Digital Commons Network^™

A Phase 2 Trial Of Cpx-351 Combined With Venetoclax In Relapsed Or Refractory Acute Myeloid Leukemia, Tapan M Kadia, Wei-Ying Jen, Alex Bataller, Alexandre Bazinet, Gautam Borthakur, Elias Jabbour, Wei Qiao, Nicholas J Short, Koichi Takahashi, Ghayas C Issa, Courtney D Dinardo, Guillermo Montalban-Bravo, Naveen Pemmaraju, Andrew Tran, Vanthana Bharathi, Sanam Loghavi, Amin M Alousi, Uday Popat, Naval G Daver, Farhad Ravandi, Hagop M Kantarjian

Faculty, Staff and Student Publications

Outcomes in patients with relapsed/refractory (RR) AML are poor. We sought to investigate if CPX-531 in combination with venetoclax (CPX + VEN) was tolerable and effective in RR AML. This was a single institution phase 1b/2 trial of CPX + VEN. Patients aged ≥ 18 years with RR AML who were fit for intensive chemotherapy were eligible. Prior venetoclax exposure was allowed. The phase 1b portion followed a 3 + 3 design to identify the recommended phase 2 dose (RP2D) for the expansion cohort. At the starting dose level of −1, prolonged myelosuppression was observed, leading to dose level −2 …

Efficacy And Safety Of Oral Decitabine/Cedazuridine In The Chronic Myelomonocytic Leukaemia Subpopulations From Phase 2 And 3 Studies, Michael R Savona, Olatoyosi Odenike, Gail J Roboz, Harshad Amin, Amy E Dezern, Elizabeth A Griffiths, Kim-Hien Dao, Amer M Zeidan, Bhavana Bhatnagar, Rena Buckstein, Brian Leber, Mary-Margaret Keating, Somedeb Ball, Aram Oganesian, Yuri Sano, Harold N Keer, Guillermo Garcia-Manero

Faculty, Staff and Student Publications

DNA methyltransferase inhibitors (DNMTis) are commonly used in treating chronic myelomonocytic leukaemia (CMML); however, data from prospective studies of DNMTis in CMML are limited. The present analysis evaluated the efficacy, safety and pharmacodynamics of the oral DNMTi decitabine/cedazuridine in the subset of patients with CMML from the phase 2 and 3 trials, which led to the approval of this agent for myelodysplastic syndromes and CMML in the United States and Canada. Potential prognostic factors also were analysed. In all, 34 patients with CMML were screened and 33 were treated. Most patients (76% [n = 25]) had myelodysplastic type-CMML and 77% …

Master Protocol Design With Hybrid Control For Efficient Early-Phase Trial Consolidation, Alexander M Kaizer, Xiaojiang Zhan, Eric Baron, Rui Sammi Tang, David S Hong, Brian P Hobbs

Faculty, Staff and Student Publications

Purpose: Master protocols represent transformations, enabling multiple therapies or diseases under a single protocol. These designs streamline therapeutic development by reducing redundancies. Suited for evolving fields such as oncology and global emergencies such as the COVID-19 pandemic, master protocols have been exemplified by studies such as RECOVERY, Solidarity, and I-SPY 2, which accelerated effective treatment identification (with I-SPY 2 focused on molecular subtypes). Recent oncology examples, such as MORPHEUS, evaluate immunotherapy combinations with shared controls. Despite advantages, their application in early-phase oncology remains underutilized amid growing regulatory emphasis on randomization for robust evidence.

Methods: US Food and Drug Administration (FDA) …

Film Measurements Of Kilovoltage X-Ray Percentage Depth Doses For Different Energies And Field Sizes, Tze Yee Lim, Dershan Luo, Jamshid Moradi-Kurdestany, Xin Wang, Ramesh Tailor

Faculty, Staff and Student Publications

Objectives: Kilovoltage x-ray therapy, which includes superficial x-rays and orthovoltage x-rays, is particularly suited for treatments near the skin surface. Radiation dose deposition at depth is typically characterized by percentage depth dose (PDD) curves. However, PDD data are difficult to obtain in kilovoltage x-ray therapy, hence clinics often rely on British Journal of Radiology Supplement 25 (BJR-25) archival data. This study measured PDD for different energies and field sizes on the Xstrahl 300 kilovoltage x-ray therapy unit for comparison with BJR-25 data.

Methods: We irradiated EBT3 film in water using different energy and field size combinations, then, compared the acquired …

Orp2 Regulates Free Cholesterol Accumulation In Hepatocytes During Mash, Jin Wu, Yudi Zhao, Liwen Qiu, Qiaoli Chen, Xiaowei Wang, Jingwen Gu, Yan Liang, Yingjie Zhang, Hong-Yu Wang, Yang Liu, Xiaoqin Wu, Shuai Chen, Feng-Jung Chen, Mingming Gao, Hongyuan Yang

Faculty, Staff and Student Publications

Background: Cholesterol crystals in hepatocytes are known to strongly associate with human metabolic dysfunction-associated steatohepatitis. However, it remains unclear which molecular pathway(s) regulates free cholesterol accumulation and the formation of cholesterol crystals in hepatocytes. In cultured cell lines, oxysterol-binding protein-related protein 2 (ORP2) functions to deliver cholesterol to the plasma membrane from endosomal compartments.

Methods: Here, we generated liver-specific ORP2 knockout (ORP2-LKO) mice and characterized their metabolic phenotypes on chow and high-fat diet.

Results: The ORP2-LKO mice developed much more severe hepatic steatosis than floxed control mice after high-fat diet feeding. They also demonstrated more severe liver inflammation and damage. …

Patient, Physician, And Assessor Blinding In Phase Iii Randomized Trials In Oncology: A Meta-Epidemiological Analysis, Gabrielle Brown, Pavlos Msaouel, Avital M Miller, Ramez Kouzy, Timothy A Lin, Joseph Abi Jaoude, Ethan B Ludmir, Alexander D Sherry

Faculty, Staff and Student Publications

Background: Blinding mitigates bias in randomized trials and may be especially crucial for surrogate endpoints, such as progression-free survival (PFS). Here, we characterize utilization of and factors associated with blinding in Phase III oncology trials with PFS primary endpoints.

Methods: Two-arm, superiority-design trials investigating systemic therapy were identified in May 2024 from ClinicalTrials.gov with no date limitation. Trials were required to have a PFS primary endpoint. The study outcomes were the presence of double-blind designs and blinded independent central review (BICR) for the primary endpoint. Ninety-five percent credible intervals for binary outcomes were estimated from beta distributions, and multivariable logistic …

Single-Cell Proteomic Analysis Reveals Multiple Myeloma Heterogeneity And The Dynamics Of The Tumor Immune Microenvironment In Precursor And Advanced States, Mohamed Kamal, Stephanie N Shishido, Jeremy Mason, Krina Patel, Elisabet E Manasanch, Robert Z Orlowski, Peter Kuhn

Faculty, Staff and Student Publications

Multiple myeloma (MM) is an aggressive hematologic malignancy arising from plasma cell (PC) proliferation in the bone marrow, progressing from its precursor states MGUS and SMM. Despite therapeutic advances, MM remains incurable, underscoring the need for better risk stratification and early detection. Tumor heterogeneity and dynamic immune microenvironment changes drive progression, yet bulk analyses overlook rare subpopulations critical to disease evolution and resistance. This study employed multiplexed targeted proteomics to characterize bone marrow aspirates (BMA) from 22 patients to observe the change in the distribution of PCs and tumor immune microenvironment (TiME) cells across MM disease states and controls. Bone …

Provider Attitudes And Perspectives On Rehabilitation For Pediatric Cancer Patients, Maria C Swartz, Eduardo Gonzalez Villarreal, Keri Schadler, Donna Kelly, Alakh P Rajan, Clark Andersen, Shiming Zhang, Stephanie J Wells, Amy Heaton, Karen M Moody

Faculty, Staff and Student Publications

PurposeTwenty percent of childhood cancer survivors experience physical function impairments, and ∼75% develop a chronic health condition. Physical and occupational therapists (PT/OTs) can mitigate these late effects, yet few children receive cancer rehabilitation (CR). This research aimed to identify provider attitudes and perspectives towards CR services for children across inpatient and outpatient settings at a cancer center.MethodsThree cardiac rehabilitation instruments were adapted to evaluate knowledge, attitudes, and perceptions regarding CR delivery. Descriptive statistics were used to summarize participant survey results.ResultsTwenty administrators, 20 physicians/advanced practice providers (APPs), and 20 PT/OTs completed surveys. All disciplines strongly agreed on the value of CR …

Bard: A Seamless Two-Stage Dose Optimization Design Integrating Backfill And Adaptive Randomization, Yixuan Zhao, Rachael Liu, Jianchang Lin, Ying Yuan

Faculty, Staff and Student Publications

One common approach for dose optimization is a two-stage design, which initially conducts dose escalation to identify the maximum tolerated dose, followed by a randomization stage where patients are assigned to two or more doses to further assess and compare their risk-benefit profiles to identify the optimal dose. A limitation of this approach is its requirement for a relatively large sample size. To address this challenge, we propose a seamless two-stage design, BARD (Backfill and Adaptive Randomization for Dose Optimization), which incorporates two key features to reduce sample size and shorten trial duration. The first feature is the integration of …

Management Of Aromatase Inhibitor-Associated Bone Loss (Aibl) In Women With Hormone-Sensitive Breast Cancer: An Updated Joint Position Statement Of The Iof, Cabs, Ects, Ieg, Esceo, Ims, And Siog, Peyman Hadji, Matty Aapro, Nasser Al-Dagri, Majed Alokail, Emmanuel Biver, Jean-Jacques Body, Maria Luisa Brandi, Janet Brown, Cyrille Confavreux, Bernard Cortet, Matthew Drake, Peter Ebeling, Erik Fink Eriksen, Ghada El-Hajj Fuleihan, Theresa A Guise, Nick C Harvey, Andreas Kurth, Bente Langdahl, Willem Lems, Radmila Matijevic, Eugene Mccloskey, Rossella Nappi, Santiago Palacios, Georg Pfeiler, Jean-Yves Reginster, René Rizzoli, Daniele Santini, Sansin Tuzun, Catherine Van Poznak, Tobias De Villiers, M Carola Zillikens, Robert Coleman

Faculty, Staff and Student Publications

Background: Women with hormone-responsive breast cancer who receive adjuvant endocrine treatment with aromatase inhibitors (AI) are known to be at higher fracture risk due to a marked increase in bone resorption. In 2017, several interdisciplinary cancer and bone societies involved in the management of women with AI-associated bone loss (AIBL) published a joint position statement comprising evidence-based recommendations and a practical management algorithm for the assessment of fracture risk and optimal treatment of this patient population.

Patients and methods: In order to provide updated recommendations that reflect recent advances in the assessment and management of AIBL since publication of the …

Tert Promoter Mutations And Survival Outcomes In Adult-Type Granulosa Cell Tumors, Allison L Brodsky, Alejandra Flores Legarreta, Bryan M Fellman, Deanna Glassman, Jeffrey How, Veena Vuttaradhi, Anil K Sood, Lois Michelle Ramondetta, David Gershenson, R Tyler Hillman

Faculty, Staff and Student Publications

Objectives: To evaluate survival outcomes among patients with adult-type granulosa cell tumors who have telomerase reverse transcriptase (TERT) promoter mutations.

Methods: This is a retrospective cohort study using the MD Anderson Rare Gynecologic Malignancy Registry. Patients with adult granulosa cell tumors who underwent molecular testing for TERT promoter and FOXL2 c.C402G mutations were included. We used descriptive statistics to compare demographic and clinical variables and estimated progression-free and overall survival with Kaplan-Meier curves. Cox proportional hazards regression and log-rank tests were employed for comparisons, with multivariable analyses adjusting for various factors.

Results: Among 70 patients, 28 (40%) had TERT+ tumors. …

Clinical And Dosimetric Considerations For Yttrium-90 Glass Microspheres Radioembolization Of Intrahepatic Cholangiocarcinoma, Metastatic Colorectal Carcinoma, And Metastatic Neuroendocrine Carcinoma: Recommendations From An International Multidisciplinary Working Group, Marnix Lam, Riad Salem, Beau Toskich, S Cheenu Kappadath, Carlo Chiesa, Kirk Fowers, Paul Haste, Joseph M Herman, Edward Kim, Thomas Leung, Siddharth A Padia, Bruno Sangro, Daniel Y Sze, Etienne Garin

Faculty, Staff and Student Publications

Purpose: The TheraSphere Global Steering Committee reconvened to review clinical data and address knowledge gaps related to treatment and dosimetry in non-HCC indications using Yttrium-90 (90Y) glass microspheres.

Methods: A PubMed search was performed. References were reviewed and adjudicated by the Delphi method. Recommendations were graded according to the degree of recommendation and strength of consensus. Dosimetry focused on a mean dose approach, i.e., aiming for an average dose over either single or multicompartment volumes of interests. Committee discussion and consensus focused on optimal patient selection, disease presentation, liver function, tumour type, tumour vascularity, and curative/palliative treatment intent for intrahepatic …

Domains Of Housing Instability And Intimate Partner Violence Risk Among U.S. Tenants, Anairany Zapata, Leila G Wood, Annalynn M Galvin, Wenyaw Chan, Timothy A Thomas, Jack Tsai, Heather K Way, Elizabeth J Mueller, Daphne C Hernandez

Faculty, Staff and Student Publications

While IPV is often studied as a predictor of housing insecurity, few U.S. studies explore how different forms of housing instability may contribute to intimate partner violence (IPV) risk. Using a mixed-methods approach and a cross-sectional design, this study examined the association between four housing instability domains and IPV among a sample of tenants that had either experienced eviction or were at high risk for eviction. Tenants in Harris and Travis counties (Texas, USA) completed an online survey (n = 1085; March–July 2024). Housing instability was assessed across four domains: homelessness, lease violations, utility hardship, and poor housing quality. …

Resting Quantitative Myocardial Perfusion-Essential Clinical Coronary Pathophysiology For Preventing Non-Beneficial Revascularization In Cardiomyopathy, K Lance Gould, Nils P Johnson

Faculty, Staff and Student Publications

No abstract provided.

Treatment With Allogenic Mesenchymal Stromal Cells For Moderate To Severe Acute Respiratory Distress Syndrome: A Double-Blind, Placebo-Controlled, Multi-Center, Phase 2b Clinical Trial (Stat), Michael A Matthay, Hanjing Zhou, Aartik Sarma, Narges Alipanah-Lechner, Carolyn Hendrickson, Lucy Z Kornblith, Martin Schreiber, David Zonies, Akram Khan, Bryce Robinson, Nicholas J Johnson, Lorraine B Ware, Oscar Guillamondegui, Jonathan Casey, Laura Moore, Bela Patel, Lillian Kao, Charles E Wade, Erin Fox, Charles Cox, Farrakh Khawanja, Robier Aguillon Prada, Sami Hossri, Rachael Callcut, Timothy Albertson, Kevin L Delucchi, Melanie Mcmillan, Charles R Langelier, Shibani Pati, David H Mckenna, Carolyn Leroux, Carolyn S Calfee, Kathleen D Liu

Faculty, Staff and Student Publications

Background: Prior clinical trials established the safety, but not the efficacy of bone marrow-derived mesenchymal stromal cells (MSCs) in the acute respiratory distress syndrome (ARDS).

Methods: We conducted a prospective, double-blind, multi-center randomized phase 2b clinical trial of one dose of intravenous MSCs (10 x 106/kg predicted body weight) versus placebo in 120 ventilated patients with ARDS (PaO2/FiO2 < 250 mmHg). The primary endpoint was change in oxygenation index (OI) over 36 hours from baseline.

Findings: Enrollment began in January 2020. Due to the Coronavirus 2019 (COVID-19) pandemic, the majority of subjects (101/120, 84%) developed ARDS from COVID-19. There were no significant baseline differences in severity of illness between patients treated with MSCs and those who received …

Glucose Metabolism And Its Direct Action In Cancer And Immune Regulation: Opportunities And Challenges For Metabolic Targeting, Bo-Syong Pan, Che-Chia Hsu, Hsin-En Wu, Yuan-Ru Chen, Xiaobo Zhou, Shu-Chi Wang, Chia-Yang Li, Hui-Kuan Lin

Faculty, Staff and Student Publications

Glucose metabolism is a pivotal hub for cellular energy production and the generation of building blocks that support cell growth, survival, and differentiation. Cancer cells undergo metabolic reprogramming to sustain rapid proliferation, survive in harsh microenvironments, and resist therapies. Beyond producing energy and building blocks to meet cancer cell demands, glucose metabolism generates numerous metabolites that serve as signaling molecules, orchestrating signaling pathways and epigenetic modifications that regulate cancer cell phenotypes and immunity. In this review, we discuss how glucose, through its metabolism and direct actions, influences diverse biological processes driving cancer progression and therapeutic resistance, while also exploring metabolic …

Use Of Cholic Acid In Smith-Lemli-Opitz Syndrome (Slos): Real-World Patient Outcomes, Edwin Ferren, Paul R Hillman, Amy Kritzer, Joseph Ray, Alvaro Serrano, Hope Northrup, Paige Roberts, Rana Dutta, Tiziano Pramparo, Pamela Vig, Robert D Steiner

Faculty, Staff and Student Publications

Background: Smith-Lemli-Opitz Syndrome (SLOS) is an autosomal recessive disorder of cholesterol biosynthesis caused by biallelic pathogenic variants in DHCR7, which encodes the enzyme 7-dehydrocholesterol reductase (DHCR7). SLOS is a multisystemic disorder affecting various aspects of health, including growth, development, behavior, and quality of life, underscoring the need for safe, efficacious interventions that limit disease burden. DHCR7 enzyme deficiency leads to a "metabolic block" resulting in decreased cholesterol production and accumulation of its precursor 7-dehydrocholesterol and the secondary isomer 8-dehydrocholesterol. Reduced cholesterol synthesis, in turn, leads to decreased levels of cholic acid (CA), an endogenous bile acid synthesized from cholesterol and …

Human Ipsc-Based Breast Cancer Model Identifies S100p-Dependent Cancer Stemness Induced By Brca1 Mutation, Jingxin Liu, Cai Zhao, Jiahao Chen, Pengguihang Zeng, Qingjian Li, Ranran Dai, Xingqiang Lai, Wenqian Song, Jianing Chen, Xixi Zhu, Xinyi Liu, Jun Sun, Jia Wang, Peihang Fang, Tengfei Wang, Wenjie Chen, Diana Guallar, Nan Cao, Jianli Zhao, Shicheng Su, Andy Peng Xiang, Yi Arial Zeng, Jie Li, Junchao Cai, Dung-Fang Lee, Jinxin Bei, Yongliang Huo, Hai Hu, Shengbao Suo, Dong-Feng Huang, Jin Bai, Junjun Ding

Faculty, Staff and Student Publications

Breast cancer is the most common malignancy in females and remains the leading cause of cancer-related deaths for women worldwide. The cellular and molecular basis of breast tumorigenesis is not completely understood partly due to the lack of human research models which simulate the development of breast cancer. Here, we developed a method for generating functional mammary-like cells (MCs) from human-induced pluripotent stem cells (iPSCs). The iPSC-MCs closely resemble human primary MCs at cellular, transcriptional, and functional levels. Using this method, a breast cancer model was generated using patient-derived iPSCs harboring germline

Adaptive Bayesian Clinical Trials: The Past, Present, And Future Of Clinical Research, Donald A Berry

Faculty, Staff and Student Publications

Background/Objectives: Decision-analytic Bayesian approaches are ideally suited for designing clinical trials. They have been used increasingly over the last 30 years in developing medical devices and drugs. A prototype trial is a bandit problem in which treating participants is as important as treating patients in clinical practice after the trial.

Methods: This article chronicles the use of the Bayesian approach in clinical trials motivated by bandit problems. It provides a comprehensive historical and practical review of Bayesian adaptive trials, with a focus on bandit-inspired designs.

Results: The 20th century saw advances in Bayesian methodology involving computer simulation. In the …

Adhd In Youth With Major Depressive Disorder In The Texas Youth Depression And Suicide Research Network (Tx-Ydsrn): Clinical Correlates And Moderators, Blake M Upshaw, Samuel D Spencer, Caitlin M Pinciotti, Vadym Zhyrov, Abu Minhajuddin, Ace A Castillo, Allyssa Abacan, Holli Slater, Rachel A Walker, Joseph C Blader, Sarah L Martin, Jeffrey D Shahidullah, Jair C Soares, Robert Andrew Harper, Madelyn Guerra, Lynnel C Goodman, Wayne K Goodman, Sarah M Wakefield, Madhukar H Trivedi, Eric A Storch

Faculty, Staff and Student Publications

Objective: Depression is a major public health concern with a 19% lifetime prevalence in youth, often precipitating other concerns, including suicidal behavior, poor school performance, and worsened peer relationships. ADHD is also common among youth and frequently presents alongside major depressive disorder (MDD), with this comorbidity associated with increased impairment. More research is needed to elucidate the clinical characteristics of this comorbidity (MDD + ADHD), especially as it relates to youth with MDD and no ADHD (MDD - ADHD). The present study examined the clinical correlates of MDD + ADHD in youth and the presence of an ADHD diagnosis as …

Conjugative Delivery Of Toxin Genes, Yang Grace Li, Daniel Haeusser, William Margolin, Peter J Christie

Faculty, Staff and Student Publications

The bacterial type IV secretion systems (T4SS) are medically problematic for their roles in the dissemination of mobile genetic elements or effector proteins, but they also have great potential for new antimicrobial therapies. Recent studies have deployed the T4SS subfamily of conjugation systems to deliver gene editing CRISPR/Cas systems to disrupt drug resistance genes or kill targeted bacterial recipients. However, the therapeutic potential of conjugative CRISPR/Cas delivery is compromised by mutations or host repair systems that diminish the efficiency with which CRISPR/Cas induces double-strand breaks in new transconjugants. Here, we compared the efficiencies of conjugation-based killing systems based on the …

Cancer Screening Uptake And Usual Source Of Care Among Hispanic Survivors Of Adolescent And Young Adult Cancer, Eunju Choi, Amy M Berkman, Clark R Andersen, John M Salsman, Andrea C Betts, Joel Milam, Kimberly A Miller, Susan K Peterson, Qian Lu, Christabel K Cheung, Lauren V Ghazal, J A Livingston, Michelle A T Hildebrandt, Susan K Parsons, David R Freyer, Michael E Roth

Faculty, Staff and Student Publications

Purpose: This study examines cancer screening rates and the usual source of care among Hispanic survivors of adolescent and young adult (AYA, aged 15-39) cancer, a group at increased risk for poor health outcomes, including secondary cancers.

Methods: National Health Interview Survey data were analyzed to identify Hispanic survivors of AYA cancer and Hispanic age- and sex-matched non-cancer controls. Breast, cervical, and colorectal screening (ever screened and up-to-date according to the U.S. Preventive Services Task Force recommendations) as well as reporting a usual source of care were compared among survivors and controls eligible for screening using multivariable logistic regressions.

Results: …

Survival-Inferred Fragility Of Statistical Significance In Phase Iii Oncology Trials, Alexander D Sherry, Yufei Liu, Pavlos Msaouel, Timothy A Lin, Alex Koong, Christine Lin, Joseph Abi Jaoude, Roshal R Patel, Ramez Kouzy, Molly B El-Alam, Avital M Miller, Mohannad Owiwi, Jonathan Ofer, David Bomze, Zachary R Mccaw, Tomer Meirson, Ethan B Ludmir

Faculty, Staff and Student Publications

In phase III oncology trials, superiority is defined by statistical significance using P thresholds. However, this approach has been criticized because P is continuous. Here, we reconstruct patient-level data for 230 phase III oncology trials to model the robustness of statistical significance by estimating the survival-inferred fragility index (SIFI), defined as the smallest number of patients changing arms that alters the statistical significance interpretation. The median SIFI was 8 patients (IQR 4-19), representing 1.4% of enrollments (IQR 0.7%-3%). As a continuous statistic, P-but not the significance interpretation-was correlated with SIFI. Moreover, overall survival endpoints were more fragile than surrogate endpoints. …

Minimal Ablative Margin Quantification Using Hepatic Arterial Versus Portal Venous Phase Ct For Colorectal Metastases Segmentation: A Dual-Center, Retrospective Analysis, Noreen S Siddiqi, Yuan-Mao Lin, Jessica Albuquerque Marques Silva, Gregor Laimer, Peter Schullian, Yannick Scharll, Alexandra M Dunker, Caleb S O'Connor, Kyle A Jones, Kristy K Brock, Reto Bale, Bruno C Odisio, Iwan Paolucci

Faculty, Staff and Student Publications

Objective: To compare the predictive value of minimal ablative margin (MAM) quantification using tumor segmentation on intraprocedural contrast-enhanced hepatic arterial (HAP) versus portal venous phase (PVP) CT on local outcomes following percutaneous thermal ablation of colorectal liver metastases (CRLM).

Methods: This dual-center retrospective study included patients undergoing thermal ablation of CRLM with intraprocedural preablation and postablation contrast-enhanced CT imaging between 2009 and 2021. Tumors were segmented in both HAP and PVP CT phases using an artificial intelligence-based auto-segmentation model and reviewed by a trained radiologist. The MAM was quantified using a biomechanical deformable image registration process. The area under the …

Feasibility Of Manufacturing And Antitumor Activity Of Til For Advanced Endometrial Cancers, Yongliang Zhang, Kathleen N Moore, Amir A Jazaeri, Judy Fang, Ilabahen Patel, Andrew Yuhas, Patrick Innamarato, Nathan Gilbert, Joseph W Dean, Behzad Damirchi, Joe Yglesias, Rongsu Qi, Michelle R Simpson-Abelson, Erwin Cammaart, Sean R R Hall, Hequn Yin

Faculty, Staff and Student Publications

Lifileucel, a tumor-infiltrating lymphocyte (TIL) cell therapy approved for advanced melanoma, demonstrates promise for treating other solid tumors, including endometrial cancer (EC). The current study evaluates the feasibility of manufacturing TILs from EC tumors using Iovance’s proprietary 22-day Gen2 manufacturing process. Key parameters, including TIL yield, viability, immune phenotype, T-cell receptor clonality, and cytotoxic activity, were assessed. Of the 11 EC tumor samples processed at research scale, 10 (91%) successfully generated >1 × 109 viable TIL cells, with a median yield of 1.1 × 1010 cells and a median viability of 82.8%. Of the four EC tumor samples processed at …

The Microtubule-Severing Enzyme Spastin Regulates Spindle Dynamics To Promote Chromosome Segregation In Trypanosoma Brucei, Thiago Souza Onofre, Qing Zhou, Ziyin Li

Faculty, Staff and Student Publications

Microtubule-severing enzymes play essential roles in diverse cellular processes, including mitosis and cytokinesis, by modulating microtubule dynamics. In the early branching Trypanosoma brucei, microtubule-severing enzymes are involved in cytokinesis and flagellum length control, but none of them have been found to regulate mitosis. Here we report the characterization of the microtubule-severing enzyme spastin in the procyclic form of T. brucei. We demonstrate that spastin severs microtubule in vitro and overexpression of spastin disrupts spindle microtubules in vivo in trypanosomes, leading to defective chromosome segregation. Knockdown of spastin impairs spindle integrity and disrupts chromosome alignment and segregation. We further show that …

Precision Care In Screening, Surveillance, And Overall Management Of Barrett's Esophagus, Yeshaswini Reddy, Madhav Desai, Bernadette Tumaliuan, Nirav Thosani

Faculty, Staff and Student Publications

Barrett's esophagus (BE), a metaplastic transformation of an esophageal squamous epithelium into an intestinal-type columnar epithelium, is the primary precursor to esophageal adenocarcinoma (EAC). Traditional management strategies have relied heavily on selective screening, tailored surveillance intervals, and early dysplasia detection and treatment algorithms. However, the heterogeneity in progression risk among BE patients necessitates a more nuanced, personalized approach involving precision care, tailoring decisions to individual patient characteristics, promises to enhance outcomes in BE through more targeted screening, personalized surveillance intervals, and risk-based therapeutic strategies. This review explores the current landscape and emerging trends in precision medicine for Barrett's esophagus, highlighting …

The Adaptation And Translation Of A Transition Readiness Scale For Young Adults With Autism Spectrum Disorder, Antonio F Pagán, Katherine A Loveland, Ron Acierno

Faculty, Staff and Student Publications

Autistic young adults often face significant challenges during the transition to adulthood, yet few tools exist to assess their readiness in a developmentally appropriate and culturally responsive manner. This study presents the development and initial validation of the Transition Readiness Scale for Autistic Adolescents and Adults (TRS-A), a brief caregiver- and self-report measure designed to assess key domains of transition readiness. Guided by qualitative input from Latino families, community stakeholders, and clinicians, the TRS-A was piloted with 114 autistic young adults, 66 Spanish-speaking parents, and 48 English-speaking parents. Psychometric analyses supported the scale's internal consistency and construct validity, mirroring the …

Timing Of Nodal Metastasis In Patients With Eyelid Sebaceous Carcinoma And Implications For Surveillance And Survival, Tracy Lu, Casey Anthony, Xinyang Jiang, Jing Ning, Ryan Goepfert, Bita Esmaeli

Faculty, Staff and Student Publications

Background/aims: Sebaceous carcinoma (SC) is a rare cancer with periocular predilection. Studies focused on patterns and timing of nodal metastasis are scarce.Our aim is to report patterns of regional nodal metastasis in patients with eyelid SC, including results of sentinel lymph node biopsy (SLNB).

Methods: All consecutive patients with eyelid SC treated during 1999-2023 were retrospectively reviewed.All subjects had standard of care surgery for eyelid SC and regional lymph node evaluation using physical examination and ultrasonography (US) at baseline and during the surveillance period. 38 patients with negative findings on US had SLNB to rule out microscopic metastasis.The frequency and …

Acute Respiratory Distress Syndrome In Patients With Lymphopenia: Results From The National Inpatient Sample (2017-2021), Arnav Garyali, Trishna Parikh, Dhruv Kumar, Ishan Gupta, Adishwar Rao, Akriti Agrawal, Sabiha Armin, Rishi Panjala, Rohan Patil, Nikhil Sriram, Sruthi Parthasarathy, Aarohi Parikh, Bindu Akkanti

Faculty, Staff and Student Publications

Background: Lymphopenia has been associated with in-hospital, early, and late mortality. We aimed to elucidate differences in baseline characteristics in patients with lymphopenia with and without acute respiratory distress syndrome (ARDS) and determine predictors of in-hospital mortality in this patient population.

Methods: Patients ≥ 18 years of age with lymphopenia were identified in the National Inpatient Sample (2017-2021) and stratified according to ARDS diagnosis. Predictors of in-hospital mortality were determined using multivariate analyses with a logistic regression model.

Results: From 183,185 patients with lymphopenia, 10,420 (5.7%) had ARDS, of which 92.8% had coronavirus disease 2019. The patients with ARDS suffered …