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Full-Text Articles in Therapeutics

Pruritus Associated With Systemic Sclerosis: A Systematic Review Of Treatment-Based Clinical Trials, Kiana Mortezaei, Rishika Pasula, Meryem Nursoy, Amit Syal, Ioannis Panayiotou, Geeta Kumari, Andreas Chatzitoffis, Pavle Milutinovic, Olajumoke O. Fadugba, Chris T. Derk Jun 2026

Pruritus Associated With Systemic Sclerosis: A Systematic Review Of Treatment-Based Clinical Trials, Kiana Mortezaei, Rishika Pasula, Meryem Nursoy, Amit Syal, Ioannis Panayiotou, Geeta Kumari, Andreas Chatzitoffis, Pavle Milutinovic, Olajumoke O. Fadugba, Chris T. Derk

Student Papers, Posters & Projects

This paper aims to examine the latest research on treatments for itch (pruritus) in people with systemic sclerosis (SSc), identify areas where information is lacking, and propose suggestions for future studies.A comprehensive literature search was performed in PubMed and Scopus in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines to identify studies evaluating therapeutic interventions for pruritus in SSc. Eligible studies included randomized controlled trials, cohort studies, and case series comprising a minimum of three patients. Data extraction encompassed study design, patient demographics, pruritus assessment instruments including validated and non-validated measures, and clinical outcomes. Studies …


Safety And Efficacy Of Individualised Exercise And Nad+ Precursor Supplementation In Patients With Friedreich’S Ataxia In The Usa: A Single-Centre, 2 × 2 Factorial, Randomised Controlled Trial, Kimberly Lin, Anna Bucha, Kara Mcsweeney, Kristin Wade, Antoneta Karaj, Jaclyn Tamaroff, Shannon O'Malley, Nicole Chung, Nicolette Cilenti, Julianne Wanner, Gabriel Adzika, Clementina Mesaros, Ian Blair, Teerapat Rojsajjakul, Suraj Serai, Jennifer Farmer, Kyle Bryant, Yingying Lu, Michael Harhay, David Weber, Stephen Paridon, Erin Seifert, Mary Putt, Payman Zamani, Joseph Baur, David Lynch, Shana Mccormack May 2026

Safety And Efficacy Of Individualised Exercise And Nad+ Precursor Supplementation In Patients With Friedreich’S Ataxia In The Usa: A Single-Centre, 2 × 2 Factorial, Randomised Controlled Trial, Kimberly Lin, Anna Bucha, Kara Mcsweeney, Kristin Wade, Antoneta Karaj, Jaclyn Tamaroff, Shannon O'Malley, Nicole Chung, Nicolette Cilenti, Julianne Wanner, Gabriel Adzika, Clementina Mesaros, Ian Blair, Teerapat Rojsajjakul, Suraj Serai, Jennifer Farmer, Kyle Bryant, Yingying Lu, Michael Harhay, David Weber, Stephen Paridon, Erin Seifert, Mary Putt, Payman Zamani, Joseph Baur, David Lynch, Shana Mccormack

Department of Pathology, Anatomy, and Cell Biology Faculty Papers

BACKGROUND:Friedreich's ataxia is a rare, chronic, progressive, neurodegenerative condition affecting multiple organ systems, including neurological, musculoskeletal, cardiac, and endocrine systems, and is marked by low cardiopulmonary fitness. We tested the effect of exercise and NAD+ precursor supplementation with nicotinamide riboside, which have each shown benefits in animal and early clinical studies, on cardiopulmonary fitness in individuals with Friedreich's ataxia.

METHODS: This 12-week, outpatient, phase 2, single-site (Children's Hospital of Philadelphia, Philadelphia, PA, USA), randomised, 2 × 2 factorial clinical trial recruited individuals aged 10–40 years with an ejection fraction of 45% or greater who were able to exercise. A …


P-15 Peptide Enhanced Bone Graft Improves Time To Fusion In Transforaminal Lumbar Interbody Fusion: A Randomized, Controlled, Investigational Device Exemption Study, James S. Harrop, Michael P. Steinmetz, John E. O'Toole, Christopher D Chaput, Rick C. Sasso, K. Brandon Strenge, Greg Maislin, Jeffrey P. Mullin, Thomas B. Freeman, Anthony Guanciale, Howard Lantner, Michael E. Janssen, David G. Schwartz, John M. Small, Wellington K. Hsu, Paul M. Arnold Dec 2025

P-15 Peptide Enhanced Bone Graft Improves Time To Fusion In Transforaminal Lumbar Interbody Fusion: A Randomized, Controlled, Investigational Device Exemption Study, James S. Harrop, Michael P. Steinmetz, John E. O'Toole, Christopher D Chaput, Rick C. Sasso, K. Brandon Strenge, Greg Maislin, Jeffrey P. Mullin, Thomas B. Freeman, Anthony Guanciale, Howard Lantner, Michael E. Janssen, David G. Schwartz, John M. Small, Wellington K. Hsu, Paul M. Arnold

Department of Neurosurgery Faculty Papers

STUDY DESIGN: Prospective, multicenter, single-blind, randomized, and controlled pivotal study.

OBJECTIVE: Compare time-to-fusion in patients treated with P-15L (PearlMatrix TM P-15 peptide enhanced bone graft) versus local autograft over 24 months and evaluate changes in pain and quality of life at 24 months relative to baseline.

SUMMARY OF BACKGROUND DATA: P-15L, an FDA-designated breakthrough device, is a composite bone graft with P-15, a 15-amino acid polypeptide that promotes cellular adhesion, proliferation, and differentiation to support bone formation.

METHODS: Patients (22-80 y) with degenerative disc disease were randomized to the investigational (P-15L) or control (local autograft) group during single-level transforaminal lumbar …


Optimizing Pharmacologic Treatment For Neonatal Opioid Withdrawal Syndrome (Optimize Now): A Symptom-Based Dosing Approach Study Protocol For A Multi-Center, Cluster Crossover Design Randomized Controlled Trial, Leslie W. Young, Denise C. Babineau, Abhik Das, Sara Demauro, Walter K. Kraft, Scott Lorch, Michele C. Walsh, Stephanie Merhar, Lori A. Devlin Aug 2025

Optimizing Pharmacologic Treatment For Neonatal Opioid Withdrawal Syndrome (Optimize Now): A Symptom-Based Dosing Approach Study Protocol For A Multi-Center, Cluster Crossover Design Randomized Controlled Trial, Leslie W. Young, Denise C. Babineau, Abhik Das, Sara Demauro, Walter K. Kraft, Scott Lorch, Michele C. Walsh, Stephanie Merhar, Lori A. Devlin

Department of Pharmacology, Physiology, and Cancer Biology Faculty Papers

BACKGROUND: Opioid use and misuse during pregnancy rose from 1.5 to 6.5 per 1000 deliveries between 1999 and 2014 and continues as a significant public health concern. A fivefold increase in neonatal opioid withdrawal syndrome (NOWS) has accompanied the increase in opioid use. The Eating, Sleeping, Consoling care approach (ESC) has been shown to improve outcomes for infants with NOWS and is quickly becoming the standard of care for infants affected by opioid use disorder. Quality improvement initiatives following the implementation of ESC provide some evidence to suggest that symptom-based (i.e., as needed, PRN, just in time) dosing of opioid …


Comparative Outcomes Of Coil Embolization And Surgical Clipping In Elderly Patients With Subarachnoid Hemorrhage: A Systematic Review And Meta-Analysis, Yohanna Idsabella Rossi, Gabriel Bolner, Jonathan Costa Dall'acqua, Fabiana Dolovitsch De Oliveira, Lucas V. Zacaria, Taís L. Denicol, Michel Frudit, Natália Vasconcellos De Oliveira Souza Aug 2025

Comparative Outcomes Of Coil Embolization And Surgical Clipping In Elderly Patients With Subarachnoid Hemorrhage: A Systematic Review And Meta-Analysis, Yohanna Idsabella Rossi, Gabriel Bolner, Jonathan Costa Dall'acqua, Fabiana Dolovitsch De Oliveira, Lucas V. Zacaria, Taís L. Denicol, Michel Frudit, Natália Vasconcellos De Oliveira Souza

Department of Neurology Faculty Papers

BACKGROUND AND OBJECTIVES: Elderly patients with subarachnoid hemorrhage (SAH) face a disproportionately high burden of morbidity and mortality. While endovascular coiling is often favored in this population, direct comparisons with surgical clipping are limited. We conducted a meta-analysis to compare outcomes of clipping versus coiling in SAH patients aged ≥ 60 years.

METHODS: A systematic search of PubMed, Embase, and Cochrane databases identified studies comparing the two treatments in this age group. The primary outcome was a composite of unfavorable outcomes (modified Rankin Scale [mRS] > 2 and mortality). Secondary outcomes included mortality, favorable outcome (mRS 0-2), rebleeding, and hospital length …


Predictors Of Final Visual Outcome In Patients With Leber Hereditary Optic Neuropathy Treated With Lenadogene Nolparvovec Gene Therapy, Robert C Sergott, Valerio Carelli, Nancy Newman, Valérie Biousse, Patrick Yu-Wai-Man, Catherine Vignal-Clermont, Constant Josse, Magali Taiel, José-Alain Sahel, Piero Barboni Jul 2025

Predictors Of Final Visual Outcome In Patients With Leber Hereditary Optic Neuropathy Treated With Lenadogene Nolparvovec Gene Therapy, Robert C Sergott, Valerio Carelli, Nancy Newman, Valérie Biousse, Patrick Yu-Wai-Man, Catherine Vignal-Clermont, Constant Josse, Magali Taiel, José-Alain Sahel, Piero Barboni

Wills Eye Hospital Papers

PURPOSE: This exploratory analysis aimed to identify predictive factors of final best-corrected visual acuity (BCVA) in patients with Leber hereditary optic neuropathy (LHON) harboring the m.11778G>A mutation who received lenadogene nolparvovec gene therapy.

METHODS: The following covariates were individually evaluated as possible factors associated with improved final BCVA: age, gender, timing of treatment, baseline BCVA value, and baseline optical coherence tomography (OCT) parameters. Univariate analyses were performed from three phase 3 studies (RESCUE, REVERSE, and REFLECT), using BCVA at 1.5 years post-treatment as the dependent variable.

RESULTS: In 113 eyes treated at least 6 months after disease onset, the …


Systemic Exogenous Progestins With Or Without Estrogens Are Associated With Decreased Rates Of Venous Procedures For Varicose Veins, Paarth Jain, Adam M. Ostrovsky, Paul J Dimuzio, Luis H. Eraso, Michael Nooromid, Dawn Salvatore, Babak Abai Jul 2025

Systemic Exogenous Progestins With Or Without Estrogens Are Associated With Decreased Rates Of Venous Procedures For Varicose Veins, Paarth Jain, Adam M. Ostrovsky, Paul J Dimuzio, Luis H. Eraso, Michael Nooromid, Dawn Salvatore, Babak Abai

Department of Surgery Faculty Papers

OBJECTIVES: Risk factors for varicose veins (VVs) such as female sex, pregnancy, and obesity are high estrogen states, yet the role of systemic progestins with or without estrogens (SPEs) in VV management is not well characterized. This study investigates how SPE use affects rates of venous procedures for patients with VV.

METHODS: The TriNetX database was queried for subjects with International Classification of Diseases, 10th edition, diagnoses of asymptomatic VV, chronic venous insufficiency, and complicated VV (inflammation or ulceration). Patients were divided into a control cohort with no subsequent SPE use, a progestin-only cohort, and a combined estrogen-progestin (CEP) cohort. …


Comparison Between Pipeline Embolization Device (Ped) Versus Flow Redirection Endoluminal Device (Fred) For Intracranial Aneurysms: A Comprehensive Systematic Review And Meta-Analysis, Seyed Maroufi, Mohammad Fallahi, Muhammad Waqas, Othman Bin-Alamer, Manisha Koneru, Joanna Roy, Jane Khalife, Hamza Shaikh, Daniel Tonetti Jun 2025

Comparison Between Pipeline Embolization Device (Ped) Versus Flow Redirection Endoluminal Device (Fred) For Intracranial Aneurysms: A Comprehensive Systematic Review And Meta-Analysis, Seyed Maroufi, Mohammad Fallahi, Muhammad Waqas, Othman Bin-Alamer, Manisha Koneru, Joanna Roy, Jane Khalife, Hamza Shaikh, Daniel Tonetti

Department of Neurosurgery Faculty Papers

OBJECTIVES: The performance of the Pipeline Embolization Device (PED) and relatively newer double-layered Flow Re-Direction Endoluminal Device (FRED) have been studied for the treatment of intracranial aneurysms, but direct comparisons between PED and FRED are limited. The current systematic review aims at comparing the efficacy and safety of PED and FRED.

METHODS: A systematic review of the literature was conducted according to the PRISMA guideline. PubMed, Embase, Scopus, Web of Science, and Cochrane Library were searched, and related records were identified. A meta-analysis of double-arm studies comparing PED and FRED was conducted on angiographic and clinical outcomes, retreatment rates, and …


Spetzler-Martin Grade I And Ii Cerebral Arteriovenous Malformations: A Propensity-Score Matched Analysis Of Resection And Stereotactic Radiosurgery In Adult Patients, Salem Tos, Mahmoud Osama, Georgios Mantziaris, Bardia Hajikarimloo, Nimer Adeeb, Sandeep Kandregula, Adam Dmytriw, Hamza Adel Salim, Basel Musmar, Kareem El Naamani, Christopher Ogilvy, Douglas Kondziolka, Ahmed Abdelsalam, Deepak Kumbhare, Sanjeev Gummadi, Cagdas Ataoglu, Ufuk Erginoglu, Muhammed Amir Essibayi, Abdullah Keles, Sandeep Muram, Daniel Sconzo, Howard Riina, Arwin Rezai, Johannes Pöppe, Rajeev Sen, Omar Alwakaa, Christoph Griessenauer, Pascal Jabbour, Stavropoula Tjoumakaris, Jan-Karl Burkhardt, Robert Starke, Mustafa Baskaya, Laligam Sekhar, Michael Levitt, David Altschul, Neil Haranhalli, Malia Mcavoy, Assala Aslan, Abdallah Abushehab, Christian Swaid, Adib Abla, Christopher Stapleton, Matthew Koch, Visish M Srinivasan, Peng Chen, Spiros Blackburn, Mark Dannenbaum, Omar Choudhri, Bryan Pukenas, Darren Orbach, Edward Smith, Markus Möhlenbruch, Ali Alaraj, Ali Aziz-Sultan, Aman Patel, Hugo Cuellar, Michael Lawton, Jacques Morcos, Bharat Guthikonda, Jason Sheehan Feb 2025

Spetzler-Martin Grade I And Ii Cerebral Arteriovenous Malformations: A Propensity-Score Matched Analysis Of Resection And Stereotactic Radiosurgery In Adult Patients, Salem Tos, Mahmoud Osama, Georgios Mantziaris, Bardia Hajikarimloo, Nimer Adeeb, Sandeep Kandregula, Adam Dmytriw, Hamza Adel Salim, Basel Musmar, Kareem El Naamani, Christopher Ogilvy, Douglas Kondziolka, Ahmed Abdelsalam, Deepak Kumbhare, Sanjeev Gummadi, Cagdas Ataoglu, Ufuk Erginoglu, Muhammed Amir Essibayi, Abdullah Keles, Sandeep Muram, Daniel Sconzo, Howard Riina, Arwin Rezai, Johannes Pöppe, Rajeev Sen, Omar Alwakaa, Christoph Griessenauer, Pascal Jabbour, Stavropoula Tjoumakaris, Jan-Karl Burkhardt, Robert Starke, Mustafa Baskaya, Laligam Sekhar, Michael Levitt, David Altschul, Neil Haranhalli, Malia Mcavoy, Assala Aslan, Abdallah Abushehab, Christian Swaid, Adib Abla, Christopher Stapleton, Matthew Koch, Visish M Srinivasan, Peng Chen, Spiros Blackburn, Mark Dannenbaum, Omar Choudhri, Bryan Pukenas, Darren Orbach, Edward Smith, Markus Möhlenbruch, Ali Alaraj, Ali Aziz-Sultan, Aman Patel, Hugo Cuellar, Michael Lawton, Jacques Morcos, Bharat Guthikonda, Jason Sheehan

Department of Neurosurgery Faculty Papers

Cerebral arteriovenous malformations (AVMs) are congenital vascular anomalies that can lead to severe complications, including hemorrhage and neurological deficits. This study compares the outcomes of microsurgical resection and stereotactic radiosurgery (SRS) for SM grade I and II AVMs. Out of a large multicenter registry, we identified 180 matched patients with SM grade I and II AVMs treated with either microsurgical resection or SRS between 2010 and 2023. The primary outcomes were AVM obliteration rates and complications; secondary outcomes included neurological status and functional outcomes measured by the modified Rankin Scale (mRS). Propensity score matching (PSM) was utilized to ensure comparability …


Incidence And Predictors Of Woven Endobridge (Web) Shape Modification Following Treatment Of Intracranial Aneurysms In A Large Multicenter Study, Nimer Adeeb, Hamza Adel Salim, Basel Musmar, Assala Aslan, Christian Swaid, Miguel Cuellar, Mahmoud Dibas, Nicole Cancelliere, Jose Danilo Bengzon Diestro, Oktay Algin, Sherief Ghozy, Sovann Lay, Adrien Guenego, Leonardo Renieri, Joseph Carnevale, Guillaume Saliou, Panagiotis Mastorakos, Kareem El Naamani, Eimad Shotar, Markus Möhlenbruch, Michael Kral, Charlotte Chung, Mohamed Salem, Ivan Lylyk, Paul Foreman, Hamza Shaikh, Vedran Župančić, Muhammad U Hafeez, Joshua Catapano, Muhammad Waqas, Muhammet Arslan, Onur Ergun, James Rabinov, Yifan Ren, Clemens Schirmer, Mariangela Piano, Anna Kühn, Caterina Michelozzi, Robert Starke, Ameer Hassan, Mark Ogilvie, Anh Nguyen, Jesse Jones, Waleed Brinjikji, Marie Nawka, Marios Psychogios, Christian Ulfert, Bryan Pukenas, Jan-Karl Burkhardt, Thien Huynh, Juan Carlos Martinez-Gutierrez, Muhammed Amir Essibayi, Sunil Sheth, Diana Slawski, Rabih Tawk, Benjamin Pulli, Boris Lubicz, Pietro Panni, Ajit Puri, Guglielmo Pero, Eytan Raz, Christoph Griessenauer, Hamed Asadi, Adnan Siddiqui, Elad Levy, Neil Haranhalli, David Altschul, Andrew Ducruet, Felipe Albuquerque, Robert Regenhardt, Christopher Stapleton, Peter Kan, Vladimir Kalousek, Pedro Lylyk, Srikanth Boddu, Jared Knopman, Stavropoula Tjoumakaris, Pascal Jabbour, Frédéric Clarençon, Nicola Limbucci, Vivek Yedavalli, Max Wintermark, Vitor Mendes Pereira, Aman Patel, Hugo Cuellar-Saenz, Adam Dmytriw Feb 2025

Incidence And Predictors Of Woven Endobridge (Web) Shape Modification Following Treatment Of Intracranial Aneurysms In A Large Multicenter Study, Nimer Adeeb, Hamza Adel Salim, Basel Musmar, Assala Aslan, Christian Swaid, Miguel Cuellar, Mahmoud Dibas, Nicole Cancelliere, Jose Danilo Bengzon Diestro, Oktay Algin, Sherief Ghozy, Sovann Lay, Adrien Guenego, Leonardo Renieri, Joseph Carnevale, Guillaume Saliou, Panagiotis Mastorakos, Kareem El Naamani, Eimad Shotar, Markus Möhlenbruch, Michael Kral, Charlotte Chung, Mohamed Salem, Ivan Lylyk, Paul Foreman, Hamza Shaikh, Vedran Župančić, Muhammad U Hafeez, Joshua Catapano, Muhammad Waqas, Muhammet Arslan, Onur Ergun, James Rabinov, Yifan Ren, Clemens Schirmer, Mariangela Piano, Anna Kühn, Caterina Michelozzi, Robert Starke, Ameer Hassan, Mark Ogilvie, Anh Nguyen, Jesse Jones, Waleed Brinjikji, Marie Nawka, Marios Psychogios, Christian Ulfert, Bryan Pukenas, Jan-Karl Burkhardt, Thien Huynh, Juan Carlos Martinez-Gutierrez, Muhammed Amir Essibayi, Sunil Sheth, Diana Slawski, Rabih Tawk, Benjamin Pulli, Boris Lubicz, Pietro Panni, Ajit Puri, Guglielmo Pero, Eytan Raz, Christoph Griessenauer, Hamed Asadi, Adnan Siddiqui, Elad Levy, Neil Haranhalli, David Altschul, Andrew Ducruet, Felipe Albuquerque, Robert Regenhardt, Christopher Stapleton, Peter Kan, Vladimir Kalousek, Pedro Lylyk, Srikanth Boddu, Jared Knopman, Stavropoula Tjoumakaris, Pascal Jabbour, Frédéric Clarençon, Nicola Limbucci, Vivek Yedavalli, Max Wintermark, Vitor Mendes Pereira, Aman Patel, Hugo Cuellar-Saenz, Adam Dmytriw

Department of Neurosurgery Faculty Papers

The Woven EndoBridge (WEB) device is FDA-approved for the treatment of bifurcation aneurysms. Despite its wide popularity, it has been under scrutiny for its association with potential aneurysm recanalization and retreatment due to device shape modification. This study aims to analyze the shape modification rate of WEB devices and identify factors associated with this phenomenon, as well as its correlation with aneurysm retreatment. We conducted a retrospective review of the WorldWide WEB Consortium database, including adult patients treated for intracranial aneurysms with the WEB device. We assessed aneurysm occlusion using the WEB Occlusion Scale and defined WEB shape modification as …


Large-Bore Mechanical Thrombectomy Versus Catheter-Directed Thrombolysis In The Management Of Intermediate-Risk Pulmonary Embolism: Primary Results Of The Peerless Randomized Controlled Trial, Wissam Jaber, Carin Gonsalves, Stefan Stortecky, Samuel Horr, Orestis Pappas, Ripal Gandhi, Keith Pereira, Jay Giri, Sameer Khandhar, Khawaja Afzal Ammar, David Lasorda, Brian Stegman, Lucas Busch, David Dexter, Ezana Azene, Nikhil Daga, Fakhir Elmasri, Chandra Kunavarapu, Mark Rea, Joseph Rossi, Joseph Campbell, Jonathan Lindquist, Adam Raskin, Jason Smith, Thomas Tamlyn, Gabriel Hernandez, Parth Rali, Torrey Schmidt, Jeffrey Bruckel, Juan Camacho, Jun Li, Samy Selim, Catalin Toma, Sukhdeep Singh Basra, Brian Bergmark, Bhavraj Khalsa, David Zlotnick, Jordan Castle, David O'Connor, C. Michael Gibson Feb 2025

Large-Bore Mechanical Thrombectomy Versus Catheter-Directed Thrombolysis In The Management Of Intermediate-Risk Pulmonary Embolism: Primary Results Of The Peerless Randomized Controlled Trial, Wissam Jaber, Carin Gonsalves, Stefan Stortecky, Samuel Horr, Orestis Pappas, Ripal Gandhi, Keith Pereira, Jay Giri, Sameer Khandhar, Khawaja Afzal Ammar, David Lasorda, Brian Stegman, Lucas Busch, David Dexter, Ezana Azene, Nikhil Daga, Fakhir Elmasri, Chandra Kunavarapu, Mark Rea, Joseph Rossi, Joseph Campbell, Jonathan Lindquist, Adam Raskin, Jason Smith, Thomas Tamlyn, Gabriel Hernandez, Parth Rali, Torrey Schmidt, Jeffrey Bruckel, Juan Camacho, Jun Li, Samy Selim, Catalin Toma, Sukhdeep Singh Basra, Brian Bergmark, Bhavraj Khalsa, David Zlotnick, Jordan Castle, David O'Connor, C. Michael Gibson

Department of Radiology Faculty Papers

BACKGROUND: There are a lack of randomized controlled trial data comparing outcomes of different catheter-based interventions for intermediate-risk pulmonary embolism.

METHODS: PEERLESS is a prospective, multicenter, randomized controlled trial that enrolled 550 patients with intermediate-risk pulmonary embolism with right ventricular dilatation and additional clinical risk factors randomized 1:1 to treatment with large-bore mechanical thrombectomy (LBMT) or catheter-directed thrombolysis (CDT). The primary end point was a hierarchal win ratio composite of the following (assessed at the sooner of hospital discharge or 7 days after the procedure): (1) all-cause mortality, (2) intracranial hemorrhage, (3) major bleeding, (4) clinical deterioration and/or escalation to …


Five-Year Outcomes Of Lenadogene Nolparvovec Gene Therapy In Leber Hereditary Optic Neuropathy, Patrick Yu-Wai-Man, Nancy Newman, Valérie Biousse, Valerio Carelli, Mark Moster, Catherine Vignal-Clermont, Thomas Klopstock, Alfredo Sadun, Robert C Sergott, Rabih Hage, Simona Degli Esposti, Chiara La Morgia, Claudia Priglinger, Rustum Karanja, Magali Taiel, José-Alain Sahel Feb 2025

Five-Year Outcomes Of Lenadogene Nolparvovec Gene Therapy In Leber Hereditary Optic Neuropathy, Patrick Yu-Wai-Man, Nancy Newman, Valérie Biousse, Valerio Carelli, Mark Moster, Catherine Vignal-Clermont, Thomas Klopstock, Alfredo Sadun, Robert C Sergott, Rabih Hage, Simona Degli Esposti, Chiara La Morgia, Claudia Priglinger, Rustum Karanja, Magali Taiel, José-Alain Sahel

Department of Neurology Faculty Papers

IMPORTANCE: Limited studies have assessed the long-term benefit/risk of gene therapy for Leber hereditary optic neuropathy (LHON).

OBJECTIVE: To determine the safety and efficacy of lenadogene nolparvovec in patients with LHON due to the MT-ND4 gene variant for up to 5 years after administration.

DESIGN, SETTING, AND PARTICIPANTS: The RESCUE and REVERSE Long-Term Follow-up Study (RESTORE), conducted from 2018 to 2022, is the 5-year follow-up study of the 2 phase 3 clinical studies RESCUE (Efficacy Study of Lenadogene Nolparvovec for the Treatment of Vision Loss Up to 6 Months From Onset in LHON Due to the MT-ND4 Mutation) and REVERSE …


Patient-Reported Outcomes: Comparing Functional Avoidance And Standard Thoracic Radiation Therapy In Lung Cancer, Spencer Poiset, Joseph Lombardo, Edward Castillo, Richard Castillo, Bernard Jones, Moyed Miften, Brian Kavanagh, Adam Dicker, Cullen Boyle, Nicole Simone, Benjamin Movsas, Inga Grills, Chad Rusthoven, Yevgeniy Vinogradskiy, Lydia Wilson Feb 2025

Patient-Reported Outcomes: Comparing Functional Avoidance And Standard Thoracic Radiation Therapy In Lung Cancer, Spencer Poiset, Joseph Lombardo, Edward Castillo, Richard Castillo, Bernard Jones, Moyed Miften, Brian Kavanagh, Adam Dicker, Cullen Boyle, Nicole Simone, Benjamin Movsas, Inga Grills, Chad Rusthoven, Yevgeniy Vinogradskiy, Lydia Wilson

Department of Radiation Oncology Faculty Papers

PURPOSE: Novel methods generate functional images using image processing techniques combined with four-dimensional computed tomography (4DCT) data (4DCT-ventilation). 4DCT-ventilation was implemented in a phase II, multicenter functional avoidance clinical trial. The work compares functional avoidance patient-reported outcomes (PROs) against historical standards.

METHODS: Patients with locally advanced lung cancer undergoing curative-intent chemoradiation were accrued. 4DCT-ventilation imaging was generated and functional avoidance treatment plans created reduced dose to functional lung. PRO instruments included Functional Assessment of Cancer Therapy Lung questionnaire and accompanying subscales (including the Trial Outcome Index [TOI]), EuroQol-5 Dimension (EQ-5D), and EQ-Visual Analog Scale (EQ-VAS). The average change from baseline …


Comparing Functional Outcomes Between 3d Printed Acetabular Cups And Traditional Prosthetic Implants In Hip Arthroplasty: A Systematic Review And Meta Analysis, Ryan St. John, Seth Spicer, Mo Hadaya, Hanna Brancaccio, Seungkyu Park, Sean Mcmillan Dec 2024

Comparing Functional Outcomes Between 3d Printed Acetabular Cups And Traditional Prosthetic Implants In Hip Arthroplasty: A Systematic Review And Meta Analysis, Ryan St. John, Seth Spicer, Mo Hadaya, Hanna Brancaccio, Seungkyu Park, Sean Mcmillan

Rowan-Virtua School of Osteopathic Medicine Departmental Research

OBJECTIVE: The primary research aim was to determine if the use of traditional or 3D printed prosthesis resulted in better functional outcome scores in hip arthroplasty.

METHODS: A systematic review and meta-analysis was conducted utilizing the PRISMA 2020 guidelines. Six databases (PubMed, Embase, Scopus, WebOfScience, and Cochrane Library, Google Scholar) were searched yielding 1117 article titles and abstracts. Rayyan.ai was used to detect duplicates (n = 246) and for manual screening for inclusion and exclusion criteria. Included were controlled studies of any publication time that assessed Harris Hip Score (HHS) at baseline and twelve months. Six papers were sought for …


Letter To "Early Do-Not-Attempt Resuscitation Orders And Neurological Outcomes In Older Out-Of-Hospital Cardiac Arrest Patient: A Multicenter Observational Study", Benjamin Tangkamolsuk, Quang La, David F Lo Dec 2024

Letter To "Early Do-Not-Attempt Resuscitation Orders And Neurological Outcomes In Older Out-Of-Hospital Cardiac Arrest Patient: A Multicenter Observational Study", Benjamin Tangkamolsuk, Quang La, David F Lo

Rowan-Virtua School of Osteopathic Medicine Departmental Research

We enjoyed reading the article by Kohri et al., titled “Early do‐not‐attempt resuscitation orders and neurological outcomes in older out‐of‐hospital cardiac arrest patients: A multicenter observational study” and would like to offer additional commentary on the article. We hope these perspectives may provide insight into areas that may require further research and improvement.


Systemic Effects Of Anti-Vegf Intravitreal Injection In Patients With Age-Related Macular Degeneration: A Multi-Institutional Real-World Study, Tzu-Yi Lin, Yi-Ting Hsieh, Sunir J. Garg, Lee-Jen Chen, Kuan-Jen Chen, Wei-Chi Wu, Chi-Chun Lai, Yih-Shiou Hwang, Eugene Yu-Chuan Kang Nov 2024

Systemic Effects Of Anti-Vegf Intravitreal Injection In Patients With Age-Related Macular Degeneration: A Multi-Institutional Real-World Study, Tzu-Yi Lin, Yi-Ting Hsieh, Sunir J. Garg, Lee-Jen Chen, Kuan-Jen Chen, Wei-Chi Wu, Chi-Chun Lai, Yih-Shiou Hwang, Eugene Yu-Chuan Kang

Wills Eye Hospital Papers

PURPOSE: In individuals aged >50 years, age-related macular degeneration (AMD) is the leading cause of irreversible blindness. Intravitreal injections of antivascular endothelial growth factor (VEGF) agents (bevacizumab, ranibizumab, and aflibercept) show good efficacy and similar incidences of systemic adverse events (SAEs). However, comparative studies between agents are limited. Our study aimed to compare the real-world SAE risks of bevacizumab, ranibizumab, and aflibercept users.

METHODS: This retrospective cohort study identified new bevacizumab, ranibizumab, and aflibercept users in a multi-institutional database in Taiwan between 2014 and 2019. Inverse probability of treatment weights (IPTW) with propensity scores was conducted to achieve homogeneity among …


Association Between Mri Findings Of Facet Arthropathy And Synovitis With Health-Related Outcome And Pain Scores Following Therapeutic Lumbar Facet Injections., Michael S Green, Michele Van Hal, Matthew Onimus, Christopher R Hoffman, Dhruv K C Goyal, Valeria Potigailo, Khuram S Kazmi Oct 2024

Association Between Mri Findings Of Facet Arthropathy And Synovitis With Health-Related Outcome And Pain Scores Following Therapeutic Lumbar Facet Injections., Michael S Green, Michele Van Hal, Matthew Onimus, Christopher R Hoffman, Dhruv K C Goyal, Valeria Potigailo, Khuram S Kazmi

Rowan-Virtua School of Osteopathic Medicine Departmental Research

Low back pain is a common complaint among adults. The facet joint is a major source of lumbar pain, and therapeutic facet injections have gained popularity as a minimally invasive treatment option. In addition, magnetic resonance imaging (MRI) utilization for diagnosing low back pain has increased significantly over the past few decades. Facet synovitis is an entity characterized by edema and inflammatory changes affecting the facet joints, adjacent bone marrow, and surrounding soft tissues. Although its underlying etiology remains poorly understood, recent reports suggest a high incidence in patients with arthropathy and arthritis. It is essential to explore potential correlations …


Antiplatelet Strategy For Patients With Acute Coronary Syndrome Undergoing Percutaneous Coronary Intervention: A Systematic Review And Network Meta-Analysis, Waqas Ullah, Harigopal Sandhyavenu, Amro Taha, Smitha Narayana Gowda, Maryam Mukhtar, Aravind Reddy Polam, Salman Zahid, David L. Fischman, Michael P. Savage, Sunil V. Rao, Mohamad Alkhouli Oct 2024

Antiplatelet Strategy For Patients With Acute Coronary Syndrome Undergoing Percutaneous Coronary Intervention: A Systematic Review And Network Meta-Analysis, Waqas Ullah, Harigopal Sandhyavenu, Amro Taha, Smitha Narayana Gowda, Maryam Mukhtar, Aravind Reddy Polam, Salman Zahid, David L. Fischman, Michael P. Savage, Sunil V. Rao, Mohamad Alkhouli

Division of Cardiology Faculty Papers

BACKGROUND: Optimal duration and choice of antiplatelet therapy in patients with acute coronary syndrome undergoing percutaneous coronary intervention remain controversial.

METHODS AND RESULTS: Digital databases (PubMed, Cochrane, and Embase) were queried to select all randomized controlled trials on a post-percutaneous coronary intervention population with acute coronary syndrome. Dual-antiplatelet therapy (DAPT) with aspirin and clopidogrel for 12 months was compared with 4 major strategies: high-potency, high- to low-potency, low-dose, and short-duration DAPT. A network meta-analysis was performed to compare the safety and efficacy of different antiplatelet strategies. This study was the second updated manuscript under the International Prospective Register of Systematic …


A Comparison Of The Safety And Efficacy Of Tapinarof And Roflumilast Topical Therapies In The Management Of Mild-To-Moderate Plaque Psoriasis, Hira Ghani, Alicia Podwojniak, Isabella J Tan, Aarushi K Parikh, Bianca Sanabria, Babar Rao Sep 2024

A Comparison Of The Safety And Efficacy Of Tapinarof And Roflumilast Topical Therapies In The Management Of Mild-To-Moderate Plaque Psoriasis, Hira Ghani, Alicia Podwojniak, Isabella J Tan, Aarushi K Parikh, Bianca Sanabria, Babar Rao

Rowan-Virtua School of Osteopathic Medicine Departmental Research

INTRODUCTION: Psoriasis is an immune-mediated inflammatory skin disease. First-line topical treatments include steroids, calcineurin inhibitors, vitamin D analogs, and anthralin. Recently, novel topical therapeutics like tapinarof and roflumilast have emerged with unique anti-inflammatory mechanisms and promising efficacy profiles.

MATERIALS AND METHODS: This review utilized PubMed, SCOPUS, and Web of Science databases to identify recent studies on tapinarof and roflumilast. Criteria focused on efficacy, safety profiles, and therapeutic roles in psoriasis treatment.

RESULTS: Four primary literature articles were identified for tapinarof and five for roflumilast. Both drugs demonstrated strong efficacy with minimal adverse events in treating mild-to-moderate plaque psoriasis. Tapinarof showed …


Clinical Outcomes For Va-Ecmo Patients Associated With Hyperlipidemia: An Analysis Of The National Inpatient Sample, Dakota B. Pastore, Tony Elias, Rafail Beshai, Kyrillos Girgis, Maziyar Daneshvar, Keith Anacker May 2024

Clinical Outcomes For Va-Ecmo Patients Associated With Hyperlipidemia: An Analysis Of The National Inpatient Sample, Dakota B. Pastore, Tony Elias, Rafail Beshai, Kyrillos Girgis, Maziyar Daneshvar, Keith Anacker

Rowan-Virtua Research Day

Introduction: Veno-arterial extracorporeal membrane oxygenation (VA-ECMO) is considered the most advanced temporary life support which provides complete hemodynamic support in addition to gas exchange. There is limited data available on the impact of hyperlipidemia (HLD) on VA-ECMO patients. We sought to examine the national inpatient sample (NIS) database to describe in-hospital outcomes among these patients.

Methods: The NIS was searched for hospitalizations of adult VA-ECMO patients with and without a concomitant diagnosis of HLD for the years 2019 and 2020. The primary outcome was inpatient mortality.

Results:This study included 3,885 VA-ECMO patients, of which 1,082 (27.8%) patients had HLD. …


Outcomes Of Thoracic Endovascular Aortic Repair (Tevar) Procedure In Octogenarians, Nonagenarians And Centenarians: A Population-Based Study, Dakota B. Pastore, Tony Elias, Rafail Beshai, Kyrillos Girgis, Maziyar Daneshvar, Keith Anacker May 2024

Outcomes Of Thoracic Endovascular Aortic Repair (Tevar) Procedure In Octogenarians, Nonagenarians And Centenarians: A Population-Based Study, Dakota B. Pastore, Tony Elias, Rafail Beshai, Kyrillos Girgis, Maziyar Daneshvar, Keith Anacker

Rowan-Virtua Research Day

Introduction: Limited data exists for patients undergoing a thoracic endovascular aortic repair (TEVAR) procedure who are older than or equal to 80 years old (YO). This study sought to examine the national inpatient sample (NIS) database to describe in-hospital outcomes among these older patients.

Methods: The NIS was searched for hospitalizations of adults who underwent a TEVAR procedure in 2019 and 2020. The study compared those aged 80 YO and over to those younger than 80 YO. The primary outcome was inpatient mortality. Secondary outcomes included hospital length of stay (LOS), and total hospital charges (TOTHCG).

Results: This study included …


A Literature Review Of Pain Control In Osteoarthritis Patients Using Blood Flow Restriction Training, John Gericke, Dakota Pastore, Tyler Pigott May 2024

A Literature Review Of Pain Control In Osteoarthritis Patients Using Blood Flow Restriction Training, John Gericke, Dakota Pastore, Tyler Pigott

Rowan-Virtua Research Day

Background: Osteoarthritis (OA) is a degenerative disease that mainly affects articular cartilage of joints. OA most commonly affects the knees and causes pain and reduces quality of life in older adults. Blood flow restriction (BFR) occludes arterial and venous flow during exercise using a blood pressure cuff placed on the proximal lower limb. This creates an intramuscular hypoxic environment which promotes muscle hypertrophy. High load training can cause pain and inflammation in knee OA patients due to increased stress. BFR can allow patients with knee OA to use lower loads to achieve beneficial effects of rehabilitation.

Methods: Data was ascertained …


Unmasking A Silent Threat: Early Detection Of Elevated Hbco Levels When The Story Doesn’T Add Up, Anna M. Delancy, Eric Maddock, James A. Espinosa, Alan Lucerna May 2024

Unmasking A Silent Threat: Early Detection Of Elevated Hbco Levels When The Story Doesn’T Add Up, Anna M. Delancy, Eric Maddock, James A. Espinosa, Alan Lucerna

Rowan-Virtua Research Day

Carbon monoxide (CO) is difficult to detect in the surrounding environment due to its colorless, odorless, and tasteless properties. Patients acutely poisoned with CO can have a myriad of presentations including headache, dizziness, and critical illness— all with a mortality rate up to 3%. When concerned for CO poisoning, blood co-oximeter testing should be performed, with treatment surrounding the idea of providing supplemental oxygen The efficacy of hyperbaric oxygen (HBO) treatment remains unclear. Here we describe an atypical scenario of a patient ultimately found to have CO poisoning, and the importance of early identification and treatment of this elusive diagnosis …


A Systematic Review On Belimumab’S Effectiveness, Improved Health Outcomes And Quality Of Life In Patients With Lupus Syndromes, Emily Meale, Alexandra Fontaine May 2024

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 …


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 May 2024

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 May 2024

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 …


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 May 2024

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


Clinical Outcomes For Impella Patients Associated With Hyperlipidemia: An Analysis Of The National Inpatient Sample, Tony Elias, Sonika Vatsa, Kyrillos Girgis, Taha Syed, Rafail Beshai May 2024

Clinical Outcomes For Impella Patients Associated With Hyperlipidemia: An Analysis Of The National Inpatient Sample, Tony Elias, Sonika Vatsa, Kyrillos Girgis, Taha Syed, Rafail Beshai

Rowan-Virtua Research Day

The Impella, a ventricular assist device, is crucial for managing severe heart failure and cardiogenic shock. Despite its widespread use, there's scant information on how hyperlipidemia affects Impella patients. To address this gap, we delved into the National Inpatient Sample Database from 2019 and 2020. Our aim was to scrutinize in-hospital outcomes among these patients. We identified 8233 Impella patients, among whom 1012 (12.3%) had hyperlipidemia. Those with hyperlipidemia displayed higher rates of hypertension, diabetes mellitus, and chronic kidney disease compared to their counterparts without hyperlipidemia. Shockingly, in-hospital mortality was notably elevated in the hyperlipidemia group, emphasizing its clinical significance. …


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 May 2024

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 …


Auriculotemporal Nerve Block For Tmj: A Systematic Review, Hasan Zia, Usmaan Al-Shehab, Ahmed Gawash, Anthony Dipalma, Dale Johnson, Musa Dolley, David F. Lo, Ryan St. John, Zain Hassam, Don D. Shamilov May 2024

Auriculotemporal Nerve Block For Tmj: A Systematic Review, Hasan Zia, Usmaan Al-Shehab, Ahmed Gawash, Anthony Dipalma, Dale Johnson, Musa Dolley, David F. Lo, Ryan St. John, Zain Hassam, Don D. Shamilov

Rowan-Virtua Research Day

The purpose of this systematic review is to evaluate auriculotemporal nerve blocks for temporomandibular joint (TMJ) disorder management. 583 articles reviewed from five databases, selected four studies for pooled analysis on visual analog scale (VAS) and maximum mouth opening (MMO) outcomes. Pooled analysis showed significant improvement in VAS (-2.27, p < 0.001) and MMO (0.94, p = 0.03) post-auriculotemporal nerve blocks for TMJ disorder. Auriculotemporal nerve blocks demonstrate potential effectiveness in TMJ disorder treatment, warranting further research on long-term effects and side effects.