) treated lesions demonstrated MRI findings concerning for local treatment failure (LTF), of which 202 /1733 (12%) …

Three-Year Outcomes Of The Abre Venous Self-Expanding Stent System In Patients With Symptomatic Iliofemoral Venous Outflow Obstruction., Stephen Black, Marc Sapoval, David J Dexter, Kathleen Gibson, Raghu Kolluri, Mahmood Razavi, Dorian J Defreitas, Hong Wang, Stephanie Brucato, Erin Murphy

Heart and Vascular Articles

PURPOSE: To report 36-month outcomes and subgroup analysis of the ABRE study evaluating the safety and effectiveness of the Abre venous self-expanding stent system for the treatment of symptomatic iliofemoral venous outflow obstruction disease.

METHODS: The ABRE study was a prospective, multicenter, nonrandomized study that enrolled and implanted Abre venous stents in 200 participants (mean age 51.5 years [SD ± 15.9], 66.5% women) with symptomatic iliofemoral venous outflow obstruction at 24 global sites. Outcomes assessed through 36 months included patency, major adverse events, stent migration, stent fracture, and quality-of-life changes. Adverse events and imaging studies were adjudicated by independent clinical …

Neoadjuvant Chemoimmunotherapy For Nsclc: A Systematic Review And Meta-Analysis, Mark Sorin, Connor Prosty, Louis Ghaleb, Kathy Nie, Khaled Katergi, Muhammad H Shahzad, Laurie-Rose Dubé, Aline Atallah, Anikka Swaby, Matthew Dankner, Trafford Crump, Logan A Walsh, Pierre O Fiset, Boris Sepesi, Patrick M Forde, Tina Cascone, Mariano Provencio, Jonathan D Spicer

Faculty, Staff and Student Publications

IMPORTANCE: To date, no meta-analyses have comprehensively assessed the association of neoadjuvant chemoimmunotherapy with clinical outcomes in non-small cell lung cancer (NSCLC) in randomized and nonrandomized settings. In addition, there exists controversy concerning the efficacy of neoadjuvant chemoimmunotherapy for patients with NSCLC with programmed cell death 1 ligand 1 (PD-L1) levels less than 1%.

OBJECTIVE: To compare neoadjuvant chemoimmunotherapy with chemotherapy by adverse events and surgical, pathological, and efficacy outcomes using recently published randomized clinical trials and nonrandomized trials.

DATA SOURCES: MEDLINE and Embase were systematically searched from January 1, 2013, to October 25, 2023, for all clinical trials of …

Mechanical Venous Thrombectomy For Deep Venous Thrombosis In Cancer Patients: A Single-Center Retrospective Study, Riya M Patel, Koustav Pal, Syed Hadi Ahmed, Joshua D Kuban, Milan Patel, Ketan Shah, Peiman Habibollahi, Zeyad Metwalli, Varshana Gurusamy, Sanjay Gupta, Cristhiam M Rojas-Hernandez, Vahid Afshar-Kharghan, Michael H Kroll, Rahul A Sheth

Faculty, Staff and Student Publications

PURPOSE: Venous thromboembolism (VTE) is a major contributor to the mortality of cancer patients. Mechanical thrombectomy (MT) is an endovascular technique that physically removes a thrombus without thrombolytics. The purpose of this study was to evaluate safety, efficacy, and clinical outcomes following MT for lower extremity DVT in cancer patients.

METHODS: This single-center, retrospective study evaluated outcomes following MT of lower extremity DVT in cancer patients from November 2019 to May 2023. The primary outcome measure was clinical success, defined as a decrease in Villalta score by at least 2 points following the intervention. Secondary outcomes included repeat intervention-free survival …

Clofarabine Monotherapy In Aggressive, Relapsed And Refractory Langerhans Cell Histiocytosis, Deevyashali Parekh, Howard Lin, Akanksha Batajoo, Erin Peckham-Gregory, Vivekanudeep Karri, Whitney Stanton, Brooks Scull, Ryan Fleishmann, Nader El-Mallawany, Olive S Eckstein, Zachary D Prudowsky, Nitya Gulati, Jennifer E Agrusa, Asra Z Ahmed, Roland Chu, Matthew S Dietz, Stanton C Goldman, Michael D Hogarty, Hamayun Imran, Stefanos Intzes, Jenny M Kim, Lisa M Kopp, Carolyn Fein Levy, Philip Neff, Pallavi M Pillai, Bryan A Sisk, Deborah E Schiff, Angela D Trobaugh-Lotrario, Kelly Walkovich, Kenneth L Mcclain, Carl E Allen

Faculty, Staff and Students Publications

Over 50% of patients with systemic LCH are not cured with front-line therapies and data to guide salvage options are limited. We describe 58 patients with LCH who were treated with clofarabine. Clofarabine monotherapy was active against LCH in this cohort including heavily pretreated patients with systemic objective response rate of 92.6%, higher in children (93.8%) than adults (83.3%). BRAFV600E+ variant allele frequency in peripheral blood correlated with positive clinical responses. Prospective multi-center trials are warranted to determine optimal dosing, long-term efficacy, late toxicities, relative cost and patient reported outcomes of clofarabine compared to alternative LCH salvage therapy strategies.

Examining The Relationship Between Anxiety Severity And Autism-Related Challenges During Cognitive Behavioral Therapy For Children With Autism, Madeleine N Fuselier, Andrew G Guzick, Jafar Bakhshaie, Jeffrey J Wood, Philip C Kendall, Connor M Kerns, Brent J Small, Wayne K Goodman, Eric A Storch

Faculty, Staff and Students Publications

PURPOSE: Using data from a randomized clinical trial evaluating cognitive behavioral therapy (CBT) for children with autism and co-occurring anxiety, this study examined the relationship between autism features and anxiety symptoms throughout CBT.

METHODS: Two multilevel mediation analyses were run which examined the mediating role of changes in anxiety for changes in two core features of autism, (a) repetitive and restrictive behaviors (RRBs) and (b) social communication/interaction impairments, between pre- and post-treatment.

RESULTS: Indirect effects between time and autism characteristics were significant for both models, indicating that as anxiety changes, so do RRBs and social communication/interaction as the outcomes respectively. …

Parent-Led Cognitive Behavioral Teletherapy For Anxiety In Autistic Youth: A Randomized Trial Comparing Two Levels Of Therapist Support, Andrew G Guzick, Sophie C Schneider, Minjee Kook, Julia Rose Iacono, Saira A Weinzimmer, Troy Quast, Sean M Olsen, Killian Rainey Hughes, Emily Jellinek-Russo, Amanda Perozo Garcia, Abigail Candelari, Leandra N Berry, Robin P Goin-Kochel, Wayne K Goodman, Eric A Storch

Center for Medical Ethics and Health Policy Staff Publications

Parent-led cognitive behavioral therapy (CBT) is an efficient, promising form of therapy that may be well suited for autistic youth with anxiety disorders, though to date it has been minimally tested. In this study, 87 autistic youth (7 to 13 years old) with anxiety disorders and their parents were randomized to two forms of parent-led CBT in which parents led their child through a guided CBT workbook across 12 weeks: one with low therapist contact (four 30-minute telehealth calls), and one with standard therapist contact (ten 60-minute telehealth calls). Anxiety, functional impairment, and autism features significantly declined across therapy, without …

Clinical Outcomes After Acute Coronary Syndromes Or Revascularization Among People Living With Hiv: A Systematic Review And Meta-Analysis, Mohammed Haji, Michael Capilupi, Michael Kwok, Nouran Ibrahim, Gerald S Bloomfield, Christopher T Longenecker, Maria C Rodriguez-Barradas, Chester N Ashong, Eric Jutkowitz, Tracey H Taveira, Michelle Richard, Jennifer L Sullivan, James L Rudolph, Wen-Chih Wu, Sebhat Erqou

Faculty, Staff and Students Publications

IMPORTANCE: Clinical outcomes after acute coronary syndromes (ACS) or percutaneous coronary interventions (PCIs) in people living with HIV have not been characterized in sufficient detail, and extant data have not been synthesized adequately.

OBJECTIVE: To better characterize clinical outcomes and postdischarge treatment of patients living with HIV after ACS or PCIs compared with patients in an HIV-negative control group.

DATA SOURCES: Ovid MEDLINE, Embase, and Web of Science were searched for all available longitudinal studies of patients living with HIV after ACS or PCIs from inception until August 2023.

STUDY SELECTION: Included studies met the following criteria: patients living with …

Chlorthalidone Vs Hydrochlorothiazide For Hypertension Treatment After Myocardial Infarction Or Stroke: A Secondary Analysis Of A Randomized Clinical Trial, Areef Ishani, Cynthia Hau, William C Cushman, Sarah M Leatherman, Robert A Lew, Peter A Glassman, Addison A Taylor, Ryan E Ferguson

Faculty, Staff and Students Publications

IMPORTANCE: Patients with prior myocardial infarction (MI) or stroke have a greater risk of recurrent cardiovascular (CV) events.

OBJECTIVE: To evaluate the association of chlorthalidone (CTD) vs hydrochlorothiazide (HCTZ) with CV outcomes and noncancer deaths in participants with and without prior MI or stroke.

DESIGN, SETTING, AND PARTICIPANTS: This was a prespecified secondary analysis of the Diuretic Comparison Project (DCP), a pragmatic randomized clinical trial conducted within 72 participating Veterans Affairs health care systems from June 2016 to June 2021, in which patients aged 65 years or older with hypertension taking HCTZ at baseline were randomized to continue HCTZ or …

Clinical, Radiological And Molecular Responses To Combination Chemotherapy With Mapk Pathway Inhibition In Relapsed And Refractory Langerhans Cell Histiocytosis, Vivekanudeep Karri, Howard Lin, Jessica Velazquez, Akanksha Batajoo, Deevyashali Parekh, Whitney Stanton, Harshal Abhyankar, Nader K El-Mallawany, Jennifer Agrusa, Olive Eckstein, Nitya Gulati, Jeffrey Schwartz, Wendy Woods-Swafford, Jaime Boyd, Anikit Saha, Carl E Allen, Kenneth L Mcclain

Faculty, Staff and Students Publications

Optimal therapeutic approaches for advanced Langerhans cell histiocytosis (LCH) are not known. We assessed the safety and efficacy of combined chemotherapy with MAPK pathway inhibition in 10 patients with refractory systemic disease and/or LCH-associated neurodegeneration. Overall response rate was 9/10 (90%) for the entire cohort: 5/5 (100%) for patients with systemic disease and 6/7 (86%) for patients with central nervous system disease. BRAFV600E+ peripheral blood fraction decreased in 5/6 (83%). Toxicities included fever, skin rash, myalgias, neuropathy, cytopenias and hypocalcaemia. Prospective trials are required to optimize combination strategies, determine potential to achieve cure and compare outcomes to chemotherapy or MAPK …

Robotic-Assisted Single-Site Abdominal Cerclage In The Bicornuate Uterus Patient With Cervical Insufficiency, Nurul Farhanah Binte Abdul Latif, Zhenkun Guan, Sylvia Asare, Xiaoming Guan

Faculty, Staff and Students Publications

Objective: To demonstrate the surgical techniques for improving safety in robotic-assisted abdominal cerclage in patients with bicornuate uteri complicated by recurrent pregnancy loss and cervical insufficiency.

Design: Stepwise demonstration with narrated video footage.

Setting: An academic tertiary care hospital.

Patients: Our patient is a 22-year-old G2P0020 with a history of recurrent pregnancy loss. During her first pregnancy, she was asymptomatic until 19 weeks and delivered because of a preterm premature rupture of membranes. A transvaginal cerclage was performed for her second pregnancy at 14 weeks, which ended at 16 weeks because of preterm premature rupture of membranes. The final magnetic …

Management And Outcomes Of Patients With Refractory Solitary Plasmacytoma After Treatment With Definitive Radiation Therapy, Penny Fang, Chelsea C Pinnix, Susan Y Wu, Hans C Lee, Krina K Patel, Neeraj Saini, Melody R Becnel, Gregory Kaufman, Sheeba K Thomas, Robert Z Orlowski, Behrang Amini, Pei Lin, Bouthaina S Dabaja, Jillian R Gunther

Faculty, Staff and Student Publications

Purpose: Radiation therapy (RT) is the standard treatment for solitary plasmacytoma (SP); however, the optimal management of RT-refractory SPs is unknown. We examined outcomes after early systemic therapy, surgical resection, or observation for patients with RT-refractory disease and assessed the potential impact of treatment selection on disease outcomes.

Methods and materials: We retrospectively reviewed patients with SP treated with definitive radiation and evaluated at a single institution with persistent disease on imaging or biopsy. Descriptive statistics were used to characterize patient and disease characteristics and treatment outcomes.

Results: Of 102 total SP patients, 17 (17%) were RT-refractory. The median RT …

Angiogenic Gene Therapy For Refractory Angina: Results Of The Exact Phase 2 Trial, Kenta Nakamura, Timothy D Henry, Jay H Traverse, David A Latter, Nahush A Mokadam, Geoffrey A Answini, Adam R Williams, Benjamin C Sun, Christopher R Burke, Faisal G Bakaeen, Marcelo F Dicarli, Bernard R Chaitman, Mark W Peterson, Dawn G Byrnes, E Magnus Ohman, Carl J Pepine, Ronald G Crystal, Todd K Rosengart, Elaine Kowalewski, Gary G Koch, Howard C Dittrich, Thomas J Povsic, Exact Trial Investigators

Faculty, Staff and Students Publications

Background: XC001 is a novel adenoviral-5 vector designed to express multiple isoforms of VEGF (vascular endothelial growth factor) and more safely and potently induce angiogenesis. The EXACT trial (Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment) assessed the safety and preliminary efficacy of XC001 in patients with no option refractory angina.

Methods: In this single-arm, multicenter, open-label trial, 32 patients with no option refractory angina received a single treatment of XC001 (1×1011 viral particles) via transepicardial delivery.

Results: There were no severe adverse events attributed to the study drug. Twenty expected severe adverse events in 13 patients …

Tenapanor As Therapy For Hyperphosphatemia In Maintenance Dialysis Patients: Results From The Optimize Study, Stuart M Sprague, Daniel E Weiner, David P Tietjen, Pablo E Pergola, Steven Fishbane, Geoffrey A Block, Arnold L Silva, Stephen Z Fadem, Robert I Lynn, George Fadda, Lynae Pagliaro, Suling Zhao, Susan Edelstein, David M Spiegel, David P Rosenbaum

Faculty, Staff and Students Publications

KEY POINTS: Tenapanor, a first-in-class local inhibitor of sodium/hydrogen exchanger isoform 3, acts as a phosphate absorption inhibitor by decreasing paracellular phosphate absorption. Tenapanor alone or with phosphate binders achieved

BACKGROUND: OPTIMIZE was a randomized, open-label study evaluating different tenapanor initiation methods. OPTIMIZE evaluated tenapanor alone and in combination with phosphate binders (PBs) to achieve target serum phosphate (P) ≤5.5 mg/dl.

METHODS: Patients with inadequately controlled P receiving maintenance dialysis from 42 US locations who were taking PBs with baseline

RESULTS: By part A end point, 34.4% (

CONCLUSIONS: Tenapanor as monotherapy or in combination with PBs effectively lowered P …

Safety And Efficacy Of Ivacaftor In Infants Aged 1 To Less Than 4 Months With Cystic Fibrosis., Paul Mcnally, Alvin Singh, Susanna A. Mccolley, Jane C. Davies, Mark Higgins, Meng Liu, Jennifer Lu, Violeta Rodriguez-Romero, Judy L. Shih, Margaret Rosenfeld, Vx15-770-124 Study Group

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Ivacaftor (IVA) has been shown to be safe and efficacious in children aged ≥4 months with cystic fibrosis (CF) and CFTR gating variants. We evaluated safety, pharmacokinetics (PK), and efficacy of IVA in a small cohort of infants aged 1 toCF.

METHODS: In this phase 3, open-label study, infants 1 toinfants, doses were adjusted at day 15 to better match median adult exposures based on individual PK measurements taken on day 4. Primary endpoints were safety and PK measurements.

RESULTS: Seven infants (residual function CFTR variants [n=5]; minimal function CFTR variants [n=2]) received ≥1 dose of IVA. Six infants …

Impact Of Revised International Staging System 2 Risk Stratification On Outcomes Of Patients With Multiple Myeloma Receiving Autologous Haematopoietic Stem Cell Transplantation, Kamal Alzahrani, Oren Pasvolsky, Zhongya Wang, Denái R Milton, Mark R Tanner, Qaiser Bashir, Samer Srour, Neeraj Saini, Paul Lin, Jeremy Ramdial, Yago Nieto, Hans C Lee, Krina K Patel, Elisabet E Manasanch, Partow Kebriaei, Sheeba K Thomas, Donna M Weber, Robert Z Orlowski, Elizabeth J Shpall, Richard Champlin, Muzaffar H Qazilbash

Faculty, Staff and Student Publications

The second revision of the International Staging System (R2-ISS) is a simple tool to risk-stratify newly diagnosed multiple myeloma (NDMM) patients. Here, we completed a retrospective analysis to evaluate the utility of R2-ISS in NDMM patients who underwent up-front autologous haematopoietic stem cell transplantation (auto-HCT). A total of 1291 patients were included, with a median age of 62 years (range 29-83). The distribution of R2-ISS stages was: 123 (10%) stage I, 471 (36%) stage II, 566 (44%) stage III and 131 (10%) stage IV. With a median follow-up of 42.2 months (range 0.3-181.0), the median PFS was 73.0, 65.2, 44.0 …

Impact Of Pretransplant Minimal Residual Disease In Patients With Multiple Myeloma And A Very Good Partial Response Or Better Receiving Autologous Hematopoietic Stem Cell Transplantation, Oren Pasvolsky, Sarah Pasyar, Roland L Bassett, Hina N Khan, Mark R Tanner, Qaiser Bashir, Samer Srour, Neeraj Saini, Paul Lin, Jeremy Ramdial, Yago Nieto, Hans C Lee, Krina K Patel, Partow Kebriaei, Sheeba K Thomas, Donna M Weber, Robert Z Orlowski, Elizabeth J Shpall, Richard E Champlin, Muzaffar H Qazilbash

Faculty, Staff and Student Publications

Background: The prognostic significance of minimal residual disease (MRD) status before autologous hematopoietic stem cell transplantation (autoHCT) in patients with multiple myeloma (MM) has not been clearly elucidated.

Methods: Retrospective single-center study of adult MM patients who achieved ≥very good partial response (VGPR) after induction therapy from 2015 to 2021 received upfront autoHCT and had available pretransplant MRD status by next-generation flow cytometry. The cohort was divided into pretransplant MRD-negative (MRDneg) and MRD-positive (MRDpos) groups.

Results: A total of 733 patients were included in our analysis; 425 were MRDneg and 308 MRDpos at autoHCT. In the MRDpos group, more patients …